PharmaVOICE Blog Post

Confronting the Challenges of Rare Disease

Posted By: Dan Limbach
March 6, 2018

The Orphan Drug Act of 1983 brought increased awareness to the need for new treatments for rare disease patients and provided incentives to pharmaceutical manufacturers, including tax credits for the costs of clinical research, seven years of patent exclusivity, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees. Of the 3,500 drug designations issued by the Office of Orphan Products Development since 1983, more than 500 have resulted in marketing approval. Still, only 5 percent of the estimated 7,000 known rare diseases have a therapy approved by the U.S. Food and Drug Administration (FDA), leaving substantial unmet medical needs for patients.

Key takeaways:

  • Learn how rare disease hurdles impact clinical trial design
  • Uncover how to engage rare disease patients in a trusting relationship
  • Review Expanded Access Program considerations in orphan disease populations
  • Understand the need to meet patients and prescribers needs(?) during and after the clinical trial

Download the white paper now for a deeper dive into these topics.

Provided by United Biosource Corporation

About the Blog Poster: Dan Limbach

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