Cathy Kelly

Blue Cross Blue Shield of Massachusetts is implementing what it says are industry-leading policies to restrain the abuse of opioid medications among its members, including a requirement that new opioid drug prescriptions written for quantities of longer than 30 days must be accompanied by a medical authorization before coverage is approved. The new policies become effective July 1.

BCBS Massachusetts said in a statement that it developed the new policies after an internal review showed that 30,000 of its members received prescriptions for short-acting pain killers lasting longer than 30 days, "a practice that many experts believe increases the chances of drug misuse, dependency, and diversion."

Massachusetts has seen a significant increase in opioid-related deaths and hospital stays over the past decade mainly due to heroin use, but in part because of the increased availability, misuse, and abuse of prescription pain killers, according to the health plan.

The new policies also mandate that all prescriptions for a short-acting opioid be obtained from just one prescriber or prescribing group and that scripts must be filled at one designated pharmacy or pharmacy chain. The restrictions appear to go further than those set by other private insurers, which first determine if a potential problem exists through claim reviews before restricting members’ access to opioid coverage.

The health plan also will require written agreements between the prescriber and the patient that address prescription management, diversion, the use of other substances while taking pain medication, and informed consent regarding the risks involved in taking prescription pain killers. Physicians and other prescribers also will be required to make a formal assessment of addiction risk and to develop treatment plans that include a "clear diagnosis, explicit goals and exploration of other treatment options for pain."

For long-acting opioids, the plan said it will apply new guidelines in approving initial prescriptions. Special accommodations, however, will be made for patients on chronic medications, those with cancer, and those who are at the end of life.

In curtailing member access to opioids, the actions by BCBS Massachusetts are in line with 2011 recommendations from the Government Accountability Office aimed at the Medicare Part D program. The GAO report also noted the growing incidence of doctor shopping, in which beneficiaries were obtaining more drugs than were medically necessary and urged Medicare to limit patients to a single physician and single pharmacy.

When called to testify before a Senate panel regarding the GAO report, officials from the Centers for Medicare and Medicaid Services cautioned that the GAO recommendations also might prevent appropriate medical use. They suggested better use of existing programs such as drug utilization review might address the situation – and pointed out that current Medicare statutes bar the kind of limitations suggested by the GAO.

Editor’s note: This story appears courtesy of "The Pink Sheet," a weekly Elsevier publication covering pharmaceutical business and policy issues. To learn more, contact customer care at 800-332-2181 or sign up for a free trial.

Blue Cross Blue Shield of Massachusetts is implementing what it says are industry-leading policies to restrain the abuse of opioid medications among its members, including a requirement that new opioid drug prescriptions written for quantities of longer than 30 days must be accompanied by a medical authorization before coverage is approved. The new policies become effective July 1.

BCBS Massachusetts said in a statement that it developed the new policies after an internal review showed that 30,000 of its members received prescriptions for short-acting pain killers lasting longer than 30 days, "a practice that many experts believe increases the chances of drug misuse, dependency, and diversion."

Massachusetts has seen a significant increase in opioid-related deaths and hospital stays over the past decade mainly due to heroin use, but in part because of the increased availability, misuse, and abuse of prescription pain killers, according to the health plan.

The new policies also mandate that all prescriptions for a short-acting opioid be obtained from just one prescriber or prescribing group and that scripts must be filled at one designated pharmacy or pharmacy chain. The restrictions appear to go further than those set by other private insurers, which first determine if a potential problem exists through claim reviews before restricting members’ access to opioid coverage.

The health plan also will require written agreements between the prescriber and the patient that address prescription management, diversion, the use of other substances while taking pain medication, and informed consent regarding the risks involved in taking prescription pain killers. Physicians and other prescribers also will be required to make a formal assessment of addiction risk and to develop treatment plans that include a "clear diagnosis, explicit goals and exploration of other treatment options for pain."

For long-acting opioids, the plan said it will apply new guidelines in approving initial prescriptions. Special accommodations, however, will be made for patients on chronic medications, those with cancer, and those who are at the end of life.

In curtailing member access to opioids, the actions by BCBS Massachusetts are in line with 2011 recommendations from the Government Accountability Office aimed at the Medicare Part D program. The GAO report also noted the growing incidence of doctor shopping, in which beneficiaries were obtaining more drugs than were medically necessary and urged Medicare to limit patients to a single physician and single pharmacy.

When called to testify before a Senate panel regarding the GAO report, officials from the Centers for Medicare and Medicaid Services cautioned that the GAO recommendations also might prevent appropriate medical use. They suggested better use of existing programs such as drug utilization review might address the situation – and pointed out that current Medicare statutes bar the kind of limitations suggested by the GAO.

Editor’s note: This story appears courtesy of "The Pink Sheet," a weekly Elsevier publication covering pharmaceutical business and policy issues. To learn more, contact customer care at 800-332-2181 or sign up for a free trial.

Blue Cross Blue Shield of Massachusetts is implementing what it says are industry-leading policies to restrain the abuse of opioid medications among its members, including a requirement that new opioid drug prescriptions written for quantities of longer than 30 days must be accompanied by a medical authorization before coverage is approved. The new policies become effective July 1.

BCBS Massachusetts said in a statement that it developed the new policies after an internal review showed that 30,000 of its members received prescriptions for short-acting pain killers lasting longer than 30 days, "a practice that many experts believe increases the chances of drug misuse, dependency, and diversion."

Massachusetts has seen a significant increase in opioid-related deaths and hospital stays over the past decade mainly due to heroin use, but in part because of the increased availability, misuse, and abuse of prescription pain killers, according to the health plan.

The new policies also mandate that all prescriptions for a short-acting opioid be obtained from just one prescriber or prescribing group and that scripts must be filled at one designated pharmacy or pharmacy chain. The restrictions appear to go further than those set by other private insurers, which first determine if a potential problem exists through claim reviews before restricting members’ access to opioid coverage.

The health plan also will require written agreements between the prescriber and the patient that address prescription management, diversion, the use of other substances while taking pain medication, and informed consent regarding the risks involved in taking prescription pain killers. Physicians and other prescribers also will be required to make a formal assessment of addiction risk and to develop treatment plans that include a "clear diagnosis, explicit goals and exploration of other treatment options for pain."

For long-acting opioids, the plan said it will apply new guidelines in approving initial prescriptions. Special accommodations, however, will be made for patients on chronic medications, those with cancer, and those who are at the end of life.

In curtailing member access to opioids, the actions by BCBS Massachusetts are in line with 2011 recommendations from the Government Accountability Office aimed at the Medicare Part D program. The GAO report also noted the growing incidence of doctor shopping, in which beneficiaries were obtaining more drugs than were medically necessary and urged Medicare to limit patients to a single physician and single pharmacy.

When called to testify before a Senate panel regarding the GAO report, officials from the Centers for Medicare and Medicaid Services cautioned that the GAO recommendations also might prevent appropriate medical use. They suggested better use of existing programs such as drug utilization review might address the situation – and pointed out that current Medicare statutes bar the kind of limitations suggested by the GAO.

Editor’s note: This story appears courtesy of "The Pink Sheet," a weekly Elsevier publication covering pharmaceutical business and policy issues. To learn more, contact customer care at 800-332-2181 or sign up for a free trial.

The Centers for Medicare and Medicaid Services will keep the status quo for now regarding Medicare Part B coverage for Roche/Genentech’s Avastin (bevacizumab) in metastatic breast cancer despite the Food and Drug Administration’s decision to formally withdraw approval for the indication, according to a CMS spokesman.

"Medicare will continue to cover Avastin. CMS will monitor the issue and evaluate coverage options as a result of action by the FDA but has no immediate plans to change coverage policies," the spokesman said in an e-mail. The FDA announced its final conclusion Nov. 18, maintaining that the safety risks associated with Avastin are not outweighed by any benefit in metastatic breast cancer (MBC).

But it also means there is a chance that local Medicare contractors may decide to deny coverage for the patients whose claims they manage now that the indication is officially off label. Administrative claims contractors are allowed to use their discretion in coverage decisions when CMS has no formal national reimbursement policy in place.

Last January, one of the largest contractors, Palmetto GBA, moved to drop coverage for Avastin in MBC but then quickly reversed itself, stating it would await a final decision by FDA before acting. At the time, Palmetto said it would continue to review relevant clinical trials and literature on the effectiveness of the drug in breast cancer and, if it determines a coverage policy change is warranted, would initiate a formal notice-and-comment process before any final decision is made.

Medicare contractors, like other payers, typically rely on medical policy guidelines in cases where a treatment is off label. One of the more influential sets of guidelines, the National Comprehensive Cancer Network Clinical Practice Guidelines for Oncology, recently reaffirmed its support for the treatment of Avastin in MBC.

