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Presented by Rare Access Action Project: Delivered every Tuesday and Friday by 12 p.m., Prescription Pulse examines the latest pharmaceutical news and policy. | | | | | |  | | By David Lim and Lauren Gardner | Presented by Rare Access Action Project | With Katherine Ellen Foley. | | | — Moderna’s Covid-19 vaccine provides robust protection six months after inoculation, the company said Thursday. But it’s still planning to make booster shots. — Novavax doesn’t plan to petition the FDA for emergency use authorization for its Covid shot until the fourth quarter. — HHS’s inspector general has launched an inquiry into FDA’s accelerated approval process for drugs amid continued blowback over its green-light to Aduhelm. It’s Friday, welcome back to Prescription Pulse. Rafael Nadal falling in love with DC sparked joy this week. Be sure to send tips to David Lim (dlim@politico.com or @davidalim), Lauren Gardner (lgardner@politico.com or @Gardner_LM) and Katherine Ellen Foley (kfoley@politico.com or @katherineefoley). | | | | A message from Rare Access Action Project: The $3.5 trillion budget would reduce lifesaving treatments and cures and make it harder for America’s 25 million rare disease patients to have access to the medications they need — and the cures they hope for. There is a better way. Find out more at RareAccessActionProject.org | | | | | | MODERNA SHOT HIGHLY EFFECTIVE 6 MONTHS OUT, BUT BOOSTERS LOOM — Despite the company’s findings on its shot’s efficacy, Moderna executives said during a second-quarter earnings call they expect immunity to wane and eventually cut into that 93 percent figure — which is why they believe a third booster dose will be necessary ahead of the winter months. The virulent Delta variant, alongside public ennui with pandemic-related restrictions and increased indoor activities as the weather cools, will likely spell more breakthrough infections, Moderna President Stephen Hoge said. The company has tested three booster candidates at half and full doses, as compared to the current two 100-microgram doses administered, and half a dose of the existing formulation appears to provide “sufficient” protection, Hoge said. However, Moderna is waiting on data from its full-dose studies of the three boosters before taking its case to the FDA. Meanwhile, Moderna expects to complete its rolling application to the FDA for licensing its two-shot regimen this month. NOVAVAX TO PUNT EUA FILING TO Q4 — The company, which has been plagued by production issues, disclosed ahead of its second-quarter earnings call Thursday that it would again push back the timeline for filing for an EUA, which had been expected in the third quarter. “Completing the work to get all of our analytics, potency and purity assays validated and qualified is progressing, despite being a time-consuming process,” spokesperson Amy Speak told POLITICO. Still, CEO Stan Erck was bullish on the U.S. government’s continued interest in getting a protein-based vaccine like Novavax’s to market, and said that the company could finalize its submission “early” in the fourth quarter. "We’re talking weeks here, we’re not talking months,” he said on an earnings call. Meanwhile: FDA is expected to approve Pfizer’s bid to fully license its Covid vaccine for individuals 16 and older by early September, POLITICO’s Sarah Owermohle and Adam Cancryn report. | | | HHS WATCHDOG TO PROBE FDA’S USE OF ACCELERATED APPROVAL PROCESS — The HHS Office of Inspector General launched an investigation into the FDA’s modified approval process that allows drugs to hit the market using surrogate endpoints. The investigation was spurred by the agency’s approval of Aduhelm, a drug targeting proteins associated with Alzheimer’s disease, which some experts argue hasn’t shown clinical benefits to patients in trials. The investigation will focus on "interactions between the FDA and outside parties as well as other aspects of the process, such as deciding on this pathway and scientific disputes," the Office of the Inspector General said, and will finalize a report in 2023. | | | | 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. | | | | | | | | SENATE PANEL APPROVES FDA SPENDING BILL — The Senate Appropriations Committee signed off Wednesday on a $25.86 billion bill, S. 2599 (117) , to fund the Department of Agriculture and the FDA. The measure, approved in a 25-5 vote, would direct $3.4 billion to the FDA. This caught our eye from the committee report: “The Committee is concerned with reports on the FDA’s objectiveness in approving new drug applications and potential conflicts of interest. The FDA exists to be a trusted regulatory body to review the merits of drug applications, and as such, the Committee instructs the Inspector General to review conflicts of interest between agency employees and companies, including consulting firms, interested in receiving drug approvals and the FDAs review of these drugs, and report back to the Committee on the results of this review.” | | | INFLUENTIAL DRUG PRICING PANEL RELEASES FINAL ADUHELM ANALYSIS — The Alzheimer’s drug from Biogen should be priced at $3,000 to $8,400 annually, representing an 85 percent to 95 percent discount from its original sticker tag, analysts from Institute for Clinical and Economic Review wrote in a report published Thursday. The assessment comes from the conclusion that the actual benefits of the drug are “insufficient,” experts write. A word for clinicians...: ICER also recommends that health care providers discussing the drug with patients should set realistic expectations for the drug, avoiding words like “improvement” or “return of quality of life” in their discussions. ...and a word for the FDA: The “insufficient” evidence label comes in part from the ambiguity over whether the FDA’s surrogate measure of success — the reduction of amyloid protein in the brain — actually improves patients’ lives. The panel suggests the FDA come up with a concrete measure of how much amyloid reduction results in better qualities of life. Meanwhile, a clinical trial quandary: As part of Aduhelm’s approval, FDA required Biogen to conduct a randomized, controlled post-marketing trial. However, experts lament that conducting this kind of trial with the drug already on the market creates ethical and practical dilemmas for the study’s designers and potential participants. | | | BARDA INVESTS IN ORAL FLU VACCINES — The Biomedical Advanced Research and Development Authority, or BARDA, awarded $750,000 in funding to US Biologics, a Memphis, Tenn.