Consider this: You’re diagnosed with a rare form of cancer that currently has no approved treatment. Your condition is worsening and your prognosis, without intervention, is poor. You feel hopeless; your family and friends, desperate.
But you learn of a new therapy, granted accelerated approval by the U.S. Food and Drug Administration (FDA), which could help keep your cancer in check and potentially improve your symptoms — and it is available now. At last, the dark clouds break and a collective weight eases. Your disease may respond to treatment and slow down. You may have more time to enjoy more of life’s big and small moments, including milestones you accepted you’d miss.
Real-world moments of potential relief like these are possible thanks to the FDA’s Accelerated Approval Program (AAP), which allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need. Accelerated approval is based on a surrogate endpoint that is reasonably likely to predict clinical benefit with the requirement that companies conduct studies to confirm the clinical benefit. Patients with hard-to-treat diseases have access to more than 300 new drugs and biologics sooner because of the AAP. It is a crucial and, sometimes, the only pathway to treatment for patients who do not have other effective options. These therapies may provide significant benefits for patients and doctors fighting difficult to treat diseases — and offer hope.
And yet, the program is under attack from those that want to alter the program or get rid of it entirely. Let me be frank: Changing or eliminating the AAP will hinder patients’ access to potentially life-saving treatments. The health outcomes will worsen for those who may be in desperate need of a new treatment for their hard-to-treat and, often, fatal disease — now and in the future.
Gaining life years
Until recently, there was no quantified understanding of the holistic benefit of the AAP for people diagnosed and living with rare and serious, unmet conditions. At Johnson & Johnson, we set out to discover that impact, examining the true value of accelerated approval therapies for our most important stakeholders: patients.
In April, a first-of-its-kind study in the Journal of the National Comprehensive Cancer Network (JNCCN) evaluated life years gained among cancer patients who have taken medicines approved via the AAP. Life years gained measures the additional years a person lives due to receiving a treatment. In this retrospective study, the likely survival benefit for patients was assessed from the time of initial accelerated approval to the time of full approval using overall survival results from subsequent studies. This groundbreaking research — agnostic of pharmaceutical manufacturer or outcome and including therapies that were later withdrawn — demonstrates that the pathway is working for patients.
From 2006 to 2022, the analysis estimated that the pathway gave approximately 911,000 patients about 263,000 life years across their use of 69 approved products for which overall survival data was available. If this timeline was extended to 2026, the life years gained would increase to 382,000 years — a 45% increase. In addition, a subgroup analysis calculated that treatments with an orphan designation gained 145,413 life years across 264,061 patients — a 16.5% gain over standard of care. This analysis determined the treatments approved via the AAP added significant time to the lives of cancer patients during the period between accelerated approval and conversion to full approval.
Although the results are a snapshot in time specific to the timeframe examined, and may not be generalizable for the future, currently approved oncology therapies now include cell and gene therapies, which have curative potential.
Recent critiques of the AAP have failed to account for a variety of issues that directly impact patient outcomes — such as focusing only on counting drugs and not the patients who used them, not calculating patient uptake and providing incomplete information which does not consider all evidence available, such as real-world evidence.
Protecting the purpose — and patients
Patients are the reason the AAP was created, and the reason to fight for its preservation and strengthen it for the future. We are committed to breaking down barriers to treatment so that all patients, regardless of geography, ethnicity, or race, are diagnosed early and have equitable access to innovative therapies. As researchers, regulators and policymakers who ultimately will shape the fate of Accelerated Approval Program, we can — and must — protect this pathway and the additional time it can potentially deliver for patients to spend with their loved ones.
Ginny Beakes-Read is Head, Global Regulatory Policy and Intelligence, at Johnson & Johnson Innovative Medicine.
