Recognizing the patient groups’ involvement in orphan drug development

In Advocacy, Clinical Development, Partnering & Investment, Regulation & Government by André Singer

For many years now, even in a well-established industry as the orphan drugs one, we have been noticing a shift in the way that pharma and biotechs are relying on a broader and more effective involvement of patient advocacy groups in orphan drug development.

Examples of this successful engagement are many. From Cystic Fibrosis Foundation‘s scientific, clinical and financial support to the development of Vertex‘s Kalydeco drug, to Parent’s Project Muscular Dystrophy‘s efforts in the public policy front for a better benefit-risk assessment for duchenne muscular dystrophy therapies in clinical trials.

Every orphan drug stakeholder – government, industry, academia, and payers – have now clearly understood the crucial role that patients and patient organizations are playing in expediting the launch of new rare disease therapies in the market.

In light of that promising collaboration, the 4th edition of the World Orphan Drug Congress USA, to be held in Washington D.C. on April 23-25, will be featuring the Patient Zone. This program will not only allow a better interaction between patient groups and other stakeholders, but also bring into discussion the most challenging issues that patient organizations face in creating awareness about the disease and accelerate research.