Leading up to Rare Disease Day 2013, Siren was pleased to write a series of articles for PharmaPhorum. We addressed key topics that are currently generating lots of discussion in the rare disorder community.
- Is Rare Disease Pricing Sustainable? Doug Paul from Medical Marketing Economics provided an overview of the history of orphan drug pricing, why rare disease therapies cost so much and whether this model is sustainable to continue moving forward.
- Rare Disease Research: The Current and Future State. Wendy White interviewed Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy, on the risk-benefit equation and the FDA move towards engaging patients.
- Patient Registries and Why They are Essential for Rare Diseases. I delved into the different types of registries, who should fund them and who owns the data.
- The Future of Social Media and Pharma: Collaboration. Katie Mihelich showed the evolution from listening to content cocreation.
- How Gene Therapy is Transforming Medicine. This is currently my favorite topic since reading the book, The Forever Fix, and with the first approval in the Western world of a gene therapy for sale, Glybera.
Please comment and let us know what you think about these topics.
Image courtesy of Steve Jurvetson on Flickr (CC BY).
- 08 April 2013 at 9:04pm
Read: "Hot Topics in the #RareDisease Community" on Siren Song: ...