Lawmakers press FDA on clinical trial holds

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Mar 28, 2023 View in browser   By Katherine Ellen Foley and David Lim With Krista Mahr and Alice Miranda Ollstein Driving the Day The House Energy and Commerce Health Subcommittee wants to know why the FDA suspends or delays so many clinical trials related to cell and gene therapies. | Getty Images E&C: WHY SO MANY CLINICAL HOLDS? Top House lawmakers wrote to Peter Marks, the director of the FDA Center for Biologics Evaluation and Review, asking why the agency so frequently issues clinical trial holds to experimental cell and gene therapies. House Energy and Commerce Health Subcommittee Chair Brett Guthrie (R-Ky.) and ranking member Anna Eshoo (D-Calif.) in a letter Sunday pointed to recent press reports that show 40 percent of all suspended or delayed clinical trials involve cell and gene therapy studies, which they suggest could be avoided by improving communication between sponsors and the FDA. “CBER should not, for example, use clinical holds as a means for FDA to gain additional time to review a clinical protocol,” Guthrie and Eshoo wrote. “Unless patients are exposed to immediate and serious risks, FDA’s regulations require it to discuss deficiencies with researchers and sponsors and attempt to satisfactorily resolve the matter. IT'S TUESDAY. WELCOME TO PRESCRIPTION PULSE. We were curious about the burning rubber smell in D.C. on Monday, which might be due to wildfires in North Carolina. Send news and tips to David Lim (dlim@politico.com or @davidalim) or Katherine Ellen Foley (kfoley@politico.com or @katherineefoley). TODAY ON OUR PULSE CHECK PODCAST, host Megan Messerly talks with POLITICO’s Natalie Fertig, who recently sat down with Dr. Wilson Compton, the deputy director of the National Institute on Drug Abuse, to chat about the known health effects of cannabis and how scientists' limited access to cannabis products sold in state-legal dispensaries is thwarting effective research. Listen to today’s Pulse Check podcast Eye on the FDA A treatment for ALS will be the topic of an upcoming FDA advisory committee meeting. | Manuel Balce Ceneta/AP Photo ADVISERS TO CONVENE ON ALS CELL THERAPY — BrainStorm Cell Therapeutics said Monday that the FDA will schedule an advisory committee meeting to discuss its cell therapy candidate NurOwn for the treatment of amyotrophic lateral sclerosis. Last November, the agency rejected BrainStorm’s application to market NurOwn, which converts patients’ stem cells into those that coax nerve growth. BrainStorm formally protested the decision, leading the FDA to hold a public meeting of the agency’s Cellular, Tissue and Gene Therapies Advisory Committee to discuss NurOwn’s application. No meeting date has been announced. BrainStorm completed Phase III clinical trials for the treatment in October 2020. The company said that NurOwn benefitted study participants by some metrics. But the FDA said in March 2021 that it disagreed with the company’s interpretation of the data. FDA CLARIFIES ADDICTION TREATMENT POLICIES — On Monday, the FDA said it is notifying pharmacists that they no longer need to verify that prescriptions for buprenorphine, a drug used to treat opioid use disorder, come from providers with the so-called “X-waiver,” Krista reports. Backstory: The X-waiver — which required medical providers to undergo lengthy training to prescribe the life-saving drug — was eliminated in the omnibus spending bill of 2023. Now, any provider federally licensed to prescribe controlled substances can prescribe the drug, which is an opioid. The FDA previously required pharmacists to ensure that prescriptions came from doctors who had the X-waiver. Why it matters: To reduce U.S. overdose deaths, the Biden administration has been trying to improve access to buprenorphine. But patients nationwide have had difficulty filling their buprenorphine prescriptions, as POLITICO reported earlier this month, because some pharmacists and distributors are wary of running afoul of the federal agencies that regulate the drug. OTC NALOXONE DECISION FORTHCOMING — The FDA is slated to make a decision by Wednesday on Emergent BioSolution’s bid for making its naloxone nasal spray, Narcan, available without a prescription. In a February advisory committee meeting, experts voted unanimously that the agency approve the drug for sale in convenience stores, gas stations and pharmacies. What we’re watching: Right now, a two-pack of Narcan costs $70. It’s unclear how the company will price it for over-the-counter purchases, but experts have said that cost will be a factor in accessibility. ONE TRIAL TO RULE THEM ALL — Companies designing cancer drugs might be able to use a single randomized controlled clinical trial for accelerated approval and confirmation of clinical benefit, which is required for full approval, instead of two separate randomized controlled studies, according to a draft guidance published by the FDA on Friday. “Given the limitations of single-arm trials, a randomized controlled trial is the preferred approach to support an application for accelerated approval. Sponsors can, as appropriate, elect to conduct a single randomized controlled trial to support an accelerated approval and to verify clinical benefit (i.e., follow a “one-trial” approach) or, they can conduct separate trials — one to support the accelerated approval and another, a confirmatory trial, to verify clinical benefit,” says the document.   