The guidelines recommend that "bevacizumab in combination with paclitaxel is an appropriate therapeutic option for metastatic breast cancer with the evidence designation 2A," which means that "based upon lower-level evidence, there is uniform NCCN consensus that the intervention is appropriate."

In the private sector, a small but growing number of regional plans previously announced plans to drop coverage for Avastin in MBC, including Blue Shield of California.

[Poll: Tell us what you think of the Avastin decision.]

This coverage is provided courtesy of "The Pink Sheet." This news organization and "The Pink Sheet" are owned by Elsevier.

The Centers for Medicare and Medicaid Services will keep the status quo for now regarding Medicare Part B coverage for Roche/Genentech’s Avastin (bevacizumab) in metastatic breast cancer despite the Food and Drug Administration’s decision to formally withdraw approval for the indication, according to a CMS spokesman.

"Medicare will continue to cover Avastin. CMS will monitor the issue and evaluate coverage options as a result of action by the FDA but has no immediate plans to change coverage policies," the spokesman said in an e-mail. The FDA announced its final conclusion Nov. 18, maintaining that the safety risks associated with Avastin are not outweighed by any benefit in metastatic breast cancer (MBC).

But it also means there is a chance that local Medicare contractors may decide to deny coverage for the patients whose claims they manage now that the indication is officially off label. Administrative claims contractors are allowed to use their discretion in coverage decisions when CMS has no formal national reimbursement policy in place.

Last January, one of the largest contractors, Palmetto GBA, moved to drop coverage for Avastin in MBC but then quickly reversed itself, stating it would await a final decision by FDA before acting. At the time, Palmetto said it would continue to review relevant clinical trials and literature on the effectiveness of the drug in breast cancer and, if it determines a coverage policy change is warranted, would initiate a formal notice-and-comment process before any final decision is made.

Medicare contractors, like other payers, typically rely on medical policy guidelines in cases where a treatment is off label. One of the more influential sets of guidelines, the National Comprehensive Cancer Network Clinical Practice Guidelines for Oncology, recently reaffirmed its support for the treatment of Avastin in MBC.

The guidelines recommend that "bevacizumab in combination with paclitaxel is an appropriate therapeutic option for metastatic breast cancer with the evidence designation 2A," which means that "based upon lower-level evidence, there is uniform NCCN consensus that the intervention is appropriate."

In the private sector, a small but growing number of regional plans previously announced plans to drop coverage for Avastin in MBC, including Blue Shield of California.

[Poll: Tell us what you think of the Avastin decision.]

This coverage is provided courtesy of "The Pink Sheet." This news organization and "The Pink Sheet" are owned by Elsevier.

The Centers for Medicare and Medicaid Services will keep the status quo for now regarding Medicare Part B coverage for Roche/Genentech’s Avastin (bevacizumab) in metastatic breast cancer despite the Food and Drug Administration’s decision to formally withdraw approval for the indication, according to a CMS spokesman.

"Medicare will continue to cover Avastin. CMS will monitor the issue and evaluate coverage options as a result of action by the FDA but has no immediate plans to change coverage policies," the spokesman said in an e-mail. The FDA announced its final conclusion Nov. 18, maintaining that the safety risks associated with Avastin are not outweighed by any benefit in metastatic breast cancer (MBC).

But it also means there is a chance that local Medicare contractors may decide to deny coverage for the patients whose claims they manage now that the indication is officially off label. Administrative claims contractors are allowed to use their discretion in coverage decisions when CMS has no formal national reimbursement policy in place.

Last January, one of the largest contractors, Palmetto GBA, moved to drop coverage for Avastin in MBC but then quickly reversed itself, stating it would await a final decision by FDA before acting. At the time, Palmetto said it would continue to review relevant clinical trials and literature on the effectiveness of the drug in breast cancer and, if it determines a coverage policy change is warranted, would initiate a formal notice-and-comment process before any final decision is made.

Medicare contractors, like other payers, typically rely on medical policy guidelines in cases where a treatment is off label. One of the more influential sets of guidelines, the National Comprehensive Cancer Network Clinical Practice Guidelines for Oncology, recently reaffirmed its support for the treatment of Avastin in MBC.

The guidelines recommend that "bevacizumab in combination with paclitaxel is an appropriate therapeutic option for metastatic breast cancer with the evidence designation 2A," which means that "based upon lower-level evidence, there is uniform NCCN consensus that the intervention is appropriate."

In the private sector, a small but growing number of regional plans previously announced plans to drop coverage for Avastin in MBC, including Blue Shield of California.

[Poll: Tell us what you think of the Avastin decision.]

This coverage is provided courtesy of "The Pink Sheet." This news organization and "The Pink Sheet" are owned by Elsevier.

The Centers for Medicare & Medicaid Services (CMS) should look for ways to ensure that financial incentives for Medicare accountable care organizations (ACOs) do not promote inappropriate shifts in prescribing from the Part B benefit to Part D, urged clinician and biotechnology groups in comments filed with the agency on June 6, 2011.

The Part B benefit covers drugs administered in a physician’s office, such as infused products. It excludes most oral and other medications that patients obtain at retail or mail-order pharmacies for use at home; these medications instead are covered under Part D. So in theory, the ACO program will encompass Part B expenditures, but not Part D. ACOs could save on the cost of drugs by shifting some prescriptions to Part D.

The comments respond to the proposed rule to establish ACOs that the CMS published on March 31, 2011. ACOs are part of a “shared savings” initiative, mandated by the Affordable Care Act, aimed at driving providers through financial incentives to better manage and coordinate healthcare delivery to improve quality and reduce costs. ACOs are slated to begin operating January 1, 2012. Under the proposed regulation, ACOs would be made up of physician practices, hospitals, and other healthcare providers. Primary care physicians are expected to serve as coordinators of individual patient care. The proposal posal does not specifically discuss potential shifts in prescribing. However, the Biotechnology Industry Organization (BIO) and others have urged the CMS to develop a process to ensure that patient care is not secondary to the pursuit of savings.

The implications of prescribing shifts

The BIO points out that the Congressional Research Service (CRS) identified prescribing shifts as a possible issue in an April 25 report on ACOs and the Medicare shared savings program. Such shifts might occur because the savings calculations used to evaluate ACOs are based on Parts A and B expenditures only, suggests the CRS, an analytic unit employed by Congress. Thus, “there may be instances where there is the appearance of cost savings as a result of providers unduly relying on Part D prescription medicines over other forms of care,” the CRS cautioned. The BIO added, “this issue may present problems from both the perspective of quality patient care as well as for Medicare program expenditures.” A shift to Part D drugs could actually increase patient outof- pocket costs, “which in turn may impact prescription drug adherence and ultimately clinical outcomes,” the comments stated.

The prospect of increasing patient costs under Part D is supported by a CMS-commissioned report released in August 2010. The report looked at the potential impact of consolidating Medicare reimbursement for drug categories with overlapping Parts B and D coverage. It found, on average, that patients would face higher costsharing if categories are consolidated under Part D, but that the Medicare program would save money, because Part D has less generous coverage rules. The study looked at oral anticancer and antiemetic drugs, insulin, vaccinations, inhalants, and rheumatoid arthritis medications.

In separate comments, the American Society of Clinical Oncology (ASCO) also targeted potential prescribing shifts in cancer therapies. “The CMS should address perverse incentives that will arise due to the proposed rule’s exclusion of Part D drugs from the costs by which ACO savings are measured,” ASCO warned. “Safeguards are needed to ensure that cancer patients receive the most appropriate therapy, regardless of whether that therapy is typically covered under Part B or under Part D.” (In addition to chemotherapy administered in a physician’s office, some oral anticancer drugs are also covered under Part B when they are direct substitutions for physician-administered treatments.)

Even the medical device and diagnostics industry registered concern with possible prescribing shifts to Part D drugs. The Advanced Medical Technology Association pointed out that providers might have an artificial incentive to substitute a pain medication, for example, when a surgical procedure covered under Part A or B would be considered standard of care.

Carving out the cost of new technologies

A number of stakeholders flagged the prospect that cost issues might also deter the adoption of cuttingedge drugs or other medical technologies by ACOs. The BIO and the Medical Device Manufacturers Association are among the groups urging CMS to consider ways to carve out new technologies from the benchmark for ACO costs and from annual expenditure performance reviews.

As proposed, the ACO program would be risk-based: an organization would receive a bonus payment if cost and quality benchmarks are met but would also face penalties if they are not. As a result, providers may be reluctant to adopt expensive new technologies, based on the comments.

