Oncology

A renewed effort to modernize and stabilize Medicare reimbursement for radiation therapy services is underway.

In mid-March, members of Congress reintroduced bipartisan federal legislation that would shift Medicare reimbursement for radiation oncology services from quantity-based payments to episode-based payments and help stabilize the declining rates of reimbursement in the field. 

The Radiation Oncology Case Rate (ROCR) Value Based Payment Program Act, sponsored by two senators and four representatives, would not only “transform” how Medicare reimburses radiation therapy services, it would also “protect access to high quality cancer care and improve outcomes for patients nationwide, while generating savings for Medicare,” according to a recent American Society for Radiation Oncology (ASTRO) press release praising the bill. 

However, the reaction among those in the field has been mixed. Whereas some radiation oncologists are aligned with the bill, others argue that the legislation was crafted without meaningful input from many who will be affected.

“There’s consensus across multiple groups within the house of radiation oncology, hospital groups, and industry, which is incredibly important,” according to Mustafa Basree, DO, a radiation oncology resident who serves on ASTRO’s government relations committee and was part of the discussion on drafting the bill. 

But, Basree acknowledged, “not everybody likes the bill.”

A core complaint is a lack of communication and input from clinicians in the field. “If we’re going to decide to design our own quality program — which is really like a dream from a clinician’s standpoint — we need a meaningful way to come together as a unified field,” said Matthew Spraker, MD, PhD, a radiation oncologist practicing in Denver. In this bill, “we’re not getting any of that.”

 

Impetus for the Bill

Amid dramatic drops in Medicare reimbursement — and with more probably on the horizon — ASTRO announced in January 2024 that the society had partnered with the American College of Radiation Oncology, the American College of Radiology, and the American Society of Clinical Oncology to lobby for payment reform. 

Cuts to Medicare reimbursement were approaching 25% at the time. These declines were related, in part, to changes in how radiation treatment was being delivered. Reimbursement has historically been based on the fraction of radiation given, but the field has increasingly embraced hypofractionated regimens and deescalated approaches, which have led to fewer billable fractions and consequently lower reimbursement. 

A recent study highlighted significant declines in reimbursement based on this shift in care. For instance, greater use of hypofractionation led to declines in reimbursement for technical services in freestanding radiation oncology offices by nearly 17% for breast cancer and 14.2% for prostate cancer between 2016 and 2022. Inflation-adjusted Medicare conversion factors fell 12.2% in hospital outpatient centers and 20.8% in freestanding offices.

These declining reimbursement rates have occurred alongside changes to radiation oncology practice patterns. A recent analysis reported a 51% increase in the number of US practices with at least 10 radiation oncologists between 2015 and 2023, and a 27% decrease in the number of solo practices during the same period. The number of practicing radiation oncologists increased by 16%, but the number of practices employing them decreased by 13%, indicating a trend toward practice consolidation. 

These changes, the analysis found, may affect patients’ access to care. In rural areas, retirement rates were higher and rates of entry of new radiation oncologists was lower compared with urban areas.

The current payment structure “has become untenable,” leading to practice consolidation that threaten patient access, especially in rural and underserved areas, a spokesperson for ASTRO told this news organization last year. 

 

What Is the ROCR Act?

The ROCR ACT was drafted by ASTRO to address these issues and reverse declines in Medicare reimbursement. 

In addition to shifting radiation oncology reimbursement from fraction-based to episode-based, the bill also aims to encourage clinicians to adoptevidence-based shorter treatment regimens, improve safety and quality by supporting new technologies, and generate savings to Medicare by eliminating outdated and costly practices that have not been shown to improve patient outcomes.

When first introduced last year, the bill did not receive a vote in Congress.

A 2025 version of the bill, introduced last month, largely aligns with the 2024 legislation but contains some “enhancements,” such as improving accreditation with increased incentive payments, outlining a revised exemption for practices with limited resources and instituting a transitional payment period for adaptive radiation therapy to allow billing to continue while a new code is created.

 

Mixed Reactions 

How has the radiation oncology community reacted to the latest ROCR Act? 

A recent survey, which included more than 500 practicing radiation oncologists, found that 61% of respondents supported implementing an episode-based payment model such as that proposed in the 2025 legislation, 17.3% neither supported nor opposed it, and 21.6% opposed the model.

“I think this supports this idea that our field would have benefited from much more open discussion in the design phases of the bill,” Spraker told this news organization.

Jason Beckta, MD, PhD, a radiation oncologist at Rutland Regional Medical Center, Vermont, agreed. 

While on board with the concept of reform and episode-based payment, given what Beckta called “the absolute absurdity of the cuts in radiation oncology,” he took issue with the lack of transparency in the rollout of the bill.

The announcement about the 2025 version of the ROCR Act came as “a complete surprise — out of nowhere — except to insiders,” Beckta said.

ASTRO held a town hall in February 2025 “featuring new information and discussion” regarding ROCR 2025, but the bill had already been finalized for submission at that point, Beckta said. 

And although Beckta and Spraker believe ASTRO had good intentions, the physicians highlighted concerns with several aspects of the bill.

“What upsets me most is the blatant regulatory capture,” Beckta said. The legislation will require all practices to be accredited by either ASTRO, the American College of Radiation Oncology, or the American College of Radiology, essentially capturing their business through a regulation or having practices “face a 2.5% penalty, which is up 1% from the prior version,” Beckta explained. 

The bill also shortens the runway to get accredited from 3 years to 2 years, Beckta noted, stressing the arduousness of the accreditation process as a hurdle for many practices.

But doing the accreditation program does not mean care will get better, Spraker said. In fact, “that is absolutely not the case.”

Another issue: Requirements for medical equipment and quality review periods seem to favor industry over patients and practices, he said, highlighting the potential role of manufacturers in determining if or when equipment updates are required.

“A field like ours has rapidly exploding technology with not-always-clear patient benefit,” said Spraker. “We’re seeing too many examples where people are leveraging that, basically to sell devices instead of to help patients,” he added. 

Furthermore, Beckta noted, the bill allows for reduced reimbursement of between 4% and 7%, depending on the circumstances — a cut to reimbursement that is being justified by saying it’s the only way the bill will get through Congress. But “it’s just a less-bad option than continued cuts to fee-for-service,” Beckta said.

ASTRO leadership has expressed strong support for the bill. In the recent ASTRO press release, Howard M. Sandler, MD, chair of the ASTRO Board of Directors, called the ROCR Act “the only viable policy solution designed to provide payment stability for the field of radiation oncology in 2026 and beyond.” 

The ROCR Act, which is broadly supported by more than 80 organizations, “represents a balanced, evidence-based policy solution to safeguard access to high value cancer treatment for Americans,” Sandler said. 

“I believe in this bill,” Basree added.

Basree touted the replacement of a fee-for-service model with a “value-based payment system, ensuring predictable, fair reimbursement for the field” as a major win for stabilizing Medicare reimbursement. The bill also includes measures to improve patient access, such as providing discounted transportation for patients — a significant need, particularly in rural areas, he explained.

Although not everyone is happy with the bill, ASTRO did aim “to build coalition of support,” Basree said. “It’s an uphill battle, for sure, but we should press forward and hope for the best,” he added.

Even with their concerns, both Spraker and Beckta are optimistic that improvements to the bill can still be made, and urge colleagues to study the bill, speak out, and engage to help promote the best possible policy.

Basree reported receiving reimbursement for meeting travel and lodging as both a Fellow and member of the Association of Residents in Radiation Oncology. Spraker and Beckta reported having no relevant disclosures.

A version of this article first appeared on Medscape.com.

A renewed effort to modernize and stabilize Medicare reimbursement for radiation therapy services is underway.

In mid-March, members of Congress reintroduced bipartisan federal legislation that would shift Medicare reimbursement for radiation oncology services from quantity-based payments to episode-based payments and help stabilize the declining rates of reimbursement in the field. 

The Radiation Oncology Case Rate (ROCR) Value Based Payment Program Act, sponsored by two senators and four representatives, would not only “transform” how Medicare reimburses radiation therapy services, it would also “protect access to high quality cancer care and improve outcomes for patients nationwide, while generating savings for Medicare,” according to a recent American Society for Radiation Oncology (ASTRO) press release praising the bill. 

However, the reaction among those in the field has been mixed. Whereas some radiation oncologists are aligned with the bill, others argue that the legislation was crafted without meaningful input from many who will be affected.

“There’s consensus across multiple groups within the house of radiation oncology, hospital groups, and industry, which is incredibly important,” according to Mustafa Basree, DO, a radiation oncology resident who serves on ASTRO’s government relations committee and was part of the discussion on drafting the bill. 

But, Basree acknowledged, “not everybody likes the bill.”

A core complaint is a lack of communication and input from clinicians in the field. “If we’re going to decide to design our own quality program — which is really like a dream from a clinician’s standpoint — we need a meaningful way to come together as a unified field,” said Matthew Spraker, MD, PhD, a radiation oncologist practicing in Denver. In this bill, “we’re not getting any of that.”

 

Impetus for the Bill

Amid dramatic drops in Medicare reimbursement — and with more probably on the horizon — ASTRO announced in January 2024 that the society had partnered with the American College of Radiation Oncology, the American College of Radiology, and the American Society of Clinical Oncology to lobby for payment reform. 

Cuts to Medicare reimbursement were approaching 25% at the time. These declines were related, in part, to changes in how radiation treatment was being delivered. Reimbursement has historically been based on the fraction of radiation given, but the field has increasingly embraced hypofractionated regimens and deescalated approaches, which have led to fewer billable fractions and consequently lower reimbursement. 

A recent study highlighted significant declines in reimbursement based on this shift in care. For instance, greater use of hypofractionation led to declines in reimbursement for technical services in freestanding radiation oncology offices by nearly 17% for breast cancer and 14.2% for prostate cancer between 2016 and 2022. Inflation-adjusted Medicare conversion factors fell 12.2% in hospital outpatient centers and 20.8% in freestanding offices.

These declining reimbursement rates have occurred alongside changes to radiation oncology practice patterns. A recent analysis reported a 51% increase in the number of US practices with at least 10 radiation oncologists between 2015 and 2023, and a 27% decrease in the number of solo practices during the same period. The number of practicing radiation oncologists increased by 16%, but the number of practices employing them decreased by 13%, indicating a trend toward practice consolidation. 

These changes, the analysis found, may affect patients’ access to care. In rural areas, retirement rates were higher and rates of entry of new radiation oncologists was lower compared with urban areas.

The current payment structure “has become untenable,” leading to practice consolidation that threaten patient access, especially in rural and underserved areas, a spokesperson for ASTRO told this news organization last year. 

 

What Is the ROCR Act?

The ROCR ACT was drafted by ASTRO to address these issues and reverse declines in Medicare reimbursement. 

In addition to shifting radiation oncology reimbursement from fraction-based to episode-based, the bill also aims to encourage clinicians to adoptevidence-based shorter treatment regimens, improve safety and quality by supporting new technologies, and generate savings to Medicare by eliminating outdated and costly practices that have not been shown to improve patient outcomes.

