2018 NORD Summit: An Advocate’s Perspective

Nov 27, 2018 | Research & Innovation

By Leslie Condon, TargetCancer Foundation Advocacy Council Chair

The National Organization for Rare Disorders was formed after a small coalition of rare disease patients came together in the late 1970’s and became a driving force that helped establish the 1983 Orphan Drug Act. Since then, the number of NORD patient organization members has grown to 280, including twenty-one rare cancer organizations in NORD’s Rare Cancer Coalition. Within NORD’s thirty-five year history, the organization established the first patient assistant program, and helped advocate and legislate for new laws, including the Rare Diseases Act in 2002 and the FDA Safety and Innovation Act in 2006.

NORD’s annual Rare Diseases and Orphan Products Summit welcomes rare disease patients and advocates, industry leaders, government representatives, and others from the rare disease community.

Rare Cancer Coalition

In 2017, NORD created the Rare Cancer Coalition in recognition of the distinctive position rare cancers hold within the medical field. This new initiative brings together the rare cancer member organizations of NORD who advocate for diseases like fibrolamellar cancer or desmoid tumors, in an effort to raise the profile of all rare cancers. During the Summit and throughout the year, these organizations have the opportunity to connect, share strategies, and collaborate on future projects.

The Rare Cancer Coalition presented its very first breakout session at this year’s NORD Summit. Corrie Painter, Associate Director of Operations and Scientific Outreach for the Broad Cancer Program, and rare cancer patient herself, started the presentation by sharing her own story, and the exciting news of the Gastroesophageal Cancer Project with the crowd. This new project, which partners TargetCancer Foundation with the Broad Institute of MIT and Harvard, is a perfect embodiment of NORD’s commitment to “patient-focused innovation,” Its ongoing success will hinge on the participation of patients impacted by esophageal or stomach/gastric cancer.

Another distinct component of the NORD Summit programming is the inclusion of presenters from the FDA. Collectively, they agree that research for rare disease was broadly supported by lawmakers on the federal level. However, recent actions taken by lawmakers as well as pending legislation have added serious complications to advancement in rare disease treatment.

Orphan Drug Act Tax Credit

One significant setback recently pushed through by the house and senate was a reform to the Orphan Drug Tax Credit. As mentioned, the original passing of the Orphan Drug Act was a momentous breakthrough for rare disease drug development. Prior to its passing, only a very small number of drugs were available to rare disease patients. The Orphan Drug Tax Credit, as a part of the original Act of 1983, gave pharmaceutical companies financial incentives to research and develop crucial, life saving treatments. This included tax relief for clinical trial research related to the development of individual drugs that have come to market. Since then, the research has steadily increased and expanded to target more rare diseases with increasingly advanced therapies.

When initially passed, the Orphan Drug Tax Credit was at 50%. However, at the end of 2017, the House and Senate tried to eliminate the tax credit completely. Rare disease advocates in the Senate fought ceaselessly to retain the credit but now only 25% of costs can be claimed by drug developers.

The reduction of such a tax credit will, undoubtedly, stall and hinder future drug development for rare diseases. Raising funds for rare disease already has many obstacles due to the low numbers of patients afflicted, the high costs of developing drugs for small populations, and the ongoing struggle to advocate to the general public about most rare disorders. This development, coupled with potential changes to pre-existing conditions protections and the looming changes to the ACA, has left patients in the rare disease community both medically and economically vulnerable.

Thankfully, the FDA continues to be a committed and passionate ally in our work. In the last decade, they’ve enhanced their support for rare disease research through a series of clearly defined actions, including the establishment of the Office of Patient Affairs, and a more streamlined system for reviewing orphan drug designations. As legislation around orphan drug development continues to evolve, the FDA will continue seeking ways to best navigate the new terrain.

One of the best ways to advocate for a rare disease such as cholangiocarcinoma or esophageal cancer, is to inform oneself of the specific issues surrounding that disease. Attending the annual NORD Summit has been for me an invaluable way to deepen my understanding of the current conversations, and to connect with other patients, advocates, and organizations to better learn from each other. I invite you to consider attending a NORD event yourself in the future.

To attend a NORD event on behalf of TargetCancer Foundation, please contact Jim Palma directly at jim@targetcancerfoundation. NORD offers travel scholarships to their annual Summit, and they also host local events all over the country, include the Greater Boston area. Please visit their website at rarediseases.org to learn more.

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