Insight journal, Insight journal - Therapy

Orphan diseases partnering: A lucrative step for drug makers

Posted on 02 September 2014

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By Shamini Thiagarajan

Increased collaborations, big pharma involvements, venture capital investments, and medical/technological breakthroughs are together changing the landscape of research and development in orphan diseases.

Key Findings

  • 2014 has already seen orphan diseases deal values total up to $631 million
  • M&A activity is high for orphan drug makers
  • Increase in big pharma involvement over the past year
  • Technology behind partnering is shifting from drug delivery to genetic approaches

As the name suggests, orphan diseases are very rare diseases affecting fewer than 200,000 individuals. In the past, the pharma industry did not pay a keen interest into the research and development of orphan drugs due to the low financial returns in producing and marketing these drugs to prevent or treat the disease.

However, with the increasing need to treat patients affected by orphan diseases, government and regulatory authorities have introduced various incentives to encourage drug makers to provide solutions. This has in turn resulted in an increase in partnering activity in the orphan diseases therapy area.

This summary article reviews the area of orphan diseases in terms of partnering and deal making, key players in the industry are highlighted and the emerging trends of the field are revealed. The piece also explores the popular types of technologies being used to target orphan diseases and concludes by examining the challenges and opportunities faced by the lucrative industry.

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