When first introduced last year, the bill did not receive a vote in Congress.

A 2025 version of the bill, introduced last month, largely aligns with the 2024 legislation but contains some “enhancements,” such as improving accreditation with increased incentive payments, outlining a revised exemption for practices with limited resources and instituting a transitional payment period for adaptive radiation therapy to allow billing to continue while a new code is created.

 

Mixed Reactions 

How has the radiation oncology community reacted to the latest ROCR Act? 

A recent survey, which included more than 500 practicing radiation oncologists, found that 61% of respondents supported implementing an episode-based payment model such as that proposed in the 2025 legislation, 17.3% neither supported nor opposed it, and 21.6% opposed the model.

“I think this supports this idea that our field would have benefited from much more open discussion in the design phases of the bill,” Spraker told this news organization.

Jason Beckta, MD, PhD, a radiation oncologist at Rutland Regional Medical Center, Vermont, agreed. 

While on board with the concept of reform and episode-based payment, given what Beckta called “the absolute absurdity of the cuts in radiation oncology,” he took issue with the lack of transparency in the rollout of the bill.

The announcement about the 2025 version of the ROCR Act came as “a complete surprise — out of nowhere — except to insiders,” Beckta said.

ASTRO held a town hall in February 2025 “featuring new information and discussion” regarding ROCR 2025, but the bill had already been finalized for submission at that point, Beckta said. 

And although Beckta and Spraker believe ASTRO had good intentions, the physicians highlighted concerns with several aspects of the bill.

“What upsets me most is the blatant regulatory capture,” Beckta said. The legislation will require all practices to be accredited by either ASTRO, the American College of Radiation Oncology, or the American College of Radiology, essentially capturing their business through a regulation or having practices “face a 2.5% penalty, which is up 1% from the prior version,” Beckta explained. 

The bill also shortens the runway to get accredited from 3 years to 2 years, Beckta noted, stressing the arduousness of the accreditation process as a hurdle for many practices.

But doing the accreditation program does not mean care will get better, Spraker said. In fact, “that is absolutely not the case.”

Another issue: Requirements for medical equipment and quality review periods seem to favor industry over patients and practices, he said, highlighting the potential role of manufacturers in determining if or when equipment updates are required.

“A field like ours has rapidly exploding technology with not-always-clear patient benefit,” said Spraker. “We’re seeing too many examples where people are leveraging that, basically to sell devices instead of to help patients,” he added. 

Furthermore, Beckta noted, the bill allows for reduced reimbursement of between 4% and 7%, depending on the circumstances — a cut to reimbursement that is being justified by saying it’s the only way the bill will get through Congress. But “it’s just a less-bad option than continued cuts to fee-for-service,” Beckta said.

ASTRO leadership has expressed strong support for the bill. In the recent ASTRO press release, Howard M. Sandler, MD, chair of the ASTRO Board of Directors, called the ROCR Act “the only viable policy solution designed to provide payment stability for the field of radiation oncology in 2026 and beyond.” 

The ROCR Act, which is broadly supported by more than 80 organizations, “represents a balanced, evidence-based policy solution to safeguard access to high value cancer treatment for Americans,” Sandler said. 

“I believe in this bill,” Basree added.

Basree touted the replacement of a fee-for-service model with a “value-based payment system, ensuring predictable, fair reimbursement for the field” as a major win for stabilizing Medicare reimbursement. The bill also includes measures to improve patient access, such as providing discounted transportation for patients — a significant need, particularly in rural areas, he explained.

Although not everyone is happy with the bill, ASTRO did aim “to build coalition of support,” Basree said. “It’s an uphill battle, for sure, but we should press forward and hope for the best,” he added.

Even with their concerns, both Spraker and Beckta are optimistic that improvements to the bill can still be made, and urge colleagues to study the bill, speak out, and engage to help promote the best possible policy.

Basree reported receiving reimbursement for meeting travel and lodging as both a Fellow and member of the Association of Residents in Radiation Oncology. Spraker and Beckta reported having no relevant disclosures.

A version of this article first appeared on Medscape.com.

A renewed effort to modernize and stabilize Medicare reimbursement for radiation therapy services is underway.

In mid-March, members of Congress reintroduced bipartisan federal legislation that would shift Medicare reimbursement for radiation oncology services from quantity-based payments to episode-based payments and help stabilize the declining rates of reimbursement in the field. 

The Radiation Oncology Case Rate (ROCR) Value Based Payment Program Act, sponsored by two senators and four representatives, would not only “transform” how Medicare reimburses radiation therapy services, it would also “protect access to high quality cancer care and improve outcomes for patients nationwide, while generating savings for Medicare,” according to a recent American Society for Radiation Oncology (ASTRO) press release praising the bill. 

However, the reaction among those in the field has been mixed. Whereas some radiation oncologists are aligned with the bill, others argue that the legislation was crafted without meaningful input from many who will be affected.

“There’s consensus across multiple groups within the house of radiation oncology, hospital groups, and industry, which is incredibly important,” according to Mustafa Basree, DO, a radiation oncology resident who serves on ASTRO’s government relations committee and was part of the discussion on drafting the bill. 

But, Basree acknowledged, “not everybody likes the bill.”

A core complaint is a lack of communication and input from clinicians in the field. “If we’re going to decide to design our own quality program — which is really like a dream from a clinician’s standpoint — we need a meaningful way to come together as a unified field,” said Matthew Spraker, MD, PhD, a radiation oncologist practicing in Denver. In this bill, “we’re not getting any of that.”

 

Impetus for the Bill

Amid dramatic drops in Medicare reimbursement — and with more probably on the horizon — ASTRO announced in January 2024 that the society had partnered with the American College of Radiation Oncology, the American College of Radiology, and the American Society of Clinical Oncology to lobby for payment reform. 

Cuts to Medicare reimbursement were approaching 25% at the time. These declines were related, in part, to changes in how radiation treatment was being delivered. Reimbursement has historically been based on the fraction of radiation given, but the field has increasingly embraced hypofractionated regimens and deescalated approaches, which have led to fewer billable fractions and consequently lower reimbursement. 

A recent study highlighted significant declines in reimbursement based on this shift in care. For instance, greater use of hypofractionation led to declines in reimbursement for technical services in freestanding radiation oncology offices by nearly 17% for breast cancer and 14.2% for prostate cancer between 2016 and 2022. Inflation-adjusted Medicare conversion factors fell 12.2% in hospital outpatient centers and 20.8% in freestanding offices.

These declining reimbursement rates have occurred alongside changes to radiation oncology practice patterns. A recent analysis reported a 51% increase in the number of US practices with at least 10 radiation oncologists between 2015 and 2023, and a 27% decrease in the number of solo practices during the same period. The number of practicing radiation oncologists increased by 16%, but the number of practices employing them decreased by 13%, indicating a trend toward practice consolidation. 

These changes, the analysis found, may affect patients’ access to care. In rural areas, retirement rates were higher and rates of entry of new radiation oncologists was lower compared with urban areas.

The current payment structure “has become untenable,” leading to practice consolidation that threaten patient access, especially in rural and underserved areas, a spokesperson for ASTRO told this news organization last year. 

 

What Is the ROCR Act?

The ROCR ACT was drafted by ASTRO to address these issues and reverse declines in Medicare reimbursement. 

In addition to shifting radiation oncology reimbursement from fraction-based to episode-based, the bill also aims to encourage clinicians to adoptevidence-based shorter treatment regimens, improve safety and quality by supporting new technologies, and generate savings to Medicare by eliminating outdated and costly practices that have not been shown to improve patient outcomes.

When first introduced last year, the bill did not receive a vote in Congress.

A 2025 version of the bill, introduced last month, largely aligns with the 2024 legislation but contains some “enhancements,” such as improving accreditation with increased incentive payments, outlining a revised exemption for practices with limited resources and instituting a transitional payment period for adaptive radiation therapy to allow billing to continue while a new code is created.

 

Mixed Reactions 

How has the radiation oncology community reacted to the latest ROCR Act? 

A recent survey, which included more than 500 practicing radiation oncologists, found that 61% of respondents supported implementing an episode-based payment model such as that proposed in the 2025 legislation, 17.3% neither supported nor opposed it, and 21.6% opposed the model.

“I think this supports this idea that our field would have benefited from much more open discussion in the design phases of the bill,” Spraker told this news organization.

Jason Beckta, MD, PhD, a radiation oncologist at Rutland Regional Medical Center, Vermont, agreed. 

While on board with the concept of reform and episode-based payment, given what Beckta called “the absolute absurdity of the cuts in radiation oncology,” he took issue with the lack of transparency in the rollout of the bill.

The announcement about the 2025 version of the ROCR Act came as “a complete surprise — out of nowhere — except to insiders,” Beckta said.

ASTRO held a town hall in February 2025 “featuring new information and discussion” regarding ROCR 2025, but the bill had already been finalized for submission at that point, Beckta said. 

And although Beckta and Spraker believe ASTRO had good intentions, the physicians highlighted concerns with several aspects of the bill.

“What upsets me most is the blatant regulatory capture,” Beckta said. The legislation will require all practices to be accredited by either ASTRO, the American College of Radiation Oncology, or the American College of Radiology, essentially capturing their business through a regulation or having practices “face a 2.5% penalty, which is up 1% from the prior version,” Beckta explained. 

The bill also shortens the runway to get accredited from 3 years to 2 years, Beckta noted, stressing the arduousness of the accreditation process as a hurdle for many practices.

But doing the accreditation program does not mean care will get better, Spraker said. In fact, “that is absolutely not the case.”

Another issue: Requirements for medical equipment and quality review periods seem to favor industry over patients and practices, he said, highlighting the potential role of manufacturers in determining if or when equipment updates are required.

“A field like ours has rapidly exploding technology with not-always-clear patient benefit,” said Spraker. “We’re seeing too many examples where people are leveraging that, basically to sell devices instead of to help patients,” he added. 

Furthermore, Beckta noted, the bill allows for reduced reimbursement of between 4% and 7%, depending on the circumstances — a cut to reimbursement that is being justified by saying it’s the only way the bill will get through Congress. But “it’s just a less-bad option than continued cuts to fee-for-service,” Beckta said.

ASTRO leadership has expressed strong support for the bill. In the recent ASTRO press release, Howard M. Sandler, MD, chair of the ASTRO Board of Directors, called the ROCR Act “the only viable policy solution designed to provide payment stability for the field of radiation oncology in 2026 and beyond.” 

The ROCR Act, which is broadly supported by more than 80 organizations, “represents a balanced, evidence-based policy solution to safeguard access to high value cancer treatment for Americans,” Sandler said. 

“I believe in this bill,” Basree added.

Basree touted the replacement of a fee-for-service model with a “value-based payment system, ensuring predictable, fair reimbursement for the field” as a major win for stabilizing Medicare reimbursement. The bill also includes measures to improve patient access, such as providing discounted transportation for patients — a significant need, particularly in rural areas, he explained.

