Author: Lori Shogren, NAF Community Program and Services Director
The first ever virtual Hill Day on March 18th was a tremendous success! This event was organized by the Alliance for a Stronger FDA which is an advocacy organization that NAF is proud to be a member of. For Alliance members and Hill staff alike, it was a new experience—Hill Day by phone. It worked remarkably well. More than 40 Alliance members were organized into seven teams (5 House, 2 Senate) and completed more than 50 meetings. I had the pleasure of joining one of the “House” teams with six other Alliance members including two representatives from FARA, Ron Bartek and Brigid Brennan, Esq. This was an exciting learning experience to gain more support for the important work of the FDA and hear about the vast impact that the FDA has on the missions of the other organizations represented on our team. Our team held meetings or connected with the following House Offices:
- Joe Fuentes, Legislative Assistant (Rep. Ken Calvert, R-CA)
- Carianne Lee, Legislative Aide (Rep. Bonnie Watson Coleman, D-NJ)
- Robert Kerr, Legislative Assistant (Rep. Chuck Fleischmann, R-TN)
- Hillary Caron, Legislative Director (Rep. Ben Ray Lujan, D-NM)
- Margaret McInnis, Legislative Assistant and Courtney Hruska, Scheduler/Office Manager (Rep. Marcy Kaptur, D-OH)
- Ryan Diffley, Legislative Assistant (Rep. Tom Graves, R-GA)
- Alex Morris, Legislative Assistant (Rep. Peter Welch, D-VT)
- Sahil Chaudhary, Health Policy Adviser (Rep. Eliot Engel, D-NY)
About Alliance for a Stronger FDA
The Alliance for a Stronger FDA works to ensure annual appropriations that will adequately fund the FDA’s essential missions. A strong FDA is essential to the nation’s health, U.S. economy, jobs, and balance of trade.
FDA performs a core function of the Federal Government in regulating:
- 100% of drugs, medical devices, diagnostics and cosmetics
- 75% of our nation’s food supply
- 20% of all consumer spending (= $2.4 trillion)
In the current pandemic, the FDA is doing all that is possible to help accelerate availability of testing and medical supplies and development of treatments and vaccines. The FDA recently issued guidance regarding the conduct of clinical trials being conducted amidst this crisis, making recommendations regarding how to conduct this important research while safeguarding the health of the participating patients who are most often especially vulnerable to the virus, as well as the clinical staff. The guidance also expresses additional flexibility on the part of the FDA in terms of how and where the trials are conducted (e.g., at homes vs. hospitals, and meeting additional challenges from such things as quarantines, site closures, travel limitations, and interruptions to the supply chain for the investigational product).
Over the last decade, congress has assigned additional responsibilities to the FDA, for example, in areas such as food safety, biosimilars, drug safety, security and innovation, pharmacy compounding, the 21st Century Cures Act and combatting the opioid crisis.
2020 Hill Day
The theme for the Hill Day was “Growing Responsibilities Require a Budget That Grows.” During the Hill Day the following “ask” of congress was explained. The FDA’s mission continues to grow; vital responsibilities have become more sophisticated and complex. Accordingly, the Alliance’s FDA “ask” for FY 21 is for an increase of $120 million in budget authority (BA) above the FY 20 appropriations level. This would bring FDA “salary and expenses” to $3.278 billion. This would be an overall increase of just under 4% compared to the FY 20 enacted levels and an increase of $72 million over the Administration FY 21 request. Funding under 21st Century Cures, $70 million, is additional.
The agency needs an increased budget, more scientific and technical staff, and better analytical tools to support scientific-based decision making and innovation in both food safety and medical products activities.
- In medical products, this means new tools to evaluate medical products that incorporate cell and gene therapy, digital health, artificial intelligence, and real-world evidence and other new technologies.
- Addressing the shortage of agency staff and expertise in 1) new high-growth scientific areas, notably the dramatic increase in products being developed with gene and cell therapy and 2) new technology-driven challenges, such as cybersecurity, artificial intelligence, digital health, and blockchain;
- Expanding the FDA’s ability to respond to public health emergencies, including coordinated efforts to speed development of diagnostics, therapeutics, and vaccines for viral and other emerging threats to public health;
- Strengthening FDA ability to address the safety of imported medical products and API’s;
- Renewing and expanding initiatives first funded in FY 19, including innovative initiatives in advanced manufacturing, outsourcing, real world evidence, compounding, generics and rare diseases.
How This Impacts the Ataxia Community
The Ataxia community along with other rare disease communities are hopeful for the development of new cell and gene therapies on the horizon. These innovative therapies are very scientifically complex. This promising field of cell and gene therapy has brought almost a thousand submissions with novel, cutting edge science to the FDA and the exciting, curative promise of those new therapies will not reach our patients unless the FDA has enough highly skilled personnel to review those submissions promptly.
Congress has been very supportive of the FDA and Congress is certainly committed to passing FY 21 appropriations bills, but faces a strong headwind with the pandemic, the economy, a Presidential election year, and partisan disputes that are unresolved. We will continue to advocate for a stronger FDA in creative and alternative ways through the COVID-19 pandemic and beyond.
The timing of this Hill Day amid an unprecedented response to a global pandemic created some notable opportunities. For one, the FDA was already on the minds of Hill staff and their understanding and appreciation of the agency’s role in response to the pandemic. In addition, there was widespread awareness of the impressive speed and mobilization of resources to test, treat, and cure COVID-19. This allowed us to inform congress of the impact that this investment and mobilization of resources could have on the development of treatments and cures if applied to rare diseases like Ataxia.