Rare Disease Day Congressional Tele-Town Hall

On February 28, 2014, the Plasma Protein Therapeutics Association (PPTA) hosted a Rare Disease Day Congressional Tele-Town Hall featuring remarks by Congressman Leonard Lance (NJ-7), Co-Chair of the Congressional Rare Disease Caucus, and Dr. Stephen Groft, Pharm. D., Former Director of the Office of Rare Disease Research, National Institutes of Health (NIH).

The town hall highlighted for Congressional staff and patient advocates, the science-driven policies and collaborative practices that are helping accelerate lifesaving treatments to rare disease patients, and provided important insights into the current state of rare disease research and advocacy.

PPTA Senior Vice President, Julie Birkofer, opened the meeting acknowledging the diversity of activities and awareness raising events taking place in honor of rare disease research and advocacy across the world. Thanking the many advocates, patients, and policymakers joining the call, Julie introduced Congressman Lance and provided a brief overview of his longstanding leadership in Congress on rare disease policies, specifically citing his support and leadership on the Medicare IVIG Access Act of 2012 (P.L. 112-242) that was signed into law on January 10, 2013, and is currently being implemented by the Centers for Medicare and Medicaid Services (CMS). Congressman Lance commended the town hall participants for helping lead the rare disease community in an extraordinary number of activities and events over the course of the week leading into Rare Disease Day, and sending the message to policymakers that rare disease research and innovation is a priority. The Congressman outlined the mission and priorities of the Congressional Rare Disease Caucus, emphasizing that all members of Congress are dedicated to advancing policies that positively impact patients living with rare diseases. The Caucus is currently supporting the Modernizing Our Drug Diagnostics Evaluation and Regulatory Network (MODDERN) Cures Act (H.R. 3091), a bill intended to incentivize the development of new treatments and the investigation of “dormant therapies” that may slow progression of, or cure, hard-to-treat diseases.  Congressman Lance is the lead sponsor of the MODDERN Cures Act.   

Concluding his remarks, Congressman Lance encouraged patient advocates to engage their representatives to support the advancement of research and innovation for rare diseases. Further underlining the important role that patients and patient advocates play in policymaking, the Congressman urged the town hall participants to convey their personal stories to lawmakers, in both their Washington, D.C., and district offices, to help them better understand the vital need to improve rare disease research and innovation.

After a brief question and answer session led by Congressman Lance, Dr. Groft provided a comprehensive update on the demographics of rare diseases in the United States, as well as the state of access for rare disease patients to treatments and  vaccines in development: "18-25 million" people are diagnosed with a rare disease in the U.S. (but we are lacking hard data on overall prevalence) and "450" drugs for those diseases are in development stages. "9,400" research projects on rare diseases are currently underway with a "$3.6 billion" budget allocation for NIH. In 2013, "258" drugs received orphan designation from the Food and Drug Administration (FDA) and "30" thus far in 2014. "Six" orphan-designated drugs have obtained marketing approval thus far in 2014. He also discussed the rising interest internationally in rare disease research and innovation, namely the first International Rare Diseases Research Consortium Conference held last year and the increasing number of global collaborations between research institutions in Europe and Asia and the NIH. 

Dr. Groft’s overall message was that of collaboration and the significant value of public-private partnership. Partnerships are paving the way for better rare disease research, diagnosis and treatment. The increase of knowledge sharing through internet and social media as well as media publicity has fostered the move to public-private partnerships within the rare disease community. Patient groups, government researchers, healthcare providers, academic researchers, and biotechnology and biopharmaceutical companies are working together both domestically and abroad to progress the researchers’ understanding of the basic science underlying many rare diseases. Notably, clinical centers of excellence are being developed for rare diseases in collaboration between patient organizations, academic centers, and NIH, increasing research and clinical development that allow for the earlier integration of the patient perspective, and ensuring a more efficient path from “bench-to-bedside and back.”

Dr. Groft stressed the importance of patient advocacy, citing patients as the true stimulus to all these accomplishments and future advancements, and of NIH-FDA collaboration and the role of patients in clinical studies. His message to rare disease innovators was communication through the whole process. He further explained, in response to a question about innovations concerning plasma protein therapies and the diseases they treat, that the healthcare providers must be made aware that these therapies are available and lifesaving.

PPTA would like to thank Congressman Lance and Dr. Groft for their time in providing this valuable look into the current state of rare disease research and advocacy.

Everett Crosland, Director, Federal Affairs
Mary Clare Kimber, Senior Manager, Regulatory Policy
Michelle Krecz, Senior Manager, Communications

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