Although not everyone is happy with the bill, ASTRO did aim “to build coalition of support,” Basree said. “It’s an uphill battle, for sure, but we should press forward and hope for the best,” he added.

Even with their concerns, both Spraker and Beckta are optimistic that improvements to the bill can still be made, and urge colleagues to study the bill, speak out, and engage to help promote the best possible policy.

Basree reported receiving reimbursement for meeting travel and lodging as both a Fellow and member of the Association of Residents in Radiation Oncology. Spraker and Beckta reported having no relevant disclosures.

A version of this article first appeared on Medscape.com.

For years, the default definitive treatment for patients with early-stage I non–small cell lung cancer (NSCLC) has been surgical resection, typically minimally invasive lobectomy with systematic lymph node dissection.

Guidelines from the National Comprehensive Cancer Network (NCCN), the American Society of Clinical Oncology, and the European Society for Medical Oncology all list surgery (in particular, lobectomy) as the primary local therapy for fit, operable patients with stage I NSCLC.

More recently, however, stereotactic body radiotherapy (SBRT), also called stereotactic ablative radiotherapy, has emerged as a definitive treatment option for stage I NSCLC, especially for older, less fit patients who are unsuitable or deemed high-risk for surgery.

“We see patients in our practice who cannot undergo surgery or who may not have adequate lung function to be able to tolerate surgery, and for these patients who are medically inoperable or surgically unresectable, radiation therapy may be a reasonable option,” Charu Aggarwal, MD, MPH, professor and lung cancer specialist, University of Pennsylvania, Philadelphia, told this news organization.

Given some encouraging data suggesting that SBRT could provide similar survival outcomes and be an alternative to surgery for operable disease, SBRT is also increasingly being considered in these early-stage patients. However, other evidence indicates that SBRT may be associated with higher rates of regional and distant recurrences and worse long-term survival, particularly in operable patients.

What may ultimately matter most is carefully selecting operable patients who undergo SBRT.

Aggarwal has encountered certain patients who are fit for surgery but would rather have radiation therapy. “This is an individual decision, and these patients are usually discussed at tumor board and in multidisciplinary discussions to really make sure that they’re making the right decision for themselves,” she explained.

 

The Pros and Cons of SBRT

SBRT is a nonsurgical approach in which precision high-dose radiation is delivered in just a few fractions — typically, 3, 5, or 8, depending on institutional protocols and tumor characteristics.

SBRT is performed on an outpatient basis, usually over 1-2 weeks, with most patients able to resume usual activities with minimal to no delay. Surgery, on the other hand, requires a hospital stay and takes most people about 2-6 weeks to return to regular activities. SBRT also avoids anesthesia and surgical incisions, both of which come with risks.

The data on SBRT in early-stage NSCLC are mixed. While some studies indicate that SBRT comes with promising survival outcomes, other research has reported worse survival and recurrence rates.

One potential reason for higher recurrence rates with SBRT is the lack of pathologic nodal staging, which only happens after surgery, as well as lower rates of nodal evaluation with endobronchial ultrasound or mediastinoscopy before surgery or SBRT. Without nodal assessments, clinicians may miss a more aggressive histology or more advanced nodal stage, which would go undertreated if patients received SBRT.

 

Latest Data in Large Cohort

A recent study published in Lung Cancer indicated that, when carefully selected, operable patients with early NSCLC have comparable survival with lobectomy or SBRT.

In the study, Dutch researchers took an in-depth look at survival and recurrence patterns in a retrospective cohort study of 2183 patients with clinical stage I NSCLC treated with minimally invasive lobectomy or SBRT. The study includes one of the largest cohorts to date, with robust data collection on baseline characteristics, comorbidities, tumor size, performance status, and follow-up.

Patients receiving SBRT were typically older (median age, 74 vs 67 years), had higher comorbidity burdens (Charlson index ≥ 5 in 57% of SBRT patients vs 23% of surgical patients), worse performance status, and lower lung function. To adjust for these differences, the researchers used propensity score weighting so the SBRT group’s baseline characteristics were comparable with those in the surgery group.

The surgery cohort had more invasive nodal evaluation: 21% underwent endobronchial ultrasound or mediastinoscopy vs only 12% in the SBRT group. The vast majority in both groups had PET-CT staging, reflecting modern imaging-based workups.

While 5-year local recurrence rates between the two groups were similar (13.1% for SBRT vs 12.1% for surgery), the 5-year regional recurrence rate was significantly higher after SBRT than lobectomy (18.1% vs 14.2%; hazard ratio [HR], 0.74), as was the distant metastasis rate (26.2% vs 20.2%; HR, 0.72).

Mortality at 30 days was higher after surgery than SBRT (1.0% vs 0.2%). And in the unadjusted analysis, 5-year overall survival was significantly better with lobectomy than SBRT (70.2% vs 40.3%).

However, when the analysis only included patients with similar baseline characteristics, overall survival was no longer significantly different in the two groups (HR, 0.89; 95% CI, 0.65-1.20). Lung cancer–specific mortality was also not significantly different between the two treatments (HR, 1.08), but the study was underpowered to detect significant differences in this outcome on the basis of a relatively low number of deaths from NSCLC.

Still, even after comparing similar patients, recurrence-free survival was notably better with surgery (HR, 0.70), due to fewer regional recurrences and distant metastases. Overall, 13% of the surgical cohort had nodal upstaging at pathology, meaning that even in clinically “node-negative” stage I disease, a subset of patients had unsuspected nodal involvement.

Patients receiving SBRT did not have pathologic nodal staging, raising the possibility of occult micrometastases. The authors noted that the proportion of SBRT patients with occult lymph node metastases is likely at least equal to that in the surgery group, but these metastases would go undetected without pathologic assessment.

Missing potential occult micrometastases in the SBRT group likely contributed to higher regional recurrence rates over time. By improving nodal staging, more patients with occult lymph node metastases who would be undertreated with SBRT may be identified before treatment, the authors said.

 

What Do Experts Say?

So, is SBRT an option for patients with stage I NSCLC?

Opinions vary.

“If you got one shot for a cure, then you want to do the surgery because that’s what results in a cure,” said Raja Flores, MD, chairman of Thoracic Surgery, Mount Sinai Health System, New York City.

Flores noted that the survival rate with surgery is high in this population. “There’s really nothing out there that can compare,” he said.

In his view, surgery “remains the gold standard.” However, “radiation could be considered in nonsurgical candidates,” he said.

The most recent NCCN guidelines align with Flores’ take. The guidelines say that SBRT is indicated for stage IA-IIA (N0) NSCLC in patients who are deemed “medically inoperable, high surgical risk as determined by thoracic surgeon, and those who decline surgery after thoracic surgical consultation.”

Clifford G. Robinson, MD, agreed. “In the United States, we largely treat patients with SBRT who are medically inoperable or high-risk operable and a much smaller proportion who decline surgery,” said Robinson, professor of radiation oncology and chief of SBRT at Washington University in St. Louis, St. Louis. “Many patients who are deemed operable are not offered SBRT.”

Still, for Robinson, determining which patients are best suited for surgery or SBRT remains unclear.

“Retrospective comparisons are fraught with problems because of confounding,” Robinson told this news organization. “That is, the healthier patients get surgery, and the less healthy ones get SBRT. No manner of fancy statistical manipulation can remove that fact.”

In fact, a recent meta-analysis found that the most significant variable predicting whether surgery or SBRT was superior in retrospective studies was whether the author was a surgeon or radiation oncologist.

Robinson noted that multiple randomized trials have attempted to randomize patients with medically operable early-stage NSCLC to surgery or SBRT and failed to accrue, largely due to patients’ “understandable unwillingness to be randomized between operative vs nonoperative interventions when most already prefer one or the other approach.”

Flores highlighted another point of caution about interpreting trial results: Not all early-stage NSCLC behaves similarly. “Some are slow-growing ‘turtles,’ and others are aggressive ‘rabbits’ — and the turtles are usually the ones that have been included in these radiotherapy trials, and that’s the danger,” he said.

While medical operability is the primary factor for deciding the treatment modality for early-stage NSCLC, there are other more subtle factors that can play into the decision.

These include prior surgery or radiotherapy to the chest, prior cancers, and social issues, such as the patient being a primary caregiver for another person and job insecurity, that might make recovery from surgery more challenging. And in rare instances, a patient may be medically fit to undergo surgery, but the cancer is technically challenging to resect due to anatomic issues or prior surgery to the chest, Robinson added.

 

A Winner?

Results from two ongoing, highly anticipated randomized trials expected in the next several years will hopefully provide additional insights and clarify ongoing uncertainties about the optimal treatment strategies for operable patients with stage I NSCLC.

STABLE-MATES is comparing outcomes after sublobar resection and SBRT in high-risk operable stage I NSCLC, and VALOR is evaluating outcomes after anatomic pulmonary resections and SBRT in patients with stage I NSCLC who have a long life expectancy and are fit enough to tolerate surgery.

But Robinson said his group believes that trying to decide on a winner is a “fool’s errand” and is instead running a pragmatic study across multiple academic and community centers around the United States and Canada where patients choose therapy based on their personal preferences and guidance from their physicians. The researchers will carefully track baseline comorbidity and frailty and assess serial quality-of-life changes over time.

“The goal is to create a calculator that a given patient might use in the future to determine what patients like them would have received, complete with expected outcomes and side effects,” Robinson said.

Robinson cautioned, however, that it “seems unlikely, given the existing literature, that one of the treatments will be truly ‘superior’ to the other one and lead to the ‘losing’ treatment fading away since both are excellent options with pros and cons.”

Aggarwal, Robinson, and Flores had no relevant disclosures.

A version of this article first appeared on Medscape.com.

For years, the default definitive treatment for patients with early-stage I non–small cell lung cancer (NSCLC) has been surgical resection, typically minimally invasive lobectomy with systematic lymph node dissection.

Guidelines from the National Comprehensive Cancer Network (NCCN), the American Society of Clinical Oncology, and the European Society for Medical Oncology all list surgery (in particular, lobectomy) as the primary local therapy for fit, operable patients with stage I NSCLC.

More recently, however, stereotactic body radiotherapy (SBRT), also called stereotactic ablative radiotherapy, has emerged as a definitive treatment option for stage I NSCLC, especially for older, less fit patients who are unsuitable or deemed high-risk for surgery.

“We see patients in our practice who cannot undergo surgery or who may not have adequate lung function to be able to tolerate surgery, and for these patients who are medically inoperable or surgically unresectable, radiation therapy may be a reasonable option,” Charu Aggarwal, MD, MPH, professor and lung cancer specialist, University of Pennsylvania, Philadelphia, told this news organization.

Given some encouraging data suggesting that SBRT could provide similar survival outcomes and be an alternative to surgery for operable disease, SBRT is also increasingly being considered in these early-stage patients. However, other evidence indicates that SBRT may be associated with higher rates of regional and distant recurrences and worse long-term survival, particularly in operable patients.

