What goes into the new ALS drug’s high cost?

Presented by the Foundation for Pharmacy Compounding: Delivered every Tuesday and Friday by 12 p.m., Prescription Pulse examines the latest pharmaceutical news and policy.

Oct 04, 2022 View in browser   By Katherine Ellen Foley and Lauren Gardner Presented by the Foundation for Pharmacy Compounding Drug Pricing The much-debated drug for ALS won approval by the FDA. | Amylyx Pharmaceuticals via AP NEW ALS DRUG COMES WITH A SIX-FIGURE PRICE TAG — Relyvrio, the novel amyotrophic lateral sclerosis drug from Amylyx, has a sticker price of $158,000 annually for patients, the company said in an investor call on Friday. Sticker shock: Patient groups and other experts were excited about having a third drug available to treat ALS, the fatal neurodegenerative condition that afflicts roughly 30,000 people in the U.S. The company expects to fill prescriptions for the drug in four to six weeks. But Amylyx's pricing of the drug raised eyebrows — particularly with the limited data on its efficacy. The drug is a combination of a widely available supplement and an existing drug for ALS. The FDA approved the product, which can be mixed into water or given via feeding tube, based on a Phase II trial with 137 patients, while also noting the dearth of treatments for the condition. In the study, the drug slowed some disease progression. An ongoing Phase III trial for the drug should finish in 2024. In August, the Institute for Clinical and Economic Review estimated that, given the data behind the drug, it would be cost-effective if priced at $9,100 to $30,600 annually. "This price balances the needs of and input from the ALS community, will be supported by insurers, allows Amylyx to sustain programs to ensure that anyone who can benefit from Relyvrio can access it, and enables us to continue our mission," Josh Cohen and Justin Klee, the co-CEOs of the company, said in a statement. But other experts disagree. "We're not talking about them setting a price to recoup their investments or anything like that," Stacie Dusetzina, an associate professor of health policy at Vanderbilt University Medical Center, told Katherine. "This is basically taking advantage of the fact that we allow the price to be what the market will bear." The good news: Amylyx is betting that public and private insurers will cover the drug and said it was committed to eliminating co-pays for those with commercial insurance. It also said the drug will be free for uninsured patients unable to pay. Dusetzina noted that, even if insurance companies cover the drug, it's still important to confirm the patient benefit from a cost standpoint. "We all pay for the premiums of our health plans," she said. It's Tuesday. Welcome to Prescription Pulse, where we're turning on our heaters for the first time this year. Send tips, feedback and your favorite tips for keeping toasty to David Lim (dlim@politico.com or @davidalim), Lauren Gardner (lgardner@politico.com or @Gardner_LM) or Katherine Ellen Foley (kfoley@politico.com or @katherineefoley). Want more Pulse? Today in our Pulse Check podcast, Megan Messerly on the Biden administration's slow start on using Medicaid to expand abortion rights. Plus, Megan Wilson on the dialysis patients and providers squaring off with health plans over a coverage bill. Listen to today's Pulse Check podcast.   A message from the Foundation for Pharmacy Compounding: Patient access to compounded hormones is at risk Compounded hormone therapy can provide personalized and flexible treatments, instead of the one-size-fits-all limitations of some commercial drugs. Millions of women and men in the U.S. rely upon compounded to provide relief from debilitating physical and cognitive symptoms. Yet, even with the widespread benefits of personalized hormone replacement options, patients and physicians are facing possible federal regulatory restrictions that would curb use of these life-supportive treatments. More...   Industry Intel Some members of Congress want to overhaul the market for antibiotics. | Joe Raedle/Getty Images WILL CONGRESS ACT ON SUPERBUGS IN THE LAME DUCK? — A bipartisan group of lawmakers is pushing leadership to prioritize legislation after the midterms that they say would help head off the next major public health crisis by overhauling the market for antibiotics, Lauren and POLITICO's Krista Mahr report. Antimicrobial resistance — when bacteria and parasites mutate to dodge tried-and-true medicines — worsened during the Covid-19 pandemic as the drugs were overprescribed. Pharma companies and infectious disease experts say the status quo in the market does little to encourage development of new drugs that must be used sparingly. That's where the legislation, dubbed the PASTEUR Act, would step in by creating a "subscription" model for antimicrobial drugs that decouples payments to drug companies from how much medicine they sell. Public health advocates say that would help them survive financially while preserving powerful new drugs for infections that don't respond to any other treatment. But whether Congress passes the bill remains to be seen. There's an opening for health legislation to move in the lame-duck session, with key FDA programs needing reauthorization after Dec. 16. But a hefty price tag and general inertia threaten to stymie those efforts. PFIZER ACQUIRES MIGRAINE DRUGMAKER — Pfizer said Monday it has acquired Biohaven Pharmaceuticals, which makes the migraine drug Nurtec, for about $11.6 billion.   