Alicia Ault

The Food and Drug Administration on Jan. 31 approved the drug ivacaftor (Kalydeco) for cystic fibrosis patients aged 6 years and older who have the G551D mutation.

According to ivacaftor maker Vertex Pharmaceuticals, the approval is one of the fastest ever at the FDA. It took only 3 months from submission of data to approval.

Ivacaftor targets the G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. CFTR proteins regulate the flow of salt and water across the cells. A defective CFTR protein causes thick, sticky mucus to build up in the lungs, digestive tract, and other parts of the body, which can lead to respiratory and digestive problems and other complications.

The Vertex drug, taken orally twice a day, is a CFTR potentiator. It targets abnormal CFTR protein channels and opens them to allow chloride ions to move into and out of the cell; that, in turn, thins the mucus and the airways become less clogged, according to Vertex.

The FDA said that about 1,200 people – 4% of the 30,000 with cystic fibrosis in the United States – have the G551D mutation. Ivacaftor can help only those with the mutation. The agency and Vertex recommend FDA-approved molecular diagnostic testing before starting the therapy.

"Kalydeco is an excellent example of the promise of personalized medicine – targeted drugs that treat patients with a specific genetic makeup," said Dr. Margaret A. Hamburg, FDA commissioner, in a statement.

Added Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, "Kalydeco is the first available treatment that targets the defective CFTR protein, which is the underlying cause of cystic fibrosis. This is a breakthrough therapy for the cystic fibrosis community because current therapies only treat the symptoms of this genetic disease."

The FDA’s approval was based on two 48-week, placebo-controlled clinical studies that enrolled 213 patients. One study was in patients aged 12 years and older, and the other was in patients aged 6-11 years.

They all had at least one copy of the G551D mutation. The ivacaftor-treated groups had significant and sustained improvements in lung function, as well as in weight gain and some quality of life measurements, compared with those who received placebo, according to Vertex. They also had fewer pulmonary exacerbations.

The most common side effects were upper respiratory tract infection, headache, stomach ache, rash, diarrhea, and dizziness.

Vertex said it would begin shipping ivacaftor to U.S. pharmacies this week.

The Food and Drug Administration on Jan. 31 approved the drug ivacaftor (Kalydeco) for cystic fibrosis patients aged 6 years and older who have the G551D mutation.

According to ivacaftor maker Vertex Pharmaceuticals, the approval is one of the fastest ever at the FDA. It took only 3 months from submission of data to approval.

Ivacaftor targets the G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. CFTR proteins regulate the flow of salt and water across the cells. A defective CFTR protein causes thick, sticky mucus to build up in the lungs, digestive tract, and other parts of the body, which can lead to respiratory and digestive problems and other complications.

The Vertex drug, taken orally twice a day, is a CFTR potentiator. It targets abnormal CFTR protein channels and opens them to allow chloride ions to move into and out of the cell; that, in turn, thins the mucus and the airways become less clogged, according to Vertex.

The FDA said that about 1,200 people – 4% of the 30,000 with cystic fibrosis in the United States – have the G551D mutation. Ivacaftor can help only those with the mutation. The agency and Vertex recommend FDA-approved molecular diagnostic testing before starting the therapy.

"Kalydeco is an excellent example of the promise of personalized medicine – targeted drugs that treat patients with a specific genetic makeup," said Dr. Margaret A. Hamburg, FDA commissioner, in a statement.

Added Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, "Kalydeco is the first available treatment that targets the defective CFTR protein, which is the underlying cause of cystic fibrosis. This is a breakthrough therapy for the cystic fibrosis community because current therapies only treat the symptoms of this genetic disease."

The FDA’s approval was based on two 48-week, placebo-controlled clinical studies that enrolled 213 patients. One study was in patients aged 12 years and older, and the other was in patients aged 6-11 years.

They all had at least one copy of the G551D mutation. The ivacaftor-treated groups had significant and sustained improvements in lung function, as well as in weight gain and some quality of life measurements, compared with those who received placebo, according to Vertex. They also had fewer pulmonary exacerbations.

The most common side effects were upper respiratory tract infection, headache, stomach ache, rash, diarrhea, and dizziness.

Vertex said it would begin shipping ivacaftor to U.S. pharmacies this week.

The Food and Drug Administration on Jan. 31 approved the drug ivacaftor (Kalydeco) for cystic fibrosis patients aged 6 years and older who have the G551D mutation.

According to ivacaftor maker Vertex Pharmaceuticals, the approval is one of the fastest ever at the FDA. It took only 3 months from submission of data to approval.

Ivacaftor targets the G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. CFTR proteins regulate the flow of salt and water across the cells. A defective CFTR protein causes thick, sticky mucus to build up in the lungs, digestive tract, and other parts of the body, which can lead to respiratory and digestive problems and other complications.

The Vertex drug, taken orally twice a day, is a CFTR potentiator. It targets abnormal CFTR protein channels and opens them to allow chloride ions to move into and out of the cell; that, in turn, thins the mucus and the airways become less clogged, according to Vertex.

The FDA said that about 1,200 people – 4% of the 30,000 with cystic fibrosis in the United States – have the G551D mutation. Ivacaftor can help only those with the mutation. The agency and Vertex recommend FDA-approved molecular diagnostic testing before starting the therapy.

"Kalydeco is an excellent example of the promise of personalized medicine – targeted drugs that treat patients with a specific genetic makeup," said Dr. Margaret A. Hamburg, FDA commissioner, in a statement.

Added Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, "Kalydeco is the first available treatment that targets the defective CFTR protein, which is the underlying cause of cystic fibrosis. This is a breakthrough therapy for the cystic fibrosis community because current therapies only treat the symptoms of this genetic disease."

The FDA’s approval was based on two 48-week, placebo-controlled clinical studies that enrolled 213 patients. One study was in patients aged 12 years and older, and the other was in patients aged 6-11 years.

They all had at least one copy of the G551D mutation. The ivacaftor-treated groups had significant and sustained improvements in lung function, as well as in weight gain and some quality of life measurements, compared with those who received placebo, according to Vertex. They also had fewer pulmonary exacerbations.

The most common side effects were upper respiratory tract infection, headache, stomach ache, rash, diarrhea, and dizziness.

Vertex said it would begin shipping ivacaftor to U.S. pharmacies this week.

The Patient-Centered Outcomes Research Institute, established by the Affordable Care Act, has issued a draft of its initial priorities.

PCORI, as it is known, is not a government agency; it is independent and nonprofit and has a 21-member board of governors. But the organization has been, and continues to be, a primary focus of objections by Republican lawmakers who have alleged that it was established to find ways to ration care.

In its mission statement, PCORI says it "helps people make informed health care decisions – and improves health care delivery and outcomes – by producing and promoting high-integrity, evidence-based information that comes from research guided by patients, caregivers, and the broader health care community."

The board of governors has met eight times since November 2010. In late January, it issued a priority list that focused on five areas:

• Assessment of Options for Prevention, Diagnosis, and Treatment.

• Improving Health Care Systems.

• Communication and Dissemination Research.

• Addressing Disparities.

• Accelerating Patient-Centered Outcomes Research and Methodological Research.

"We want to hear from patients, caregivers, providers, and the wider health care community on whether our draft priorities and initial research agenda capture the broad areas where more evidence-based information is needed to make better decisions," PCORI Board Chair Eugene Washington, said in a statement.

Dr. Joe Selby, PCORI executive director, added, "This initial agenda does not limit which conditions or treatments will be studied. It is a starting point."

The organization did spell out in its draft where it thinks research should focus in each of the five broad areas. For instance, research comparing prevention, diagnosis, and treatment options should focus on "clinical options with emphasis on patient preferences and decision making."

To accelerate patient-centered research, proposals should hone in on "use of registries and clinical data networks to support research about patient-centered outcomes."

The draft is very likely to change. The organization is seeking public comment until March 15. It will hold a "national forum" in the District of Columbia on Feb. 27. PCORI said that once it gathers all the public feedback, it will publish a report summarizing the input and how it led to changes in the draft.

The research priorities then have to be approved by the PCORI board of governors. The organization said it will issue its first grants in May.

PCORI expects to have about $150 million in funding in 2012. Beginning in 2013, it will be funded by a tax on each insured American – through private insurance or Medicare. It will receive an estimated $500 million a year.

Meanwhile, the consulting company Avalere Health is predicting where the Centers for Medicare and Medicaid Services will head with its comparative effectiveness research in 2012. The CMS conducts analyses of various technologies and therapies in making its National Coverage Decisions (NCDs).

In 2008, the CMS issued a list of 20 potential topics for public comment. So far, the agency has acted on 7 of those topics, Avalere noted in its report.

Avalere expects that in 2012, the CMS will focus heavily on oncology and molecular diagnostics, with a concentration on non–small-cell lung cancer therapies, localized prostate cancer treatments, pharmacogenomic testing for breast and colon cancers, and prostate and lung cancer screening.

The consulting company said that oncology is ripe for exploration by the CMS, in part because of an influx of costly new therapies and diagnostic tests, in particular, those that tailor a therapy to the patient. Forty-six percent of the 241 genetic tests that are approved and available for clinical use are for cancer, according to Avalere.

There have been no NCDs on molecular tests for oncology, but there have been decisions by local Medicare contractors, mostly negative.