What may ultimately matter most is carefully selecting operable patients who undergo SBRT.

Aggarwal has encountered certain patients who are fit for surgery but would rather have radiation therapy. “This is an individual decision, and these patients are usually discussed at tumor board and in multidisciplinary discussions to really make sure that they’re making the right decision for themselves,” she explained.

 

The Pros and Cons of SBRT

SBRT is a nonsurgical approach in which precision high-dose radiation is delivered in just a few fractions — typically, 3, 5, or 8, depending on institutional protocols and tumor characteristics.

SBRT is performed on an outpatient basis, usually over 1-2 weeks, with most patients able to resume usual activities with minimal to no delay. Surgery, on the other hand, requires a hospital stay and takes most people about 2-6 weeks to return to regular activities. SBRT also avoids anesthesia and surgical incisions, both of which come with risks.

The data on SBRT in early-stage NSCLC are mixed. While some studies indicate that SBRT comes with promising survival outcomes, other research has reported worse survival and recurrence rates.

One potential reason for higher recurrence rates with SBRT is the lack of pathologic nodal staging, which only happens after surgery, as well as lower rates of nodal evaluation with endobronchial ultrasound or mediastinoscopy before surgery or SBRT. Without nodal assessments, clinicians may miss a more aggressive histology or more advanced nodal stage, which would go undertreated if patients received SBRT.

 

Latest Data in Large Cohort

A recent study published in Lung Cancer indicated that, when carefully selected, operable patients with early NSCLC have comparable survival with lobectomy or SBRT.

In the study, Dutch researchers took an in-depth look at survival and recurrence patterns in a retrospective cohort study of 2183 patients with clinical stage I NSCLC treated with minimally invasive lobectomy or SBRT. The study includes one of the largest cohorts to date, with robust data collection on baseline characteristics, comorbidities, tumor size, performance status, and follow-up.

Patients receiving SBRT were typically older (median age, 74 vs 67 years), had higher comorbidity burdens (Charlson index ≥ 5 in 57% of SBRT patients vs 23% of surgical patients), worse performance status, and lower lung function. To adjust for these differences, the researchers used propensity score weighting so the SBRT group’s baseline characteristics were comparable with those in the surgery group.

The surgery cohort had more invasive nodal evaluation: 21% underwent endobronchial ultrasound or mediastinoscopy vs only 12% in the SBRT group. The vast majority in both groups had PET-CT staging, reflecting modern imaging-based workups.

While 5-year local recurrence rates between the two groups were similar (13.1% for SBRT vs 12.1% for surgery), the 5-year regional recurrence rate was significantly higher after SBRT than lobectomy (18.1% vs 14.2%; hazard ratio [HR], 0.74), as was the distant metastasis rate (26.2% vs 20.2%; HR, 0.72).

Mortality at 30 days was higher after surgery than SBRT (1.0% vs 0.2%). And in the unadjusted analysis, 5-year overall survival was significantly better with lobectomy than SBRT (70.2% vs 40.3%).

However, when the analysis only included patients with similar baseline characteristics, overall survival was no longer significantly different in the two groups (HR, 0.89; 95% CI, 0.65-1.20). Lung cancer–specific mortality was also not significantly different between the two treatments (HR, 1.08), but the study was underpowered to detect significant differences in this outcome on the basis of a relatively low number of deaths from NSCLC.

Still, even after comparing similar patients, recurrence-free survival was notably better with surgery (HR, 0.70), due to fewer regional recurrences and distant metastases. Overall, 13% of the surgical cohort had nodal upstaging at pathology, meaning that even in clinically “node-negative” stage I disease, a subset of patients had unsuspected nodal involvement.

Patients receiving SBRT did not have pathologic nodal staging, raising the possibility of occult micrometastases. The authors noted that the proportion of SBRT patients with occult lymph node metastases is likely at least equal to that in the surgery group, but these metastases would go undetected without pathologic assessment.

Missing potential occult micrometastases in the SBRT group likely contributed to higher regional recurrence rates over time. By improving nodal staging, more patients with occult lymph node metastases who would be undertreated with SBRT may be identified before treatment, the authors said.

 

What Do Experts Say?

So, is SBRT an option for patients with stage I NSCLC?

Opinions vary.

“If you got one shot for a cure, then you want to do the surgery because that’s what results in a cure,” said Raja Flores, MD, chairman of Thoracic Surgery, Mount Sinai Health System, New York City.

Flores noted that the survival rate with surgery is high in this population. “There’s really nothing out there that can compare,” he said.

In his view, surgery “remains the gold standard.” However, “radiation could be considered in nonsurgical candidates,” he said.

The most recent NCCN guidelines align with Flores’ take. The guidelines say that SBRT is indicated for stage IA-IIA (N0) NSCLC in patients who are deemed “medically inoperable, high surgical risk as determined by thoracic surgeon, and those who decline surgery after thoracic surgical consultation.”

Clifford G. Robinson, MD, agreed. “In the United States, we largely treat patients with SBRT who are medically inoperable or high-risk operable and a much smaller proportion who decline surgery,” said Robinson, professor of radiation oncology and chief of SBRT at Washington University in St. Louis, St. Louis. “Many patients who are deemed operable are not offered SBRT.”

Still, for Robinson, determining which patients are best suited for surgery or SBRT remains unclear.

“Retrospective comparisons are fraught with problems because of confounding,” Robinson told this news organization. “That is, the healthier patients get surgery, and the less healthy ones get SBRT. No manner of fancy statistical manipulation can remove that fact.”

In fact, a recent meta-analysis found that the most significant variable predicting whether surgery or SBRT was superior in retrospective studies was whether the author was a surgeon or radiation oncologist.

Robinson noted that multiple randomized trials have attempted to randomize patients with medically operable early-stage NSCLC to surgery or SBRT and failed to accrue, largely due to patients’ “understandable unwillingness to be randomized between operative vs nonoperative interventions when most already prefer one or the other approach.”

Flores highlighted another point of caution about interpreting trial results: Not all early-stage NSCLC behaves similarly. “Some are slow-growing ‘turtles,’ and others are aggressive ‘rabbits’ — and the turtles are usually the ones that have been included in these radiotherapy trials, and that’s the danger,” he said.

While medical operability is the primary factor for deciding the treatment modality for early-stage NSCLC, there are other more subtle factors that can play into the decision.

These include prior surgery or radiotherapy to the chest, prior cancers, and social issues, such as the patient being a primary caregiver for another person and job insecurity, that might make recovery from surgery more challenging. And in rare instances, a patient may be medically fit to undergo surgery, but the cancer is technically challenging to resect due to anatomic issues or prior surgery to the chest, Robinson added.

 

A Winner?

Results from two ongoing, highly anticipated randomized trials expected in the next several years will hopefully provide additional insights and clarify ongoing uncertainties about the optimal treatment strategies for operable patients with stage I NSCLC.

STABLE-MATES is comparing outcomes after sublobar resection and SBRT in high-risk operable stage I NSCLC, and VALOR is evaluating outcomes after anatomic pulmonary resections and SBRT in patients with stage I NSCLC who have a long life expectancy and are fit enough to tolerate surgery.

But Robinson said his group believes that trying to decide on a winner is a “fool’s errand” and is instead running a pragmatic study across multiple academic and community centers around the United States and Canada where patients choose therapy based on their personal preferences and guidance from their physicians. The researchers will carefully track baseline comorbidity and frailty and assess serial quality-of-life changes over time.

“The goal is to create a calculator that a given patient might use in the future to determine what patients like them would have received, complete with expected outcomes and side effects,” Robinson said.

Robinson cautioned, however, that it “seems unlikely, given the existing literature, that one of the treatments will be truly ‘superior’ to the other one and lead to the ‘losing’ treatment fading away since both are excellent options with pros and cons.”

Aggarwal, Robinson, and Flores had no relevant disclosures.

A version of this article first appeared on Medscape.com.

For years, the default definitive treatment for patients with early-stage I non–small cell lung cancer (NSCLC) has been surgical resection, typically minimally invasive lobectomy with systematic lymph node dissection.

Guidelines from the National Comprehensive Cancer Network (NCCN), the American Society of Clinical Oncology, and the European Society for Medical Oncology all list surgery (in particular, lobectomy) as the primary local therapy for fit, operable patients with stage I NSCLC.

More recently, however, stereotactic body radiotherapy (SBRT), also called stereotactic ablative radiotherapy, has emerged as a definitive treatment option for stage I NSCLC, especially for older, less fit patients who are unsuitable or deemed high-risk for surgery.

“We see patients in our practice who cannot undergo surgery or who may not have adequate lung function to be able to tolerate surgery, and for these patients who are medically inoperable or surgically unresectable, radiation therapy may be a reasonable option,” Charu Aggarwal, MD, MPH, professor and lung cancer specialist, University of Pennsylvania, Philadelphia, told this news organization.

Given some encouraging data suggesting that SBRT could provide similar survival outcomes and be an alternative to surgery for operable disease, SBRT is also increasingly being considered in these early-stage patients. However, other evidence indicates that SBRT may be associated with higher rates of regional and distant recurrences and worse long-term survival, particularly in operable patients.

What may ultimately matter most is carefully selecting operable patients who undergo SBRT.

Aggarwal has encountered certain patients who are fit for surgery but would rather have radiation therapy. “This is an individual decision, and these patients are usually discussed at tumor board and in multidisciplinary discussions to really make sure that they’re making the right decision for themselves,” she explained.

 

The Pros and Cons of SBRT

SBRT is a nonsurgical approach in which precision high-dose radiation is delivered in just a few fractions — typically, 3, 5, or 8, depending on institutional protocols and tumor characteristics.

SBRT is performed on an outpatient basis, usually over 1-2 weeks, with most patients able to resume usual activities with minimal to no delay. Surgery, on the other hand, requires a hospital stay and takes most people about 2-6 weeks to return to regular activities. SBRT also avoids anesthesia and surgical incisions, both of which come with risks.

The data on SBRT in early-stage NSCLC are mixed. While some studies indicate that SBRT comes with promising survival outcomes, other research has reported worse survival and recurrence rates.

One potential reason for higher recurrence rates with SBRT is the lack of pathologic nodal staging, which only happens after surgery, as well as lower rates of nodal evaluation with endobronchial ultrasound or mediastinoscopy before surgery or SBRT. Without nodal assessments, clinicians may miss a more aggressive histology or more advanced nodal stage, which would go undertreated if patients received SBRT.

 

Latest Data in Large Cohort

A recent study published in Lung Cancer indicated that, when carefully selected, operable patients with early NSCLC have comparable survival with lobectomy or SBRT.

In the study, Dutch researchers took an in-depth look at survival and recurrence patterns in a retrospective cohort study of 2183 patients with clinical stage I NSCLC treated with minimally invasive lobectomy or SBRT. The study includes one of the largest cohorts to date, with robust data collection on baseline characteristics, comorbidities, tumor size, performance status, and follow-up.

