This is my first visit to San Francisco, the city by the bay, home of the Golden Gate Bridge and Tony Bennett’s song “I Left My Heart in San Francisco,” a song from my younger days and one that, even today, sometimes gets in my head and repeats and repeats.
Some time back I was invited to participate in the Rare Disease Leaders’ Forum, participating in a working group to discuss how the role and business model of patient groups is evolving, the funding models, de-risking specific programs or areas of drug development, building patient registries and natural history data and its relevance to Phase IV, post marketing surveillance, and what might be a recipe for success (and failure) in rare diseases. As the working groups for these discussions evolved, I was invited to chair a workshop on entering and collaborating with patient advocates, a discussion centered around piecing together an assembly line of information to make potential rare disease opportunities more tangible and to discuss lobbying for reimbursement and how we might ensure a sustainable model for price and access throughout the product lifecycle. These are complex issues but there are solutions and key reforms that need to take place to ensure access.
I’m looking forward to these discussions and will report back what I learn.
Pat Furlong, Founding President, CEO
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