To ensure beneficiary access to new therapies and to retain incentives for innovation, the stakeholders have suggested that the CMS adapt the Medicare payment mechanisms already used for new technologies in the hospital setting. Medicare’s hospital payment scheme includes mechanisms for add-on (inpatient) or pass-through (outpatient) payments. They provide payment for new and relatively expensive treatments in their first few years on the market in addition to the reimbursement rate set for their therapeutic category. The system is designed to account for the fact that the CMS does not yet know with certainty how much the new treatment will add to hospital charges and costs. And the extra reimbursement is intended to make it more likely that hospitals can afford to “try out” new technologies while the CMS collects the data on cost and use patterns that will ultimately allow it to develop a more permanent payment level.

The BIO has suggested that the carveout should apply regardless of the healthcare setting and should include drugs and biologics provided in the physician’s office. “Such congruity is necessary to ensure that the policy does not create an incentive to perform procedures in the hospital rather than the physician’s office,” according to the comments.

Quality metrics and warfarin

In comments on other aspects of the proposal, the BIO has recommended that the CMS should establish a process to rapidly update the quality measures used to evaluate ACO performance. The proposal outlines a number of quality measures, including whether patients at risk of serious disease or frail elderly patients are taking drugs for managing chronic disease. The agency also will track patient measurements, such as cholesterol levels and hemoglobin A1c values, to monitor the effectiveness of disease management. The quality measures are expected to improve adherence to drugs for chronic disease.

Nevertheless, the BIO takes issue with the quality metric assessing adherence to warfarin therapy in patients with heart failure and paroxysmal or chronic atrial fibrillation; the concern is that it could discourage the use of newer agents. “This [warfarin] measure already is outdated,” the BIO stated. “There are more advanced therapeutic options available for these patients today as well as additional therapies in development. Leaving the measure in place may force ACOs to use a therapy that is no longer the only clinically appropriate, or even recommended, choice.”

The latest newcomer to the category is dabigatran (Pradaxa). Two other oral anticoagulants are in late-stage development: rivaroxaban (Xarelto) and apixaban. The biotech group’s concerns reflect the tension in the anticoagulant market segment among developers of newer agents and payer concerns over whether the advantages offered by such drugs can justify their increased cost.

A more prominent role for specialists

The ACO proposal is designed to emphasize the role of primary care providers. However, the BIO has advised that the CMS also ensure that specialists play an important part in the operation of ACOs, such as during the development of clinical guidelines and processes. Under the proposed rule, ACOs would be required to “develop and implement evidence-based medical practice or clinical guidelines and processes for delivering care consistent with the goals of better care for individuals, better health for populations, and lower growth in expenditures.”

ASCO echoed the BIO recommendation. “Given the prevalence of cancer in the Medicare community … [the] CMS should require ACOs to secure substantial input from specialists practicing in the community.” ASCO also requested that the CMS ensure that oncology practices do not face undue financial or procedural challenges under the ACO program.

“Oncologists already face a significant form of financial risk arising from the need to purchase and administer chemotherapy drugs within an environment in which Medicare and other health insurers often provide ambiguous coverage policies, payment rates that are inadequate to cover acquisition costs and slow preauthorization decisions,” ASCO stated. Furthermore, ASCO noted that Medicare contractors frequently require “excessive documentation” on the effectiveness of off-label drug indications, even when the indication is supported in the peer-reviewed literature.

The Centers for Medicare & Medicaid Services (CMS) should look for ways to ensure that financial incentives for Medicare accountable care organizations (ACOs) do not promote inappropriate shifts in prescribing from the Part B benefit to Part D, urged clinician and biotechnology groups in comments filed with the agency on June 6, 2011.

The Part B benefit covers drugs administered in a physician’s office, such as infused products. It excludes most oral and other medications that patients obtain at retail or mail-order pharmacies for use at home; these medications instead are covered under Part D. So in theory, the ACO program will encompass Part B expenditures, but not Part D. ACOs could save on the cost of drugs by shifting some prescriptions to Part D.

The comments respond to the proposed rule to establish ACOs that the CMS published on March 31, 2011. ACOs are part of a “shared savings” initiative, mandated by the Affordable Care Act, aimed at driving providers through financial incentives to better manage and coordinate healthcare delivery to improve quality and reduce costs. ACOs are slated to begin operating January 1, 2012. Under the proposed regulation, ACOs would be made up of physician practices, hospitals, and other healthcare providers. Primary care physicians are expected to serve as coordinators of individual patient care. The proposal posal does not specifically discuss potential shifts in prescribing. However, the Biotechnology Industry Organization (BIO) and others have urged the CMS to develop a process to ensure that patient care is not secondary to the pursuit of savings.

The implications of prescribing shifts

The BIO points out that the Congressional Research Service (CRS) identified prescribing shifts as a possible issue in an April 25 report on ACOs and the Medicare shared savings program. Such shifts might occur because the savings calculations used to evaluate ACOs are based on Parts A and B expenditures only, suggests the CRS, an analytic unit employed by Congress. Thus, “there may be instances where there is the appearance of cost savings as a result of providers unduly relying on Part D prescription medicines over other forms of care,” the CRS cautioned. The BIO added, “this issue may present problems from both the perspective of quality patient care as well as for Medicare program expenditures.” A shift to Part D drugs could actually increase patient outof- pocket costs, “which in turn may impact prescription drug adherence and ultimately clinical outcomes,” the comments stated.

The prospect of increasing patient costs under Part D is supported by a CMS-commissioned report released in August 2010. The report looked at the potential impact of consolidating Medicare reimbursement for drug categories with overlapping Parts B and D coverage. It found, on average, that patients would face higher costsharing if categories are consolidated under Part D, but that the Medicare program would save money, because Part D has less generous coverage rules. The study looked at oral anticancer and antiemetic drugs, insulin, vaccinations, inhalants, and rheumatoid arthritis medications.

In separate comments, the American Society of Clinical Oncology (ASCO) also targeted potential prescribing shifts in cancer therapies. “The CMS should address perverse incentives that will arise due to the proposed rule’s exclusion of Part D drugs from the costs by which ACO savings are measured,” ASCO warned. “Safeguards are needed to ensure that cancer patients receive the most appropriate therapy, regardless of whether that therapy is typically covered under Part B or under Part D.” (In addition to chemotherapy administered in a physician’s office, some oral anticancer drugs are also covered under Part B when they are direct substitutions for physician-administered treatments.)

Even the medical device and diagnostics industry registered concern with possible prescribing shifts to Part D drugs. The Advanced Medical Technology Association pointed out that providers might have an artificial incentive to substitute a pain medication, for example, when a surgical procedure covered under Part A or B would be considered standard of care.

Carving out the cost of new technologies

A number of stakeholders flagged the prospect that cost issues might also deter the adoption of cuttingedge drugs or other medical technologies by ACOs. The BIO and the Medical Device Manufacturers Association are among the groups urging CMS to consider ways to carve out new technologies from the benchmark for ACO costs and from annual expenditure performance reviews.

As proposed, the ACO program would be risk-based: an organization would receive a bonus payment if cost and quality benchmarks are met but would also face penalties if they are not. As a result, providers may be reluctant to adopt expensive new technologies, based on the comments.

To ensure beneficiary access to new therapies and to retain incentives for innovation, the stakeholders have suggested that the CMS adapt the Medicare payment mechanisms already used for new technologies in the hospital setting. Medicare’s hospital payment scheme includes mechanisms for add-on (inpatient) or pass-through (outpatient) payments. They provide payment for new and relatively expensive treatments in their first few years on the market in addition to the reimbursement rate set for their therapeutic category. The system is designed to account for the fact that the CMS does not yet know with certainty how much the new treatment will add to hospital charges and costs. And the extra reimbursement is intended to make it more likely that hospitals can afford to “try out” new technologies while the CMS collects the data on cost and use patterns that will ultimately allow it to develop a more permanent payment level.

The BIO has suggested that the carveout should apply regardless of the healthcare setting and should include drugs and biologics provided in the physician’s office. “Such congruity is necessary to ensure that the policy does not create an incentive to perform procedures in the hospital rather than the physician’s office,” according to the comments.

Quality metrics and warfarin

In comments on other aspects of the proposal, the BIO has recommended that the CMS should establish a process to rapidly update the quality measures used to evaluate ACO performance. The proposal outlines a number of quality measures, including whether patients at risk of serious disease or frail elderly patients are taking drugs for managing chronic disease. The agency also will track patient measurements, such as cholesterol levels and hemoglobin A1c values, to monitor the effectiveness of disease management. The quality measures are expected to improve adherence to drugs for chronic disease.

