Orphan Drug Development

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Orphan Drug Development Industry Perspective
Food Drug Administration in the
U.S. Copenhagen, Denmark November 5, 2008 John
Wilson, Jr., PhD, MPH Senior Vice President,
Clinical Development
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Orphan Drug Development First Principle

• A rationally motivated pharmaceutical company
  focuses its resources on those endeavors that
  will yield the greatest returns.
• David D. Rohde

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Another Important Principle

• When all else is equal, the largest number of
  patients for a given drug or indication get the
  greatest attention by the pharmaceutical company.
  

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Historical Notes

• Prior to the Orphan Drug Act, there was little
  incentive to develop orphan drugs
• All about recouping RD costs plus profit
• Many potential orphan compounds could not be
  patented

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Efficient Markets

• Prior to the Orphan Drug Act, the market was
  working normally and fairly
• Unless you happened to have a rare disease
• 5 million Americans suffer from a rare disease
• 5 million 1.6 of the U.S. population

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Orphan Drug Development Hard to Study

• Orphan Drug Challenges in study
• Nature of disease
• RD efficiencies
• Lack of profit potential
• Tort Liability
• Uncertainty and unpredictability stacked onto an
  uncertain and unpredictable business model

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Orphan Drug Act

• Incentives!!
• Most significant
• 7-year marketing exclusivity
• What does this mean?
• Not the same as traditional patent
• Only for the rare disease for which it was
  approved
• Another sponsor could receive approval for
  another use, even another rare disease

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Industry Expectations

• Protocol design assistance
• Marketing exclusivity
• Limited monopoly
• Faster FDA review time
• Well, not exactly..

Date
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Some Realities

• Usually related to Development rather than
  Discovery
• Potential orphans discovered during drug
  development for larger populations
• Many halt at this stage, due to no data

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Some Recent Successes

• Tuberculosis
• Neonatal respiratory distress syndrome
• Hairy cell leukemia
• Cystic Fibrosis
• Refractory childhood ALL
• (Acute Lymphocytic Leukemia)

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Joint EMEA/FDA Application Process

• Simultaneous Orphan Designation
• Application
• One Form
• One Process

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