The Patient-Centered Outcomes Research Institute, established by the Affordable Care Act, has issued a draft of its initial priorities.

PCORI, as it is known, is not a government agency; it is independent and nonprofit and has a 21-member board of governors. But the organization has been, and continues to be, a primary focus of objections by Republican lawmakers who have alleged that it was established to find ways to ration care.

In its mission statement, PCORI says it "helps people make informed health care decisions – and improves health care delivery and outcomes – by producing and promoting high-integrity, evidence-based information that comes from research guided by patients, caregivers, and the broader health care community."

The board of governors has met eight times since November 2010. In late January, it issued a priority list that focused on five areas:

• Assessment of Options for Prevention, Diagnosis, and Treatment.

• Improving Health Care Systems.

• Communication and Dissemination Research.

• Addressing Disparities.

• Accelerating Patient-Centered Outcomes Research and Methodological Research.

"We want to hear from patients, caregivers, providers, and the wider health care community on whether our draft priorities and initial research agenda capture the broad areas where more evidence-based information is needed to make better decisions," PCORI Board Chair Eugene Washington, said in a statement.

Dr. Joe Selby, PCORI executive director, added, "This initial agenda does not limit which conditions or treatments will be studied. It is a starting point."

The organization did spell out in its draft where it thinks research should focus in each of the five broad areas. For instance, research comparing prevention, diagnosis, and treatment options should focus on "clinical options with emphasis on patient preferences and decision making."

To accelerate patient-centered research, proposals should hone in on "use of registries and clinical data networks to support research about patient-centered outcomes."

The draft is very likely to change. The organization is seeking public comment until March 15. It will hold a "national forum" in the District of Columbia on Feb. 27. PCORI said that once it gathers all the public feedback, it will publish a report summarizing the input and how it led to changes in the draft.

The research priorities then have to be approved by the PCORI board of governors. The organization said it will issue its first grants in May.

PCORI expects to have about $150 million in funding in 2012. Beginning in 2013, it will be funded by a tax on each insured American – through private insurance or Medicare. It will receive an estimated $500 million a year.

Meanwhile, the consulting company Avalere Health is predicting where the Centers for Medicare and Medicaid Services will head with its comparative effectiveness research in 2012. The CMS conducts analyses of various technologies and therapies in making its National Coverage Decisions (NCDs).

In 2008, the CMS issued a list of 20 potential topics for public comment. So far, the agency has acted on 7 of those topics, Avalere noted in its report.

Avalere expects that in 2012, the CMS will focus heavily on oncology and molecular diagnostics, with a concentration on non–small-cell lung cancer therapies, localized prostate cancer treatments, pharmacogenomic testing for breast and colon cancers, and prostate and lung cancer screening.

The consulting company said that oncology is ripe for exploration by the CMS, in part because of an influx of costly new therapies and diagnostic tests, in particular, those that tailor a therapy to the patient. Forty-six percent of the 241 genetic tests that are approved and available for clinical use are for cancer, according to Avalere.

There have been no NCDs on molecular tests for oncology, but there have been decisions by local Medicare contractors, mostly negative.

The Patient-Centered Outcomes Research Institute, established by the Affordable Care Act, has issued a draft of its initial priorities.

PCORI, as it is known, is not a government agency; it is independent and nonprofit and has a 21-member board of governors. But the organization has been, and continues to be, a primary focus of objections by Republican lawmakers who have alleged that it was established to find ways to ration care.

In its mission statement, PCORI says it "helps people make informed health care decisions – and improves health care delivery and outcomes – by producing and promoting high-integrity, evidence-based information that comes from research guided by patients, caregivers, and the broader health care community."

The board of governors has met eight times since November 2010. In late January, it issued a priority list that focused on five areas:

• Assessment of Options for Prevention, Diagnosis, and Treatment.

• Improving Health Care Systems.

• Communication and Dissemination Research.

• Addressing Disparities.

• Accelerating Patient-Centered Outcomes Research and Methodological Research.

"We want to hear from patients, caregivers, providers, and the wider health care community on whether our draft priorities and initial research agenda capture the broad areas where more evidence-based information is needed to make better decisions," PCORI Board Chair Eugene Washington, said in a statement.

Dr. Joe Selby, PCORI executive director, added, "This initial agenda does not limit which conditions or treatments will be studied. It is a starting point."

The organization did spell out in its draft where it thinks research should focus in each of the five broad areas. For instance, research comparing prevention, diagnosis, and treatment options should focus on "clinical options with emphasis on patient preferences and decision making."

To accelerate patient-centered research, proposals should hone in on "use of registries and clinical data networks to support research about patient-centered outcomes."

The draft is very likely to change. The organization is seeking public comment until March 15. It will hold a "national forum" in the District of Columbia on Feb. 27. PCORI said that once it gathers all the public feedback, it will publish a report summarizing the input and how it led to changes in the draft.

The research priorities then have to be approved by the PCORI board of governors. The organization said it will issue its first grants in May.

PCORI expects to have about $150 million in funding in 2012. Beginning in 2013, it will be funded by a tax on each insured American – through private insurance or Medicare. It will receive an estimated $500 million a year.

Meanwhile, the consulting company Avalere Health is predicting where the Centers for Medicare and Medicaid Services will head with its comparative effectiveness research in 2012. The CMS conducts analyses of various technologies and therapies in making its National Coverage Decisions (NCDs).

In 2008, the CMS issued a list of 20 potential topics for public comment. So far, the agency has acted on 7 of those topics, Avalere noted in its report.

Avalere expects that in 2012, the CMS will focus heavily on oncology and molecular diagnostics, with a concentration on non–small-cell lung cancer therapies, localized prostate cancer treatments, pharmacogenomic testing for breast and colon cancers, and prostate and lung cancer screening.

The consulting company said that oncology is ripe for exploration by the CMS, in part because of an influx of costly new therapies and diagnostic tests, in particular, those that tailor a therapy to the patient. Forty-six percent of the 241 genetic tests that are approved and available for clinical use are for cancer, according to Avalere.

There have been no NCDs on molecular tests for oncology, but there have been decisions by local Medicare contractors, mostly negative.

In another example of the continuing scrutiny being given to cardiac procedures, a cardiologist has filed a whistleblower suit against former colleagues, alleging that they knowingly bilked federal health programs over a 4-year period, from 2001-2005.

The suit was filed by Dr. Tullio Emanuele in the U.S. District Court for the Western District of Pennsylvania. It was under seal until Oct. 2011, and was served on the defendants on Jan. 17. The defendants have 20 days to respond.

Dr. Emanuele is now based in Kentucky, but had worked in an Erie-based practice, Medicor Associates. In his suit, he claims that his former colleagues at Medicor and at Flagship Cardiac, Vascular, and Thoracic Surgery of Erie – along with the UPMC Hamot Medical Center of Erie – engaged in billing practices that defrauded Medicare, Medicaid, and Tricare, among other insurers.

Carly Manino, manager of media relations at UPMC Hamot, said that the hospital would not comment. "It’s our policy not to comment on pending litigation," she said.

Whistleblower or "qui tam" suits are filed under the False Claims Act. Such suits allow private citizens with knowledge of false claims against the government to bring a lawsuit on behalf of the United States and to share in any recovery, according to the U.S. Department of Justice.

So far, the federal government has declined to intervene in the case, but the suit can be advanced by private litigators, according to Andy Stone, cocounsel on the Emanuele suit. The government may intervene at a later point, said Mr. Stone, whose practice is in Pittsburgh. The government can seek triple the amount of damages determined by the courts. Mr. Stone said that it’s not clear how much might be at stake in the case before discovery, but that given the potential number of claims, it could be millions of dollars.

The Pennsylvania suit also accuses the cardiologists and the hospital of violating the antikickback statutes that prohibit improper patient referrals.

In addition, the suit alleges that the named physicians performed unnecessary medical procedures, including cardiac catheterizations and vascular surgeries.

Mr. Stone said that he believes that cases alleging overuse of stents or other percutaneous interventions may be brought more often.

"These are important cases because the dollar amounts are big. The incentives are there – and substantial patient risk. You put those two things together and that makes for a real serious abuse situation," he said.

Indeed, the Emanuele suit seems to be the latest in a high-profile battle over the appropriateness of cardiac procedures, particularly stent placement. Last spring, Greensburg, Pa.–based Excela Health said that it had found that stents had been unnecessarily placed in almost 200 patients. In a turnaround, that health system has just won accreditation under the Accreditation for Cardiovascular Excellence (ACE) program that is sponsored by the Society for Cardiovascular Angiography and Interventions and the American College of Cardiology Foundation.

Those two organizations, along with the Maryland Chapter of the ACC, have been working to head off regulation of stent procedures, according to an editorial in the Journal of the American College of Cardiology. Instead, they are hoping to convince the state to require all Maryland hospitals to submit to a mandatory accrediting process through the groups’ ACE program.

The state of Maryland began looking into stenting as the result of another qui tam suit filed by three cardiologists against St. Joseph Medical Center in Towson, Md. The hospital settled for $22 million. The suit alleged that St. Joseph paid kickbacks in return for referrals from MidAtlantic Cardiovascular Associates.

St. Joseph also settled charges that cardiologist Mark Midei performed unnecessary stent procedures.

