WASHINGTON—Passage of legislation aimed at speeding up Food and Drug Administration approvals, combined with an incoming president who has pledged to “cut red tape” at the agency, is expected to usher in a new, more industry-friendly era of drug and device regulation.
The Senate on Wednesday cleared the measure known as the 21st Century Cures bill, which lays out in detail speedier and more pro-industry methods for the FDA to approve new drugs and medical devices. Its supporters, such as PhRMA, the drug-industry association, and AdvaMed, representing device makers, praise it as a way to bring about more innovation and get treatments to patients faster.
Republicans have been pushing for the changes for the past few years, arguing that the FDA takes too long to study scientific evidence and sometimes wrongly insists on large, multiyear clinical studies that delay important treatments to patients.
The bill’s mechanisms allow the FDA to use shorter and simpler studies more widely. Under the measure, certain new antibiotics could see shorter trials, and a fairly wide range of drugs could get additional approvals for new uses based on relatively low amounts of evidence, such as data summaries and data from company registries.
THE TRUMP TRANSITION
Sen. Lamar Alexander (R., Tenn.), chairman of the Senate Health Committee, said the bill “will help us take advantage of the breathtaking advances in biomedical research and bring those innovations to doctors’ offices and patients’ medicine cabinets around the country.”
President Barack Obama said after the vote he would sign the measure “as soon as it reaches my desk.”
The bill includes funding for the “cancer moonshot” program led by Vice President Joe Biden and a precision-medicine initiative endorsed by Francis Collins, the director of the National Institutes of Health, which would receive $4.3 billion over 10 years under the bill.
The White House, too, has said it “supports FDA’s efforts to modernize clinical trial design.”
But the legislation has drawn strong criticism from some medical experts and consumer advocates, who say the legislation in combination with the expressed views of Mr. Trumpwill amount to an unfortunate mix.
“The worry is that this could be a potentially toxic combination, a kind of one-two punch,” said Jerry Avorn, a medical professor at Harvard Medical School. “The bill instructs the FDA to use nonconventional methods to approve drugs, in the hands of a new FDA commissioner with a deregulatory bent.”
Critics and supporters alike agree the measure will give the new administration broad latitude to shape the drug and device approval process as it sees appropriate, and both fully expect the incoming Trump administration to embrace the approach laid out in the Cures bill.
President-elect Donald Trump hasn’t been specific about whom he will select as FDA commissioner. But he has talked about significantly scaling back federal regulations, including food-safety measures.
In a campaign document laying out his priorities for his first 100 days, Mr. Trump said part of his health-care agenda would include “cutting red tape at the FDA.”
“There are over 4,000 drugs awaiting approval, and we especially want to speed the approval of lifesaving medications,” he said.
In medical products, the legislation mostly will allow the FDA to use smaller studies, less scientifically rigorous research and “real-world evidence” for approval of several drugs and medical devices.
For instance, that real-world evidence is expected to include company registries of patients and other evidence that falls short of the current gold-standard—clinical trials with patients randomly assigned to a study drug, or to a placebo or standard treatment. Consumer groups say it will allow companies to put greater pressure on the FDA to regulate combination drug-device products as devices, which are often subject to a relatively lower standard of evidence than drugs.
The measure also will allow far wider use of simpler goals in medical studies called “surrogate endpoints.” This can mean, for instance, measuring biomarkers in the blood or changes in a tumor, rather than harder to prove outcomes, such as measuring the death rate. One of the main areas in which such surrogate goals are now used is for cancer drugs, and consumer advocates say this experience raises concerns based on some recent research.
Researchers from the National Cancer Institute and Oregon Health Sciences University recently looked at all 36 cancer drugs approved by the FDA from January 2008 through December 31, 2012. The drugs were approved based on factors like tumor size or period of time in which the disease didn’t progress. The researchers, Drs. Chul Kim and Vinay Prasad, concluded in the 2015 research in JAMA Internal Medicine that five drugs ultimately showed a survival benefit, 18 didn’t, and that 13 have unknown survival benefits.
Already, more FDA approvals get faster treatment through mechanisms called accelerated, fast-track and expedited approvals.
“We were already seeing weakening of FDA standards under the Obama administration,” said attorney Sarah Sorscher of the Public Citizen Health Research Group. She cited the recent example of the FDA approving a muscular-dystrophy drug that didn’t prove any benefit in outcomes of patients.
The FDA had no comment.
