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Orphan Drugs In The Pipeline For Rare Diseases: January 2013 Report

January 18, 2013

A January 2013 Report, from the Analysis Group and sponsored by Pharmaceutical Research and Manufacturers of America (PhrMA), is titled, “Innovation in the Biopharmaceutical Pipeline: A Multidimensional View”.

The Analysis Group did a study based on a review of data from several sources:

  1.   EvaluatePharma data from 1986 forward
  2.   Proprietary commercial database containing information on > 4,500 companies & 50,000 products
  3.   ClinicalTrials.gov data
  4.   Orphan drug designations from the FDA Orphan Drug Product database.

The Report uses the following metrics to describe the drug development pipeline:

  1.   Total # of medicines in development
  2.   Potential 1st-in-class medicines
  3.   Medicines targeting rare “orphan diseases”
  4.   Medicines targeting diseases for which there have been no recently approved therapies
  5.   Medicines that use a “personalized medicine” approach
  6.   Medicines that are the 1st to apply new scientific strategies to diseases.

According to this Report, the following are statistics related to rare diseases and orphan drugs:

  1.   From 1984 – 2011, the FDA reports granting a total of 2,626 orphan designations
  2.   From 1984 – 2011, subtracting 831 projects that have either been approved or withdrawn, gives a total of 1,795 orphan designations for projects in the development pipeline
  3.   From 1984 – 2011, > 350 drugs with an orphan designation are approved for marketing
  4.   In the 10 years before 1984, < 10 drugs with an orphan designation are approved and marketed
  5.   In the most recent 5-year period, orphan drug approvals account for 30% of approvals & the average population size for an orphan designation is about 39,000 patients.

References

Analysis Group, “Innovation in the Biopharmaceutical Pipeline: A Multidimensional View”

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