In another example of the continuing scrutiny being given to cardiac procedures, a cardiologist has filed a whistleblower suit against former colleagues, alleging that they knowingly bilked federal health programs over a 4-year period, from 2001-2005.

The suit was filed by Dr. Tullio Emanuele in the U.S. District Court for the Western District of Pennsylvania. It was under seal until Oct. 2011, and was served on the defendants on Jan. 17. The defendants have 20 days to respond.

Dr. Emanuele is now based in Kentucky, but had worked in an Erie-based practice, Medicor Associates. In his suit, he claims that his former colleagues at Medicor and at Flagship Cardiac, Vascular, and Thoracic Surgery of Erie – along with the UPMC Hamot Medical Center of Erie – engaged in billing practices that defrauded Medicare, Medicaid, and Tricare, among other insurers.

Carly Manino, manager of media relations at UPMC Hamot, said that the hospital would not comment. "It’s our policy not to comment on pending litigation," she said.

Whistleblower or "qui tam" suits are filed under the False Claims Act. Such suits allow private citizens with knowledge of false claims against the government to bring a lawsuit on behalf of the United States and to share in any recovery, according to the U.S. Department of Justice.

So far, the federal government has declined to intervene in the case, but the suit can be advanced by private litigators, according to Andy Stone, cocounsel on the Emanuele suit. The government may intervene at a later point, said Mr. Stone, whose practice is in Pittsburgh. The government can seek triple the amount of damages determined by the courts. Mr. Stone said that it’s not clear how much might be at stake in the case before discovery, but that given the potential number of claims, it could be millions of dollars.

The Pennsylvania suit also accuses the cardiologists and the hospital of violating the antikickback statutes that prohibit improper patient referrals.

In addition, the suit alleges that the named physicians performed unnecessary medical procedures, including cardiac catheterizations and vascular surgeries.

Mr. Stone said that he believes that cases alleging overuse of stents or other percutaneous interventions may be brought more often.

"These are important cases because the dollar amounts are big. The incentives are there – and substantial patient risk. You put those two things together and that makes for a real serious abuse situation," he said.

Indeed, the Emanuele suit seems to be the latest in a high-profile battle over the appropriateness of cardiac procedures, particularly stent placement. Last spring, Greensburg, Pa.–based Excela Health said that it had found that stents had been unnecessarily placed in almost 200 patients. In a turnaround, that health system has just won accreditation under the Accreditation for Cardiovascular Excellence (ACE) program that is sponsored by the Society for Cardiovascular Angiography and Interventions and the American College of Cardiology Foundation.

Those two organizations, along with the Maryland Chapter of the ACC, have been working to head off regulation of stent procedures, according to an editorial in the Journal of the American College of Cardiology. Instead, they are hoping to convince the state to require all Maryland hospitals to submit to a mandatory accrediting process through the groups’ ACE program.

The state of Maryland began looking into stenting as the result of another qui tam suit filed by three cardiologists against St. Joseph Medical Center in Towson, Md. The hospital settled for $22 million. The suit alleged that St. Joseph paid kickbacks in return for referrals from MidAtlantic Cardiovascular Associates.

St. Joseph also settled charges that cardiologist Mark Midei performed unnecessary stent procedures.

In another example of the continuing scrutiny being given to cardiac procedures, a cardiologist has filed a whistleblower suit against former colleagues, alleging that they knowingly bilked federal health programs over a 4-year period, from 2001-2005.

The suit was filed by Dr. Tullio Emanuele in the U.S. District Court for the Western District of Pennsylvania. It was under seal until Oct. 2011, and was served on the defendants on Jan. 17. The defendants have 20 days to respond.

Dr. Emanuele is now based in Kentucky, but had worked in an Erie-based practice, Medicor Associates. In his suit, he claims that his former colleagues at Medicor and at Flagship Cardiac, Vascular, and Thoracic Surgery of Erie – along with the UPMC Hamot Medical Center of Erie – engaged in billing practices that defrauded Medicare, Medicaid, and Tricare, among other insurers.

Carly Manino, manager of media relations at UPMC Hamot, said that the hospital would not comment. "It’s our policy not to comment on pending litigation," she said.

Whistleblower or "qui tam" suits are filed under the False Claims Act. Such suits allow private citizens with knowledge of false claims against the government to bring a lawsuit on behalf of the United States and to share in any recovery, according to the U.S. Department of Justice.

So far, the federal government has declined to intervene in the case, but the suit can be advanced by private litigators, according to Andy Stone, cocounsel on the Emanuele suit. The government may intervene at a later point, said Mr. Stone, whose practice is in Pittsburgh. The government can seek triple the amount of damages determined by the courts. Mr. Stone said that it’s not clear how much might be at stake in the case before discovery, but that given the potential number of claims, it could be millions of dollars.

The Pennsylvania suit also accuses the cardiologists and the hospital of violating the antikickback statutes that prohibit improper patient referrals.

In addition, the suit alleges that the named physicians performed unnecessary medical procedures, including cardiac catheterizations and vascular surgeries.

Mr. Stone said that he believes that cases alleging overuse of stents or other percutaneous interventions may be brought more often.

"These are important cases because the dollar amounts are big. The incentives are there – and substantial patient risk. You put those two things together and that makes for a real serious abuse situation," he said.

Indeed, the Emanuele suit seems to be the latest in a high-profile battle over the appropriateness of cardiac procedures, particularly stent placement. Last spring, Greensburg, Pa.–based Excela Health said that it had found that stents had been unnecessarily placed in almost 200 patients. In a turnaround, that health system has just won accreditation under the Accreditation for Cardiovascular Excellence (ACE) program that is sponsored by the Society for Cardiovascular Angiography and Interventions and the American College of Cardiology Foundation.

Those two organizations, along with the Maryland Chapter of the ACC, have been working to head off regulation of stent procedures, according to an editorial in the Journal of the American College of Cardiology. Instead, they are hoping to convince the state to require all Maryland hospitals to submit to a mandatory accrediting process through the groups’ ACE program.

The state of Maryland began looking into stenting as the result of another qui tam suit filed by three cardiologists against St. Joseph Medical Center in Towson, Md. The hospital settled for $22 million. The suit alleged that St. Joseph paid kickbacks in return for referrals from MidAtlantic Cardiovascular Associates.

St. Joseph also settled charges that cardiologist Mark Midei performed unnecessary stent procedures.

The Food and Drug Administration has approved vismodegib, an oral, once-daily medication for adults with locally advanced and metastatic advanced basal cell carcinoma on Jan. 30.

Vismodegib (Erivedge) is the first medication approved for metastatic BCC and was approved ahead of its March 8 statutory review date, according to a statement from the FDA.

The drug works by inhibiting the hedgehog pathway, which is active in most BCCs. "Our understanding of molecular pathways involved in cancer, such as the hedgehog pathway, has enabled the development of targeted drugs for specific diseases," said Dr. Richard Pazdur, director of the FDA’s Office of Hematology and Oncology Products, in a statement.

"Today’s approval provides a new treatment for people with advanced basal cell carcinoma who, until now, had no approved medicines to help shrink disfiguring or potentially life-threatening lesions," Dr. Hal Barron, Genentech’s chief medical officer and head of global product development, said in a statement.

The FDA based its approval on a single-arm, multicenter phase II study, ERIVANCE BCC. The open-label study enrolled 104 patients with locally advanced or metastatic BCC but, according to the FDA, approval was based on evaluations of 96 patients. Study participants took 150 mg vismodegib once daily.

The primary end point was objective response rate, that is, the percentage of patients who experienced complete and partial shrinkage or disappearance of the cancerous lesions after treatment. A total of 43% of patients with locally advanced BCC had partial or complete response (27 of 63) and 30% with metastatic disease had a partial response (10 of 33), according to Genentech. The median duration of response was 7.6 months.

The most common side effects were muscle spasms, hair loss, weight loss, nausea, diarrhea, fatigue, distorted sense of taste or loss of taste, decreased appetite, constipation, vomiting, and aching joints.

Vismodegib will carry a boxed warning about a potential risk of death or severe birth effects to a fetus. The FDA is not requiring a birth defects risk management program for vismodegib, but Genentech is advising female patients to use "highly effective" birth control before, during, and for 7 months after the last dose of treatment. Men should use a condom with spermicide, even if they have had a vasectomy, during sex with female partners during treatment and for 2 months after the last dose.

The company said that pregnant women are being encouraged to participate in the Erivedge pregnancy pharmacovigilance program, which collects information about exposure to that drug during pregnancy and the effects on the mother and her unborn child.

Vismodegib will be available in the United States within a few weeks and will be distributed through specialty pharmacies, according to Genentech.

The Food and Drug Administration has approved vismodegib, an oral, once-daily medication for adults with locally advanced and metastatic advanced basal cell carcinoma on Jan. 30.

Vismodegib (Erivedge) is the first medication approved for metastatic BCC and was approved ahead of its March 8 statutory review date, according to a statement from the FDA.

The drug works by inhibiting the hedgehog pathway, which is active in most BCCs. "Our understanding of molecular pathways involved in cancer, such as the hedgehog pathway, has enabled the development of targeted drugs for specific diseases," said Dr. Richard Pazdur, director of the FDA’s Office of Hematology and Oncology Products, in a statement.