Nevertheless, the BIO takes issue with the quality metric assessing adherence to warfarin therapy in patients with heart failure and paroxysmal or chronic atrial fibrillation; the concern is that it could discourage the use of newer agents. “This [warfarin] measure already is outdated,” the BIO stated. “There are more advanced therapeutic options available for these patients today as well as additional therapies in development. Leaving the measure in place may force ACOs to use a therapy that is no longer the only clinically appropriate, or even recommended, choice.”

The latest newcomer to the category is dabigatran (Pradaxa). Two other oral anticoagulants are in late-stage development: rivaroxaban (Xarelto) and apixaban. The biotech group’s concerns reflect the tension in the anticoagulant market segment among developers of newer agents and payer concerns over whether the advantages offered by such drugs can justify their increased cost.

A more prominent role for specialists

The ACO proposal is designed to emphasize the role of primary care providers. However, the BIO has advised that the CMS also ensure that specialists play an important part in the operation of ACOs, such as during the development of clinical guidelines and processes. Under the proposed rule, ACOs would be required to “develop and implement evidence-based medical practice or clinical guidelines and processes for delivering care consistent with the goals of better care for individuals, better health for populations, and lower growth in expenditures.”

ASCO echoed the BIO recommendation. “Given the prevalence of cancer in the Medicare community … [the] CMS should require ACOs to secure substantial input from specialists practicing in the community.” ASCO also requested that the CMS ensure that oncology practices do not face undue financial or procedural challenges under the ACO program.

“Oncologists already face a significant form of financial risk arising from the need to purchase and administer chemotherapy drugs within an environment in which Medicare and other health insurers often provide ambiguous coverage policies, payment rates that are inadequate to cover acquisition costs and slow preauthorization decisions,” ASCO stated. Furthermore, ASCO noted that Medicare contractors frequently require “excessive documentation” on the effectiveness of off-label drug indications, even when the indication is supported in the peer-reviewed literature.

The Centers for Medicare & Medicaid Services (CMS) should look for ways to ensure that financial incentives for Medicare accountable care organizations (ACOs) do not promote inappropriate shifts in prescribing from the Part B benefit to Part D, urged clinician and biotechnology groups in comments filed with the agency on June 6, 2011.

The Part B benefit covers drugs administered in a physician’s office, such as infused products. It excludes most oral and other medications that patients obtain at retail or mail-order pharmacies for use at home; these medications instead are covered under Part D. So in theory, the ACO program will encompass Part B expenditures, but not Part D. ACOs could save on the cost of drugs by shifting some prescriptions to Part D.

The comments respond to the proposed rule to establish ACOs that the CMS published on March 31, 2011. ACOs are part of a “shared savings” initiative, mandated by the Affordable Care Act, aimed at driving providers through financial incentives to better manage and coordinate healthcare delivery to improve quality and reduce costs. ACOs are slated to begin operating January 1, 2012. Under the proposed regulation, ACOs would be made up of physician practices, hospitals, and other healthcare providers. Primary care physicians are expected to serve as coordinators of individual patient care. The proposal posal does not specifically discuss potential shifts in prescribing. However, the Biotechnology Industry Organization (BIO) and others have urged the CMS to develop a process to ensure that patient care is not secondary to the pursuit of savings.

The implications of prescribing shifts

The BIO points out that the Congressional Research Service (CRS) identified prescribing shifts as a possible issue in an April 25 report on ACOs and the Medicare shared savings program. Such shifts might occur because the savings calculations used to evaluate ACOs are based on Parts A and B expenditures only, suggests the CRS, an analytic unit employed by Congress. Thus, “there may be instances where there is the appearance of cost savings as a result of providers unduly relying on Part D prescription medicines over other forms of care,” the CRS cautioned. The BIO added, “this issue may present problems from both the perspective of quality patient care as well as for Medicare program expenditures.” A shift to Part D drugs could actually increase patient outof- pocket costs, “which in turn may impact prescription drug adherence and ultimately clinical outcomes,” the comments stated.

The prospect of increasing patient costs under Part D is supported by a CMS-commissioned report released in August 2010. The report looked at the potential impact of consolidating Medicare reimbursement for drug categories with overlapping Parts B and D coverage. It found, on average, that patients would face higher costsharing if categories are consolidated under Part D, but that the Medicare program would save money, because Part D has less generous coverage rules. The study looked at oral anticancer and antiemetic drugs, insulin, vaccinations, inhalants, and rheumatoid arthritis medications.

In separate comments, the American Society of Clinical Oncology (ASCO) also targeted potential prescribing shifts in cancer therapies. “The CMS should address perverse incentives that will arise due to the proposed rule’s exclusion of Part D drugs from the costs by which ACO savings are measured,” ASCO warned. “Safeguards are needed to ensure that cancer patients receive the most appropriate therapy, regardless of whether that therapy is typically covered under Part B or under Part D.” (In addition to chemotherapy administered in a physician’s office, some oral anticancer drugs are also covered under Part B when they are direct substitutions for physician-administered treatments.)

Even the medical device and diagnostics industry registered concern with possible prescribing shifts to Part D drugs. The Advanced Medical Technology Association pointed out that providers might have an artificial incentive to substitute a pain medication, for example, when a surgical procedure covered under Part A or B would be considered standard of care.

Carving out the cost of new technologies

A number of stakeholders flagged the prospect that cost issues might also deter the adoption of cuttingedge drugs or other medical technologies by ACOs. The BIO and the Medical Device Manufacturers Association are among the groups urging CMS to consider ways to carve out new technologies from the benchmark for ACO costs and from annual expenditure performance reviews.

As proposed, the ACO program would be risk-based: an organization would receive a bonus payment if cost and quality benchmarks are met but would also face penalties if they are not. As a result, providers may be reluctant to adopt expensive new technologies, based on the comments.

To ensure beneficiary access to new therapies and to retain incentives for innovation, the stakeholders have suggested that the CMS adapt the Medicare payment mechanisms already used for new technologies in the hospital setting. Medicare’s hospital payment scheme includes mechanisms for add-on (inpatient) or pass-through (outpatient) payments. They provide payment for new and relatively expensive treatments in their first few years on the market in addition to the reimbursement rate set for their therapeutic category. The system is designed to account for the fact that the CMS does not yet know with certainty how much the new treatment will add to hospital charges and costs. And the extra reimbursement is intended to make it more likely that hospitals can afford to “try out” new technologies while the CMS collects the data on cost and use patterns that will ultimately allow it to develop a more permanent payment level.

The BIO has suggested that the carveout should apply regardless of the healthcare setting and should include drugs and biologics provided in the physician’s office. “Such congruity is necessary to ensure that the policy does not create an incentive to perform procedures in the hospital rather than the physician’s office,” according to the comments.

Quality metrics and warfarin

In comments on other aspects of the proposal, the BIO has recommended that the CMS should establish a process to rapidly update the quality measures used to evaluate ACO performance. The proposal outlines a number of quality measures, including whether patients at risk of serious disease or frail elderly patients are taking drugs for managing chronic disease. The agency also will track patient measurements, such as cholesterol levels and hemoglobin A1c values, to monitor the effectiveness of disease management. The quality measures are expected to improve adherence to drugs for chronic disease.

Nevertheless, the BIO takes issue with the quality metric assessing adherence to warfarin therapy in patients with heart failure and paroxysmal or chronic atrial fibrillation; the concern is that it could discourage the use of newer agents. “This [warfarin] measure already is outdated,” the BIO stated. “There are more advanced therapeutic options available for these patients today as well as additional therapies in development. Leaving the measure in place may force ACOs to use a therapy that is no longer the only clinically appropriate, or even recommended, choice.”

The latest newcomer to the category is dabigatran (Pradaxa). Two other oral anticoagulants are in late-stage development: rivaroxaban (Xarelto) and apixaban. The biotech group’s concerns reflect the tension in the anticoagulant market segment among developers of newer agents and payer concerns over whether the advantages offered by such drugs can justify their increased cost.

A more prominent role for specialists

The ACO proposal is designed to emphasize the role of primary care providers. However, the BIO has advised that the CMS also ensure that specialists play an important part in the operation of ACOs, such as during the development of clinical guidelines and processes. Under the proposed rule, ACOs would be required to “develop and implement evidence-based medical practice or clinical guidelines and processes for delivering care consistent with the goals of better care for individuals, better health for populations, and lower growth in expenditures.”

ASCO echoed the BIO recommendation. “Given the prevalence of cancer in the Medicare community … [the] CMS should require ACOs to secure substantial input from specialists practicing in the community.” ASCO also requested that the CMS ensure that oncology practices do not face undue financial or procedural challenges under the ACO program.

