we’ve created a white paper that summarizes the insights and experiences that were collected
For companies that operate in the rare disease space, compassionate use policies are critical. Rare disease patients and caregivers are highly motivated to access treatments and they are skilled at using social media, online petitions, blogs, and videos to generate support. Maintaining good relationships with the patient community requires not only having a well-articulated policy for compassionate use, but also communicating about this sensitive topic in an effective way.
Siren’s workshop at the 2014 World Orphan Drug Congress (WODC) generated a rich and informative discussion about this timely issue, with experts providing the perspective of industry and advocacy groups. Panelists included:
- Barbara Wuebbels, vice president of patient advocacy and medical affairs at Audentes Therapeutics
- Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy
- Ben Lenail, co-founder and business strategy officer of ALD Connect
- Julie Raskin, executive director of Congenital Hyperinsulinism International
- Carrie Burke, director of alliance development at Shire Pharmaceuticals
- Nicole Boice, founder and president of Global Genes/Rare Project
Now we’ve created a white paper that summarizes the insights and experiences that were collected, offers instructive case studies, and provides recommendations.
This is the first in a series of white papers that Siren is creating to share insights from the WODC workshop with you. Future topics include Effective Collaboration Between Patient Groups and Pharma and Trends in Global Advocacy. If you sign up (link to sign up) for our email list, we’ll be sure to notify you as soon as they’re available.
Download our white paper.