"Today’s approval provides a new treatment for people with advanced basal cell carcinoma who, until now, had no approved medicines to help shrink disfiguring or potentially life-threatening lesions," Dr. Hal Barron, Genentech’s chief medical officer and head of global product development, said in a statement.

The FDA based its approval on a single-arm, multicenter phase II study, ERIVANCE BCC. The open-label study enrolled 104 patients with locally advanced or metastatic BCC but, according to the FDA, approval was based on evaluations of 96 patients. Study participants took 150 mg vismodegib once daily.

The primary end point was objective response rate, that is, the percentage of patients who experienced complete and partial shrinkage or disappearance of the cancerous lesions after treatment. A total of 43% of patients with locally advanced BCC had partial or complete response (27 of 63) and 30% with metastatic disease had a partial response (10 of 33), according to Genentech. The median duration of response was 7.6 months.

The most common side effects were muscle spasms, hair loss, weight loss, nausea, diarrhea, fatigue, distorted sense of taste or loss of taste, decreased appetite, constipation, vomiting, and aching joints.

Vismodegib will carry a boxed warning about a potential risk of death or severe birth effects to a fetus. The FDA is not requiring a birth defects risk management program for vismodegib, but Genentech is advising female patients to use "highly effective" birth control before, during, and for 7 months after the last dose of treatment. Men should use a condom with spermicide, even if they have had a vasectomy, during sex with female partners during treatment and for 2 months after the last dose.

The company said that pregnant women are being encouraged to participate in the Erivedge pregnancy pharmacovigilance program, which collects information about exposure to that drug during pregnancy and the effects on the mother and her unborn child.

Vismodegib will be available in the United States within a few weeks and will be distributed through specialty pharmacies, according to Genentech.

The Food and Drug Administration has approved vismodegib, an oral, once-daily medication for adults with locally advanced and metastatic advanced basal cell carcinoma on Jan. 30.

Vismodegib (Erivedge) is the first medication approved for metastatic BCC and was approved ahead of its March 8 statutory review date, according to a statement from the FDA.

The drug works by inhibiting the hedgehog pathway, which is active in most BCCs. "Our understanding of molecular pathways involved in cancer, such as the hedgehog pathway, has enabled the development of targeted drugs for specific diseases," said Dr. Richard Pazdur, director of the FDA’s Office of Hematology and Oncology Products, in a statement.

"Today’s approval provides a new treatment for people with advanced basal cell carcinoma who, until now, had no approved medicines to help shrink disfiguring or potentially life-threatening lesions," Dr. Hal Barron, Genentech’s chief medical officer and head of global product development, said in a statement.

The FDA based its approval on a single-arm, multicenter phase II study, ERIVANCE BCC. The open-label study enrolled 104 patients with locally advanced or metastatic BCC but, according to the FDA, approval was based on evaluations of 96 patients. Study participants took 150 mg vismodegib once daily.

The primary end point was objective response rate, that is, the percentage of patients who experienced complete and partial shrinkage or disappearance of the cancerous lesions after treatment. A total of 43% of patients with locally advanced BCC had partial or complete response (27 of 63) and 30% with metastatic disease had a partial response (10 of 33), according to Genentech. The median duration of response was 7.6 months.

The most common side effects were muscle spasms, hair loss, weight loss, nausea, diarrhea, fatigue, distorted sense of taste or loss of taste, decreased appetite, constipation, vomiting, and aching joints.

Vismodegib will carry a boxed warning about a potential risk of death or severe birth effects to a fetus. The FDA is not requiring a birth defects risk management program for vismodegib, but Genentech is advising female patients to use "highly effective" birth control before, during, and for 7 months after the last dose of treatment. Men should use a condom with spermicide, even if they have had a vasectomy, during sex with female partners during treatment and for 2 months after the last dose.

The company said that pregnant women are being encouraged to participate in the Erivedge pregnancy pharmacovigilance program, which collects information about exposure to that drug during pregnancy and the effects on the mother and her unborn child.

Vismodegib will be available in the United States within a few weeks and will be distributed through specialty pharmacies, according to Genentech.

Although the Republican primaries this year have been anything but predictable, one thing is fairly certain: Health care issues will play a more prominent role in the Florida primary on Jan. 31 than they have so far.

Florida is a coveted win for the GOP contenders in part because 50 delegates are at stake, but also because the state is more diverse – racially, ethnically, and politically – than Iowa, New Hampshire, and South Carolina, where primaries have already been held. As such, it’s considered a better reflection of the general election.

With its large elderly population and hard-hit economy, social welfare programs like Social Security, Medicare, and Medicaid are very important to Florida voters.

"Medicare is certainly the third rail in Florida among the sizeable senior population, but so too is Medicaid," said Daniel A. Smith, Ph.D., professor of political science at the University of Florida, Gainesville.

Many seniors depend on both Medicare and Medicaid for their health care needs: Medicare for basic doctor and hospital care and Medicaid for long-term care, Dr. Smith pointed out.

That means Republican candidates should consider treading lightly when they talk about Social Security, Medicare, or Medicaid, he said, noting that in 1992, Ross Perot’s presidential bid was derailed in Florida after he said he would favor having wealthier Americans pay more for Social Security and Medicare.

"Retirees aren’t interested in dismantling the welfare state," Mr. Smith said. "Even those Floridians of means are not interested in having their benefits cut."

Health Care Platforms

At the Republican debate in Tampa on Jan. 23, Rick Santorum went after putative front-runner Mitt Romney for creating what he called a "government-run" health care plan when Mr. Romney was governor of Massachusetts. Mr. Santorum, a former senator from Pennsylvania, called that plan "RomneyCare," a play on the dismissive "Obamacare" moniker that Republicans have given to the Affordable Care Act.

In the past, Mr. Santorum, who at press time was polling a distant third in Florida, has supported a plan by Rep. Paul Ryan (R-Wisc.) to essentially privatize Medicare. On his official campaign website, he says his first priority as President would be to repeal the Affordable Care Act. He also has said he would encourage the purchase of health insurance across state lines, push for block grants to states for Medicaid, and bolster health savings accounts. Mr. Santorum also backs medical liability reform.

Like Mr. Santorum, Newt Gingrich is calling for more competition in health care, block grants for Medicaid, and a repeal of the ACA. But the former Speaker of the House presents a more detailed plan for health care that includes reforming the Food and Drug Administration, investing more in health research, and putting a premium on quality of care.

Mr. Gingrich and Mr. Romney have traded the lead in Florida for the last several months, with Mr. Romney leading fairly broadly before the South Carolina primary on Jan. 21. Then, just as Mr. Romney won New Hampshire, Mr. Santorum was belatedly declared the winner in Iowa. Mr. Gingrich handily beat Mr. Romney in South Carolina, 40% to 28%.

According to the University of Florida’s Dr. Smith, Mr. Romney has actively courted health care executives in Florida. But it may not be enough to fend off continued brickbats thrown at him for the Massachusetts plan. He has repeatedly disavowed the notion that the plan was the model for the Affordable Care Act. On his website he says his first priority will be a repeal of Obamacare. Mr. Romney, like the other candidates, says he supports less regulation, more competition, and medical liability reform.

Although his platform makes no overt mention of Medicare, analysis by the Center on Budget and Policy Priorities noted that, if enacted, Mr. Romney’s proposal to cap total spending and balance the budget would lead to a 17%-24% cut to Medicare by 2016.

Interestingly, the American Federation of State, County and Municipal Employees has purchased air time in Florida to attack Mr. Romney for what it calls "Medicare fraud."

"Retirees aren’t interested in dismantling the welfare state."

The 30-second spot says that, while Mr. Romney worked for the Damon Corp., the company defrauded Medicare. The spot then goes on to morph Mr. Romney’s face into that of Florida Governor Rick Scott’s, with the tag line, "Sound familiar?"

Gov. Scott, a Republican, was the CEO of HCA (then called Columbia/HCA) during a period when the company was found guilty of defrauding Medicare. He was forced to resign and the company paid a record fine to the government. Gov. Scott has seen his approval ratings in Florida bottom out, in part because he proposed to finance public education through reductions in Medicaid payments to hospitals.

Gov. Scott has not endorsed any of the GOP candidates. "He knows that his poll numbers are quite toxic. As a result I don’t think his endorsement behooves anyone right now," Dr. Smith said.

The Doctors Stand Clear

Several major physicians’ organizations have so far demurred on endorsing any of the candidates. A spokesperson said that the Florida Medical Association did not feel it was appropriate to even comment on a potential endorsement, given that its membership holds a variety of views.

Dr. John A. Gross, a member of the board of the Florida Academy of Family Physicians (that state’s chapter of the American Academy of Family Physicians), said that the FAFP would not be endorsing any candidates at this time either.

The FAFP is "looking for candidates that are willing to stand up for the patient," said Dr. Gross, who chairs the group’s government relations committee. Family physicians would be interested in candidates who support the patient-centered medical home and new models that provide high quality care.

Tort reform also is a huge issue for Florida physicians, Dr. Gross noted. In a recent poll, physicians in the state estimated that 1 of every 3 health care dollars in Florida is spent on so-called defensive medical costs. The poll was conducted by Patients for Fair Compensation, a nonprofit established by the for-profit physician staffing company Jackson Healthcare.

