The U.S. House of Representatives last night passed by a wide bipartisan margin the 21st Century Cures Act, sending to the Senate a measure intended to speed up approval of new drugs and maintain U.S. leadership in research, while tackling a host of disease and health priorities ranging from cancer to the opioid epidemic.
By a 396 to 26 vote, the House approved an amended version of the legislation it passed last year, only to see the measure stall in the Senate after key committees decided to support only portions rather than the whole bill. For the current bill to fare better in that chamber, it will need to overcome emerging opposition promised by two Democratic senators, Elizabeth Warren (D-MA) and Bernie Sanders (D-VT).
Included in the bill is $4.8 billion for the NIH, specifically to fund three Obama administration research efforts. The “Cancer Moonshot” headed by Vice President Joe Biden would receive $1.8 billion, while $1.6 billion would go toward the BRAIN initiative, and $1.4 billion, the Precision Medicine Initiative.
The bill also includes $500 million over 10 years for the FDA, in part for use toward filling 600 vacant positions and carrying out new rules designed to quicken reviews of new drugs—such as a streamlined review process for combination drug-device products.
21st Century Cures would also require the FDA to include a statement regarding any patient experience data that was used at the time a drug is approved. Patient experience data is defined as “data collected by any persons (including patients, family members, and caregivers of patients, patient advocacy organizations, disease research foundations, researchers, and drug manufacturers).” The FDA would be required to issue guidance on how patient experience data is to be collected.
Also, the FDA would be allowed to support approval of a new indication for a previously approved drug by evaluating “real world” evidence from clinical experience in place of evidence from clinical trials and to establish a streamlined data review program. The agency would be required to issue guidance addressing the use of novel statistical modeling in clinical trials and in the development and review of drugs.
Under 21st Century Cures, manufacturers and distributors of investigational drugs for “serious” conditions would have to publish their policies on expanded access or “compassionate use.”
Also included in the amended 21st Century Cures Act:
- $1 billion over 2 years for grants to states toward programs to prevent and treat opioid abuse
- Creation of a “Next Generation of Researchers Initiative” in the Office of the Director at the NIH to “coordinate, develop, modify, and prioritize” policies and programs intended to improve funding opportunities for new researchers
- Development of a 6-year NIH Research Strategic Plan
- Creation of a new Assistant Secretary for Mental Health and Substance Use, replacing the current position of Administrator at the U.S. Substance Abuse and Mental Health Services Administration (SAMHSA). The bill would also establish a National Mental Health and Substance Use Policy Lab to promote evidence-based grant making within SAMHSA.
Groups representing the biopharma industry and research advocates praised the new legislation.
“The 21st Century Cures Act passed by the House of Representatives today is an important victory for patients and for the next generation of medical innovation,” Biotechnology Innovation Organization (BIO) President and CEO James C. Greenwood said in a statement. “The legislation advances important patient-centered policies that can speed the pace of drug development, while authorizing essential funding for scientific discovery and the promotion of biomedical advancements that can help transform healthcare for patients with the promise of next generation modern medicines.”
Mary Woolley, president and CEO of research advocacy group Research!America called the amended 21st Century Cures Act in a statement “a triumph for patients and a true testament to what can be accomplished when patients and other research stakeholders engage with policymakers and commit to finding solutions to our most pressing health threats.”
While disappointed that the House opted to hold off on future increases in the Prevention and Public Health Fund to help pay for the measure, Woolley lauded the chamber for including funding to combat opioid addiction and mental illness, as well as funding for the Obama administration’s Cancer Moonshot, Precision Medicine, and BRAIN initiatives.
“We are grateful that members of Congress from both sides of the aisle persevered until they found a common path to faster medical progress,” Woolley added.