Post by icemandios on Jun 8, 2023 2:36:18 GMT
FDA commissioner says drug prices are too high, calls for better evidence generation
Nicole DeFeudis
Editor
FDA Commissioner Rob Califf told BIO CEO Rachel King on Wednesday that it’s “impossible to argue that the US is doing well in health,” highlighting high drug prices and misinformation as two key challenges.
The commissioner also took the opportunity to discuss accelerated approval reforms, attrition at the agency and the Inflation Reduction Act, which came into focus earlier this week when Merck filed a lawsuit calling parts of the drug pricing law a sham. Industry critics have argued that the legislation will have a chilling effect on innovation.
When asked about the IRA, Califf responded that “the prices of drugs are too high.”
“We have to come up with a better system. And the IRA is an effort to do that,” he said, later adding that “all of these big laws have imperfections,” but he’s “not at liberty to talk about specific details of what I think is imperfect.”
He discussed ramping up communications with CMS, not just as part of the IRA, but “on multiple fronts,” adding that “one thing we’re talking about is what to do about obesity drugs right now because CMS is prohibited from paying for obesity drugs if that’s the only indication.”
“There’s something missing in the equation by which we translate all this amazing innovation into products and services that help the population,” he said generally.
CMS said earlier this year that it intends to work closely with the FDA on accelerated approval payment reforms in a pilot program designed to “reduce Medicare spending on drugs that have no confirmed clinical benefit.”
While Califf noted that he’s “totally in favor” of the accelerated approval pathway in serious, life-threatening circumstances of unmet need, he added that “by and large, the industry has not done a great job with some of the follow-up studies” that are required for full approval.
“That’s created a bit of an appearance problem and we need to fix that,” he said.
Califf has made accelerated approval reforms a key priority, arguing last year that “we need more teeth,” especially around swifter confirmatory studies. A majority of confirmatory trials are late and delayed past the FDA’s requested completion dates, according to a research letter published earlier this year by law and medical professors in JAMA Health Forum. In an October report, HHS’ inspector general said that Medicare and Medicaid spent more than $18 billion from 2018 to 2021 for therapies with incomplete confirmatory trials past their original planned completion dates.
Under the Food and Drug Omnibus Reform Act (FDORA), the FDA has new authority to require the launch of confirmatory trials prior to an accelerated approval, and the ability to expedite the withdrawal process when a confirmatory trial doesn’t show benefit.
When asked about reimbursement decisions for accelerated approvals, Califf said Wednesday, “We have to face the reality that an accelerated approval is not based on clinical outcomes. It’s based on reasonably likely non-validated surrogate endpoints.”
“My main desire is to really rev up our evidence generation system so that we actually know quickly where a treatment is effective,” he added.
“We need to really ramp up the generation of evidence, and I’m seeking your help. Please criticize the FDA,” Califf said. “Think of us as umpires. We’d love to see a great game played by super players.”
Editor
FDA Commissioner Rob Califf told BIO CEO Rachel King on Wednesday that it’s “impossible to argue that the US is doing well in health,” highlighting high drug prices and misinformation as two key challenges.
The commissioner also took the opportunity to discuss accelerated approval reforms, attrition at the agency and the Inflation Reduction Act, which came into focus earlier this week when Merck filed a lawsuit calling parts of the drug pricing law a sham. Industry critics have argued that the legislation will have a chilling effect on innovation.
When asked about the IRA, Califf responded that “the prices of drugs are too high.”
“We have to come up with a better system. And the IRA is an effort to do that,” he said, later adding that “all of these big laws have imperfections,” but he’s “not at liberty to talk about specific details of what I think is imperfect.”
He discussed ramping up communications with CMS, not just as part of the IRA, but “on multiple fronts,” adding that “one thing we’re talking about is what to do about obesity drugs right now because CMS is prohibited from paying for obesity drugs if that’s the only indication.”
“There’s something missing in the equation by which we translate all this amazing innovation into products and services that help the population,” he said generally.
CMS said earlier this year that it intends to work closely with the FDA on accelerated approval payment reforms in a pilot program designed to “reduce Medicare spending on drugs that have no confirmed clinical benefit.”
While Califf noted that he’s “totally in favor” of the accelerated approval pathway in serious, life-threatening circumstances of unmet need, he added that “by and large, the industry has not done a great job with some of the follow-up studies” that are required for full approval.
“That’s created a bit of an appearance problem and we need to fix that,” he said.
Califf has made accelerated approval reforms a key priority, arguing last year that “we need more teeth,” especially around swifter confirmatory studies. A majority of confirmatory trials are late and delayed past the FDA’s requested completion dates, according to a research letter published earlier this year by law and medical professors in JAMA Health Forum. In an October report, HHS’ inspector general said that Medicare and Medicaid spent more than $18 billion from 2018 to 2021 for therapies with incomplete confirmatory trials past their original planned completion dates.
Under the Food and Drug Omnibus Reform Act (FDORA), the FDA has new authority to require the launch of confirmatory trials prior to an accelerated approval, and the ability to expedite the withdrawal process when a confirmatory trial doesn’t show benefit.
When asked about reimbursement decisions for accelerated approvals, Califf said Wednesday, “We have to face the reality that an accelerated approval is not based on clinical outcomes. It’s based on reasonably likely non-validated surrogate endpoints.”
“My main desire is to really rev up our evidence generation system so that we actually know quickly where a treatment is effective,” he added.
“We need to really ramp up the generation of evidence, and I’m seeking your help. Please criticize the FDA,” Califf said. “Think of us as umpires. We’d love to see a great game played by super players.”