“Oncologists already face a significant form of financial risk arising from the need to purchase and administer chemotherapy drugs within an environment in which Medicare and other health insurers often provide ambiguous coverage policies, payment rates that are inadequate to cover acquisition costs and slow preauthorization decisions,” ASCO stated. Furthermore, ASCO noted that Medicare contractors frequently require “excessive documentation” on the effectiveness of off-label drug indications, even when the indication is supported in the peer-reviewed literature.

Medicare would cover sipuleucel-T for Food and Drug Administration–approved uses, the Centers for Medicare and Medicaid Services proposed in a draft national coverage decision released March 30.

Off-label use of the novel therapeutic prostate cancer vaccine, marketed by Dendreon under the trade name Provenge, would be left to the discretion of local Medicare claims carriers, the agency said.

The draft national coverage determination (NCD) would not change the reimbursement situation for on-label uses for Provenge. As of early January, all 15 Medicare subcontractors in the United States were covering the vaccine for its approved indications, according to Dendreon. Provenge was approved by the FDA in April 2010 for patients with asymptomatic or minimally symptomatic, metastatic, castrate-resistant (hormone-refractory) prostate cancer.

For off-label uses, the draft NCD listed a number of reasons why the agency decided against prohibiting Medicare coverage. First, because there is currently no evidence to support additional indications, the CMS said that it is "hopeful that unlabeled uses in the near future will take place only in the context of bona fide clinical studies."

However, "if this turns out to be an overly optimistic viewpoint," according to the draft, the agency may "reconsider this NCD to ensure that Medicare coverage is restricted to uses that are supported by robust evidence."

Other reasons for not barring off-label reimbursement relate to providing contractors with the flexibility to quickly adapt coverage requirements as physicians gain experience with the vaccine, according to the draft decision.

Because the "potency of the administered preparation itself is unique from patient to patient and may even differ in a given patient from administration to administration," future evidence may demonstrate "improvement in patient health outcomes as a result of incremental improvements to the current labeled process," according to the draft decision. The process of administration involves autotransfusion of the patient’s own treated white blood cells.

Should future evidence demonstrate improvements in patients who do not meet the current labeled indication, "we want our administrative contractors to have the flexibility to determine local coverage without the need to reconsider an NCD."

The CMS noted that it considered requiring that coverage for Provenge be premised on the collection of outcomes evidence (using "coverage with evidence development" provisions), but decided against that course because the data are already strong enough to support on-label uses and there is no evidence to support uses off label.

Panelists on the Medicare Evidence Development and Coverage Advisory Committee urged further data collection for Provenge during a November meeting. A number of members suggested building on the patient registry that Dendreon will establish to monitor Provenge safety as a way to collect information that can fill some of the evidence gaps.

The Provenge registry, which will include a minimum of 1,500 patients, was originally intended to track incidences of cerebrovascular adverse events linked to treatment; it is part of a REMS (Risk Evaluation and Mitigation Strategy) required by the FDA as a condition of approval.

Following the MedCAC meeting and a subsequent meeting with the CMS, Dendreon committed to collecting additional data in the registry.

A large proportion of patients in the registry are expected to be on Medicare, Dendreon said. As a result, "there will be the opportunity ... to compare outcomes across age groups, and also to compare the survival of individuals in the registry stratified by age to the outcomes of similar patients in the pivotal studies of Provenge and other publicly available databases."

The registry will support further analysis of whether black men may benefit more than other groups from Provenge, which was suggested in the pivotal clinical studies on the therapy, the company added. Some 5%-10% of treated patients are expected to be black.

"The findings will be considered both in comparison to the pooled survival estimate from the pivotal trials in this sub-group, and to the survival of subjects of other races in the registry. Each of these comparisons may lend further insight into the question of whether Provenge is substantially more beneficial in this or any other sub-group than it is in the general population."

The company will also catalog the type and timing of therapies that patients receive prior to and subsequent to Provenge.

"We will include these treatments as covariates in models of overall survival, allowing us to generate hypotheses about potentially positive or negative interactions between Provenge and such therapies," according to the company.

The CMS will accept comments on the draft guidance for 30 days and release a final national coverage decision by June 30.

This coverage is provided courtesy of “The Pink Sheet.” “The Pink Sheet” and Internal Medicine News Digital Network are both owned by Elsevier.

Medicare would cover sipuleucel-T for Food and Drug Administration–approved uses, the Centers for Medicare and Medicaid Services proposed in a draft national coverage decision released March 30.

Off-label use of the novel therapeutic prostate cancer vaccine, marketed by Dendreon under the trade name Provenge, would be left to the discretion of local Medicare claims carriers, the agency said.

The draft national coverage determination (NCD) would not change the reimbursement situation for on-label uses for Provenge. As of early January, all 15 Medicare subcontractors in the United States were covering the vaccine for its approved indications, according to Dendreon. Provenge was approved by the FDA in April 2010 for patients with asymptomatic or minimally symptomatic, metastatic, castrate-resistant (hormone-refractory) prostate cancer.

For off-label uses, the draft NCD listed a number of reasons why the agency decided against prohibiting Medicare coverage. First, because there is currently no evidence to support additional indications, the CMS said that it is "hopeful that unlabeled uses in the near future will take place only in the context of bona fide clinical studies."

However, "if this turns out to be an overly optimistic viewpoint," according to the draft, the agency may "reconsider this NCD to ensure that Medicare coverage is restricted to uses that are supported by robust evidence."

Other reasons for not barring off-label reimbursement relate to providing contractors with the flexibility to quickly adapt coverage requirements as physicians gain experience with the vaccine, according to the draft decision.

Because the "potency of the administered preparation itself is unique from patient to patient and may even differ in a given patient from administration to administration," future evidence may demonstrate "improvement in patient health outcomes as a result of incremental improvements to the current labeled process," according to the draft decision. The process of administration involves autotransfusion of the patient’s own treated white blood cells.

Should future evidence demonstrate improvements in patients who do not meet the current labeled indication, "we want our administrative contractors to have the flexibility to determine local coverage without the need to reconsider an NCD."

The CMS noted that it considered requiring that coverage for Provenge be premised on the collection of outcomes evidence (using "coverage with evidence development" provisions), but decided against that course because the data are already strong enough to support on-label uses and there is no evidence to support uses off label.

Panelists on the Medicare Evidence Development and Coverage Advisory Committee urged further data collection for Provenge during a November meeting. A number of members suggested building on the patient registry that Dendreon will establish to monitor Provenge safety as a way to collect information that can fill some of the evidence gaps.

The Provenge registry, which will include a minimum of 1,500 patients, was originally intended to track incidences of cerebrovascular adverse events linked to treatment; it is part of a REMS (Risk Evaluation and Mitigation Strategy) required by the FDA as a condition of approval.

Following the MedCAC meeting and a subsequent meeting with the CMS, Dendreon committed to collecting additional data in the registry.

A large proportion of patients in the registry are expected to be on Medicare, Dendreon said. As a result, "there will be the opportunity ... to compare outcomes across age groups, and also to compare the survival of individuals in the registry stratified by age to the outcomes of similar patients in the pivotal studies of Provenge and other publicly available databases."

The registry will support further analysis of whether black men may benefit more than other groups from Provenge, which was suggested in the pivotal clinical studies on the therapy, the company added. Some 5%-10% of treated patients are expected to be black.

"The findings will be considered both in comparison to the pooled survival estimate from the pivotal trials in this sub-group, and to the survival of subjects of other races in the registry. Each of these comparisons may lend further insight into the question of whether Provenge is substantially more beneficial in this or any other sub-group than it is in the general population."

The company will also catalog the type and timing of therapies that patients receive prior to and subsequent to Provenge.

"We will include these treatments as covariates in models of overall survival, allowing us to generate hypotheses about potentially positive or negative interactions between Provenge and such therapies," according to the company.

The CMS will accept comments on the draft guidance for 30 days and release a final national coverage decision by June 30.

This coverage is provided courtesy of “The Pink Sheet.” “The Pink Sheet” and Internal Medicine News Digital Network are both owned by Elsevier.

Medicare would cover sipuleucel-T for Food and Drug Administration–approved uses, the Centers for Medicare and Medicaid Services proposed in a draft national coverage decision released March 30.

Off-label use of the novel therapeutic prostate cancer vaccine, marketed by Dendreon under the trade name Provenge, would be left to the discretion of local Medicare claims carriers, the agency said.

The draft national coverage determination (NCD) would not change the reimbursement situation for on-label uses for Provenge. As of early January, all 15 Medicare subcontractors in the United States were covering the vaccine for its approved indications, according to Dendreon. Provenge was approved by the FDA in April 2010 for patients with asymptomatic or minimally symptomatic, metastatic, castrate-resistant (hormone-refractory) prostate cancer.