The director of the Public Citizen health group, Michael Carome, said, “Industry will be emboldened by this legislation, and under a deregulation-minded commissioner, will seek further changes in the FDA regulatory scheme.”
With all these changes, the drug industry, with estimated U.S. retail sales of $325 billion annually, and the $150 billion-a-year device industry, are poised to benefit. The industry group PhRMA said the legislation will “improve the discovery and development of new medicines for patients. The legislation includes pro-patient, science-based reforms which enhance the competitive market for biopharmaceuticals.”
Medical-device startups will benefit from a requirement for the FDA to more quickly approve “breakthrough” products that are significant advances over existing treatments, said Stryker Corp. Chief Executive Kevin Lobo in an interview. Mr. Lobo said such provisions could incentivize the development of new devices to treat neurological conditions, a small but growing portion of Stryker’s business.
Still, the law is unlikely to have a significant financial impact on large device companies like Stryker, based in Kalamazoo, Mich., Mr. Lobo said. The bulk of Stryker’s $10 billion in revenue last year came from hip and knee replacement implants, and other established technologies are already approved under less-stringent FDA reviews.
The measure includes a number of provisions that helped secure broad support in the House and Senate. The House passed it Nov. 30, with a 392-26 vote, and the Senate on Wednesday cleared the measure 94-5. In addition to the NIH money, it would make $1 billion available for prevention and treatment of opioid addiction, and it would make mental-health treatment more widely accessible to people with mental illness.
Sen. Jeanne Shaheen (D., N.H.) said in an interview that she supported the bill for what it can do to lessen the “public-health emergency” of opioid overdoses, saying that with treatment and counseling, “it is pretty compelling that people can get off addiction.”
Sen. Susan Collins (R., Maine) said, “I doubt there is a family in America who will not be touched by this important legislation in some way.”
My name is Samual Lau. I am a long-term value investor and a zealous disciple of Ben Graham. And I am a MBA graduated in May 2010 from Carnegie Mellon University. My concentrations are Finance, Strategy and Marketing.
TimelessInvestor
Drug deregulation is coming: Ease FDA Drug and Device Approvals
Drug deregulation is coming: Ease FDA Drug and Device Approvals – this could be big for drug industry, and benefit VRX greatly
http://www.wsj.com/articles/senate-clears-bill-to-ease-fda-drug-and-device-approvals-1481139236
Senate Clears Bill to Ease FDA Drug and Device Approvals
Measure includes money for NIH, cancer moonshot, precision medicine initiative
By
WASHINGTON—Passage of legislation aimed at speeding up Food and Drug Administration approvals, combined with an incoming president who has pledged to “cut red tape” at the agency, is expected to usher in a new, more industry-friendly era of drug and device regulation.
The Senate on Wednesday cleared the measure known as the 21st Century Cures bill, which lays out in detail speedier and more pro-industry methods for the FDA to approve new drugs and medical devices. Its supporters, such as PhRMA, the drug-industry association, and AdvaMed, representing device makers, praise it as a way to bring about more innovation and get treatments to patients faster.
Republicans have been pushing for the changes for the past few years, arguing that the FDA takes too long to study scientific evidence and sometimes wrongly insists on large, multiyear clinical studies that delay important treatments to patients.
The bill’s mechanisms allow the FDA to use shorter and simpler studies more widely. Under the measure, certain new antibiotics could see shorter trials, and a fairly wide range of drugs could get additional approvals for new uses based on relatively low amounts of evidence, such as data summaries and data from company registries.
THE TRUMP TRANSITION
Sen. Lamar Alexander (R., Tenn.), chairman of the Senate Health Committee, said the bill “will help us take advantage of the breathtaking advances in biomedical research and bring those innovations to doctors’ offices and patients’ medicine cabinets around the country.”
MORE U.S. NEWS
President Barack Obama said after the vote he would sign the measure “as soon as it reaches my desk.”
The bill includes funding for the “cancer moonshot” program led by Vice President Joe Biden and a precision-medicine initiative endorsed by Francis Collins, the director of the National Institutes of Health, which would receive $4.3 billion over 10 years under the bill.
The White House, too, has said it “supports FDA’s efforts to modernize clinical trial design.”
But the legislation has drawn strong criticism from some medical experts and consumer advocates, who say the legislation in combination with the expressed views of Mr. Trumpwill amount to an unfortunate mix.
“The worry is that this could be a potentially toxic combination, a kind of one-two punch,” said Jerry Avorn, a medical professor at Harvard Medical School. “The bill instructs the FDA to use nonconventional methods to approve drugs, in the hands of a new FDA commissioner with a deregulatory bent.”
