Timezone: (GMT) Western Europe Time, London, Lisbon, Casablanca
There are about 7,000 rare diseases identified today. While these conditions affect relatively few people individually, taken together they affect an estimated 250 million patients worldwide. New research technologies have greatly accelerated the identification of these conditions as well as potential specifically targeted therapies. Consequently, over the past decade, the market for orphan drugs has increased about 26 percent, and there has been a rise in government programs devoted to these disorders. The recent reauthorisation of the FDA Prescription Drug User Fee Act (PDUFA-V) contains specific provisions to increase services to this community. There is an urgent need to develop appropriate endpoints to evaluate the effectiveness of new treatments for rare disorders, including Patient-Reported Outcomes (PROs) and other forms of Clinical Outcomes Assessment (COA). Given the nature of these conditions, existing measures may be inadequate to assess outcomes for these patients and innovative methods for developing and validating new measures are needed.
The objectives of this webinar are to:
Katy Benjamin, Director, Patient Reported Outcomes
Noelle Dietrich, Associate Outcomes Researcher