JOIN POLITICO ON 4/5 FOR THE 2023 RECAST POWER LIST: America’s demographics and power dynamics are changing — and POLITICO is recasting how it covers the intersection of race, identity, politics and policy. Join us for a conversation on the themes of the 2023 Recast Power List that will examine America’s decision-making tables, who gets to sit at them, and the challenges that still need to be addressed. REGISTER HERE.     In Congress MORE QUESTIONS FOR THE FDA — Top Republicans on the House Energy and Commerce Committee are asking the FDA for a slate of data on recent drug shortages for key medications, including albuterol, methotrexate, amoxicillin and acetaminophen. “The FDA has also requested additional legal authority from Congress to gain improved access to medical supply-chain information,” Energy and Commerce Committee Chair Cathy McMorris Rodgers (R-Wash.), Oversight Subcommittee Chair Morgan Griffith (R-Va.) and Guthrie wrote to the FDA on Monday. “However, it is not clear to the Committee that FDA is effectively using its existing authorities.” The three lawmakers also wrote in a separate letter that the Energy and Commerce Committee is investigating the “adequacy” of the FDA’s oversight of agency-funded research “that may pose significant biosafety or biosecurity risks.” At a hearing last week, Democrats in the Senate Homeland Security and Governmental Affairs Committee raised concerns about the threat of drug shortages and noted in a newly published report that shortages had increased by 30 percent between 2021 and 2022. In the Courts OHIO SUES EXPRESS SCRIPTS, PRIME THERAPEUTICS — A lawsuit filed by Ohio Attorney General Dave Yost on Monday contends that pharmacy benefit managers Express Scripts and Prime Therapeutics used Ascent Health Services, a group purchasing organization, to share information about each other’s pricing, discount and rebate data. “Armed with this wealth of competitively-sensitive pricing and negotiations information about each other and additional rivals, Express Scripts, Prime Therapeutics, and Ascent’s PBM customers have been able to act in concert to harmonize their manufacturer negotiations and demands, effectively eliminating all competition between themselves and further ensuring they continue to profit from supracompetitive drug prices,” the lawsuit states. Yost’s lawsuit, filed in the Delaware County Common Pleas Court, claims the companies violated Ohio’s antitrust law, the Valentine Act, which prohibits price-fixing and other measures that restrain competition. Express Scripts did not respond to a request for comment. “Prime does not comment on pending litigation,” Prime Therapeutics spokesperson Jenine Anderson wrote in an email. AMGEN V. SANOFI SCOTUS SHOWDOWN — The Supreme Court heard arguments on Monday in Amgen Inc. v. Sanofi, a closely watched lawsuit in which justices are being asked to determine whether Amgen’s patent for cholesterol-lowering antibodies is too broad. The lawsuit could have widespread impacts on patent law, not only on drugmakers but also possibly other corporate patent holders, but it is difficult to tell how the court is leaning, according to Kisuk Lee, a pharmaceutical patent attorney at Harness IP. “It’s hard to tell; the Supreme Court was quite neutral,” Lee told Prescription Pulse. One possible outcome is that the court proposes a new way of assessing whether a patent enables a person or a company to make and use an invention, he said. Coronavirus BIONTECH FORECASTS VACCINE SALES — In an earnings statement Monday, BioNTech executives said they expect Covid-19 vaccines to bring in roughly $5.4 billion in 2023, down from more than $18 billion the year before. The company cited the anticipated switch to a commercial market as one reason it expects decreased sales. FDA FURTHER DETAILS COVID TRANSITION PLAN — On Friday, the FDA’s medical device center issued two guidances laying out how companies with Covid-19 products under emergency use authorization or enforcement discretion should plan to transition their devices to traditional marketing authorization. “CDRH encourages those who want to continue marketing their device and need to prepare a marketing submission to use this transition period wisely and submit a pre-submission, if needed,” said Jeff Shuren, director of the Center for Devices and Radiological Health, and William Maisel, director of CDRH’s Office of Product Evaluation and Quality. Document Drawer The FDA published final guidance for the safety and performance of soft contact lenses.   STEP INSIDE THE WEST WING: What's really happening in West Wing offices? Find out who's up, who's down, and who really has the president’s ear in our West Wing Playbook newsletter, the insider's guide to the Biden White House and Cabinet. For buzzy nuggets and details that you won't find anywhere else, subscribe today.       Follow us on Twitter David Lim @davidalim Lauren Gardner @Gardner_LM Katherine Ellen Foley @katherineefoley   Follow us

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