The World Orphan Drug Congress USA last week consisted of three days jam-packed with presentations and networking. While the event focused on rare diseases and orphan drugs, the scope was still wide and addressed issues ranging from pricing to patient registries to various country regulations.
The highlights for me were the patient/caregiver testimonies scattered throughout the conference which reminded everyone why we were in the room. The presentation by Yi-Ou Wang from the nonprofit organization China-Dolls Fund was especially powerful as she described the lack of social awareness and support for rare diseases in China.
I had the chance to talk with a few empowered caregivers: Melissa Hogan from Saving Case, Lori Sames from Hannah’s Hope Fund, and Kelli Foster from The Mastocytosis Society. I was also pleased to finally meet two legends within the rare disease industry: John Crowley from Amicus Therapeutics and Pat Furlong from Parent Project Muscular Dystrophy.
The theme that was woven through many of the presentations was the idea of harmony—the state of being in agreement. Kinnari Patel, Bristol-Myers Squibb, started the first day by noting that harmonization across all rare disease stakeholders is essential for success. There are a variety of levels of harmonization required, and many of the speakers are working on facilitating these partnerships.
Harmonization between companies
Eric Mosbrooker from Alexion and Mahesh Kolar of Pfizer both talked about the benefit of “big pharma” and small biotechs partnering to bring rare disease therapies to market. Kolar noted that larger pharmaceutical companies can offer global reach, regulatory and reimbursement expertise and manufacturing capabilities.
Harmonization around research
Dr. Nurjana Bachman, Children’s Hospital Boston, discussed that academic medical centers are a place where collaboration is happening between research and industry. She noted that academia is successfully getting involved much earlier with drug development.
What will it take to create a sustainable orphan drug system? Wills Hughes-Wilson, Swedish Orphan Biovitrum, felt that collaboration and cooperation were essential due to the small amounts of rare disease data.
Harmonization on patient registries
Chairing a panel on patient registries, Furlong noted that with multiple drug companies having their own private registries for the same disease it’s very confusing to patients. The panel discussed the benefits that can come from sharing data. According to Dr. Richard Moscicki, Genzyme would be willing to partner with others and collaborate on registries.
Harmonization between governments
Yann Le Cam, EURORDIS, said, “rare diseases are an international public health challenge and need international collaboration.” He talked about the need to have more coordination between the FDA and the EMA. He is part of an international team working on collaboration on policy guidelines. Also needed are rare disease treatment guidelines, access to specialists, and patient and clinical data.
Harmonization with patients
Hans Schikan, Prosensa, talked about the importance of communicating with patients when designing clinical trials. Megan O’Boyle echoed the cooperative feelings of other patients in the room: “We want to help pharma help our kids in any way we can. We will give you our data and help.” O’Boyle has spearheaded the formation of an international Phelan-McDermid Syndrome registry, which in one year has signed-up almost half the diagnosed individuals.
The conference left me with the feeling that there is much work to be done, but also the sense of how far we’ve come. Angus Russell commented that it’s time for an industry shift to put patients at the center, which is reflected in Shire’s tag line “To be as brave as the people we help.”
For more information on orphan drugs and rare diseases, check out the World Orphan Drug Congress USA. Siren Interactive was a silver sponsor of the 2012 event.