| | | | |  | | By Lauren Gardner | | | |  The FDA's speedy approval of the ALS drug Relyvrio has spurred questions about the agency's reliance on regulatory flexibility. | Amylyx Pharmaceuticals via AP | FLEXING FLEXIBILITY — The FDA’s approach to speed approval of a neurological drug, later proven ineffective, is under scrutiny as the agency faces ongoing pressure from biotechs and patient advocates that want to get potential breakthrough treatments to market as quickly as possible. Amylyx Pharmaceuticals said its Relyvrio, approved in 2022 for amyotrophic lateral sclerosis, failed in a late-stage clinical trial, which would typically be completed before full FDA approval. The agency instead green-lit the product with the understanding that the company would consider pulling it if it couldn’t prove any patient benefit in a Phase III study. FDA officials have signaled a willingness to find novel ways to approve drugs — especially those geared toward rare and debilitating conditions that might not have blockbuster potential — for which it can be difficult or time-consuming to collect data supporting a medication’s efficacy. It’s possible that Relyvrio could be considered a test case of how to use what the FDA calls “regulatory flexibility.” Regulatory experts say the FDA has been inconsistent in deploying this flexibility, with some fretting the agency could overcompensate in future scenarios when a drugmaker might not be amenable to ceasing production. “My worry is that, without a clear off-ramp like there is with accelerated approval, that if FDA relies on regulatory flexibility for other approvals … that companies won’t be as willing to play ball,” said Dr. Reshma Ramachandran, assistant professor at Yale School of Medicine. Because Relyvrio met the clinical endpoint — a key sign of success — in its midstage trial, it wasn’t a candidate for accelerated approval, which the FDA grants on data showing the promise of successful trials. In Relyvrio’s case, the FDA essentially asked drugmaker Amylyx to agree to consider pulling the drug if the late-stage trial failed, said Holly Fernandez Lynch, a bioethics professor at the University of Pennsylvania’s Perelman School of Medicine. In this instance, Fernandez Lynch said, “it was not a slam-dunk case in either direction” for the FDA, but it was within its authority to approve Relyvrio with the information it had at the time. “This might be an example of successful regulatory flexibility, even though the drug didn’t pan out,” she added. FDA response: “If the data are as described by the company, showing no evidence of effectiveness, we expect that the company will move expeditiously towards a voluntary withdrawal, and we will work with the company to facilitate this process,” agency spokesperson April Grant said in a statement. IT’S TUESDAY. WELCOME BACK TO PRESCRIPTION PULSE. Are you coming to our health care summit on Wednesday? Say hi to Lauren! Send news and tips to Lauren Gardner (lgardner@politico.com or @Gardner_LM) or David Lim (dlim@politico.com or @davidalim).
| | | | A message from Express Scripts by Evernorth: With drug prices on the rise, Express Scripts by Evernorth is redefining and reimagining what's possible for the Pharmacy Benefit. Prescriptions come with an easy-to-understand savings statement, detailing the drug’s cost, the consumer cost, and the value generated by Express Scripts. All of this is made simple with one, clear fee. And if we don’t reach our client’s goals, they pay less – guaranteed. Simplicity, savings, and trust – that’s our pharmacy benefit. | | | | | SPECIAL APPEARANCE: Watch USAID's Samantha Power beam live into POLITICO's annual Health Care Summit on Wednesday, March 13, for an exclusive conversation on global health issues, including President Joe Biden's humanitarian aid bridge into Gaza. RSVP required to attend, or watch here. | | | | | | 
In his annual budget request Monday, President Joe Biden proposed increased funding for the FDA. | Evan Vucci/AP Photo | FDA BUDGET BUMP PROPOSED — President Joe Biden proposed $7.2 billion in funding for the FDA in fiscal 2025, a $495 million increase over fiscal 2023 spending levels. Nearly $3.5 billion would come from user fees paid by drug and device manufacturers to offset what the agency spends on the drug approval process, with the administration asking for a $114 million boost to the tobacco user fee program. The rest would come from Congress, Biden requested a 4 percent bump, excluding user fees, over fiscal 2023 discretionary spending for the FDA. Because the federal government is still operating under a continuing resolution, fiscal 2024 levels are not final. Biden’s budget blueprint also includes a legislative proposal to allow biosimilar drugs — copycat versions of biologic medicines, or those derived from cells — to be used interchangeably with their name-brand counterparts without the FDA making a formal determination. Currently, the FDA requires biosimilar manufacturers to submit additional data to support their products being labeled “interchangeable,” which allows pharmacies to swap that version for the branded medicine without needing a doctor's approval. POLITICO’s Chelsea Cirruzzo has more on the budget’s impact on health policy, including an expansion of Medicare’s drug price negotiation program. But the usual caveats apply: The president’s budget in the final year of his term is essentially a wishlist, and any major asks are likely on life support heading into the November election. ICYMI: LILLY DRUG DELAYED — The FDA will hold an unexpected advisory committee meeting on Eli Lilly’s proposed Alzheimer’s disease drug, donanemab, delaying a regulatory decision for months, the company said Friday. A meeting date has not yet been announced.
| | | | JOIN US ON 3/21 FOR A TALK ON FINANCIAL LITERACY: Americans from all communities should be able to save, build wealth, and escape generational poverty, but doing so requires financial literacy. How can government and industry ensure access to digital financial tools to help all Americans achieve this? Join POLITICO on March 21 as we explore how Congress, regulators, financial institutions and nonprofits are working to improve financial literacy education for all. REGISTER HERE. | | | | | | | | | BLUEBIRD INKS COVERAGE DEAL — Bluebird Bio has signed its first Medicaid outcomes-based agreement with Michigan to cover Lyfgenia, its gene therapy to treat sickle cell disease. Medicaid insures about half of those living with the disease in the U.S., the company said. It’s pursuing such contracts to encourage state Medicaid agencies to cover the $3.1 million one-time treatment cost by linking reimbursement to how well patients do with the drug. The company has developed contract options for payers, such as Medicaid, that offer “meaningful risk-sharing tied to” disease-associated pain crises that lead to hospitalization, which Bluebird Bio said is “a claims-based metric that is directly correlated with clinical benefit.”
| | | | A message from Express Scripts by Evernorth:   | | | | | Medical device industry group AdvaMed hired law and lobbying firm Alston & Bird to work on coverage and payment issues for artificial intelligence-based technology.
| | | | DON’T MISS POLITICO’S HEALTH CARE SUMMIT: The stakes are high as America's health care community strives to meet the evolving needs of patients and practitioners, adopt new technologies and navigate skeptical public attitudes toward science. Join POLITICO’s annual Health Care Summit on March 13 where we will discuss the future of medicine, including the latest in health tech, new drugs and brain treatments, diagnostics, health equity, workforce strains and more. REGISTER HERE. | | | | | | | | | The FDA’s label expansion for the weight-loss drug Wegovy to include treating cardiovascular risks could convince more insurers to cover the pricey medication, The Washington Post reports.
| | The FDA revised draft guidance Monday to help drugmakers develop treatments for the early stages of Alzheimer’s disease.
| | | | A message from Express Scripts by Evernorth: With drug prices on the rise, Express Scripts by Evernorth is redefining and reimagining what's possible for the Pharmacy Benefit.
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