It's the end of another week! I hope you have been staying safe and cool during this intense summer heat. Before you give yourself to the bliss of another weekend, we want to make sure you’re abreast of the major developments in healthcare. In this post, we’ll delve into the FDA's draft guidance encouraging inclusion of patients with incurable cancers in clinical trials, Biogen's tussle with ICER over Aduhelm's list price, and Eli Lilly's Alzheimer's drug and their COVID-19 antibody treatment.
The FDA issued a draft guidance encouraging industry to include patients with incurable cancers (when there is no potential for cure or for prolonged/near normal survival) in cancer clinical trials, regardless of whether they have received existing alternative treatment options. Historically, many clinical trials have required that participating patients previously received multiple therapies. The FDA believes patients with incurable cancers, if provided adequate information to make an informed decision, should be eligible to participate in oncology clinical trials. If there is no scientific rationale for excluding these patients, then clinical trial eligibility criteria should be broadened to include these patients, with appropriate informed consent. This draft guidance is part of the FDA’s broader initiative to encourage rational expanded patient eligibility for oncology clinical trials.
The Institute for Clinical and Economic Review (ICER) said in a revised cost-effectiveness analysis that Aduhelm’s price tag of $56,000 is “not in reasonable alignment with its clinical benefits.” In ICER’s view, Aduhelm would need an 85% to 95% discount from its current list price, which would set the price at $3,000 to $8,400 a year. ICER was clear however, that a projected price of potentially $70,000 for Aduhelm would be fair if the treatment halted the progression of dementia in Alzheimer’s patients. The group pointed to the conflicting late-stage trials in patients with early-stage and mild Alzheimer’s. In the EMERGE trial, patients who got the highest dose of aduhelm experienced a 22% improvement on a clinical dementia scale over placebo after 78 weeks. But the same patient group in another study had worse outcomes than the placebo group on that same measure. The efficacy of aduhelm is the heart of the issue of approval, and price. However, the accelerated approval does extend the opportunity for aduhelm to prove itself and offers Alzheimer’s patients a drug that could change the course of their disease. Biogen understands the weight of the approval and doesn't believe ICER's report reflects the value of treatment or appropriately factors in the burden Alzheimer’s has on society. The company has vigorously defended Aduhelm’s list price for weeks, with CEO Michel Vounatsos saying it’s "substantiated by the value it is expected to bring to patients, caregivers and society.” ICER is set to meet with Biogen on July 15 to discuss its findings.
Eli Lilly said last week that it will seek a fast U.S. approval for its drug to treat Alzheimer’s disease, following the same regulatory pathway established by Biogen. They intend to submit their drug, donanemab, to the Food and Drug Administration (FDA) by the end of the year. Lilly will request the agency grant the drug accelerated approval based on its ability to reduce amyloid plaques in the brain. This is the same FDA pathway that Biogen used to win approval for its Alzheimer’s drug Aduhelm last month. The Lilly filing will be based on the results of a successful mid-stage clinical trial called TRAILBLAZER-ALZ that was completed and presented last March. While the TRAILBLAZER study results showed donanemab slowed the progression of Alzheimer’s compared to a placebo. Lilly previously said they would defer seeking approval until they had results from a larger Phase 3 trial. That Phase 3 study is currently enrolling patients. But the FDA’s controversial decision to approve Biogen’s Aduhelm has motivated Lilly to change its stance. The decision was also likely influenced by the FDA’s decision to grant breakthrough therapy designation to donanemab based on the TRAILBLAZER study results. If the FDA accepts Lilly’s accelerated approval filing for donanemab, the drug could reach the market in the middle of next year, setting up a situation where two treatments for Alzheimer’s disease are available to patients. But Lilly’s decision to seek an early filing, while seemingly justified by Biogen’s similar approach, is also likely to further debate over the FDA’s relaxed regulatory standards for new Alzheimer’s drugs. Last Thursday, Biogen shares were down 8%, representing a loss of approximately $5 billion in market value, as investors saw Lilly as potentially short-circuiting the Aduhelm commercial launch. Lilly shares were up 8%, adding $17 billion in market value.
Eli Lilly's COVID-19 antibody cocktail has already been on the outs in several states over fears of reduced variant efficacy. Now, the U.S. has decided to extend that policy nationwide. With a pair of troubling coronavirus variants on the rise, the U.S. is pausing the national distribution of Eli Lilly’s COVID-19 antibody combo of bamlanivimab and etesevimab until further notice. The Centers for Disease Control and Prevention has warned that together, the SARS-COV-2 Gamma variant, first identified in Brazil, and the Beta variant, which originally surfaced in South Africa, now make up more than 11% of sequenced U.S. coronavirus cases. That figure is ticking up, the CDC said. In vitro assays have shown that bamlanivimab and etesevimab together are not active against either variant. In lieu of Lilly's antibody combo, the U.S. recommends healthcare providers switch to other authorized monoclonal antibody drugs like Regeneron's cocktail REGEN-COV and GlaxoSmithKline and Vir Biotechnology's solo agent sotrovimab.