- - Tuesday, June 29, 2021

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.

While individuals diagnosed with ALS living in Canada and Europe will likely have access to this promising therapy by the end of 2021, ALS patients in the United States will not receive the same timely access to this therapy.

Instead, the FDA requested that Amylyx conduct an additional placebo-controlled clinical trial before the company submits a New Drug Application for FDA review. Despite Amylyx’s evidence of clinical value and measurable improvements in disease management, FDA‘s slow and overly burdensome regulatory pathways will prevent patients with no treatment options and little time to wait, from accessing a potentially life-saving drug.

When there is a life-threatening disease with no effective treatment, patient groups have been clear they are willing to trade a greater degree of risk for earlier access to meaningful treatments. Given advances in science, it’s also logical for FDA drug approval pathways to be continually rethought and reinvented to advance efficient access to meaningful treatments for patients with progressive diseases that, left untreated or under-treated, will negatively impact their daily lives or lead to premature death.

This should not be controversial, but sadly it is. Currently, the FDA is embroiled in an emotional dispute over a drug, aducanumab, which was recently approved to treat Alzheimer’s disease. Some tout FDA‘s decision as a signal for more regulatory flexibility and innovation, praising the approval as a victory for patients with no available treatment options. Others argue that the FDA‘s gold standard has been jeopardized, putting patients at risk. No matter what side of the issue you fall on, we all have the same end goal: providing patients with safe and effective treatments as soon as possible.

This particular drug was approved under an accelerated approval pathway, which is useful for bringing certain drugs to patients, but can be stronger. Delivering promising therapies to patients faster does not have to mean lowering FDA‘s gold standard, but it does require increased patient safety, data monitoring, reliance on real-world evidence, and patient-focused drug development for those dying and ready to assume higher risk.

The Promising Pathway Act is the legislative solution to support the FDA in giving those struggling with serious and life-threatening illnesses a fighting chance to receive timely access to innovative treatments that still show substantial evidence of safety and relevant early evidence of positive therapeutic outcomes.

To accomplish this, the Promising Pathway Act requires the FDA to establish a rolling, real-time, priority review pathway to evaluate provisional approval applications for drugs intended to treat, prevent, or diagnose serious or life-threatening diseases or conditions. Under the Promising Pathway Act, provisional approval is granted by the FDA to drugs demonstrating substantial evidence of safety and relevant early evidence of positive therapeutic outcomes. Drug sponsors would be allowed and encouraged to use scientifically substantiated surrogate endpoints other than those already validated by the FDA.

The Promising Pathway Act will increase innovation in clinical trial design and encourage sponsors to use real world data to determine the benefits of the drug, and it will do so without reducing the FDA‘s standard of effectiveness.

It’s time to advance drug development for life-threatening diseases, and we can secure access to safe, promising therapies for patients while maintaining the FDA‘s role in foundational and necessary safety and efficacy standards. Congress must act with urgency to pass the Promising Pathway Act.

• U.S. Senator Mike Braun, Indiana Republican, serves on the Aging Committee, Agriculture Committee, Budget Committee, Appropriations Committee, and the Health, Education, Labor and Pensions Committee (HELP). Prior to the Senate, he was the founder and CEO of Meyer Distributing, a company he built in his hometown of Jasper that employs hundreds of Americans across the country.

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