Patients receiving SBRT were typically older (median age, 74 vs 67 years), had higher comorbidity burdens (Charlson index ≥ 5 in 57% of SBRT patients vs 23% of surgical patients), worse performance status, and lower lung function. To adjust for these differences, the researchers used propensity score weighting so the SBRT group’s baseline characteristics were comparable with those in the surgery group.

The surgery cohort had more invasive nodal evaluation: 21% underwent endobronchial ultrasound or mediastinoscopy vs only 12% in the SBRT group. The vast majority in both groups had PET-CT staging, reflecting modern imaging-based workups.

While 5-year local recurrence rates between the two groups were similar (13.1% for SBRT vs 12.1% for surgery), the 5-year regional recurrence rate was significantly higher after SBRT than lobectomy (18.1% vs 14.2%; hazard ratio [HR], 0.74), as was the distant metastasis rate (26.2% vs 20.2%; HR, 0.72).

Mortality at 30 days was higher after surgery than SBRT (1.0% vs 0.2%). And in the unadjusted analysis, 5-year overall survival was significantly better with lobectomy than SBRT (70.2% vs 40.3%).

However, when the analysis only included patients with similar baseline characteristics, overall survival was no longer significantly different in the two groups (HR, 0.89; 95% CI, 0.65-1.20). Lung cancer–specific mortality was also not significantly different between the two treatments (HR, 1.08), but the study was underpowered to detect significant differences in this outcome on the basis of a relatively low number of deaths from NSCLC.

Still, even after comparing similar patients, recurrence-free survival was notably better with surgery (HR, 0.70), due to fewer regional recurrences and distant metastases. Overall, 13% of the surgical cohort had nodal upstaging at pathology, meaning that even in clinically “node-negative” stage I disease, a subset of patients had unsuspected nodal involvement.

Patients receiving SBRT did not have pathologic nodal staging, raising the possibility of occult micrometastases. The authors noted that the proportion of SBRT patients with occult lymph node metastases is likely at least equal to that in the surgery group, but these metastases would go undetected without pathologic assessment.

Missing potential occult micrometastases in the SBRT group likely contributed to higher regional recurrence rates over time. By improving nodal staging, more patients with occult lymph node metastases who would be undertreated with SBRT may be identified before treatment, the authors said.

 

What Do Experts Say?

So, is SBRT an option for patients with stage I NSCLC?

Opinions vary.

“If you got one shot for a cure, then you want to do the surgery because that’s what results in a cure,” said Raja Flores, MD, chairman of Thoracic Surgery, Mount Sinai Health System, New York City.

Flores noted that the survival rate with surgery is high in this population. “There’s really nothing out there that can compare,” he said.

In his view, surgery “remains the gold standard.” However, “radiation could be considered in nonsurgical candidates,” he said.

The most recent NCCN guidelines align with Flores’ take. The guidelines say that SBRT is indicated for stage IA-IIA (N0) NSCLC in patients who are deemed “medically inoperable, high surgical risk as determined by thoracic surgeon, and those who decline surgery after thoracic surgical consultation.”

Clifford G. Robinson, MD, agreed. “In the United States, we largely treat patients with SBRT who are medically inoperable or high-risk operable and a much smaller proportion who decline surgery,” said Robinson, professor of radiation oncology and chief of SBRT at Washington University in St. Louis, St. Louis. “Many patients who are deemed operable are not offered SBRT.”

Still, for Robinson, determining which patients are best suited for surgery or SBRT remains unclear.

“Retrospective comparisons are fraught with problems because of confounding,” Robinson told this news organization. “That is, the healthier patients get surgery, and the less healthy ones get SBRT. No manner of fancy statistical manipulation can remove that fact.”

In fact, a recent meta-analysis found that the most significant variable predicting whether surgery or SBRT was superior in retrospective studies was whether the author was a surgeon or radiation oncologist.

Robinson noted that multiple randomized trials have attempted to randomize patients with medically operable early-stage NSCLC to surgery or SBRT and failed to accrue, largely due to patients’ “understandable unwillingness to be randomized between operative vs nonoperative interventions when most already prefer one or the other approach.”

Flores highlighted another point of caution about interpreting trial results: Not all early-stage NSCLC behaves similarly. “Some are slow-growing ‘turtles,’ and others are aggressive ‘rabbits’ — and the turtles are usually the ones that have been included in these radiotherapy trials, and that’s the danger,” he said.

While medical operability is the primary factor for deciding the treatment modality for early-stage NSCLC, there are other more subtle factors that can play into the decision.

These include prior surgery or radiotherapy to the chest, prior cancers, and social issues, such as the patient being a primary caregiver for another person and job insecurity, that might make recovery from surgery more challenging. And in rare instances, a patient may be medically fit to undergo surgery, but the cancer is technically challenging to resect due to anatomic issues or prior surgery to the chest, Robinson added.

 

A Winner?

Results from two ongoing, highly anticipated randomized trials expected in the next several years will hopefully provide additional insights and clarify ongoing uncertainties about the optimal treatment strategies for operable patients with stage I NSCLC.

STABLE-MATES is comparing outcomes after sublobar resection and SBRT in high-risk operable stage I NSCLC, and VALOR is evaluating outcomes after anatomic pulmonary resections and SBRT in patients with stage I NSCLC who have a long life expectancy and are fit enough to tolerate surgery.

But Robinson said his group believes that trying to decide on a winner is a “fool’s errand” and is instead running a pragmatic study across multiple academic and community centers around the United States and Canada where patients choose therapy based on their personal preferences and guidance from their physicians. The researchers will carefully track baseline comorbidity and frailty and assess serial quality-of-life changes over time.

“The goal is to create a calculator that a given patient might use in the future to determine what patients like them would have received, complete with expected outcomes and side effects,” Robinson said.

Robinson cautioned, however, that it “seems unlikely, given the existing literature, that one of the treatments will be truly ‘superior’ to the other one and lead to the ‘losing’ treatment fading away since both are excellent options with pros and cons.”

Aggarwal, Robinson, and Flores had no relevant disclosures.

A version of this article first appeared on Medscape.com.

ORLANDO, Fla. — A study of automated three-dimensional total-body photography (3D TBP) found that it improved “hit” rates of positive malignant biopsies and reduced unnecessary biopsies of skin lesions but left unanswered questions about the practicality of its widespread use and cost-effectiveness.

“We did observe improved biopsy practices and outcomes,” said Jordan Phillipps, MD, a dermatology resident at Mayo Clinic in Jacksonville, Florida, who reported the results of the study during a late-breaker session at the American Academy of Dermatology (AAD) 2025 Annual Meeting.

“We observed reduced unnecessary biopsies, which was driven by benign and premalignant, particularly actinic keratosis, lesions,” Phillipps said. “We observed improved malignancy detection, which was profoundly driven by nonmelanoma skin cancers.”

Study Design and Results

The retrospective study included 410 adult patients who had at least two sessions with the Vectra WB360 3D TBP imaging system at a dedicated 3D imaging clinic at Mayo Clinic in Rochester, Minnesota Patient eligibility for the 3D clinic requires a previous melanoma diagnosis. All study participants also underwent dermoscopy, Phillipps said. Their average age was 51.6 years, and 53% were women.

The study accounted for 5981 total patient encounters, including 1150 dedicated Vectra imaging sessions, Phillipps said. In this group, 3006 biopsies were performed, of which 56% were benign, 32% were malignant, and 12% were premalignant. The study also separately evaluated lesion type, focusing on keratinocytic and pigmented lesions.

Most of the keratinocytic lesions were nonmelanoma skin cancers, he said, whereas the pigmented lesions were mostly benign.

“The intervention did significantly reduce biopsies per encounter by 35%, and this was driven by benign lesions and premalignant lesions, particularly actinic keratosis lesions,” Phillipps said.

Previous studies of automated TBP have been hampered by small study populations, he said, and this is one of the largest studies of the Vectra WB360 device. “Nonmelanoma skin cancers are underreported,” Phillipps said, noting that most studies focus on melanoma and pigmented lesions. “Our aim was to assess the effect of Vectra implementation on biopsy practice and outcomes,” he explained.

For malignant lesions, the investigators observed an improvement in malignancy detection, a modest 1.6% increase in hit rates of positive malignant biopsies, and a modest 1.3% decrease in the number needed to biopsy, he said.

A subgroup analysis of pigmented and keratinocytic lesions demonstrated that improved malignancy detection is “profoundly driven by nonmelanoma skin cancers” of 71% per biopsy, Phillipps said, along with “sizable” increases in the hit rate (+17%) and a reduction in the number of biopsies (–14%).

Melanoma detection decreased by 62% per biopsy. Phillipps said the reduction was probably because of the study methodology, specifically the eligibility requirement of having had a previous melanoma diagnosis. “These patients typically develop only one primary melanoma,” Phillipps said. To test this, the investigators compared melanoma hit rates with a matched, unexposed cohort that did not have Vectra imaging. They found that the hit rates were similar. “So this was reassuring that we weren’t missing any melanomas,” Phillipps said.

The results also showed improved efficacy for detecting severely dysplastic nevi, for which the hit rate increased by 16% and the number needed to biopsy decreased by 13%. “Actinic keratoses lesions were biopsied less,” he said, noting a 50% decrease. Both benign keratinocytic lesions, predominantly seborrheic keratosis and benign lichenoid keratosis, and benign pigmented (benign nevi) lesions were biopsied less.

 

Limitations and Questions

The highly selective nature of the patient population was a limitation of the study, Phillipps noted, along with financial and logistical challenges that impede the generalizability of the findings. Overall, he said, the study emphasized that 3D TBP is effective in skin cancer screening and diagnosis, notably beyond pigmented lesions.

Kristina Callis Duffin, MD, MS, chair of dermatology at The University of Utah, Salt Lake City, Utah, called the findings “exciting” but noted that the study did not compare results to the gold standard of clinician-performed skin screenings. “That absolutely would be the important way to do it, through a randomized trial,” she said, “but that’s a hard study to do.”

The cost-effectiveness of total-body imaging also needs to be evaluated, Duffin said. “You really have to look at a number of factors in terms of protection compared to a human gold standard, the rates of biopsies,” she said. “There are a lot of things to unpack; that cost-effectiveness has to be balanced with a more accurate diagnosis and reduction of morbidity with multiple biopsies.” 

Phillipps reported no relevant financial relationships. Duffin disclosed financial relationships with AbbVie, Alumis, Amgen, Boehringer Ingelheim, Bristol-Myers Squibb, Celgene, Eli Lilly, FIDE, Janssen Pharmaceuticals, Novartis, and Pfizer.

A version of this article first appeared on Medscape.com.

ORLANDO, Fla. — A study of automated three-dimensional total-body photography (3D TBP) found that it improved “hit” rates of positive malignant biopsies and reduced unnecessary biopsies of skin lesions but left unanswered questions about the practicality of its widespread use and cost-effectiveness.