Dr. Gross said that medical liability reform is a priority issue for the FAFP every year.

The latest bill to address tort reform was introduced in mid-January in the Florida House and Senate.

Abortion is also a hot-button issue in Florida. The state is regarded as anti-choice by NARAL Pro-Choice America.

Mr. Santorum describes himself as strongly pro-life. Mr. Gingrich has said he supports ending federal subsidies for abortion and defunding Planned Parenthood. Mr. Romney had a much-publicized change in position, seemingly going from supporting a woman’s right to choose to being against abortion. He does not have an official stance listed on his campaign website.

This is the first in a series of articles looking at the Republican presidential primaries through the eyes of physicians. Next up: The race moves on to Minnesota and Colorado, which hold their primaries Feb. 7.

Although the Republican primaries this year have been anything but predictable, one thing is fairly certain: Health care issues will play a more prominent role in the Florida primary on Jan. 31 than they have so far.

Florida is a coveted win for the GOP contenders in part because 50 delegates are at stake, but also because the state is more diverse – racially, ethnically, and politically – than Iowa, New Hampshire, and South Carolina, where primaries have already been held. As such, it’s considered a better reflection of the general election.

With its large elderly population and hard-hit economy, social welfare programs like Social Security, Medicare, and Medicaid are very important to Florida voters.

"Medicare is certainly the third rail in Florida among the sizeable senior population, but so too is Medicaid," said Daniel A. Smith, Ph.D., professor of political science at the University of Florida, Gainesville.

Many seniors depend on both Medicare and Medicaid for their health care needs: Medicare for basic doctor and hospital care and Medicaid for long-term care, Dr. Smith pointed out.

That means Republican candidates should consider treading lightly when they talk about Social Security, Medicare, or Medicaid, he said, noting that in 1992, Ross Perot’s presidential bid was derailed in Florida after he said he would favor having wealthier Americans pay more for Social Security and Medicare.

"Retirees aren’t interested in dismantling the welfare state," Mr. Smith said. "Even those Floridians of means are not interested in having their benefits cut."

Health Care Platforms

At the Republican debate in Tampa on Jan. 23, Rick Santorum went after putative front-runner Mitt Romney for creating what he called a "government-run" health care plan when Mr. Romney was governor of Massachusetts. Mr. Santorum, a former senator from Pennsylvania, called that plan "RomneyCare," a play on the dismissive "Obamacare" moniker that Republicans have given to the Affordable Care Act.

In the past, Mr. Santorum, who at press time was polling a distant third in Florida, has supported a plan by Rep. Paul Ryan (R-Wisc.) to essentially privatize Medicare. On his official campaign website, he says his first priority as President would be to repeal the Affordable Care Act. He also has said he would encourage the purchase of health insurance across state lines, push for block grants to states for Medicaid, and bolster health savings accounts. Mr. Santorum also backs medical liability reform.

Like Mr. Santorum, Newt Gingrich is calling for more competition in health care, block grants for Medicaid, and a repeal of the ACA. But the former Speaker of the House presents a more detailed plan for health care that includes reforming the Food and Drug Administration, investing more in health research, and putting a premium on quality of care.

Mr. Gingrich and Mr. Romney have traded the lead in Florida for the last several months, with Mr. Romney leading fairly broadly before the South Carolina primary on Jan. 21. Then, just as Mr. Romney won New Hampshire, Mr. Santorum was belatedly declared the winner in Iowa. Mr. Gingrich handily beat Mr. Romney in South Carolina, 40% to 28%.

According to the University of Florida’s Dr. Smith, Mr. Romney has actively courted health care executives in Florida. But it may not be enough to fend off continued brickbats thrown at him for the Massachusetts plan. He has repeatedly disavowed the notion that the plan was the model for the Affordable Care Act. On his website he says his first priority will be a repeal of Obamacare. Mr. Romney, like the other candidates, says he supports less regulation, more competition, and medical liability reform.

Although his platform makes no overt mention of Medicare, analysis by the Center on Budget and Policy Priorities noted that, if enacted, Mr. Romney’s proposal to cap total spending and balance the budget would lead to a 17%-24% cut to Medicare by 2016.

Interestingly, the American Federation of State, County and Municipal Employees has purchased air time in Florida to attack Mr. Romney for what it calls "Medicare fraud."

"Retirees aren’t interested in dismantling the welfare state."

The 30-second spot says that, while Mr. Romney worked for the Damon Corp., the company defrauded Medicare. The spot then goes on to morph Mr. Romney’s face into that of Florida Governor Rick Scott’s, with the tag line, "Sound familiar?"

Gov. Scott, a Republican, was the CEO of HCA (then called Columbia/HCA) during a period when the company was found guilty of defrauding Medicare. He was forced to resign and the company paid a record fine to the government. Gov. Scott has seen his approval ratings in Florida bottom out, in part because he proposed to finance public education through reductions in Medicaid payments to hospitals.

Gov. Scott has not endorsed any of the GOP candidates. "He knows that his poll numbers are quite toxic. As a result I don’t think his endorsement behooves anyone right now," Dr. Smith said.

The Doctors Stand Clear

Several major physicians’ organizations have so far demurred on endorsing any of the candidates. A spokesperson said that the Florida Medical Association did not feel it was appropriate to even comment on a potential endorsement, given that its membership holds a variety of views.

Dr. John A. Gross, a member of the board of the Florida Academy of Family Physicians (that state’s chapter of the American Academy of Family Physicians), said that the FAFP would not be endorsing any candidates at this time either.

The FAFP is "looking for candidates that are willing to stand up for the patient," said Dr. Gross, who chairs the group’s government relations committee. Family physicians would be interested in candidates who support the patient-centered medical home and new models that provide high quality care.

Tort reform also is a huge issue for Florida physicians, Dr. Gross noted. In a recent poll, physicians in the state estimated that 1 of every 3 health care dollars in Florida is spent on so-called defensive medical costs. The poll was conducted by Patients for Fair Compensation, a nonprofit established by the for-profit physician staffing company Jackson Healthcare.

Dr. Gross said that medical liability reform is a priority issue for the FAFP every year.

The latest bill to address tort reform was introduced in mid-January in the Florida House and Senate.

Abortion is also a hot-button issue in Florida. The state is regarded as anti-choice by NARAL Pro-Choice America.

Mr. Santorum describes himself as strongly pro-life. Mr. Gingrich has said he supports ending federal subsidies for abortion and defunding Planned Parenthood. Mr. Romney had a much-publicized change in position, seemingly going from supporting a woman’s right to choose to being against abortion. He does not have an official stance listed on his campaign website.

This is the first in a series of articles looking at the Republican presidential primaries through the eyes of physicians. Next up: The race moves on to Minnesota and Colorado, which hold their primaries Feb. 7.

Although the Republican primaries this year have been anything but predictable, one thing is fairly certain: Health care issues will play a more prominent role in the Florida primary on Jan. 31 than they have so far.

Florida is a coveted win for the GOP contenders in part because 50 delegates are at stake, but also because the state is more diverse – racially, ethnically, and politically – than Iowa, New Hampshire, and South Carolina, where primaries have already been held. As such, it’s considered a better reflection of the general election.

With its large elderly population and hard-hit economy, social welfare programs like Social Security, Medicare, and Medicaid are very important to Florida voters.

"Medicare is certainly the third rail in Florida among the sizeable senior population, but so too is Medicaid," said Daniel A. Smith, Ph.D., professor of political science at the University of Florida, Gainesville.

Many seniors depend on both Medicare and Medicaid for their health care needs: Medicare for basic doctor and hospital care and Medicaid for long-term care, Dr. Smith pointed out.

That means Republican candidates should consider treading lightly when they talk about Social Security, Medicare, or Medicaid, he said, noting that in 1992, Ross Perot’s presidential bid was derailed in Florida after he said he would favor having wealthier Americans pay more for Social Security and Medicare.

"Retirees aren’t interested in dismantling the welfare state," Mr. Smith said. "Even those Floridians of means are not interested in having their benefits cut."

Health Care Platforms

At the Republican debate in Tampa on Jan. 23, Rick Santorum went after putative front-runner Mitt Romney for creating what he called a "government-run" health care plan when Mr. Romney was governor of Massachusetts. Mr. Santorum, a former senator from Pennsylvania, called that plan "RomneyCare," a play on the dismissive "Obamacare" moniker that Republicans have given to the Affordable Care Act.

In the past, Mr. Santorum, who at press time was polling a distant third in Florida, has supported a plan by Rep. Paul Ryan (R-Wisc.) to essentially privatize Medicare. On his official campaign website, he says his first priority as President would be to repeal the Affordable Care Act. He also has said he would encourage the purchase of health insurance across state lines, push for block grants to states for Medicaid, and bolster health savings accounts. Mr. Santorum also backs medical liability reform.

Like Mr. Santorum, Newt Gingrich is calling for more competition in health care, block grants for Medicaid, and a repeal of the ACA. But the former Speaker of the House presents a more detailed plan for health care that includes reforming the Food and Drug Administration, investing more in health research, and putting a premium on quality of care.

