The Act signed into Law Brings the Patient Voice into Sharp Focus
On December 13th, President Obama signed into law the 21st Century Cures Act, a game-changing bill for medical innovation. We are very grateful to Representatives Fred Upton (R-MI) and Diana DeGette (D-CO), as well as members of their staff, for their impressive work and commitment to this bill and for incorporating the feedback from groups like ours in the rare disease community.
“Passage of the 21st Century Cures Act is the culmination of several years of hard work and advocacy by many rare disease patient advocates and patient advocacy organizations,” said Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD). The Amyloidosis Research Consortium and our committed patient advocates are proud to have played a part in seeing this Act come into law.
The bill includes many provisions that will enhance and improve the discovery, development, and delivery of orphan therapies for rare disease patients, including:
• Streamlining of U.S. Food and Drug Administration (FDA) review of genetically targeted and protein variant therapies for rare diseases;
• Creation of funds in the amount of $4.8 billion over 10 years for the National Institutes of Health (NIH) to include funds for the Precision Medicine Initiative, and the Cancer Moonshot;
• Further expansion of the Patient-Focused Drug Development Initiative and requirements for the FDA to report on how patient experience data was used in regulatory review.
The ARC is the first patient-led foundation to hold a Patient-Focused Drug Development meeting in parallel with the FDA’s initiative. This new law rightly recognizes that patients should play an essential role in the development of drugs and devices to diagnose and treat diseases. Patients are in a unique position to provide essential insights about what it is like to live with and fight their disease. The 21st Century Cures Act will enhance these ongoing efforts to better incorporate the patient’s voice into the FDA’s decision-making process.