No Time to Waste

June 11, 2021
Weekly Columns

Those with a life-threatening illness or condition know that time is of the essence. This week, I reintroduced a bipartisan bill to cut red tape so patients with terminal conditions may quickly access lifesaving treatments with positive clinical data.

The Promising Pathway Act will allow innovative therapies with encouraging clinical trial data to treat patients outside the Food and Drug Administration’s (FDA) rigid trial requirements, promoting innovation, and sparking competition between smaller and larger pharmaceutical companies.

It is common for small companies to complete meaningful Phase II clinical trials, only to run out of funds before they are able to test the drugs in larger populations and eventually bring it to market. Without new investments, larger companies buy the therapy, complete the clinical trials, and market the drug themselves. Through the Promising Pathway Act, the small company that has proven the safety and efficacy of the therapy will be able to market the drug to individuals outside of the clinical trials, and receive needed investment to finish the late-stage clinical trials. This increases competition between the small and large companies, helps lower drug prices, and brings new therapies to market faster.

This bill is especially important for those suffering from terminal diseases, such as ALS. A patient diagnosed with ALS is typically given no more than 3 years to live. That is not enough time to wait for the FDA to fully approve a therapy that could potentially save his or her life. Through this bill, effective therapies for ALS will more quickly become available to those who desperately need them.

Unlike the established Accelerated Approval process or President Trump’s Right to Try process, the Promising Pathway Act is the only policy that expands treatment options, brings effective therapies to patients quicker, and spurs pharmaceutical competition. Furthermore, the bill ensures that insurance companies must cover the therapy’s costs in the same way they would any fully FDA-approved treatment, relieving patients of the cost burden.

Americans with potentially terminal illnesses deserve every opportunity to change the course of their diagnosis. I am honored to play a small part in that by reintroducing the Promising Pathway Act.