Mr. Gingrich and Mr. Romney have traded the lead in Florida for the last several months, with Mr. Romney leading fairly broadly before the South Carolina primary on Jan. 21. Then, just as Mr. Romney won New Hampshire, Mr. Santorum was belatedly declared the winner in Iowa. Mr. Gingrich handily beat Mr. Romney in South Carolina, 40% to 28%.

According to the University of Florida’s Dr. Smith, Mr. Romney has actively courted health care executives in Florida. But it may not be enough to fend off continued brickbats thrown at him for the Massachusetts plan. He has repeatedly disavowed the notion that the plan was the model for the Affordable Care Act. On his website he says his first priority will be a repeal of Obamacare. Mr. Romney, like the other candidates, says he supports less regulation, more competition, and medical liability reform.

Although his platform makes no overt mention of Medicare, analysis by the Center on Budget and Policy Priorities noted that, if enacted, Mr. Romney’s proposal to cap total spending and balance the budget would lead to a 17%-24% cut to Medicare by 2016.

Interestingly, the American Federation of State, County and Municipal Employees has purchased air time in Florida to attack Mr. Romney for what it calls "Medicare fraud."

"Retirees aren’t interested in dismantling the welfare state."

The 30-second spot says that, while Mr. Romney worked for the Damon Corp., the company defrauded Medicare. The spot then goes on to morph Mr. Romney’s face into that of Florida Governor Rick Scott’s, with the tag line, "Sound familiar?"

Gov. Scott, a Republican, was the CEO of HCA (then called Columbia/HCA) during a period when the company was found guilty of defrauding Medicare. He was forced to resign and the company paid a record fine to the government. Gov. Scott has seen his approval ratings in Florida bottom out, in part because he proposed to finance public education through reductions in Medicaid payments to hospitals.

Gov. Scott has not endorsed any of the GOP candidates. "He knows that his poll numbers are quite toxic. As a result I don’t think his endorsement behooves anyone right now," Dr. Smith said.

The Doctors Stand Clear

Several major physicians’ organizations have so far demurred on endorsing any of the candidates. A spokesperson said that the Florida Medical Association did not feel it was appropriate to even comment on a potential endorsement, given that its membership holds a variety of views.

Dr. John A. Gross, a member of the board of the Florida Academy of Family Physicians (that state’s chapter of the American Academy of Family Physicians), said that the FAFP would not be endorsing any candidates at this time either.

The FAFP is "looking for candidates that are willing to stand up for the patient," said Dr. Gross, who chairs the group’s government relations committee. Family physicians would be interested in candidates who support the patient-centered medical home and new models that provide high quality care.

Tort reform also is a huge issue for Florida physicians, Dr. Gross noted. In a recent poll, physicians in the state estimated that 1 of every 3 health care dollars in Florida is spent on so-called defensive medical costs. The poll was conducted by Patients for Fair Compensation, a nonprofit established by the for-profit physician staffing company Jackson Healthcare.

Dr. Gross said that medical liability reform is a priority issue for the FAFP every year.

The latest bill to address tort reform was introduced in mid-January in the Florida House and Senate.

Abortion is also a hot-button issue in Florida. The state is regarded as anti-choice by NARAL Pro-Choice America.

Mr. Santorum describes himself as strongly pro-life. Mr. Gingrich has said he supports ending federal subsidies for abortion and defunding Planned Parenthood. Mr. Romney had a much-publicized change in position, seemingly going from supporting a woman’s right to choose to being against abortion. He does not have an official stance listed on his campaign website.

This is the first in a series of articles looking at the Republican presidential primaries through the eyes of physicians. Next up: The race moves on to Minnesota and Colorado, which hold their primaries Feb. 7.

NEW ORLEANS – The American Medical Association’s policy-making body wrapped up 2 and a half days of meetings Nov. 15 by calling for a delay in implementation of the ICD-10 coding system, and reiterating its intention to seek the ability to privately broker fees with Medicare patients.

Physicians have long sought to be allowed to engage in so-called "private contracting" with Medicare patients. Under that scenario, Medicare patients could use their benefits to see a physician that does not accept Medicare. Currently, patients have to pay for the entire visit out of pocket if their physician does not accept Medicare.

The AMA says private contracting will empower patients – and, the group is supporting legislation sponsored by Rep. Tom Price (R-Ga.) that would allow such private contracting. The Medicare Patient Empowerment Act (H.R. 1700) was introduced last May; a companion bill was introduced in the Senate by Lisa Murkowski (R-Alaska).

The AMA House of Delegates reaffirmed support of the legislation, but also called upon the AMA to start a grassroots campaign to get patients involved.

Alabama delegate Dr. Jeff Terry and president of the Medical Association of the State of Alabama said, "A grassroots campaign will allow our politicians to do the right thing." Such a campaign will let politicians know "that we don’t want our congressmen to walk out on a political limb not supported by the public."

The emphasis on private contracting was in part a symptom of the high-pressure environment physicians find themselves in, facing a potential 27% cut in Medicare payments as well as high-cost requirements to implement electronic medical records, said Dr. Robert Wah, chairman of the AMA Board of Trustees.

"The fact that patients may be able to take their benefit where they’d like to, to see the doctors they want to see, is a potential safety valve to all this pressure being exerted on physician practices and patients in the Medicare system," Dr. Wah said in an interview. "So we’re actively pushing at the local level for people to go to their congressmen and ask them to sign on to this legislation."

The push back against the coming implementation of the new ICD-10 coding system in 2013 is also a symptom of physicians feeling overwhelmed by their changing practice environment, Dr. Wah said. The House approved a resolution sponsored by the Alabama and Mississippi delegations, to take action to stop the implementation.

The ICD-10 will consist of 69,000 codes, as compared with the 14,000 in ICD-9, currently in use. "At a time when we are working to get the best value possible for our health care dollar, this massive and expensive undertaking will add administrative expense and create unnecessary workflow disruptions," AMA President Peter W. Carmel said in a statement.

The ICD-10 system has been in development for 10 years but has been delayed several times already. The House of Delegates called on the AMA to mount "a vigorous campaign to seek delay of the implementation date," according to Dr. Wah. ☐

NEW ORLEANS – The American Medical Association’s policy-making body wrapped up 2 and a half days of meetings Nov. 15 by calling for a delay in implementation of the ICD-10 coding system, and reiterating its intention to seek the ability to privately broker fees with Medicare patients.

Physicians have long sought to be allowed to engage in so-called "private contracting" with Medicare patients. Under that scenario, Medicare patients could use their benefits to see a physician that does not accept Medicare. Currently, patients have to pay for the entire visit out of pocket if their physician does not accept Medicare.

The AMA says private contracting will empower patients – and, the group is supporting legislation sponsored by Rep. Tom Price (R-Ga.) that would allow such private contracting. The Medicare Patient Empowerment Act (H.R. 1700) was introduced last May; a companion bill was introduced in the Senate by Lisa Murkowski (R-Alaska).

The AMA House of Delegates reaffirmed support of the legislation, but also called upon the AMA to start a grassroots campaign to get patients involved.

Alabama delegate Dr. Jeff Terry and president of the Medical Association of the State of Alabama said, "A grassroots campaign will allow our politicians to do the right thing." Such a campaign will let politicians know "that we don’t want our congressmen to walk out on a political limb not supported by the public."

The emphasis on private contracting was in part a symptom of the high-pressure environment physicians find themselves in, facing a potential 27% cut in Medicare payments as well as high-cost requirements to implement electronic medical records, said Dr. Robert Wah, chairman of the AMA Board of Trustees.

"The fact that patients may be able to take their benefit where they’d like to, to see the doctors they want to see, is a potential safety valve to all this pressure being exerted on physician practices and patients in the Medicare system," Dr. Wah said in an interview. "So we’re actively pushing at the local level for people to go to their congressmen and ask them to sign on to this legislation."

The push back against the coming implementation of the new ICD-10 coding system in 2013 is also a symptom of physicians feeling overwhelmed by their changing practice environment, Dr. Wah said. The House approved a resolution sponsored by the Alabama and Mississippi delegations, to take action to stop the implementation.

The ICD-10 will consist of 69,000 codes, as compared with the 14,000 in ICD-9, currently in use. "At a time when we are working to get the best value possible for our health care dollar, this massive and expensive undertaking will add administrative expense and create unnecessary workflow disruptions," AMA President Peter W. Carmel said in a statement.

The ICD-10 system has been in development for 10 years but has been delayed several times already. The House of Delegates called on the AMA to mount "a vigorous campaign to seek delay of the implementation date," according to Dr. Wah. ☐

NEW ORLEANS – The American Medical Association’s policy-making body wrapped up 2 and a half days of meetings Nov. 15 by calling for a delay in implementation of the ICD-10 coding system, and reiterating its intention to seek the ability to privately broker fees with Medicare patients.

Physicians have long sought to be allowed to engage in so-called "private contracting" with Medicare patients. Under that scenario, Medicare patients could use their benefits to see a physician that does not accept Medicare. Currently, patients have to pay for the entire visit out of pocket if their physician does not accept Medicare.

The AMA says private contracting will empower patients – and, the group is supporting legislation sponsored by Rep. Tom Price (R-Ga.) that would allow such private contracting. The Medicare Patient Empowerment Act (H.R. 1700) was introduced last May; a companion bill was introduced in the Senate by Lisa Murkowski (R-Alaska).

