PharmaVOICE Blog Post

Great Information on Rare Diseases and Orphan Drugs

Posted By: Dan Limbach
February 8, 2016

If you work in the rare disease space, you will be very interested in this blog post. Publicis Touchpount Solutions recently published an outstanding white paper on rare diseases and orphan drugs. It’s packed with valuable statistics, research findings, images, and charts. Compared to most white papers, it’s a fairly deep dive, with 25 pages in total, including links to additional reference materials.

Orphan Drugs Success: Engaging Key Stakeholders to Deliver Positive Outcomes

Here are some of the more interesting things I took away from the paper.

  • Rare diseases affect as many as 30 million Americans
  • There are 8,000 identified rare diseases , growing by 250 annually
  • Up to 80% of rare diseases have a genetic origin, which we are understanding better every day
  • Rare diseases cause 35% of deaths occurring within the first 12 months of life
  • About half of all people suffering from rare diseases are children
  • The patient population and clinical trial size is much smaller for orphan drugs, along with a higher FDA approval rate
  • By 2018, the rare disease market could grow to $127 billion at double the growth rate of non-generic prescription drugs
  • Many orphan drugs are blockbusters, at over $1 billion in annual sales
  • People working in this space are among the most dedicated in the industry
  • Commercialization teams can utilize a 200-point questionnaire to develop their strategy

Find out how you can improve your chances for success in the rare disease space.

Download the white paper now, and let me know what you think of it in the comments.

Get the White Paper

About the Blog Poster: Dan Limbach

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