For off-label uses, the draft NCD listed a number of reasons why the agency decided against prohibiting Medicare coverage. First, because there is currently no evidence to support additional indications, the CMS said that it is "hopeful that unlabeled uses in the near future will take place only in the context of bona fide clinical studies."

However, "if this turns out to be an overly optimistic viewpoint," according to the draft, the agency may "reconsider this NCD to ensure that Medicare coverage is restricted to uses that are supported by robust evidence."

Other reasons for not barring off-label reimbursement relate to providing contractors with the flexibility to quickly adapt coverage requirements as physicians gain experience with the vaccine, according to the draft decision.

Because the "potency of the administered preparation itself is unique from patient to patient and may even differ in a given patient from administration to administration," future evidence may demonstrate "improvement in patient health outcomes as a result of incremental improvements to the current labeled process," according to the draft decision. The process of administration involves autotransfusion of the patient’s own treated white blood cells.

Should future evidence demonstrate improvements in patients who do not meet the current labeled indication, "we want our administrative contractors to have the flexibility to determine local coverage without the need to reconsider an NCD."

The CMS noted that it considered requiring that coverage for Provenge be premised on the collection of outcomes evidence (using "coverage with evidence development" provisions), but decided against that course because the data are already strong enough to support on-label uses and there is no evidence to support uses off label.

Panelists on the Medicare Evidence Development and Coverage Advisory Committee urged further data collection for Provenge during a November meeting. A number of members suggested building on the patient registry that Dendreon will establish to monitor Provenge safety as a way to collect information that can fill some of the evidence gaps.

The Provenge registry, which will include a minimum of 1,500 patients, was originally intended to track incidences of cerebrovascular adverse events linked to treatment; it is part of a REMS (Risk Evaluation and Mitigation Strategy) required by the FDA as a condition of approval.

Following the MedCAC meeting and a subsequent meeting with the CMS, Dendreon committed to collecting additional data in the registry.

A large proportion of patients in the registry are expected to be on Medicare, Dendreon said. As a result, "there will be the opportunity ... to compare outcomes across age groups, and also to compare the survival of individuals in the registry stratified by age to the outcomes of similar patients in the pivotal studies of Provenge and other publicly available databases."

The registry will support further analysis of whether black men may benefit more than other groups from Provenge, which was suggested in the pivotal clinical studies on the therapy, the company added. Some 5%-10% of treated patients are expected to be black.

"The findings will be considered both in comparison to the pooled survival estimate from the pivotal trials in this sub-group, and to the survival of subjects of other races in the registry. Each of these comparisons may lend further insight into the question of whether Provenge is substantially more beneficial in this or any other sub-group than it is in the general population."

The company will also catalog the type and timing of therapies that patients receive prior to and subsequent to Provenge.

"We will include these treatments as covariates in models of overall survival, allowing us to generate hypotheses about potentially positive or negative interactions between Provenge and such therapies," according to the company.

The CMS will accept comments on the draft guidance for 30 days and release a final national coverage decision by June 30.

This coverage is provided courtesy of “The Pink Sheet.” “The Pink Sheet” and Internal Medicine News Digital Network are both owned by Elsevier.

Medicare would cover sipuleucel-T for Food and Drug Administration–approved uses, the Centers for Medicare and Medicaid Services proposed in a draft national coverage decision released March 30.

Off-label use of the novel therapeutic prostate cancer vaccine, marketed by Dendreon under the trade name Provenge, would be left to the discretion of local Medicare claims carriers, the agency said.

The draft national coverage determination (NCD) would not change the reimbursement situation for on-label uses for Provenge. As of early January, all 15 Medicare subcontractors in the United States were covering the vaccine for its approved indications, according to Dendreon. Provenge was approved by the FDA in April 2010 for patients with asymptomatic or minimally symptomatic, metastatic, castrate-resistant (hormone-refractory) prostate cancer.

For off-label uses, the draft NCD listed a number of reasons why the agency decided against prohibiting Medicare coverage. First, because there is currently no evidence to support additional indications, the CMS said that it is "hopeful that unlabeled uses in the near future will take place only in the context of bona fide clinical studies."

However, "if this turns out to be an overly optimistic viewpoint," according to the draft, the agency may "reconsider this NCD to ensure that Medicare coverage is restricted to uses that are supported by robust evidence."

Other reasons for not barring off-label reimbursement relate to providing contractors with the flexibility to quickly adapt coverage requirements as physicians gain experience with the vaccine, according to the draft decision.

Because the "potency of the administered preparation itself is unique from patient to patient and may even differ in a given patient from administration to administration," future evidence may demonstrate "improvement in patient health outcomes as a result of incremental improvements to the current labeled process," according to the draft decision. The process of administration involves autotransfusion of the patient’s own treated white blood cells.

Should future evidence demonstrate improvements in patients who do not meet the current labeled indication, "we want our administrative contractors to have the flexibility to determine local coverage without the need to reconsider an NCD."

The CMS noted that it considered requiring that coverage for Provenge be premised on the collection of outcomes evidence (using "coverage with evidence development" provisions), but decided against that course because the data are already strong enough to support on-label uses and there is no evidence to support uses off label.

Panelists on the Medicare Evidence Development and Coverage Advisory Committee urged further data collection for Provenge during a November meeting. A number of members suggested building on the patient registry that Dendreon will establish to monitor Provenge safety as a way to collect information that can fill some of the evidence gaps.

The Provenge registry, which will include a minimum of 1,500 patients, was originally intended to track incidences of cerebrovascular adverse events linked to treatment; it is part of a REMS (Risk Evaluation and Mitigation Strategy) required by the FDA as a condition of approval.

Following the MedCAC meeting and a subsequent meeting with the CMS, Dendreon committed to collecting additional data in the registry.

A large proportion of patients in the registry are expected to be on Medicare, Dendreon said. As a result, "there will be the opportunity ... to compare outcomes across age groups, and also to compare the survival of individuals in the registry stratified by age to the outcomes of similar patients in the pivotal studies of Provenge and other publicly available databases."

The registry will support further analysis of whether black men may benefit more than other groups from Provenge, which was suggested in the pivotal clinical studies on the therapy, the company added. Some 5%-10% of treated patients are expected to be black.

"The findings will be considered both in comparison to the pooled survival estimate from the pivotal trials in this sub-group, and to the survival of subjects of other races in the registry. Each of these comparisons may lend further insight into the question of whether Provenge is substantially more beneficial in this or any other sub-group than it is in the general population."

The company will also catalog the type and timing of therapies that patients receive prior to and subsequent to Provenge.

"We will include these treatments as covariates in models of overall survival, allowing us to generate hypotheses about potentially positive or negative interactions between Provenge and such therapies," according to the company.

The CMS will accept comments on the draft guidance for 30 days and release a final national coverage decision by June 30.

This coverage is provided courtesy of “The Pink Sheet.” “The Pink Sheet” and Internal Medicine News Digital Network are both owned by Elsevier.

Medicare would cover sipuleucel-T for Food and Drug Administration–approved uses, the Centers for Medicare and Medicaid Services proposed in a draft national coverage decision released March 30.

Off-label use of the novel therapeutic prostate cancer vaccine, marketed by Dendreon under the trade name Provenge, would be left to the discretion of local Medicare claims carriers, the agency said.

The draft national coverage determination (NCD) would not change the reimbursement situation for on-label uses for Provenge. As of early January, all 15 Medicare subcontractors in the United States were covering the vaccine for its approved indications, according to Dendreon. Provenge was approved by the FDA in April 2010 for patients with asymptomatic or minimally symptomatic, metastatic, castrate-resistant (hormone-refractory) prostate cancer.

For off-label uses, the draft NCD listed a number of reasons why the agency decided against prohibiting Medicare coverage. First, because there is currently no evidence to support additional indications, the CMS said that it is "hopeful that unlabeled uses in the near future will take place only in the context of bona fide clinical studies."

However, "if this turns out to be an overly optimistic viewpoint," according to the draft, the agency may "reconsider this NCD to ensure that Medicare coverage is restricted to uses that are supported by robust evidence."

Other reasons for not barring off-label reimbursement relate to providing contractors with the flexibility to quickly adapt coverage requirements as physicians gain experience with the vaccine, according to the draft decision.

Because the "potency of the administered preparation itself is unique from patient to patient and may even differ in a given patient from administration to administration," future evidence may demonstrate "improvement in patient health outcomes as a result of incremental improvements to the current labeled process," according to the draft decision. The process of administration involves autotransfusion of the patient’s own treated white blood cells.