“We did observe improved biopsy practices and outcomes,” said Jordan Phillipps, MD, a dermatology resident at Mayo Clinic in Jacksonville, Florida, who reported the results of the study during a late-breaker session at the American Academy of Dermatology (AAD) 2025 Annual Meeting.

“We observed reduced unnecessary biopsies, which was driven by benign and premalignant, particularly actinic keratosis, lesions,” Phillipps said. “We observed improved malignancy detection, which was profoundly driven by nonmelanoma skin cancers.”

Study Design and Results

The retrospective study included 410 adult patients who had at least two sessions with the Vectra WB360 3D TBP imaging system at a dedicated 3D imaging clinic at Mayo Clinic in Rochester, Minnesota Patient eligibility for the 3D clinic requires a previous melanoma diagnosis. All study participants also underwent dermoscopy, Phillipps said. Their average age was 51.6 years, and 53% were women.

The study accounted for 5981 total patient encounters, including 1150 dedicated Vectra imaging sessions, Phillipps said. In this group, 3006 biopsies were performed, of which 56% were benign, 32% were malignant, and 12% were premalignant. The study also separately evaluated lesion type, focusing on keratinocytic and pigmented lesions.

Most of the keratinocytic lesions were nonmelanoma skin cancers, he said, whereas the pigmented lesions were mostly benign.

“The intervention did significantly reduce biopsies per encounter by 35%, and this was driven by benign lesions and premalignant lesions, particularly actinic keratosis lesions,” Phillipps said.

Previous studies of automated TBP have been hampered by small study populations, he said, and this is one of the largest studies of the Vectra WB360 device. “Nonmelanoma skin cancers are underreported,” Phillipps said, noting that most studies focus on melanoma and pigmented lesions. “Our aim was to assess the effect of Vectra implementation on biopsy practice and outcomes,” he explained.

For malignant lesions, the investigators observed an improvement in malignancy detection, a modest 1.6% increase in hit rates of positive malignant biopsies, and a modest 1.3% decrease in the number needed to biopsy, he said.

A subgroup analysis of pigmented and keratinocytic lesions demonstrated that improved malignancy detection is “profoundly driven by nonmelanoma skin cancers” of 71% per biopsy, Phillipps said, along with “sizable” increases in the hit rate (+17%) and a reduction in the number of biopsies (–14%).

Melanoma detection decreased by 62% per biopsy. Phillipps said the reduction was probably because of the study methodology, specifically the eligibility requirement of having had a previous melanoma diagnosis. “These patients typically develop only one primary melanoma,” Phillipps said. To test this, the investigators compared melanoma hit rates with a matched, unexposed cohort that did not have Vectra imaging. They found that the hit rates were similar. “So this was reassuring that we weren’t missing any melanomas,” Phillipps said.

The results also showed improved efficacy for detecting severely dysplastic nevi, for which the hit rate increased by 16% and the number needed to biopsy decreased by 13%. “Actinic keratoses lesions were biopsied less,” he said, noting a 50% decrease. Both benign keratinocytic lesions, predominantly seborrheic keratosis and benign lichenoid keratosis, and benign pigmented (benign nevi) lesions were biopsied less.

 

Limitations and Questions

The highly selective nature of the patient population was a limitation of the study, Phillipps noted, along with financial and logistical challenges that impede the generalizability of the findings. Overall, he said, the study emphasized that 3D TBP is effective in skin cancer screening and diagnosis, notably beyond pigmented lesions.

Kristina Callis Duffin, MD, MS, chair of dermatology at The University of Utah, Salt Lake City, Utah, called the findings “exciting” but noted that the study did not compare results to the gold standard of clinician-performed skin screenings. “That absolutely would be the important way to do it, through a randomized trial,” she said, “but that’s a hard study to do.”

The cost-effectiveness of total-body imaging also needs to be evaluated, Duffin said. “You really have to look at a number of factors in terms of protection compared to a human gold standard, the rates of biopsies,” she said. “There are a lot of things to unpack; that cost-effectiveness has to be balanced with a more accurate diagnosis and reduction of morbidity with multiple biopsies.” 

Phillipps reported no relevant financial relationships. Duffin disclosed financial relationships with AbbVie, Alumis, Amgen, Boehringer Ingelheim, Bristol-Myers Squibb, Celgene, Eli Lilly, FIDE, Janssen Pharmaceuticals, Novartis, and Pfizer.

A version of this article first appeared on Medscape.com.

ORLANDO, Fla. — A study of automated three-dimensional total-body photography (3D TBP) found that it improved “hit” rates of positive malignant biopsies and reduced unnecessary biopsies of skin lesions but left unanswered questions about the practicality of its widespread use and cost-effectiveness.

“We did observe improved biopsy practices and outcomes,” said Jordan Phillipps, MD, a dermatology resident at Mayo Clinic in Jacksonville, Florida, who reported the results of the study during a late-breaker session at the American Academy of Dermatology (AAD) 2025 Annual Meeting.

“We observed reduced unnecessary biopsies, which was driven by benign and premalignant, particularly actinic keratosis, lesions,” Phillipps said. “We observed improved malignancy detection, which was profoundly driven by nonmelanoma skin cancers.”

Study Design and Results

The retrospective study included 410 adult patients who had at least two sessions with the Vectra WB360 3D TBP imaging system at a dedicated 3D imaging clinic at Mayo Clinic in Rochester, Minnesota Patient eligibility for the 3D clinic requires a previous melanoma diagnosis. All study participants also underwent dermoscopy, Phillipps said. Their average age was 51.6 years, and 53% were women.

The study accounted for 5981 total patient encounters, including 1150 dedicated Vectra imaging sessions, Phillipps said. In this group, 3006 biopsies were performed, of which 56% were benign, 32% were malignant, and 12% were premalignant. The study also separately evaluated lesion type, focusing on keratinocytic and pigmented lesions.

Most of the keratinocytic lesions were nonmelanoma skin cancers, he said, whereas the pigmented lesions were mostly benign.

“The intervention did significantly reduce biopsies per encounter by 35%, and this was driven by benign lesions and premalignant lesions, particularly actinic keratosis lesions,” Phillipps said.

Previous studies of automated TBP have been hampered by small study populations, he said, and this is one of the largest studies of the Vectra WB360 device. “Nonmelanoma skin cancers are underreported,” Phillipps said, noting that most studies focus on melanoma and pigmented lesions. “Our aim was to assess the effect of Vectra implementation on biopsy practice and outcomes,” he explained.

For malignant lesions, the investigators observed an improvement in malignancy detection, a modest 1.6% increase in hit rates of positive malignant biopsies, and a modest 1.3% decrease in the number needed to biopsy, he said.

A subgroup analysis of pigmented and keratinocytic lesions demonstrated that improved malignancy detection is “profoundly driven by nonmelanoma skin cancers” of 71% per biopsy, Phillipps said, along with “sizable” increases in the hit rate (+17%) and a reduction in the number of biopsies (–14%).

Melanoma detection decreased by 62% per biopsy. Phillipps said the reduction was probably because of the study methodology, specifically the eligibility requirement of having had a previous melanoma diagnosis. “These patients typically develop only one primary melanoma,” Phillipps said. To test this, the investigators compared melanoma hit rates with a matched, unexposed cohort that did not have Vectra imaging. They found that the hit rates were similar. “So this was reassuring that we weren’t missing any melanomas,” Phillipps said.

The results also showed improved efficacy for detecting severely dysplastic nevi, for which the hit rate increased by 16% and the number needed to biopsy decreased by 13%. “Actinic keratoses lesions were biopsied less,” he said, noting a 50% decrease. Both benign keratinocytic lesions, predominantly seborrheic keratosis and benign lichenoid keratosis, and benign pigmented (benign nevi) lesions were biopsied less.

 

Limitations and Questions

The highly selective nature of the patient population was a limitation of the study, Phillipps noted, along with financial and logistical challenges that impede the generalizability of the findings. Overall, he said, the study emphasized that 3D TBP is effective in skin cancer screening and diagnosis, notably beyond pigmented lesions.

Kristina Callis Duffin, MD, MS, chair of dermatology at The University of Utah, Salt Lake City, Utah, called the findings “exciting” but noted that the study did not compare results to the gold standard of clinician-performed skin screenings. “That absolutely would be the important way to do it, through a randomized trial,” she said, “but that’s a hard study to do.”

The cost-effectiveness of total-body imaging also needs to be evaluated, Duffin said. “You really have to look at a number of factors in terms of protection compared to a human gold standard, the rates of biopsies,” she said. “There are a lot of things to unpack; that cost-effectiveness has to be balanced with a more accurate diagnosis and reduction of morbidity with multiple biopsies.” 

Phillipps reported no relevant financial relationships. Duffin disclosed financial relationships with AbbVie, Alumis, Amgen, Boehringer Ingelheim, Bristol-Myers Squibb, Celgene, Eli Lilly, FIDE, Janssen Pharmaceuticals, Novartis, and Pfizer.

A version of this article first appeared on Medscape.com.

TOPLINE:

One in six patients in phase 2 cancer trials received treatments that were eventually approved by the Food and Drug Administration (FDA), a new analysis found. This proportion increased to 1 in 5 when considering National Comprehensive Cancer Network (NCCN) off-label recommendations and decreased to about 1 in 11 for approved regimens considered to have a substantial clinical benefit.

METHODOLOGY:

• Patients enroll in phase 2 oncology trials seeking access to promising new treatments, but the risk-benefit assessments and the likelihood of receiving a therapy that ultimately gains FDA approval remain unclear. Previous research suggests that the odds are 1 in 83 patients for those enrolled in a phase 1 cancer trial.
• Researchers randomly selected 400 phase 2 cancer trials initiated between November 2012 and November 2015 (to give enough time for an approval to occur); these trials included more than 25,000 patients across 608 specific treatment cohorts testing 332 drugs.
• The primary endpoint was the proportion of patients enrolled in phase 2 trials who received a treatment regimen that later attained FDA approval — defined as the “therapeutic proportion.”
• A secondary endpoint was determining the therapeutic proportion based on the therapeutic value of drugs. The three benchmarks were FDA approval alone, FDA approval plus NCCN off-label recommendations, and FDA approval for drugs considered to have a substantial clinical benefit, based on the European Society for Medical Oncology-Magnitude of Clinical Benefit Scale (ESMO-MCBS).

TAKEAWAY:

• A total of 4045 patients received a treatment regimen that advanced to FDA approval, corresponding to a therapeutic proportion of 16.2%.
• The therapeutic proportion increased to 19.4% when considering NCCN off-label recommendations and decreased to 9.3% for FDA-approved regimens considered to have a substantial clinical benefit, based on the ESMO-MCBS.
• The proportion of patients who participated in a trial in which the drug-indication pairing went on to phase 3 testing was 32.5%.
• Enrollment in a trial featuring biomarker enrichment, an immunotherapy drug, a large phase 2 cohort, and a nonrandomized, industry-sponsored trial all showed a trend toward a higher therapeutic proportion.