The AMA House of Delegates reaffirmed support of the legislation, but also called upon the AMA to start a grassroots campaign to get patients involved.

Alabama delegate Dr. Jeff Terry and president of the Medical Association of the State of Alabama said, "A grassroots campaign will allow our politicians to do the right thing." Such a campaign will let politicians know "that we don’t want our congressmen to walk out on a political limb not supported by the public."

The emphasis on private contracting was in part a symptom of the high-pressure environment physicians find themselves in, facing a potential 27% cut in Medicare payments as well as high-cost requirements to implement electronic medical records, said Dr. Robert Wah, chairman of the AMA Board of Trustees.

"The fact that patients may be able to take their benefit where they’d like to, to see the doctors they want to see, is a potential safety valve to all this pressure being exerted on physician practices and patients in the Medicare system," Dr. Wah said in an interview. "So we’re actively pushing at the local level for people to go to their congressmen and ask them to sign on to this legislation."

The push back against the coming implementation of the new ICD-10 coding system in 2013 is also a symptom of physicians feeling overwhelmed by their changing practice environment, Dr. Wah said. The House approved a resolution sponsored by the Alabama and Mississippi delegations, to take action to stop the implementation.

The ICD-10 will consist of 69,000 codes, as compared with the 14,000 in ICD-9, currently in use. "At a time when we are working to get the best value possible for our health care dollar, this massive and expensive undertaking will add administrative expense and create unnecessary workflow disruptions," AMA President Peter W. Carmel said in a statement.

The ICD-10 system has been in development for 10 years but has been delayed several times already. The House of Delegates called on the AMA to mount "a vigorous campaign to seek delay of the implementation date," according to Dr. Wah. ☐

New Jersey Governor Chris Christie (R) has signed a bill that repeals the state’s so-called "BoTax." The bill (S-1988/A-3646) phases out a 6% tax on gross receipts from cosmetic procedures that was instituted in 2004. Examples of procedures that were taxable included cosmetic surgery, hair transplants, cosmetic injections, cosmetic soft tissue fillers, dermabrasion, chemical peels, laser hair removal, laser skin resurfacing, laser treatment of leg veins, sclerotherapy, and cosmetic dentistry.

Governor's Office/Jody Somers

The tax will be lowered to 4% on March 1 and to 2% on July 1, and will be completely eliminated as of July 1, 2013. The state was collecting about $11 million a year from the tax. The collections were put into a fund for medical care for the poor. A previous effort to repeal the tax was vetoed by former Governor Jon Corzine (D).

New Jersey Governor Chris Christie (R) has signed a bill that repeals the state’s so-called "BoTax." The bill (S-1988/A-3646) phases out a 6% tax on gross receipts from cosmetic procedures that was instituted in 2004. Examples of procedures that were taxable included cosmetic surgery, hair transplants, cosmetic injections, cosmetic soft tissue fillers, dermabrasion, chemical peels, laser hair removal, laser skin resurfacing, laser treatment of leg veins, sclerotherapy, and cosmetic dentistry.

Governor's Office/Jody Somers

The tax will be lowered to 4% on March 1 and to 2% on July 1, and will be completely eliminated as of July 1, 2013. The state was collecting about $11 million a year from the tax. The collections were put into a fund for medical care for the poor. A previous effort to repeal the tax was vetoed by former Governor Jon Corzine (D).

New Jersey Governor Chris Christie (R) has signed a bill that repeals the state’s so-called "BoTax." The bill (S-1988/A-3646) phases out a 6% tax on gross receipts from cosmetic procedures that was instituted in 2004. Examples of procedures that were taxable included cosmetic surgery, hair transplants, cosmetic injections, cosmetic soft tissue fillers, dermabrasion, chemical peels, laser hair removal, laser skin resurfacing, laser treatment of leg veins, sclerotherapy, and cosmetic dentistry.

Governor's Office/Jody Somers

The tax will be lowered to 4% on March 1 and to 2% on July 1, and will be completely eliminated as of July 1, 2013. The state was collecting about $11 million a year from the tax. The collections were put into a fund for medical care for the poor. A previous effort to repeal the tax was vetoed by former Governor Jon Corzine (D).

Glucarpidase, an intravenously delivered recombinant enzyme, was approved on Jan. 17 for the treatment of patients with toxic levels of methotrexate in their blood due to kidney failure, according to the Food and Drug Administration.

Patients receiving high doses of methotrexate can develop renal failure, which allows the chemotherapy drug to accumulate. Glucarpidase (Voraxaze) rapidly breaks down methotrexate into a form that can then be eliminated from the body, according to the agency.

In addition to kidney failure, prolonged exposure to high levels of methotrexate can result in "liver damage, severe mouth sores, damage to the lining of the intestine, skin rashes, and death due to low blood counts," Dr. Richard Pazdur, director of the FDA’s Office of Hematology and Oncology Products, said in a statement. "Voraxaze is an important new treatment option for cancer patients aimed at preventing these toxicities associated with sustained high levels of methotrexate," he added.

According to glucarpidase’s manufacturer, BTG Plc, high-dose methotrexate is used to treat or prevent the recurrence of certain types of cancer, such as osteosarcoma, leukemia, and lymphoma.

Methotrexate also is used to treat rheumatologic conditions, such as rheumatoid arthritis.

The enzyme had been available to some patients under the FDA’s investigational new drug designation. In 2008, Protherics Inc., which previously owned the rights to glucarpidase, was warned by the FDA against promoting the drug before it was approved.

Glucarpidase received orphan drug status, which put it on a priority review track.

Approval was based on two studies: a single clinical study of 22 patients that evaluated effectiveness and a 290-patient trial that evaluated safety.

According to the FDA, treatment was considered successful if methotrexate levels fell below a critical level within 15 minutes and stayed below the critical level for 8 days. That result was seen in 10 of the 22 patients. BTG said that there was a 95% reduction in methotrexate concentration from pretreatment baseline levels, maintained for up to 8 days, in all evaluable patients.

The most common side effects were hypotension, headache, nausea, vomiting, flushing, and paraesthesia.

Louise Makin, chief executive officer of BTG, said in a statement that glucarpidase "is the first product BTG has taken through to approval in the U.S. and we look forward to its launch over coming months."

Glucarpidase, an intravenously delivered recombinant enzyme, was approved on Jan. 17 for the treatment of patients with toxic levels of methotrexate in their blood due to kidney failure, according to the Food and Drug Administration.

Patients receiving high doses of methotrexate can develop renal failure, which allows the chemotherapy drug to accumulate. Glucarpidase (Voraxaze) rapidly breaks down methotrexate into a form that can then be eliminated from the body, according to the agency.

In addition to kidney failure, prolonged exposure to high levels of methotrexate can result in "liver damage, severe mouth sores, damage to the lining of the intestine, skin rashes, and death due to low blood counts," Dr. Richard Pazdur, director of the FDA’s Office of Hematology and Oncology Products, said in a statement. "Voraxaze is an important new treatment option for cancer patients aimed at preventing these toxicities associated with sustained high levels of methotrexate," he added.

According to glucarpidase’s manufacturer, BTG Plc, high-dose methotrexate is used to treat or prevent the recurrence of certain types of cancer, such as osteosarcoma, leukemia, and lymphoma.

Methotrexate also is used to treat rheumatologic conditions, such as rheumatoid arthritis.

The enzyme had been available to some patients under the FDA’s investigational new drug designation. In 2008, Protherics Inc., which previously owned the rights to glucarpidase, was warned by the FDA against promoting the drug before it was approved.

Glucarpidase received orphan drug status, which put it on a priority review track.

Approval was based on two studies: a single clinical study of 22 patients that evaluated effectiveness and a 290-patient trial that evaluated safety.

According to the FDA, treatment was considered successful if methotrexate levels fell below a critical level within 15 minutes and stayed below the critical level for 8 days. That result was seen in 10 of the 22 patients. BTG said that there was a 95% reduction in methotrexate concentration from pretreatment baseline levels, maintained for up to 8 days, in all evaluable patients.

The most common side effects were hypotension, headache, nausea, vomiting, flushing, and paraesthesia.

Louise Makin, chief executive officer of BTG, said in a statement that glucarpidase "is the first product BTG has taken through to approval in the U.S. and we look forward to its launch over coming months."

Glucarpidase, an intravenously delivered recombinant enzyme, was approved on Jan. 17 for the treatment of patients with toxic levels of methotrexate in their blood due to kidney failure, according to the Food and Drug Administration.

Patients receiving high doses of methotrexate can develop renal failure, which allows the chemotherapy drug to accumulate. Glucarpidase (Voraxaze) rapidly breaks down methotrexate into a form that can then be eliminated from the body, according to the agency.

In addition to kidney failure, prolonged exposure to high levels of methotrexate can result in "liver damage, severe mouth sores, damage to the lining of the intestine, skin rashes, and death due to low blood counts," Dr. Richard Pazdur, director of the FDA’s Office of Hematology and Oncology Products, said in a statement. "Voraxaze is an important new treatment option for cancer patients aimed at preventing these toxicities associated with sustained high levels of methotrexate," he added.

According to glucarpidase’s manufacturer, BTG Plc, high-dose methotrexate is used to treat or prevent the recurrence of certain types of cancer, such as osteosarcoma, leukemia, and lymphoma.

