https://www.statnews.com/2022/05/19/take-a-stand-for-those-who-cant-with-the-promising-pathway-act/

Every five years since 1992, Congress has reauthorized the U.S. Food and Drug Administration (FDA) user fee package to authorize the FDA to collect funds from companies that produce human drug and biological products. Over the last 30 years, this process has allowed the FDA, medical 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.
 
In 2007, the FDA and medical industry began to recognize the importance of incorporating the patient perspective into drug development. Similarly, Congress took steps to include patient advocate witnesses in Committee hearings related to user fees to ensure that the patient voice was 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. 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 HELP Committee failed to 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 impact them the most.  
 
This is particularly devastating as new and promising therapies for patients with serious and life-threatening diseases are advancing every day.  If we’re going to keep up we need to rethink our review pathways at the FDA as they are built around an outdated paradigm from the 20th century. The Promising Pathway Act is an attempt to do this  while maintaining the FDA’s safety and efficacy standards. 
 
Currently, 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 and other terminal illnesses like Duchenne Muscular Dystrophy (DMD), Parkinson’s, Alzheimer’s, and childhood Diffuse Intrinsic Pontine Glioma (DIPG).
 
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” which is a measurement determined to predict clinical benefit to patients. Increasingly, FDA has demanded that the “validated endpoint” be a validated biomarker. This means that many promising treatments are forced to go through a decade worth of trials before any approval is possible.
 
This is based on a blind adherence to 20th century regulation that was used in cancer and HIV which are totally different diseases. As a result, the FDA not only deters investment in important new science, but more importantly this means many Americans suffering from terminal diseases will lose their fight to those diseases while waiting for treatments to be approved.
 
While patients diagnosed with ALS living in Canada and Europe currently have access to promising therapies through conditional approval, Americans with ALS and other rare or rapidly progressing terminal diseases are denied the same timely access. The FDA’s failure to modernize their pathways to meet the urgent need of 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 FDA’s “gold standard.” Specifically 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 as well as the pharmaceutical industry to give those 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 provisional approval would be granted by the FDA to drugs demonstrating 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 will increase, for example, innovation in clinical trial design and encourage sponsors to use real world data to determine the benefits of the 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 a way that is safe but also urgent. We 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 CEO and Founder of I AM ALS. Senator Mike Braun of Indiana serves on the U.S. Senate Health, Education, Labor and Pensions Committee.
 

Issues