IGT Statement on ICER’s Analysis of the Accelerated Approval Pathway

WASHINGTON, DC (April 27, 2021) – Today, the Institute for Gene Therapies (IGT) Chairman Congressman Erik Paulsen issued the following statement regarding the Institute for Clinical and Economic Review (ICER) analysis of the accelerated approval pathway:


“The Food & Drug Administration’s accelerated approval pathway has given patients with debilitating and life-threatening diseases the chance to live longer, healthier lives. Beginning in the 1990s, this pathway served as a lifeline to people living with HIV/AIDS as well as cancer patients who had no other approved treatments. Today, the pathway also means that many rare disease patients have therapies available to them years earlier than otherwise would have been permitted. The accelerated approval pathway upholds FDA’s gold standard for safety and efficacy and is an important tool to bring transformative gene therapies to patients.


The potential policy reforms put forth by the Institute for Clinical and Economic Review are an exercise in creating solutions to a problem that doesn’t exist. Within each reform discussed in the report, ICER itself acknowledges potential negative consequences – from unworkability and increasing FDA’s burden to causing a strong disincentive to develop drugs for rare diseases. The recommendations ignore the overwhelming success of the accelerated approval pathway and would, in effect, undo the decades of progress FDA and Congress have made in bringing new treatments to the most vulnerable patients. We urge policymakers to consider those patient communities that are relying on a strong accelerated approval pathway for their only therapeutic option. Weakening the pathway risks delaying new therapies, stifling innovation, and hampering scientific progress.”


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About the Institute for Gene Therapies

The Institute for Gene Therapies brings together experts from across the healthcare system to advocate for a modernized policy framework that encourages innovations and promotes patient access to the treatments they need. We represent innovators and patients, business leaders and academics working to ensure policies reflect medical advances, creating a new reality for patients. Follow us on Twitter @gene_therapies.