view collaboration as a four-legged table in order to gather support, drug approval and access
The World Orphan Drug Congress (WODC) brings together patient organizations and industry leaders, researchers and academics, members of the FDA and Congress, and many other active individuals in the rare community. On an annual basis, WODC offers attendees an opportunity to make new connections and review the latest topics in advancing awareness and treatment of rare disorders. The discussion topics were wide ranging, covering innovations in genomic testing and diagnostic tools, clinical research outcome identification, pathways to approval for new therapies, granting early access and globalizing access to treatment. One consistent message that radiated throughout was the need for collaboration to make progress.
Trends in the Orphan Space
Stephanie Okey, MS, Head of North America, Genetic Diseases and SVP, General Manager US Genetic Diseases at Genzyme, delivered a keynote address focusing on trends in the orphan disease space. She cited the importance of perspective, people, and communication. With increased costs in health spending, there are still major gaps in specialty support of the majority of conditions. Rare diseases need people, particularly patients, to bring attention to these gaps. Communication allows for people to share their perspectives, growing knowledge about the disease, which can build specialty support and pave the way for treatment options and access.
Collaborating to Define Endpoints
The benefit-risk analysis conducted byParent Project Muscular Dystrophy (PPMD) was cited by various attendees and presenters as a strong example of collaboration to define clinical trial endpoints with the hopes of pushing research forward and building a case to approve new therapies within better defined outcomes. Pat Furlong, Founding President and CEO of PPMD led a session on this evaluation system and credited the active parent participation and guidance from the FDA in making the report possible. This initiative is now a strong point of reference for clinical and industry researchers, as well as the FDA in evaluating the effectiveness of new therapies in development.
Marlene Haffner, veteran of the FDA and President and CEO of Haffner Associates, LLC, noted that the FDA is very interested in learning what patients think as it helps provide guidance on clinical trial endpoints. Proactive engagement with patients, advocates, and regulatory agencies will help better define research goals up front to establish quality clinical trials. Understanding the natural history of the disease and being able to predict progression is very important, which requires industry and academic collaboration with patients and organizations.
Insights on the Role of Advocacy Organizations
Finding common ground to address critical issues in patient advocacy engagement and collaboration was the topic of a pre-conference workshop hosted by Siren Interactive and moderated by Wendy White, CEO, which fit well with the overall message of WODC. A rotating panel of advocacy and industry leaders addressed the biggest issues in patient advocacy, industry engagement, strong examples of collaboration, and the future of patient advocacy. Coming out of the session, the attendees and panelists expanded upon an idea initiated by Barbara Wuebbels, VP of Patient Advocacy and Medical Affairs at Audentes Therapeutics, who suggested that we view collaboration as a four-legged table in order to gather support, drug approval and access. Patients/organizations, government, academia, and industry are each viewed as a leg of the table; without one leg, the table will not stand. A white paper is being developed in follow up to this workshop and will be available soon. Stay tuned!