SUBSCRIBE TO POWER SWITCH: The energy landscape is profoundly transforming. Power Switch is a daily newsletter that unlocks the most important stories driving the energy sector and the political forces shaping critical decisions about your energy future, from production to storage, distribution to consumption. Don't miss out on Power Switch, your guide to the politics of energy transformation in America and around the world. SUBSCRIBE TODAY.     Coronavirus FDA: EVUSHELD MAY NOT PROTECT AGAINST ALL VARIANTS — The FDA updated its fact sheet for Evusheld on Monday, warning health care providers that the Covid prophylactic has various degrees of reduced neutralizing activity against different Omicron subvariants. Providers should monitor which variants are circulating widely in their communities and evaluate patients for antiviral treatments if they develop the disease, the agency said. Around the Agencies IRA'S BIOSIMILARS PROVISION GOES INTO EFFECT — The Biden administration began implementing one of the first pieces of the Inflation Reduction Act on Monday, temporarily increasing Medicare payments for certain biosimilars. The goal of the five-year bonus Part B payment is to "foster competition in the drug marketplace for conditions such as diabetes, cancer and immune disorders" while improving access, said CMS Administrator Chiquita Brooks-LaSure. ICYMI: The White House touted an HHS analysis on Friday showing that more than 1,200 prescription drugs could have been subjected to the law's Medicare inflation rebates if the law had been in place from July 2021 through July 2022. Oct. 1 marked the first day of the new law's 12-month period over which manufacturers must later pay rebates should a Medicare Part D drug's price hike exceed inflation.   A message from the Foundation for Pharmacy Compounding:   Eye on the FDA FDA OPIOID REVIEW BREADCRUMBS — Commissioner Robert Califf on Friday tweeted a blog post under his name offering an update on implementation of 2017 recommendations from the National Academies of Sciences, Engineering, and Medicine on how the agency should approve and monitor opioids. Of note: The FDA is "exploring the need" for new authority to require makers of new opioids to prove their drugs offer safety advantages over existing products. This is similar to what Califf's told Congress in the spring , noting he lacks the power to demand that those manufacturers prove new drugs are superior to what's on the market. Currently, the FDA must compare the new drug to a placebo. The Ohio State review of the FDA's opioids work will include recommendations for the agency to take under both new and existing authorities, Califf wrote. Tobacco ALTRIA ENDS ITS EXCLUSIVITY WITH JUUL — On Friday, Altria shared that it would end its noncompete agreement with the e-cigarette maker Juul, which has been in place since 2018 when Altria purchased a 35 percent share of the startup for $12.8 billion. The original agreement between the two companies prohibited Juul from taking investments of more than 5 percent from other tobacco companies and prohibited Altria from developing its own e-cigarette or investing in e-cig makers. Altria dissolved the agreement on the basis that it valued its share of Juul as $450 million, or less than 10 percent of its initial investment, in June. In total, Altria estimates Juul's worth at $1.3 billion. "This decision gives us more flexibility as it increases the financial and strategic options we can pursue to secure our business and address the impact of the FDA's now stayed order," a spokesperson from Juul told Katherine.   STAY AHEAD OF THE CURVE: Our Future Pulse newsletter will continue to bring you the biggest stories at the intersection of technology and healthcare, but now five times a week. Want to know what's next in health care? Sign up for our Future Pulse newsletter. If you aren't already subscribed, follow this link to start receiving Future Pulse.     Pharma Moves Monica Bertagnolli began her tenure Monday as the National Cancer Institute's 16th director. Casey Quinn is now a professional staff member for Republicans on the House Ways and Means Health Subcommittee. She previously served as health policy adviser for Rep. Brad Wenstrup (R-Ohio). Quick Hits The New York public health department was already stretched thin — then it got hit with three simultaneous disease outbreaks, POLITICO's Erin Banco reports.   A message from the Foundation for Pharmacy Compounding: Federal rules threaten patient access to compounded hormone replacement therapy that has benefitted millions "In a world full of choices, we should ALL have the choice to use what we and our doctors feel is best for our bodies." – Jami W. Millions of women in the U.S., like Jami, rely upon compounded hormone replacement therapy to provide relief from debilitating symptoms. She's also among thousands who've raised concerns about potential federal regulations that would curb use of these life-supportive treatments. Why are patients like Jami worried about losing access to the compounded hormone therapy their physicians determined is best for them? In 2020, the National Academies of Science, Engineering and Medicine published a study questioning compounded bioidentical hormone therapy. Others have disputed those findings. If implemented by the FDA, the report's recommendations would harm patients and their prescribers' ability to care for them. More...     Follow us on Twitter David Lim @davidalim Lauren Gardner @Gardner_LM Katherine Ellen Foley @katherineefoley   Follow us

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