Available and affordable cutting-edge drugs, devices, and treatments have the potential to transform the lives of Americans who have life-threatening diseases.
Congress is working to foster bipartisan agreement on legislation to help promote groundbreaking medical research and advancement. One of these proposals is my “Patient-Focused Impact Assessment Act.”
The bill would give patients a stronger voice during the development of new drug products and therapies. Patients are in a unique position to offer input as the Food and Drug Administration (FDA) conducts its drug review process. The Senate Health, Education, Labor, and Pensions (HELP) Committee has agreed, unanimously approving my bill this month.
Specifically, my bill would require the development of new guidelines to allow patients and advocates to submit relevant data to the agency. FDA would also report on the use of patient data and feedback in its review of drug applications. I believe transparency and accountability within the review process can strengthen patient participation in FDA decisions, ultimately benefiting all stakeholders.
A Focus on Innovation
My “Patient-Focused Impact Assessment Act” is part of what HELP Chairman Lamar Alexander (R-Tenn.) has called the “innovation project.” This initiative is focusing on legislative proposals designed to accelerate biomedical innovation by addressing the way in which medical research is conducted.
My bill also builds on the goals of the “Food and Drug Administration Safety and Innovation Act.” This law, passed in 2012, authorizes the agency to seek greater stakeholder involvement in the drug approval process. It encourages getting breakthrough drugs and therapies to the patient faster. The FDA needs to develop concrete tools that show patients are being heard. Patients’ time and resources are valuable and should be used wisely as new therapies are being approved.
Building on Successes
The potential to transform lives is worth our energy and attention. For the past 15 years, I have worked to support medical research that could lead to a cure for muscular dystrophy. I authored the original MD CARE Act in 2001, which established the first research programs at the National Institutes of Health for addressing Duchenne muscular dystrophy. Duchenne, which affects 1 in every 3,500 young boys, is the most common fatal genetic disorder diagnosed in childhood.
MD CARE was reauthorized in 2008, and important amendments were added in 2014, expanding research to include older patients and other forms of muscular dystrophy. Since MD CARE was enacted, the lifespan of muscular dystrophy patients has increased by more than 10 years. New drugs and therapies are greatly improving quality of life, and there is room for these successes to grow.
It is no wonder that Parent Project Muscular Dystrophy and dozens of other advocacy groups have endorsed the “Patient-Focused Impact Assessment Act.” Patients should have a powerful voice in the drugs and therapies they must use every day. These firsthand experiences can be used to strengthen medical research in positive and potentially life-changing ways.

By Senator Roger Wicker