Every five years since 1992, Congress has reauthorized the Food and Drug Administration user fee package to allow the agency to collect funds from companies that produce human drugs and biological products. Over the last 30 years, this process has helped the FDA, the biomedical industry, and Congress to rethink, redesign, and modernize existing FDA programs and pathways as well as introduce new regulatory policies to advance drug development and approvals.
But it still isn’t fast or innovative enough to help Americans living with rare or life-threatening and progressive diseases.
In 2007, the FDA and medical industry began to recognize the importance of incorporating the patient perspective into drug development. Congress took steps to include patient advocate witnesses in committee hearings related to user fees to ensure that patients’ voices were represented. This year, the FDA and pharmaceutical industry commitment agreement letter contained a number of provisions to encourage pilot programs to advance the integration of patient perspectives into drug evaluations, clinical measurements, and approvals. But this begs the question: Why are we still discussing pilot programs 15 years after recognizing the importance of patient perspectives, rather than implementing substantive regulatory reform?
While these commitments are an important step forward for patients, especially those with rare or life-threatening and progressive diseases, the Senate Committee on Health, Education, Labor, and Pensions (HELP) did not allow patients to testify about the latest set of reforms and proposals during the 2022 user fee hearing. As a result, patients were denied the opportunity to weigh in on regulatory decisions that will ultimately affect them the most.
This is particularly devastating because new and promising therapies for patients with serious and life-threatening diseases are advancing every day. Congress and the FDA need to rethink the agency’s review pathways, since they are built around an outdated paradigm from the 20th century. The Promising Pathway Act, which one of us (M.B.) reintroduced to the Senate, is an attempt to do this while maintaining the FDA’s safety and efficacy standards.
The FDA’s failure to provide a true swift pathway to approve safe and effective treatments for devastating diseases means the difference between life and death for patients with ALS, which one of us (B.W), is living with, and other terminal illnesses like Duchenne muscular dystrophy, Parkinson’s disease, Alzheimer’s disease, and childhood diffuse intrinsic pontine glioma.
Here’s how current FDA review pathways work: New medicines must meet the same standard for safety and efficacy that a drug like Viagra must meet. FDA defines “efficacy” based on clinical studies, and the results have to show that the drug works on what’s called a validated endpoint — a measurement determined to predict clinical benefit to patients. The FDA is increasingly demanding that the validated endpoint be a validated biomarker, meaning that many promising treatments must go through a decade worth of trials before any approval is possible.
This approach is based on blind adherence to 20th century regulation, which fails to provide manufacturers with the regulatory clarity, predictability, or flexibility to use 21st century tools and techniques in clinical trials that support efficient drug review and approval. As a result, the FDA not only deters investment in important new science but, more importantly, this means that many Americans living with terminal diseases will lose their fight to those diseases while waiting for treatments to be approved.
People living in Canada and Europe who have been diagnosed with ALS currently have access to promising therapies through conditional approval, while Americans with ALS and other rare or rapidly progressing terminal diseases are denied the same timely access. The FDA’s failure to modernize its pathways to meet the urgent need of people with these diseases will cost tens of thousands of American lives and threatens to derail drug development and approval.
Supporting faster access to safe and effective therapies can be accomplished without sacrificing the FDA’s gold standard for drug approvals. This can be done by bolstering post-market surveillance requirements for drug sponsors while allowing faster access to promising therapies for those with life-threatening, rapidly progressing diseases.
The Promising Pathway Act was crafted with the input of thousands of patients and the pharmaceutical industry to give people struggling with serious and life-threatening illnesses a fighting chance to receive timely access to innovative treatments.
It 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, life-threatening diseases. Under this act, the FDA would grant provisional approval to drugs that demonstrate substantial evidence of safety and relevant early evidence of positive therapeutic outcomes. Crucially, drug sponsors would be allowed and encouraged to use and develop different, rigorous scientific measurements to demonstrate safety and efficacy in addition to those currently accepted by the FDA.
This would increase, for example, innovation in clinical trial design and encourage sponsors to use real-world data to determine the benefits of a drug without reducing the FDA’s standard of safety or effectiveness.
It is time to advance drug development for life-threatening diseases. All Americans deserve the chance to fight for their lives and to do so in ways that are safe but also urgent. Congress can secure access to safe, promising therapies for patients while maintaining the FDA’s role in foundational and necessary safety and efficacy standards. This is why the Promising Pathway Act was introduced and why we fight every day to realize the promise of transforming these life-threatening, rapidly progressing diseases from terminal to chronic.
Brian Wallach is the co-founder and board member of I AM ALS and the co-founder and board co-chair of Synapticure, a personalized full-stack teleneurology company, starting with ALS. Senator Mike Braun (R-Ind.) serves on the U.S. Senate Health, Education, Labor and Pensions Committee.
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