Methotrexate also is used to treat rheumatologic conditions, such as rheumatoid arthritis.

The enzyme had been available to some patients under the FDA’s investigational new drug designation. In 2008, Protherics Inc., which previously owned the rights to glucarpidase, was warned by the FDA against promoting the drug before it was approved.

Glucarpidase received orphan drug status, which put it on a priority review track.

Approval was based on two studies: a single clinical study of 22 patients that evaluated effectiveness and a 290-patient trial that evaluated safety.

According to the FDA, treatment was considered successful if methotrexate levels fell below a critical level within 15 minutes and stayed below the critical level for 8 days. That result was seen in 10 of the 22 patients. BTG said that there was a 95% reduction in methotrexate concentration from pretreatment baseline levels, maintained for up to 8 days, in all evaluable patients.

The most common side effects were hypotension, headache, nausea, vomiting, flushing, and paraesthesia.

Louise Makin, chief executive officer of BTG, said in a statement that glucarpidase "is the first product BTG has taken through to approval in the U.S. and we look forward to its launch over coming months."

The Food and Drug Administration has sent recommendations to Congress on establishing user fee programs for generic and biosimilar drugs.

Legislation is required to create the new programs, which would be modeled on the 20-year-old Prescription Drug User Fee Act (PDUFA), according to the agency.

Under PDUFA, the FDA collects fees for certain applications for a new drugs or biologics, and it also assesses fees on certain manufacturing establishments. In fiscal 2012, for instance, a new drug application that includes clinical data costs $1.8 million; establishment fees are $520,000. In return, the FDA agrees to review applications according to set deadlines.

"Human drug user fees have revolutionized the drug review process in the United States since they were adopted 20 years ago, allowing the FDA to speed the application review process without compromising the agency’s high standards," said FDA Commissioner Margaret Hamburg in a statement.

The PDUFA requires congressional reauthorization every 5 years. The current program expires in September, so the FDA also sent recommendations to Capitol Hill on how the next PDUFA program should look.

A generic drug user fee program is crucial because the agency is being flooded with applications, according to the agency. Generics now account for two-thirds of prescriptions written in the United States. The FDA receives 800-900 generic-drug-related applications each year. With user fees, the agency aims to eliminate a backlog of reviews and "achieve parity between surveillance inspections of foreign and domestic establishments by the 2017 fiscal year." Many generic products are manufactured overseas.

The Generic Pharmaceutical Association said that under the agency plan, the industry would pay $299 million a year for 5 years, beginning in fiscal 2012. In a statement, GPhA lauded the agency for completing its recommendations.

"This is an important landmark that could not have been achieved without the extraordinary efforts of the FDA, my colleagues in the generic industry, and all other stakeholders," said GPhA president and CEO Ralph G. Neas.

For biosimilar drugs – essentially generic forms of biologic products – the FDA said that the fees it recommended would apply to products in development now, to "generate revenue in the near-term and to provide FDA with the resources needed to support development-phase meetings with sponsors of biosimilar biological product candidates."

The agency expects more of these products to come online in the future, in part because the Affordable Care Act created a quicker path to market through a subtitle of the law, the Biologics Price Competition and Innovation Act (BPCI).

The Biotechnology Industry Organization (BIO) did not comment specifically on the biosimilar user fee program, but it has expressed support for the program, with caveats, in the past.

BIO president and CEO Jim Greenwood commended the FDA for completing recommendations on the PDUFA reauthorization, noting in a statement that the law had "contributed to the approval of more than 1,200 new medicines and initially reduced review times for the newest, most innovative drugs by more than a year."

Mr. Greenwood added, "In light of the scientific and regulatory complexities of modern drug development, the PDUFA V recommendations include a set of enhancements that seek to reinforce FDA’s review performance and get back-to-basics for patients with the goal of minimizing development and review issues that can delay patient access to needed treatments."

The Food and Drug Administration has sent recommendations to Congress on establishing user fee programs for generic and biosimilar drugs.

Legislation is required to create the new programs, which would be modeled on the 20-year-old Prescription Drug User Fee Act (PDUFA), according to the agency.

Under PDUFA, the FDA collects fees for certain applications for a new drugs or biologics, and it also assesses fees on certain manufacturing establishments. In fiscal 2012, for instance, a new drug application that includes clinical data costs $1.8 million; establishment fees are $520,000. In return, the FDA agrees to review applications according to set deadlines.

"Human drug user fees have revolutionized the drug review process in the United States since they were adopted 20 years ago, allowing the FDA to speed the application review process without compromising the agency’s high standards," said FDA Commissioner Margaret Hamburg in a statement.

The PDUFA requires congressional reauthorization every 5 years. The current program expires in September, so the FDA also sent recommendations to Capitol Hill on how the next PDUFA program should look.

A generic drug user fee program is crucial because the agency is being flooded with applications, according to the agency. Generics now account for two-thirds of prescriptions written in the United States. The FDA receives 800-900 generic-drug-related applications each year. With user fees, the agency aims to eliminate a backlog of reviews and "achieve parity between surveillance inspections of foreign and domestic establishments by the 2017 fiscal year." Many generic products are manufactured overseas.

The Generic Pharmaceutical Association said that under the agency plan, the industry would pay $299 million a year for 5 years, beginning in fiscal 2012. In a statement, GPhA lauded the agency for completing its recommendations.

"This is an important landmark that could not have been achieved without the extraordinary efforts of the FDA, my colleagues in the generic industry, and all other stakeholders," said GPhA president and CEO Ralph G. Neas.

For biosimilar drugs – essentially generic forms of biologic products – the FDA said that the fees it recommended would apply to products in development now, to "generate revenue in the near-term and to provide FDA with the resources needed to support development-phase meetings with sponsors of biosimilar biological product candidates."

The agency expects more of these products to come online in the future, in part because the Affordable Care Act created a quicker path to market through a subtitle of the law, the Biologics Price Competition and Innovation Act (BPCI).

The Biotechnology Industry Organization (BIO) did not comment specifically on the biosimilar user fee program, but it has expressed support for the program, with caveats, in the past.

BIO president and CEO Jim Greenwood commended the FDA for completing recommendations on the PDUFA reauthorization, noting in a statement that the law had "contributed to the approval of more than 1,200 new medicines and initially reduced review times for the newest, most innovative drugs by more than a year."

Mr. Greenwood added, "In light of the scientific and regulatory complexities of modern drug development, the PDUFA V recommendations include a set of enhancements that seek to reinforce FDA’s review performance and get back-to-basics for patients with the goal of minimizing development and review issues that can delay patient access to needed treatments."

The Food and Drug Administration has sent recommendations to Congress on establishing user fee programs for generic and biosimilar drugs.

Legislation is required to create the new programs, which would be modeled on the 20-year-old Prescription Drug User Fee Act (PDUFA), according to the agency.

Under PDUFA, the FDA collects fees for certain applications for a new drugs or biologics, and it also assesses fees on certain manufacturing establishments. In fiscal 2012, for instance, a new drug application that includes clinical data costs $1.8 million; establishment fees are $520,000. In return, the FDA agrees to review applications according to set deadlines.

"Human drug user fees have revolutionized the drug review process in the United States since they were adopted 20 years ago, allowing the FDA to speed the application review process without compromising the agency’s high standards," said FDA Commissioner Margaret Hamburg in a statement.

The PDUFA requires congressional reauthorization every 5 years. The current program expires in September, so the FDA also sent recommendations to Capitol Hill on how the next PDUFA program should look.

A generic drug user fee program is crucial because the agency is being flooded with applications, according to the agency. Generics now account for two-thirds of prescriptions written in the United States. The FDA receives 800-900 generic-drug-related applications each year. With user fees, the agency aims to eliminate a backlog of reviews and "achieve parity between surveillance inspections of foreign and domestic establishments by the 2017 fiscal year." Many generic products are manufactured overseas.

The Generic Pharmaceutical Association said that under the agency plan, the industry would pay $299 million a year for 5 years, beginning in fiscal 2012. In a statement, GPhA lauded the agency for completing its recommendations.

"This is an important landmark that could not have been achieved without the extraordinary efforts of the FDA, my colleagues in the generic industry, and all other stakeholders," said GPhA president and CEO Ralph G. Neas.

For biosimilar drugs – essentially generic forms of biologic products – the FDA said that the fees it recommended would apply to products in development now, to "generate revenue in the near-term and to provide FDA with the resources needed to support development-phase meetings with sponsors of biosimilar biological product candidates."

The agency expects more of these products to come online in the future, in part because the Affordable Care Act created a quicker path to market through a subtitle of the law, the Biologics Price Competition and Innovation Act (BPCI).

The Biotechnology Industry Organization (BIO) did not comment specifically on the biosimilar user fee program, but it has expressed support for the program, with caveats, in the past.

BIO president and CEO Jim Greenwood commended the FDA for completing recommendations on the PDUFA reauthorization, noting in a statement that the law had "contributed to the approval of more than 1,200 new medicines and initially reduced review times for the newest, most innovative drugs by more than a year."

Mr. Greenwood added, "In light of the scientific and regulatory complexities of modern drug development, the PDUFA V recommendations include a set of enhancements that seek to reinforce FDA’s review performance and get back-to-basics for patients with the goal of minimizing development and review issues that can delay patient access to needed treatments."