Patient Reported Outcomes for the Evaluation of Rare Diseases: Developing a New Paradigm

14th February, 2013
Time: 15:00 - 16:00

Location: Webinar
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:  

  • Describe the present landscape of rare disease research, regulation and reimbursement  
  • Provide a background on the nature of PRO endpoints  
  • Describe the methodological challenges in developing PROs and other COAs for rare diseases  
  • Discuss potential next steps in finding solutions to these challenges 

Katy Benjamin, Director, Patient Reported Outcomes 

Noelle Dietrich, Associate Outcomes Researcher