Orphan Drug Development: Opportunities and Challenges from a Midsized to Large Company Perspective


Orphan diseases may be one of the last frontiers in drug development. With an estimated 50 million or more people affected across Europe and North America by over 6,000 rare diseases, the unmet medical need is daunting. This research explores the question of whether the opportunities outweigh the challenges and provide incentives for midsized to large pharmaceutical and biotechnology companies to invest in this area.

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Correspondence to Rosann Reinhart MS, MBA.

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Reinhart, R., Madrzjewski, K. Orphan Drug Development: Opportunities and Challenges from a Midsized to Large Company Perspective. Ther Innov Regul Sci 44, 101–109 (2010). https://doi.org/10.1177/009286151004400201

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Key Words

  • Orphan drug
  • Rare disease
  • Orphan legislation
  • Orphan designation