- In rare disease, patients look ‘upstream’ with hope for development of new treatments and access to trials and ’downstream’ for better, faster diagnoses and access to treatments. No other disease category faces the same sorts of challenges all along the continuum. Recent market conditions have halted development of a number of promising potential therapies, and new policies, coverage decisions, financial pressures and other factors have generated additional gaps, delays and difficulties in access to newly approved medicines.
- New approaches that are emerging from discovery to treatment in response to these challenges.
Moderator:
Dr. Ron Jortner, Founder/CEO, Aspire Biosciences
Panelist:
Julie Powell, Director, Patient Advocacy and Public Affairs, Pleco Therapeutics
Lindsay Randall, Founder, CEO, Mother, Arthur’s Quest
Hazel Dawson, Market Access Director, UK & Ireland, Alexion Pharma
Robert Donnell, EVP, Medicines Access, Smartway Pharma