The Importance of Patient Advocates for Orphan Drugs

April 19, 2013
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I attended the World Orphan Drug Congress last week in Washington, DC, and Siren presented the workshop, “A Roadmap for Patient Advocacy Investment.” In the rare disease space, patient engagement is critical to every step of a drug’s development, including: filling clinical trials, getting FDA approval, obtaining reimbursement, getting patients diagnosed and promoting adherence.

I attended the World Orphan Drug Congress last week in Washington, DC, and Siren presented the workshop, “A Roadmap for Patient Advocacy Investment.” In the rare disease space, patient engagement is critical to every step of a drug’s development, including: filling clinical trials, getting FDA approval, obtaining reimbursement, getting patients diagnosed and promoting adherence.

Wendy White moderated with presentations from Jamie Ring of Genzyme, a Sanofi Company and Diane Goetz of PTC Therapeutics. Pat Furlong of Project Muscular Dystrophy and Lori Sames of Hannah’s Hope Fund provided the patient advocacy perspective. The event was overflowing with attendees–a mix of representatives from biopharmaceutical companies and advocacy organizations. Liz Donohue from Coordination of Rare Diseases at Sanford (CoRDS) shared her takeaways.

Megan O’Boyle from the Phelan-McDermid Syndrome talked about her highlights.

Lori’s recommendation to other patient advocates on how to work with pharma.

See Siren’s YouTube channel for more videos from the World Orphan Drug Congress including a review of the importance of patient registries.