3/31/2025
Summary
The FDA’s Accelerated Approval (AA) program enables early approval of drugs that treat serious conditions by bypassing unmet regulatory conditions. Despite risks, the recent approval of Humacyte’s bioengineered blood vessels has sparked concerns over how medical devices are approved during this process.
Earlier this month, the United States Food and Drug Administration (FDA) granted accelerated approval for Humacyte’s bioengineered blood vessels. The approval passed despite a clinical review warning about the device’s “catastrophic, life-threatening failures” and certain “risks [that] are unacceptable compared to current alternate treatment options.” This incident raises significant concerns about the FDA’s Accelerated Approval (AA) program.
The AA program was designed to speed up access to potentially life-saving innovations, and has led to the approval of over 300 medical devices since December 2024. However, a study on AA-approved cancer drugs from 2013-2017 found that only 43% of the drugs showed clinical benefits in follow-up trials. This statistic raises serious doubts about the effectiveness of AA approved drugs.
Researchers have previously expressed concerns about the FDA’s rapid approval process. A 2022 study published in the American Journal of Law & Medicine reported that “over sixty-four percent of approved drugs between 2015-2017 participated in expedited approval pathways … it emphasizes the growing FDA trend to approve drugs with abridged evidence of efficacy.” Another study highlighted this issue, finding that 20% of medical devices approved via expedited pathways from 1992 to 2018 later failed FDA requirements.
Many factors contribute to this pressure to approve medical devices quickly. Biotech companies often face intense market competition with high investor demands, thus they push for faster reviews. Additionally, patients with limited options advocate for quicker access to potentially life-saving treatments. This puts healthcare providers in the challenging position of recommending devices with limited data about long-term outcomes.
The FDA’s AA program also raises economic concerns. New devices typically come with premium pricing, potentially exacerbating healthcare disparities. For example, drugs approved by AA experienced price increases 26% more often than non-AA medicines over a 10 year period.
Gregg Gonsalves, an epidemiology professor at Yale, noted that drugs with no known clinical benefits are frequently being prescribed to treat certain diseases. In fact, the peer reviewed medical journal JAMA conducted a study that found that, “many drugs granted FDA accelerated approval lack evidence of clinical benefit, even after being converted to standard approval, yet they account for substantial CMS [Centers for Medicare and Medicaid Services] spending.” Specifically, they reported that 59% of CMS spending was reserved for AA drugs whose confirmatory trials evaluated surrogate end points–biomarkers intended to substitute for clinical outcomes.” This challenge is particularly relevant for patients who only have experimental treatment options. In cases like this, balancing a device’s efficacy with expedited approval is critical, as patients may not have time to wait.
It is with this mindset that some researchers argue that the benefits of AA outweigh the risks. Julie Patterson, a pharmaceutical PhD, shared: “Because of AA, patients with cancer and their clinicians have treatment options earlier than they otherwise would, and this has likely led to more than 250,000 life-years gained … most drugs meet confirmation benchmarks and continue to unlock value for patients and society.”
As medical technologies continue to advance, the ethical implications of fast-tracked approvals become increasingly complex. Much like Swiss bioethicists Mattia Andreoletti and Alessandro Blasimme emphasized in their paper: “It is ethically permissible (and sometimes even required) to derogate from typical regulatory constraints in special circumstances. At the same time, specific criteria must be fulfilled to reconcile the need to expedite the approval of potentially life-changing treatments with appropriate ethical safeguards.”
The FDA may be taking this knowledge to heart, as they announced new guidelines for post-market surveillance in 2022 to mitigate potential issues in rapidly approved devices. The guideline requires manufacturers to “conduct postmarket surveillance at the time of approval or clearance or at any time thereafter.” Although no studies have investigated the effectiveness of this new 2022 guideline it is a step in the right direction to preserve the ethicality of the AA program.