Download: PPMD on benefit-risk assessments in rare disorders

In Partnering & Investment, Reports, Whitepapers by Caroline Hornby

parent project muscular dystrophy on benefit-risk assessment in rare disorders

Pat Furlong of Parent Project Muscular Dystrophy kindly shared this whitepaper, ‘Benefit-Risk Assessments in Rare Disorders: The case for therapeutic development in Duchenne Muscular Dystrophy as the prototype for new approaches.’

Download a copy here.

“Benefit-risk assessments are essential to the Food and Drug Administration’s (FDA) regulatory process for approving therapeutic products, and to informing the patient and physician selection or rejection of approved products. This paper examines the similarities and differences between benefit-risk assessments for common, progressive disorders vs. rare, progressive diseases. In doing so, the paper offers recommendations for a distinct framework for benefit-risk assessments in rare diseases, to recognize the unique aspects associated with such decisions in the rare disease sector.”

The paper goes on to explore:

  • Comprehensive conceptual and operational approaches to benefit-risk considerations
  • Considerations in rare disorder PFDD
  • Duchenne as a prototype for rare disorder benefit-risk assessment
  • Survey data regarding Duchenne views on benefit and risk
  • The case for Duchenne Muscular Dystrophy as the prototype for rare disease benefit-risk regulator decision making

To learn more about this download a copy today.

Pat will be participating at the upcoming World Orphan Drug Congress USA 2014.