IN PRACTICE:

“By entering a phase 2 trial, a patient has a one in six chance of receiving a treatment that will later be approved for their condition,” the authors wrote. “The proportions described here, when juxtaposed with those estimated previously for phase 1 trials, suggest a striking improvement for a patient’s therapeutic prospects. This suggests that phase 1 trials do a good job at protecting patients downstream from unsafe and ineffective cancer treatments.”

In an editorial accompanying the study, Howard S. Hochster, MD, of the Rutgers Cancer Institute in New Brunswick, New Jersey, suggested that the 16.2% therapeutic proportion reported may be understated. For instance, “if using the criterion of drugs that were FDA approved in any indication and dose, the proportion of patient benefit in these trials rises to 38%, with a 51% benefit rate considering inclusion in NCCN guidelines,” he wrote.

 

SOURCE:

This study, led by Charlotte Ouimet, MSc, Department of Equity, Ethics and Policy, McGill University School of Population and Global Health, Montréal, Québec, Canada, was published online in Journal of the National Cancer Institute.

LIMITATIONS:

The longitudinal design of this study required using a historical cohort of phase 2 clinical trials, which may not reflect current drug development patterns. This study was underpowered to determine trial characteristics that predicted higher therapeutic proportions. Furthermore, the exclusion of cytotoxic drugs from the analysis resulted in a somewhat restricted view of overall drug development.

DISCLOSURES:

This study was supported by the Canadian Institutes of Health Research. The authors reported having no relevant conflicts of interest.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

One in six patients in phase 2 cancer trials received treatments that were eventually approved by the Food and Drug Administration (FDA), a new analysis found. This proportion increased to 1 in 5 when considering National Comprehensive Cancer Network (NCCN) off-label recommendations and decreased to about 1 in 11 for approved regimens considered to have a substantial clinical benefit.

METHODOLOGY:

• Patients enroll in phase 2 oncology trials seeking access to promising new treatments, but the risk-benefit assessments and the likelihood of receiving a therapy that ultimately gains FDA approval remain unclear. Previous research suggests that the odds are 1 in 83 patients for those enrolled in a phase 1 cancer trial.
• Researchers randomly selected 400 phase 2 cancer trials initiated between November 2012 and November 2015 (to give enough time for an approval to occur); these trials included more than 25,000 patients across 608 specific treatment cohorts testing 332 drugs.
• The primary endpoint was the proportion of patients enrolled in phase 2 trials who received a treatment regimen that later attained FDA approval — defined as the “therapeutic proportion.”
• A secondary endpoint was determining the therapeutic proportion based on the therapeutic value of drugs. The three benchmarks were FDA approval alone, FDA approval plus NCCN off-label recommendations, and FDA approval for drugs considered to have a substantial clinical benefit, based on the European Society for Medical Oncology-Magnitude of Clinical Benefit Scale (ESMO-MCBS).

TAKEAWAY:

• A total of 4045 patients received a treatment regimen that advanced to FDA approval, corresponding to a therapeutic proportion of 16.2%.
• The therapeutic proportion increased to 19.4% when considering NCCN off-label recommendations and decreased to 9.3% for FDA-approved regimens considered to have a substantial clinical benefit, based on the ESMO-MCBS.
• The proportion of patients who participated in a trial in which the drug-indication pairing went on to phase 3 testing was 32.5%.
• Enrollment in a trial featuring biomarker enrichment, an immunotherapy drug, a large phase 2 cohort, and a nonrandomized, industry-sponsored trial all showed a trend toward a higher therapeutic proportion.

IN PRACTICE:

“By entering a phase 2 trial, a patient has a one in six chance of receiving a treatment that will later be approved for their condition,” the authors wrote. “The proportions described here, when juxtaposed with those estimated previously for phase 1 trials, suggest a striking improvement for a patient’s therapeutic prospects. This suggests that phase 1 trials do a good job at protecting patients downstream from unsafe and ineffective cancer treatments.”

In an editorial accompanying the study, Howard S. Hochster, MD, of the Rutgers Cancer Institute in New Brunswick, New Jersey, suggested that the 16.2% therapeutic proportion reported may be understated. For instance, “if using the criterion of drugs that were FDA approved in any indication and dose, the proportion of patient benefit in these trials rises to 38%, with a 51% benefit rate considering inclusion in NCCN guidelines,” he wrote.

 

SOURCE:

This study, led by Charlotte Ouimet, MSc, Department of Equity, Ethics and Policy, McGill University School of Population and Global Health, Montréal, Québec, Canada, was published online in Journal of the National Cancer Institute.

LIMITATIONS:

The longitudinal design of this study required using a historical cohort of phase 2 clinical trials, which may not reflect current drug development patterns. This study was underpowered to determine trial characteristics that predicted higher therapeutic proportions. Furthermore, the exclusion of cytotoxic drugs from the analysis resulted in a somewhat restricted view of overall drug development.

DISCLOSURES:

This study was supported by the Canadian Institutes of Health Research. The authors reported having no relevant conflicts of interest.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

One in six patients in phase 2 cancer trials received treatments that were eventually approved by the Food and Drug Administration (FDA), a new analysis found. This proportion increased to 1 in 5 when considering National Comprehensive Cancer Network (NCCN) off-label recommendations and decreased to about 1 in 11 for approved regimens considered to have a substantial clinical benefit.

METHODOLOGY:

• Patients enroll in phase 2 oncology trials seeking access to promising new treatments, but the risk-benefit assessments and the likelihood of receiving a therapy that ultimately gains FDA approval remain unclear. Previous research suggests that the odds are 1 in 83 patients for those enrolled in a phase 1 cancer trial.
• Researchers randomly selected 400 phase 2 cancer trials initiated between November 2012 and November 2015 (to give enough time for an approval to occur); these trials included more than 25,000 patients across 608 specific treatment cohorts testing 332 drugs.
• The primary endpoint was the proportion of patients enrolled in phase 2 trials who received a treatment regimen that later attained FDA approval — defined as the “therapeutic proportion.”
• A secondary endpoint was determining the therapeutic proportion based on the therapeutic value of drugs. The three benchmarks were FDA approval alone, FDA approval plus NCCN off-label recommendations, and FDA approval for drugs considered to have a substantial clinical benefit, based on the European Society for Medical Oncology-Magnitude of Clinical Benefit Scale (ESMO-MCBS).

TAKEAWAY:

• A total of 4045 patients received a treatment regimen that advanced to FDA approval, corresponding to a therapeutic proportion of 16.2%.
• The therapeutic proportion increased to 19.4% when considering NCCN off-label recommendations and decreased to 9.3% for FDA-approved regimens considered to have a substantial clinical benefit, based on the ESMO-MCBS.
• The proportion of patients who participated in a trial in which the drug-indication pairing went on to phase 3 testing was 32.5%.
• Enrollment in a trial featuring biomarker enrichment, an immunotherapy drug, a large phase 2 cohort, and a nonrandomized, industry-sponsored trial all showed a trend toward a higher therapeutic proportion.

IN PRACTICE:

“By entering a phase 2 trial, a patient has a one in six chance of receiving a treatment that will later be approved for their condition,” the authors wrote. “The proportions described here, when juxtaposed with those estimated previously for phase 1 trials, suggest a striking improvement for a patient’s therapeutic prospects. This suggests that phase 1 trials do a good job at protecting patients downstream from unsafe and ineffective cancer treatments.”

In an editorial accompanying the study, Howard S. Hochster, MD, of the Rutgers Cancer Institute in New Brunswick, New Jersey, suggested that the 16.2% therapeutic proportion reported may be understated. For instance, “if using the criterion of drugs that were FDA approved in any indication and dose, the proportion of patient benefit in these trials rises to 38%, with a 51% benefit rate considering inclusion in NCCN guidelines,” he wrote.

 

SOURCE:

This study, led by Charlotte Ouimet, MSc, Department of Equity, Ethics and Policy, McGill University School of Population and Global Health, Montréal, Québec, Canada, was published online in Journal of the National Cancer Institute.

LIMITATIONS:

The longitudinal design of this study required using a historical cohort of phase 2 clinical trials, which may not reflect current drug development patterns. This study was underpowered to determine trial characteristics that predicted higher therapeutic proportions. Furthermore, the exclusion of cytotoxic drugs from the analysis resulted in a somewhat restricted view of overall drug development.

DISCLOSURES:

This study was supported by the Canadian Institutes of Health Research. The authors reported having no relevant conflicts of interest.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

Last month, the United States Food and Drug Administration (FDA) approved two new drugs and two biosimilars as well as halted commercialization for a hemophilia treatment.

Here’s a deeper look of what happened last month.

 

New Drugs

1. The FDA has approved mirdametinib (Gomekli, SpringWorks Therapeutics, Inc.) for adult and pediatric patients 2 years or older with neurofibromatosis type 1 and symptomatic plexiform neurofibromas that are not amenable to complete resection.

Approval for this agent was based on overall response rate findings from a multicenter, single-arm, phase 2b trial. The trial, which enrolled 58 adults and 56 pediatric patients with this rare disease, reported confirmed overall response rates of 41% among adults and 52% among children.

Adverse reactions occurring in at least 25% of adults included rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue. Mirdametinib can also cause ocular toxicity. Treatment should be withheld, discontinued, or the dosage reduced based on the severity of these adverse reactions, according to the FDA notice.

2. The FDA has approved vimseltinib (Romvimza, Deciphera Pharmaceuticals, LLC) to treat adult patients with symptomatic tenosynovial giant cell tumors who will not benefit from surgical resection.

Vimseltinib was approved based on findings from the MOTION trial, which included 123 patients randomly assigned 2:1 to vimseltinib 30 mg twice weekly or to placebo for 24 weeks. At 25 weeks, the objective response rate was 40% in the vimseltinib arm and 0% in the placebo arm. The median duration of response was not reached in the vimseltinib arm. Patients receiving vimseltinib also demonstrated significant improvements in active range of motion, physical functioning, and pain at this time. After another 6 months of follow-up, 58% of responders had a duration of response of 9 months or longer.

Treatment-emergent adverse events in MOTION were largely of grade 1 or 2. The most common adverse reactions, occurring in at least 20% of patients, included increased aspartate aminotransferase, periorbital edema, fatigue, rash, and cholesterol.

 

New or Expanded Indications

1. The FDA has approved a supplemental Biologics License Application for brentuximab vedotin (Adcetris, Seagen Inc.), in combination with lenalidomide and rituximab, for adults with relapsed or refractory large B-cell lymphoma, after at least two prior lines of therapy, who are ineligible for stem cell transplant or chimeric antigen receptor T-cell therapy. This includes patients with diffuse large B-cell lymphoma (DLBCL) not otherwise specified, DLBCL arising from indolent lymphoma, or high-grade B-cell lymphoma.

