New and promising therapies for patients with serious life-threatening diseases are advancing, but Congress must modernize review pathways at the FDA and do so in a manner that maintains its role in foundational and necessary safety and efficacy standards.
The Promising Pathway Act (S. 1644/H.R. 3761) is the solution.
FDA drug approval is the gold standard of safety, but Congress must do a better job supporting FDA professionals in doing what they do best while giving them the tools necessary to be more nimble. Approval of safe and effective treatments for devastating diseases is the difference between life and death for patients with terminal diseases, and it’s critical for FDA to have the authority to be both thorough and agile. We learned from the pandemic that FDA professionals are, without out a doubt, capable of both.
When it comes to rare diseases, these professionals are confined by an antiquated regulatory system. Under existing FDA review pathways, all new medicines must meet the same gold standard for safety and efficacy. FDA defines efficacy based on studies showing the drug has an effect on a validated surrogate endpoint or measurements reasonably determined to predict clinical benefit to patients, known as biomarkers.
Generally, endpoints must be studied for years, even decades, before gaining the FDA‘s seal of approval for use in clinical research. Unfortunately, the statutory framework Congress imposes on the FDA results in a strict and unbending reliance on validated surrogate endpoints, which in turn leads to limitations on clinical trials designed for rare or lesser-studied diseases, such as amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy, rare cancers, and other terminal diseases. Congress must update existing law and 20th century regulation and better support investment in cutting-edge science when it comes to terminal diseases.
We owe this to these Americans struggling with life-threatening diseases for which there is no cure.
Consider this example. In June of this year, Amylyx Pharmaceuticals (a developer of ALS therapies) submitted a potential new ALS treatment for approval in Canada and Europe, known as AMX0035. Amylyx’s clinical trial studying the safety and efficacy of AMX0035 showed a slowed progression of the disease in patients with ALS and also a reduced risk of death by 44 percent, extending ALS patients’ lives by an average of six months.