Should future evidence demonstrate improvements in patients who do not meet the current labeled indication, "we want our administrative contractors to have the flexibility to determine local coverage without the need to reconsider an NCD."

The CMS noted that it considered requiring that coverage for Provenge be premised on the collection of outcomes evidence (using "coverage with evidence development" provisions), but decided against that course because the data are already strong enough to support on-label uses and there is no evidence to support uses off label.

Panelists on the Medicare Evidence Development and Coverage Advisory Committee urged further data collection for Provenge during a November meeting. A number of members suggested building on the patient registry that Dendreon will establish to monitor Provenge safety as a way to collect information that can fill some of the evidence gaps.

The Provenge registry, which will include a minimum of 1,500 patients, was originally intended to track incidences of cerebrovascular adverse events linked to treatment; it is part of a REMS (Risk Evaluation and Mitigation Strategy) required by the FDA as a condition of approval.

Following the MedCAC meeting and a subsequent meeting with the CMS, Dendreon committed to collecting additional data in the registry.

A large proportion of patients in the registry are expected to be on Medicare, Dendreon said. As a result, "there will be the opportunity ... to compare outcomes across age groups, and also to compare the survival of individuals in the registry stratified by age to the outcomes of similar patients in the pivotal studies of Provenge and other publicly available databases."

The registry will support further analysis of whether black men may benefit more than other groups from Provenge, which was suggested in the pivotal clinical studies on the therapy, the company added. Some 5%-10% of treated patients are expected to be black.

"The findings will be considered both in comparison to the pooled survival estimate from the pivotal trials in this sub-group, and to the survival of subjects of other races in the registry. Each of these comparisons may lend further insight into the question of whether Provenge is substantially more beneficial in this or any other sub-group than it is in the general population."

The company will also catalog the type and timing of therapies that patients receive prior to and subsequent to Provenge.

"We will include these treatments as covariates in models of overall survival, allowing us to generate hypotheses about potentially positive or negative interactions between Provenge and such therapies," according to the company.

The CMS will accept comments on the draft guidance for 30 days and release a final national coverage decision by June 30.

This coverage is provided courtesy of “The Pink Sheet.” “The Pink Sheet” and Internal Medicine News Digital Network are both owned by Elsevier.

Medicare would cover sipuleucel-T for Food and Drug Administration–approved uses, the Centers for Medicare and Medicaid Services proposed in a draft national coverage decision released March 30.

Off-label use of the novel therapeutic prostate cancer vaccine, marketed by Dendreon under the trade name Provenge, would be left to the discretion of local Medicare claims carriers, the agency said.

The draft national coverage determination (NCD) would not change the reimbursement situation for on-label uses for Provenge. As of early January, all 15 Medicare subcontractors in the United States were covering the vaccine for its approved indications, according to Dendreon. Provenge was approved by the FDA in April 2010 for patients with asymptomatic or minimally symptomatic, metastatic, castrate-resistant (hormone-refractory) prostate cancer.

For off-label uses, the draft NCD listed a number of reasons why the agency decided against prohibiting Medicare coverage. First, because there is currently no evidence to support additional indications, the CMS said that it is "hopeful that unlabeled uses in the near future will take place only in the context of bona fide clinical studies."

However, "if this turns out to be an overly optimistic viewpoint," according to the draft, the agency may "reconsider this NCD to ensure that Medicare coverage is restricted to uses that are supported by robust evidence."

Other reasons for not barring off-label reimbursement relate to providing contractors with the flexibility to quickly adapt coverage requirements as physicians gain experience with the vaccine, according to the draft decision.

Because the "potency of the administered preparation itself is unique from patient to patient and may even differ in a given patient from administration to administration," future evidence may demonstrate "improvement in patient health outcomes as a result of incremental improvements to the current labeled process," according to the draft decision. The process of administration involves autotransfusion of the patient’s own treated white blood cells.

Should future evidence demonstrate improvements in patients who do not meet the current labeled indication, "we want our administrative contractors to have the flexibility to determine local coverage without the need to reconsider an NCD."

The CMS noted that it considered requiring that coverage for Provenge be premised on the collection of outcomes evidence (using "coverage with evidence development" provisions), but decided against that course because the data are already strong enough to support on-label uses and there is no evidence to support uses off label.

Panelists on the Medicare Evidence Development and Coverage Advisory Committee urged further data collection for Provenge during a November meeting. A number of members suggested building on the patient registry that Dendreon will establish to monitor Provenge safety as a way to collect information that can fill some of the evidence gaps.

The Provenge registry, which will include a minimum of 1,500 patients, was originally intended to track incidences of cerebrovascular adverse events linked to treatment; it is part of a REMS (Risk Evaluation and Mitigation Strategy) required by the FDA as a condition of approval.

Following the MedCAC meeting and a subsequent meeting with the CMS, Dendreon committed to collecting additional data in the registry.

A large proportion of patients in the registry are expected to be on Medicare, Dendreon said. As a result, "there will be the opportunity ... to compare outcomes across age groups, and also to compare the survival of individuals in the registry stratified by age to the outcomes of similar patients in the pivotal studies of Provenge and other publicly available databases."

The registry will support further analysis of whether black men may benefit more than other groups from Provenge, which was suggested in the pivotal clinical studies on the therapy, the company added. Some 5%-10% of treated patients are expected to be black.

"The findings will be considered both in comparison to the pooled survival estimate from the pivotal trials in this sub-group, and to the survival of subjects of other races in the registry. Each of these comparisons may lend further insight into the question of whether Provenge is substantially more beneficial in this or any other sub-group than it is in the general population."

The company will also catalog the type and timing of therapies that patients receive prior to and subsequent to Provenge.

"We will include these treatments as covariates in models of overall survival, allowing us to generate hypotheses about potentially positive or negative interactions between Provenge and such therapies," according to the company.

The CMS will accept comments on the draft guidance for 30 days and release a final national coverage decision by June 30.

This coverage is provided courtesy of “The Pink Sheet.” “The Pink Sheet” and Internal Medicine News Digital Network are both owned by Elsevier.

The existing clinical data on Dendreon’s prostate cancer vaccine Provenge (sipuleucel-T) cannot be "generalizable" to off-label uses, most members of the Medicare Evidence Development & Coverage Advisory Committee agreed at a Nov. 17 meeting.

The meeting was called by the Centers for Medicare and Medicaid Services to consider whether Provenge is a "reasonable and necessary" therapy and thus eligible for Medicare coverage. The treatment is approved for treating asymptomatic or minimally symptomatic prostate cancer that is metastatic and resistant to standard hormone treatment.

The committee’s review is part of a national coverage analysis on Provenge and may be followed by a national coverage determination. Off-label use is one significant issue for the agency. A proposed decision on the agency’s analysis is scheduled to be released by March 3, 2011, and a final decision will be released in June 2011.

The committee voted on a number of questions designed to probe the strength of the available evidence for both off-label and on-label use. In answering questions posed by CMS, members were asked to rate their confidence in the data on a scale ranging from "low" to "high."

The majority of members signaled that the data could not support use of the therapy in patients whose prostate cancer has not metastasized; in patients who have metastatic, castrate-resistant disease but whose symptoms are more severe than minimally symptomatic; or in patients who have metastatic prostate cancer but who have not failed hormonal therapy.

The committee was more confident about the data supporting on-label uses. The majority voted that the data are reasonably adequate to conclude Provenge improves overall patient survival and can help to avoid the "burdens," including side effects, associated with chemotherapy. The committee expressed less confidence that the data conclusively show Provenge improves the control of disease-related symptoms.

MEDCAC members felt the clinical trial data can be extrapolated to use of Provenge in community-based settings and were moderately confident that the research findings were generalizable to demographic groups underrepresented by the patients participating in the studies, including Medicare beneficiaries and minorities, such as African Americans.

"The Pink Sheet" and Hospitalist News Digital Network are both owned by Elsevier.

The existing clinical data on Dendreon’s prostate cancer vaccine Provenge (sipuleucel-T) cannot be "generalizable" to off-label uses, most members of the Medicare Evidence Development & Coverage Advisory Committee agreed at a Nov. 17 meeting.

The meeting was called by the Centers for Medicare and Medicaid Services to consider whether Provenge is a "reasonable and necessary" therapy and thus eligible for Medicare coverage. The treatment is approved for treating asymptomatic or minimally symptomatic prostate cancer that is metastatic and resistant to standard hormone treatment.

The committee’s review is part of a national coverage analysis on Provenge and may be followed by a national coverage determination. Off-label use is one significant issue for the agency. A proposed decision on the agency’s analysis is scheduled to be released by March 3, 2011, and a final decision will be released in June 2011.