-based company, to begin pre-clinical testing of two forms of oral flu vaccines. This investment falls under BARDA’s Division of Research Innovation and Ventures, which in part funds the development of more accessible vaccines that don’t require needles, syringes, vials and cold-chain distribution. “Current influenza vaccines have complex manufacturing processes and cold chain requirements that, in a pandemic, may result in critical delays in making vaccination available to the U.S. and the world,” BARDA said in a press release. US Biologics previously focused on developing oral vaccines for wildlife and livestock. The company is pivoting its platform for clinical use, which involves adapting it to humans’ “hostile, species-specific” digestive tract, Jolieke van Oosterwijk, US Biologic’s chief science officer, told POLITICO. Previously, BARDA awarded Vaxart, a San Francisco-based biotech company, $14 million for the development of its oral flu vaccine; it’s currently in Phase II clinical trials. | | | |   | | | | | | FDA DEBUTS NEW DEVICE SHORTAGE REPORTING PORTAL — Medical device manufacturers can now notify the FDA of potential shortages via a new online portal. The agency is asking for an estimated duration, if the Covid-19 pandemic has affected the ability of firms to make or distribute products, how much inventory is on hand, and other information. SHUREN SAYS FDA PARTICIPATING IN MORE COLLABORATIVE COMMUNITIES — Top device regulator Jeff Shuren announced Wednesday that the FDA is now partaking in 12 external groups focused on “charting paths to accelerate and address regulatory science and other knowledge gaps to aid in medical device review and oversight.” See the list here. TAP WATER A NO-NO IN NETI POTS, FDA WARNS — People using nasal irrigation devices like Neti Pots should not use tap water to flush out sinuses due to a rare risk of fatal infection, FDA cautioned Wednesday. Instead, distilled or sterile water, water passed through certain filters that can trap infectious organisms, or boiled and cooled tap water should be used. PHILIPS RECALLS MORE THAN 16K VENTILATORS — Philips Respironics is recalling certain V60 and V60 Plus Ventilators due to a risk patients may get reduced oxygen, according to an FDA alert Tuesday. ILLUMINA TO BEEF UP DC LOBBYING AMID ANTITRUST CHALLENGE — Illumina CEO Francis deSouza told investors Thursday that the company — which is attempting to acquire Grail despite FTC and European Commission concerns — “can do better” establishing a rapport with Capitol Hill and federal agencies. “One of the things we have learned is we don't have a DC presence, and we don't have employees on the Hill that are telling the Illumina story to the various agencies, to the other folks on the Hill,” deSouza said. “And it's become clear to us that we need a bigger presence there and so we’re starting to build our presence.” | | | HeartFlow announced Wednesday John Farquhar will be the company’s new chief operating officer. AAHomecare is enlisting The Nickles Group to lobby Capitol Hill and federal agencies. | | | FLORIDA WEIGHS VOUCHERS FOR KIDS WHO WANT TO AVOID MASK MANDATES — Florida’s Department of Education pitched an emergency rule on Thursday that would allow parents to transfer their children to a private school or even another school district under the “Hope Scholarship” voucher program to avoid mask mandates, POLITICO’s Andrew Atterbury reports. The state’s seven-member board will vote on the rule today. | | | | Be a Policy Pro. POLITICO Pro has a free policy resource center filled with our best practices on building relationships with state and federal representatives, demonstrating ROI, and influencing policy through digital storytelling. Read our free guides today . | | | | | | | | FRANCE TO OFFER COVID VACCINE BOOSTER TO VULNERABLE POPULATIONS — France will offer seniors and vulnerable people a booster dose of the COVID-19 vaccine, going against the call from the World Health Organization for a moratorium on third doses, POLITICO Europe’s Helen Collis reports. Israel, Hungary, Germany and the U.K. have announced similar programs. | | | FDA: Don’t worry about CDC test withdrawal — On a “town hall” meeting call with diagnostics manufacturers this week, the FDA offered up a simple plea to companies: There’s no need to worry about the impending withdrawal of the CDC’s Emergency Use Authorization for its Real-Time RT-PCR Diagnostic Panel. That diagnostic test was first authorized in February 2020 at the start of the pandemic and is intended for use in high-complexity laboratories. The withdrawal was a voluntary decision on the part of the CDC, the FDA said. CDC has indicated the withdrawal is meant to allow it to transition to the use of multiplex assays in its laboratories, which can identify and differentiate between SARS-CoV-2 and influenza A/B. The FDA did caution manufacturers that while the withdrawal doesn’t signal any shift, companies that are using the CDC’s test to validate new tests should work to complete that testing before the CDC’s test is withdrawn in January 2022. | | | ProPublica’s Neil Bedi takes a deep dive into the FDA’s response to safety issues with Medtronic’s HeartWare Ventricular Assist Device. | | | FDA will hold a virtual public workshop on applications of complex and innovative trial design in pediatric clinical trials on Sept. 1-2. FDA is extending the comment period for its draft guidance on remanufacturing of medical devices by 30 days. | | | | A message from Rare Access Action Project: The $3.5 trillion budget would reduce lifesaving treatments and cures. That’s right. Congress is debating a bill that would make it harder for America’s 25 million rare disease patients to have access to the medications and treatments they need — and the cures they hope for.
Rare disease research has made incredible progress in recent years and Congress wants to stop that to pay for pet projects. There is a better way, one that works without forcing European-style price controls. We must protect the innovations that will save vulnerable lives.
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