I recently participated in two panels hosted by Patients Rising Now about the Food and Drug Administration’s (FDA) Accelerated Approval Program, which allows for the earlier approval of drugs that treat serious conditions. As a life sciences consultant, a trained pharmacist, and a rare disease patient and advocate, I was honored to join health care experts and other patients in meaningful dialogue about the importance of this regulatory pathway. While the panels spoke to different audiences – patients and legislative staffers – both focused on the importance of developing treatments for rare and terminal diseases.
Here are a few key takeaways from the important conversations:
Accelerated approvals can benefit patients who need them the most.
Robin Strongin from the National Consumers League opened the Patients Rising Now Hill Briefing with the history of the accelerated approval pathway and how its creation stemmed from patient activism during the AIDS epidemic in the 1980s and 1990s. Patients collaborated with the FDA and providers to allow quicker approvals using early signs of clinical benefit while maintaining robust safety assessments. In the 30 years since, we have seen 278 accelerated approvals, mostly across rare and terminal diseases. One study shows that cancer treatments approved under this pathway were available approximately three to four years earlier.
Fellow panelist Mel Mann shared his experience as the second patient enrolled in a leukemia clinical trial, after being given only a handful of years left to live. He is since a 28-year terminal leukemia survivor—28 years that allowed him to live a life with his family, watch his daughter grow up to become a physician, and for him to become a patient advocate for clinical trials, cancer patients, and health equity. That leukemia trial was for a medicine that came to market earlier through an accelerated approval indication, and—he underscored—it is this regulatory pathway that allows more life-changing treatments and outcomes for patients who need them most.
Increased barriers to treatment are dangerous for patients and the future of health care.
Both panels could not ignore the recent decision by the Centers for Medicare and Medicaid Services (CMS) to restrict Medicare coverage for certain Alzheimer's treatments. Panelists discussed the implications for creating additional barriers to therapy after FDA approval, noting how such decisions can limit access patients have to current medications as well as future investment in research and development.
John Stanford, Executive Director of Incubate Coalition, brought this perspective to life: “Biopharmaceutical and biotech investment comes with a great deal of risk, yet the benefit to patients nationwide is unmatched when the system works properly. Regrettably, CMS’ decision could upend this successful system by cutting off investment in the research and development of treatments for Alzheimer’s disease—and many other diseases with unmet medical needs.”
Such precedent, argued the panelists, threatens the current and future treatment options for patients with the highest unmet medical needs. They further underscored that innovation requires coordinated efforts from policymakers, health care stakeholders, and patients, much in the same way the accelerated approval pathway was founded.
Both events reinforced the need to continue highlighting the importance and value of this program and why we as an industry must build on its successes to increase patient access to needed therapies.
 Beaver JA, Howie LJ, Pelosof L, et al. A 25-Year Experience of US Food and Drug Administration Accelerated Approval of Malignant Hematology and Oncology Drugs and Biologics: A Review. JAMA oncology. 2018;4(6):849-856