Posted on June 15th, 2015
by Roberto Bellini
The World Orphan Drug Conference (WODC) took place on April 23rd-24th, 2015. This conference is an annual gauge of the rare disease subsector’s temperature and it was booming in 2015.
In its fifth year, WODC has grown from a tiny get-together to three full conference streams including an exhibition hall. While overall participation was also up to 700 delegates, the real benefit of the conference comes from its diversity: there is just as much participation from foundations, patients and caregivers and patient organizations, as there is from industry and suppliers.
This mix leads to interesting perspectives and I’m excited to share my takeaway messages from the conference with you.
“Ideal for Entrepreneurs”
Henri Termeer, of Genzyme fame, and often referred to as the grandfather of the rare disease industry, kicked off the conference with a video address. Mr. Termeer, who clearly synthesized certain elements of our industry in a manner that only a visionary could, especially resonated with the entrepreneurs in the room stating, “Drug development for rare diseases will be fragmented. This is the ideal environment for entrepreneurs.”
There are so many rare diseases (>7,000 identified) with so much unmet medical need, it seems unlikely to see the large pharmas dominate these spheres as they have done with the large indications (eg, diabetes, cardiovascular).
The relative modest development and commercial costs to rare disease drugs means small entrepreneurial biotechs don’t necessarily need the cash from partnering transactions. They also don’t necessarily need the development expertise from big pharma either; many of the indications have never had clinical studies so there is no prior indication expertise to leverage.
Patients Driving Drug Development
Patients and their organizations (whether organized as a disease foundation or not) are everything at WODC and generally speaking, they seem to be empowered. It makes sense because there appears to be nothing these groups are not capable of. This includes the more traditional roles such as patient support, providing information and increasing disease awareness.
Even more importantly, these organizations are becoming driving forces for developing rare disease drugs. Patient advocacy is propelling change at the government and regulatory level. Several organizations told me that the FDA has approached them to better understand their disease and how it affects patients. Patient organizations are also starting patient registries, collecting natural history data and even building lists of patients ready to enter clinical trials.
It’s easy to understand why some of the most well attended talks were those concerning pricing and reimbursement. The industry understands that there is no rare disease drug development without payers reimbursing these drugs at super premium levels.
The bad news is that payers expressed frustration with high pricing and price increases. The term “broken system” was bandied about the most often. The examples given included Sovaldi (‘Gilead added no value other than setting a high price’), Gleevec (‘High pricing can’t stay stable as rare disease drugs reach broader audiences’), and the significant price increases being taken in generics and specialty drugs.
The good news is that most of the criticism is being directed toward the system in general and specifically towards mostly non-orphan areas. If the system is broken, it seems the methods to fix it will likely not be focused towards rare disease drugs. A couple of ideas from the panel: better management of drug use including formulary exclusions when necessary and increased focus on use of generics and biosimilars (‘a Rituxan biosimilar would pay for half this room’).
It was definitely another interesting year at WODC. I also live-tweeted from the event several discussions so please check my Twitter profile @rbellini to see tweets on these subjects and more. I also want to thank a number of people who tweeted and re-tweeted about the conference, including: Sean Russell (@SRussellPremier), Chris M. Lindgren (@ChrisMLindgren), Tomasz Sablinski (@TSablinski),Total Orphan Drugs (@OrphanConf), Tarquin Coles (@TarquinColes), Dr. Michael Torres (@Mykalt45) and Pelstein Lab (@PerlsteinLab).
I’m looking forward to next year!
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