Approval was based on the randomized, double-blind, placebo-controlled ECHELON-3 trial, which randomly assigned patients 1:1 to receive lenalidomide and rituximab plus either brentuximab vedotin or placebo until disease progression or unacceptable toxicity. Researchers reported a median overall survival of 13.8 months in the treatment group vs 8.5 months in the placebo group (hazard ratio, 0.63).

2. The FDA has approved the Biologics License Application for Ospomyv and Xbryk (Samsung Bioepis Co.) — biosimilars referencing denosumab (Prolia and Xgeva, respectively) — to treat osteoporosis and cancer-related bone loss.

Ospomyv and Xbryk have been approved for use in all indications of the approved reference drugs. Specifically, Xbryk is indicated for the prevention of skeletal-related events in patients with bone metastases from solid tumors or multiple myeloma, and Ospomyv is indicated in several populations of patients with osteoporosis at high risk for fracture.

“The FDA approval of Ospomyv and Xbryk marks a key step in improving patient access and alleviating treatment cost for patients with osteoporosis and cancer-related bone loss in the United States,” Byoungin Jung, vice president at Samsung Bioepis, said in the news release.

 

Drug Commercialization Halt

Pfizer announced last month that it will halt the global development and commercialization of its hemophilia gene therapy fidanacogene elaparvovec (Beqvez). The company cited several reasons for the discontinuation, including low demand from patients and doctors.

Beqvez is a one-time therapy approved in the United States last April to treat adults with moderate to severe hemophilia B, a rare bleeding disorder that affects almost 4 in 100,000 men in the United States.

The significant price tag is one reason hematologists have cited for the low uptake. Another barrier is that “we don’t know the long-term outcomes” associated with the drug, pediatric hematologist Ben Samelson-Jones, MD, PhD, of the Perelman School of Medicine at the University of Pennsylvania and Children’s Hospital of Philadelphia, Philadelphia, told this news organization earlier this year.

Other issues include the prospect of newer treatment advances in the hemophilia space and logistical challenges. “There’s just a lot of logistics to getting an institution ready to provide this type of therapy,” Samelson-Jones added.

A version of this article first appeared on Medscape.com.

Last month, the United States Food and Drug Administration (FDA) approved two new drugs and two biosimilars as well as halted commercialization for a hemophilia treatment.

Here’s a deeper look of what happened last month.

 

New Drugs

1. The FDA has approved mirdametinib (Gomekli, SpringWorks Therapeutics, Inc.) for adult and pediatric patients 2 years or older with neurofibromatosis type 1 and symptomatic plexiform neurofibromas that are not amenable to complete resection.

Approval for this agent was based on overall response rate findings from a multicenter, single-arm, phase 2b trial. The trial, which enrolled 58 adults and 56 pediatric patients with this rare disease, reported confirmed overall response rates of 41% among adults and 52% among children.

Adverse reactions occurring in at least 25% of adults included rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue. Mirdametinib can also cause ocular toxicity. Treatment should be withheld, discontinued, or the dosage reduced based on the severity of these adverse reactions, according to the FDA notice.

2. The FDA has approved vimseltinib (Romvimza, Deciphera Pharmaceuticals, LLC) to treat adult patients with symptomatic tenosynovial giant cell tumors who will not benefit from surgical resection.

Vimseltinib was approved based on findings from the MOTION trial, which included 123 patients randomly assigned 2:1 to vimseltinib 30 mg twice weekly or to placebo for 24 weeks. At 25 weeks, the objective response rate was 40% in the vimseltinib arm and 0% in the placebo arm. The median duration of response was not reached in the vimseltinib arm. Patients receiving vimseltinib also demonstrated significant improvements in active range of motion, physical functioning, and pain at this time. After another 6 months of follow-up, 58% of responders had a duration of response of 9 months or longer.

Treatment-emergent adverse events in MOTION were largely of grade 1 or 2. The most common adverse reactions, occurring in at least 20% of patients, included increased aspartate aminotransferase, periorbital edema, fatigue, rash, and cholesterol.

 

New or Expanded Indications

1. The FDA has approved a supplemental Biologics License Application for brentuximab vedotin (Adcetris, Seagen Inc.), in combination with lenalidomide and rituximab, for adults with relapsed or refractory large B-cell lymphoma, after at least two prior lines of therapy, who are ineligible for stem cell transplant or chimeric antigen receptor T-cell therapy. This includes patients with diffuse large B-cell lymphoma (DLBCL) not otherwise specified, DLBCL arising from indolent lymphoma, or high-grade B-cell lymphoma.

Approval was based on the randomized, double-blind, placebo-controlled ECHELON-3 trial, which randomly assigned patients 1:1 to receive lenalidomide and rituximab plus either brentuximab vedotin or placebo until disease progression or unacceptable toxicity. Researchers reported a median overall survival of 13.8 months in the treatment group vs 8.5 months in the placebo group (hazard ratio, 0.63).

2. The FDA has approved the Biologics License Application for Ospomyv and Xbryk (Samsung Bioepis Co.) — biosimilars referencing denosumab (Prolia and Xgeva, respectively) — to treat osteoporosis and cancer-related bone loss.

Ospomyv and Xbryk have been approved for use in all indications of the approved reference drugs. Specifically, Xbryk is indicated for the prevention of skeletal-related events in patients with bone metastases from solid tumors or multiple myeloma, and Ospomyv is indicated in several populations of patients with osteoporosis at high risk for fracture.

“The FDA approval of Ospomyv and Xbryk marks a key step in improving patient access and alleviating treatment cost for patients with osteoporosis and cancer-related bone loss in the United States,” Byoungin Jung, vice president at Samsung Bioepis, said in the news release.

 

Drug Commercialization Halt

Pfizer announced last month that it will halt the global development and commercialization of its hemophilia gene therapy fidanacogene elaparvovec (Beqvez). The company cited several reasons for the discontinuation, including low demand from patients and doctors.

Beqvez is a one-time therapy approved in the United States last April to treat adults with moderate to severe hemophilia B, a rare bleeding disorder that affects almost 4 in 100,000 men in the United States.

The significant price tag is one reason hematologists have cited for the low uptake. Another barrier is that “we don’t know the long-term outcomes” associated with the drug, pediatric hematologist Ben Samelson-Jones, MD, PhD, of the Perelman School of Medicine at the University of Pennsylvania and Children’s Hospital of Philadelphia, Philadelphia, told this news organization earlier this year.

Other issues include the prospect of newer treatment advances in the hemophilia space and logistical challenges. “There’s just a lot of logistics to getting an institution ready to provide this type of therapy,” Samelson-Jones added.

A version of this article first appeared on Medscape.com.

Last month, the United States Food and Drug Administration (FDA) approved two new drugs and two biosimilars as well as halted commercialization for a hemophilia treatment.

Here’s a deeper look of what happened last month.

 

New Drugs

1. The FDA has approved mirdametinib (Gomekli, SpringWorks Therapeutics, Inc.) for adult and pediatric patients 2 years or older with neurofibromatosis type 1 and symptomatic plexiform neurofibromas that are not amenable to complete resection.

Approval for this agent was based on overall response rate findings from a multicenter, single-arm, phase 2b trial. The trial, which enrolled 58 adults and 56 pediatric patients with this rare disease, reported confirmed overall response rates of 41% among adults and 52% among children.

Adverse reactions occurring in at least 25% of adults included rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue. Mirdametinib can also cause ocular toxicity. Treatment should be withheld, discontinued, or the dosage reduced based on the severity of these adverse reactions, according to the FDA notice.

2. The FDA has approved vimseltinib (Romvimza, Deciphera Pharmaceuticals, LLC) to treat adult patients with symptomatic tenosynovial giant cell tumors who will not benefit from surgical resection.

Vimseltinib was approved based on findings from the MOTION trial, which included 123 patients randomly assigned 2:1 to vimseltinib 30 mg twice weekly or to placebo for 24 weeks. At 25 weeks, the objective response rate was 40% in the vimseltinib arm and 0% in the placebo arm. The median duration of response was not reached in the vimseltinib arm. Patients receiving vimseltinib also demonstrated significant improvements in active range of motion, physical functioning, and pain at this time. After another 6 months of follow-up, 58% of responders had a duration of response of 9 months or longer.

Treatment-emergent adverse events in MOTION were largely of grade 1 or 2. The most common adverse reactions, occurring in at least 20% of patients, included increased aspartate aminotransferase, periorbital edema, fatigue, rash, and cholesterol.

 

New or Expanded Indications

1. The FDA has approved a supplemental Biologics License Application for brentuximab vedotin (Adcetris, Seagen Inc.), in combination with lenalidomide and rituximab, for adults with relapsed or refractory large B-cell lymphoma, after at least two prior lines of therapy, who are ineligible for stem cell transplant or chimeric antigen receptor T-cell therapy. This includes patients with diffuse large B-cell lymphoma (DLBCL) not otherwise specified, DLBCL arising from indolent lymphoma, or high-grade B-cell lymphoma.

Approval was based on the randomized, double-blind, placebo-controlled ECHELON-3 trial, which randomly assigned patients 1:1 to receive lenalidomide and rituximab plus either brentuximab vedotin or placebo until disease progression or unacceptable toxicity. Researchers reported a median overall survival of 13.8 months in the treatment group vs 8.5 months in the placebo group (hazard ratio, 0.63).

2. The FDA has approved the Biologics License Application for Ospomyv and Xbryk (Samsung Bioepis Co.) — biosimilars referencing denosumab (Prolia and Xgeva, respectively) — to treat osteoporosis and cancer-related bone loss.

Ospomyv and Xbryk have been approved for use in all indications of the approved reference drugs. Specifically, Xbryk is indicated for the prevention of skeletal-related events in patients with bone metastases from solid tumors or multiple myeloma, and Ospomyv is indicated in several populations of patients with osteoporosis at high risk for fracture.

“The FDA approval of Ospomyv and Xbryk marks a key step in improving patient access and alleviating treatment cost for patients with osteoporosis and cancer-related bone loss in the United States,” Byoungin Jung, vice president at Samsung Bioepis, said in the news release.

 

Drug Commercialization Halt

Pfizer announced last month that it will halt the global development and commercialization of its hemophilia gene therapy fidanacogene elaparvovec (Beqvez). The company cited several reasons for the discontinuation, including low demand from patients and doctors.

Beqvez is a one-time therapy approved in the United States last April to treat adults with moderate to severe hemophilia B, a rare bleeding disorder that affects almost 4 in 100,000 men in the United States.

The significant price tag is one reason hematologists have cited for the low uptake. Another barrier is that “we don’t know the long-term outcomes” associated with the drug, pediatric hematologist Ben Samelson-Jones, MD, PhD, of the Perelman School of Medicine at the University of Pennsylvania and Children’s Hospital of Philadelphia, Philadelphia, told this news organization earlier this year.

Other issues include the prospect of newer treatment advances in the hemophilia space and logistical challenges. “There’s just a lot of logistics to getting an institution ready to provide this type of therapy,” Samelson-Jones added.

A version of this article first appeared on Medscape.com.