Critics and supporters alike agree the measure will give the new administration broad latitude to shape the drug and device approval process as it sees appropriate, and both fully expect the incoming Trump administration to embrace the approach laid out in the Cures bill.
President-elect Donald Trump hasn’t been specific about whom he will select as FDA commissioner. But he has talked about significantly scaling back federal regulations, including food-safety measures.
In a campaign document laying out his priorities for his first 100 days, Mr. Trump said part of his health-care agenda would include “cutting red tape at the FDA.”
“There are over 4,000 drugs awaiting approval, and we especially want to speed the approval of lifesaving medications,” he said.
In medical products, the legislation mostly will allow the FDA to use smaller studies, less scientifically rigorous research and “real-world evidence” for approval of several drugs and medical devices.
For instance, that real-world evidence is expected to include company registries of patients and other evidence that falls short of the current gold-standard—clinical trials with patients randomly assigned to a study drug, or to a placebo or standard treatment. Consumer groups say it will allow companies to put greater pressure on the FDA to regulate combination drug-device products as devices, which are often subject to a relatively lower standard of evidence than drugs.
The measure also will allow far wider use of simpler goals in medical studies called “surrogate endpoints.” This can mean, for instance, measuring biomarkers in the blood or changes in a tumor, rather than harder to prove outcomes, such as measuring the death rate. One of the main areas in which such surrogate goals are now used is for cancer drugs, and consumer advocates say this experience raises concerns based on some recent research.
Researchers from the National Cancer Institute and Oregon Health Sciences University recently looked at all 36 cancer drugs approved by the FDA from January 2008 through December 31, 2012. The drugs were approved based on factors like tumor size or period of time in which the disease didn’t progress. The researchers, Drs. Chul Kim and Vinay Prasad, concluded in the 2015 research in JAMA Internal Medicine that five drugs ultimately showed a survival benefit, 18 didn’t, and that 13 have unknown survival benefits.
Already, more FDA approvals get faster treatment through mechanisms called accelerated, fast-track and expedited approvals.
“We were already seeing weakening of FDA standards under the Obama administration,” said attorney Sarah Sorscher of the Public Citizen Health Research Group. She cited the recent example of the FDA approving a muscular-dystrophy drug that didn’t prove any benefit in outcomes of patients.
The FDA had no comment.
The director of the Public Citizen health group, Michael Carome, said, “Industry will be emboldened by this legislation, and under a deregulation-minded commissioner, will seek further changes in the FDA regulatory scheme.”
With all these changes, the drug industry, with estimated U.S. retail sales of $325 billion annually, and the $150 billion-a-year device industry, are poised to benefit. The industry group PhRMA said the legislation will “improve the discovery and development of new medicines for patients. The legislation includes pro-patient, science-based reforms which enhance the competitive market for biopharmaceuticals.”
Medical-device startups will benefit from a requirement for the FDA to more quickly approve “breakthrough” products that are significant advances over existing treatments, said Stryker Corp. Chief Executive Kevin Lobo in an interview. Mr. Lobo said such provisions could incentivize the development of new devices to treat neurological conditions, a small but growing portion of Stryker’s business.
Still, the law is unlikely to have a significant financial impact on large device companies like Stryker, based in Kalamazoo, Mich., Mr. Lobo said. The bulk of Stryker’s $10 billion in revenue last year came from hip and knee replacement implants, and other established technologies are already approved under less-stringent FDA reviews.
The measure includes a number of provisions that helped secure broad support in the House and Senate. The House passed it Nov. 30, with a 392-26 vote, and the Senate on Wednesday cleared the measure 94-5. In addition to the NIH money, it would make $1 billion available for prevention and treatment of opioid addiction, and it would make mental-health treatment more widely accessible to people with mental illness.
Sen. Jeanne Shaheen (D., N.H.) said in an interview that she supported the bill for what it can do to lessen the “public-health emergency” of opioid overdoses, saying that with treatment and counseling, “it is pretty compelling that people can get off addiction.”
Sen. Susan Collins (R., Maine) said, “I doubt there is a family in America who will not be touched by this important legislation in some way.”
—Joseph Walker contributed to this article.
Write to Thomas M. Burton at tom.burton@wsj.com
About Timeless Investor
My name is Samual Lau. I am a long-term value investor and a zealous disciple of Ben Graham. And I am a MBA graduated in May 2010 from Carnegie Mellon University. My concentrations are Finance, Strategy and Marketing.