The committee voted on a number of questions designed to probe the strength of the available evidence for both off-label and on-label use. In answering questions posed by CMS, members were asked to rate their confidence in the data on a scale ranging from "low" to "high."

The majority of members signaled that the data could not support use of the therapy in patients whose prostate cancer has not metastasized; in patients who have metastatic, castrate-resistant disease but whose symptoms are more severe than minimally symptomatic; or in patients who have metastatic prostate cancer but who have not failed hormonal therapy.

The committee was more confident about the data supporting on-label uses. The majority voted that the data are reasonably adequate to conclude Provenge improves overall patient survival and can help to avoid the "burdens," including side effects, associated with chemotherapy. The committee expressed less confidence that the data conclusively show Provenge improves the control of disease-related symptoms.

MEDCAC members felt the clinical trial data can be extrapolated to use of Provenge in community-based settings and were moderately confident that the research findings were generalizable to demographic groups underrepresented by the patients participating in the studies, including Medicare beneficiaries and minorities, such as African Americans.

"The Pink Sheet" and Hospitalist News Digital Network are both owned by Elsevier.

The existing clinical data on Dendreon’s prostate cancer vaccine Provenge (sipuleucel-T) cannot be "generalizable" to off-label uses, most members of the Medicare Evidence Development & Coverage Advisory Committee agreed at a Nov. 17 meeting.

The meeting was called by the Centers for Medicare and Medicaid Services to consider whether Provenge is a "reasonable and necessary" therapy and thus eligible for Medicare coverage. The treatment is approved for treating asymptomatic or minimally symptomatic prostate cancer that is metastatic and resistant to standard hormone treatment.

The committee’s review is part of a national coverage analysis on Provenge and may be followed by a national coverage determination. Off-label use is one significant issue for the agency. A proposed decision on the agency’s analysis is scheduled to be released by March 3, 2011, and a final decision will be released in June 2011.

The committee voted on a number of questions designed to probe the strength of the available evidence for both off-label and on-label use. In answering questions posed by CMS, members were asked to rate their confidence in the data on a scale ranging from "low" to "high."

The majority of members signaled that the data could not support use of the therapy in patients whose prostate cancer has not metastasized; in patients who have metastatic, castrate-resistant disease but whose symptoms are more severe than minimally symptomatic; or in patients who have metastatic prostate cancer but who have not failed hormonal therapy.

The committee was more confident about the data supporting on-label uses. The majority voted that the data are reasonably adequate to conclude Provenge improves overall patient survival and can help to avoid the "burdens," including side effects, associated with chemotherapy. The committee expressed less confidence that the data conclusively show Provenge improves the control of disease-related symptoms.

MEDCAC members felt the clinical trial data can be extrapolated to use of Provenge in community-based settings and were moderately confident that the research findings were generalizable to demographic groups underrepresented by the patients participating in the studies, including Medicare beneficiaries and minorities, such as African Americans.

"The Pink Sheet" and Hospitalist News Digital Network are both owned by Elsevier.

The existing clinical data on Dendreon’s prostate cancer vaccine Provenge (sipuleucel-T) cannot be "generalizable" to off-label uses, most members of the Medicare Evidence Development & Coverage Advisory Committee agreed at a Nov. 17 meeting.

The meeting was called by the Centers for Medicare and Medicaid Services to consider whether Provenge is a "reasonable and necessary" therapy and thus eligible for Medicare coverage. The treatment is approved for treating asymptomatic or minimally symptomatic prostate cancer that is metastatic and resistant to standard hormone treatment.

The committee’s review is part of a national coverage analysis on Provenge and may be followed by a national coverage determination. Off-label use is one significant issue for the agency. A proposed decision on the agency’s analysis is scheduled to be released by March 3, 2011, and a final decision will be released in June 2011.

The committee voted on a number of questions designed to probe the strength of the available evidence for both off-label and on-label use. In answering questions posed by CMS, members were asked to rate their confidence in the data on a scale ranging from "low" to "high."

The majority of members signaled that the data could not support use of the therapy in patients whose prostate cancer has not metastasized; in patients who have metastatic, castrate-resistant disease but whose symptoms are more severe than minimally symptomatic; or in patients who have metastatic prostate cancer but who have not failed hormonal therapy.

The committee was more confident about the data supporting on-label uses. The majority voted that the data are reasonably adequate to conclude Provenge improves overall patient survival and can help to avoid the "burdens," including side effects, associated with chemotherapy. The committee expressed less confidence that the data conclusively show Provenge improves the control of disease-related symptoms.

MEDCAC members felt the clinical trial data can be extrapolated to use of Provenge in community-based settings and were moderately confident that the research findings were generalizable to demographic groups underrepresented by the patients participating in the studies, including Medicare beneficiaries and minorities, such as African Americans.

"The Pink Sheet" and Internal Medicine News Digital Network are both owned by Elsevier.

The existing clinical data on Dendreon’s prostate cancer vaccine Provenge (sipuleucel-T) cannot be "generalizable" to off-label uses, most members of the Medicare Evidence Development & Coverage Advisory Committee agreed at a Nov. 17 meeting.

The meeting was called by the Centers for Medicare and Medicaid Services to consider whether Provenge is a "reasonable and necessary" therapy and thus eligible for Medicare coverage. The treatment is approved for treating asymptomatic or minimally symptomatic prostate cancer that is metastatic and resistant to standard hormone treatment.

The committee’s review is part of a national coverage analysis on Provenge and may be followed by a national coverage determination. Off-label use is one significant issue for the agency. A proposed decision on the agency’s analysis is scheduled to be released by March 3, 2011, and a final decision will be released in June 2011.

The committee voted on a number of questions designed to probe the strength of the available evidence for both off-label and on-label use. In answering questions posed by CMS, members were asked to rate their confidence in the data on a scale ranging from "low" to "high."

The majority of members signaled that the data could not support use of the therapy in patients whose prostate cancer has not metastasized; in patients who have metastatic, castrate-resistant disease but whose symptoms are more severe than minimally symptomatic; or in patients who have metastatic prostate cancer but who have not failed hormonal therapy.

The committee was more confident about the data supporting on-label uses. The majority voted that the data are reasonably adequate to conclude Provenge improves overall patient survival and can help to avoid the "burdens," including side effects, associated with chemotherapy. The committee expressed less confidence that the data conclusively show Provenge improves the control of disease-related symptoms.

MEDCAC members felt the clinical trial data can be extrapolated to use of Provenge in community-based settings and were moderately confident that the research findings were generalizable to demographic groups underrepresented by the patients participating in the studies, including Medicare beneficiaries and minorities, such as African Americans.

"The Pink Sheet" and Internal Medicine News Digital Network are both owned by Elsevier.

The existing clinical data on Dendreon’s prostate cancer vaccine Provenge (sipuleucel-T) cannot be "generalizable" to off-label uses, most members of the Medicare Evidence Development & Coverage Advisory Committee agreed at a Nov. 17 meeting.

The meeting was called by the Centers for Medicare and Medicaid Services to consider whether Provenge is a "reasonable and necessary" therapy and thus eligible for Medicare coverage. The treatment is approved for treating asymptomatic or minimally symptomatic prostate cancer that is metastatic and resistant to standard hormone treatment.

The committee’s review is part of a national coverage analysis on Provenge and may be followed by a national coverage determination. Off-label use is one significant issue for the agency. A proposed decision on the agency’s analysis is scheduled to be released by March 3, 2011, and a final decision will be released in June 2011.

The committee voted on a number of questions designed to probe the strength of the available evidence for both off-label and on-label use. In answering questions posed by CMS, members were asked to rate their confidence in the data on a scale ranging from "low" to "high."

The majority of members signaled that the data could not support use of the therapy in patients whose prostate cancer has not metastasized; in patients who have metastatic, castrate-resistant disease but whose symptoms are more severe than minimally symptomatic; or in patients who have metastatic prostate cancer but who have not failed hormonal therapy.

The committee was more confident about the data supporting on-label uses. The majority voted that the data are reasonably adequate to conclude Provenge improves overall patient survival and can help to avoid the "burdens," including side effects, associated with chemotherapy. The committee expressed less confidence that the data conclusively show Provenge improves the control of disease-related symptoms.

MEDCAC members felt the clinical trial data can be extrapolated to use of Provenge in community-based settings and were moderately confident that the research findings were generalizable to demographic groups underrepresented by the patients participating in the studies, including Medicare beneficiaries and minorities, such as African Americans.

"The Pink Sheet" and Internal Medicine News Digital Network are both owned by Elsevier.