Obama signs 21st Century Cures into law
On Dec. 13, President Obama officially signed 21st Century Cure into law. The bill passed through Congress in the past two weeks with strong bipartisan support, with the House voting in favor 392-26 and the Senate in favor 94-5. This legislation includes policy provisions that will strengthen the biomedical innovation system and empower our nation’s emerging scientists and researchers.
Energy and Commerce Committee Chairman Fred Upton (R-MI) and Rep. Diana DeGette (D-CO) sponsored this legislation in the House, and Health, Education, Labor and Pensions Committee (HELP) Chairman Lamar Alexander and Ranking Member Patty Murray (D-WA) sponsored this legislation in the Senate.
Why is 21st Century Cures important to PKD patients?
This bill provides $4.8 billion in new funding for the National Institutes of Health (NIH) and $500 million in new funding for the Food and Drug Administration (FDA). 21st Century Cures also assures that the patient voice will be included in the drug development process and that the FDA will consider how a disease impacts patients’ lives and patient treatment preferences when considering new treatments for approval. This bill also provides a provision to allow dialysis patients to enroll in Medicare Advantage plans.
How does 21st Century Cures benefit patients with a rare disease, like ARPKD?
In addition to the increased funding to NIH and FDA, here are some of the highlights of the bill that are of particular interest to the rare disease community:
- Reauthorization of the Rare Pediatric Disease Priority Review Voucher program through 2020
- Improved biomarker qualification
- Strengthened patient engagement at the FDA through the Patient Focused Impact Assessment Act
The OPEN ACT was dropped from 21st Century Cures. This would have provided an incentive for companies to repurpose existing drugs for rare disease indications.
What are the next steps?
First, we hope that PKD advocates and their friends will thank those in the House and Senate who support 21st Century Cures. If you are not certain how your representatives voted, you can check:
Now that the Cures bill has been signed into law, the U.S. Department of Health and Human Services (HHS), NIH, and FDA will begin to draft regulations and guidelines to carry out the new Cures Act. This will take time.
In the meantime, the PKD Foundation and other patient groups need to make certain that NIH and FDA have the funds and other necessary resources to ensure the implementation of Cures when all pieces are in place.
2017 brings a New Congress, new administration and new legislative proposals and priorities. We will keep you up to date on how you can continue to help advance PKD patients’ interests.