Orphan Diseases… a new pharmaceutical strategy?

Posted by cdavenport on Friday Dec 4, 2009 Under Pharmaceutical Business

As the old “blockbuster” mentality  fades in the wake of relatively weak pipelines, increased emphasis on drug safety, slow sales growth, and the loss of patent protection on older blockbusters, it would appear that the orphan-disease niche (i.e., low volume: high cost) has become an increasingly attractive lure to major pharmaceutical companies.   While there may be few patients with each disease, David Simmons – president of Pfizer’s established product’s business unit- said “collectively, it’s a very large patient population with a great unmet medical need.”  In addition to Pfizer, other major pharmaceutical companies entering this space include GlaxoSmithKline and Novartis.   Source:  New York Times.

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One Response to “Orphan Diseases… a new pharmaceutical strategy?”

  1. bindhu michael Says:

    Orphan drug designation is on the rise and big pharmas are increasingly getting in this action. Here are some numbers: In the decade before the passage of the Orphan Drug Act in 1983, there were only 10 orphan drug approvals. After 1983, there are more than 300 orphan drug approvals. Very recently, Pfizer acquired rights to Protalix’s Gaucher drug, GSK has a deal with Prosensa to develop drugs for Duchenne muscular dystrophy, Novartis got FDA approval to sell Ilaris for cryopyrin-associated periodic syndrome (with only 300 patients in the US).

    I think orphan drug development is a wonderful thing for the 20–30 million people who are affected by 6800 orphan diseases. Most orphan drug development was done by academicians and small companies with not much financial, logistical or technical capabilities. Big pharmas getting involved in orphan drug development gives hope that more diseases (and more people) will find a cure. There is increasing focus on personalized medicine, and orphan drug development seems to be quite similar to the personalization approach (since the designation of orphan drug means less than 200,000 patients in the US). For more about that, see this new article: Drug development for orphan diseases in the context of personalized medicine, George J. Brewer, Translational Research, Volume 154, Issue 6, December 2009, Pages 314-322

    Orphan drug act states “there is no reasonable expectation that costs of research and development of the drug for the indication can be recovered by sales of the drug in the United States”. No wonder there is criticism about the pharma/biotech making huge profits from orphan drugs! Criticisms include pricing too high, misusing the orphan drug designation in numerous ways such as off label use of orphan drugs, using the orphan drug designation for exisiting drug with a widespread existing use. Examples: It is reported that Genzyme made over $800 million from imiglucerase in 1 year although there are only 20,000 Gaucher patients in the US. It is also reported that Epoitin alfa and recombinant human growth hormone have generated huge revenues for Amgen, Pfizer etc from off-label use. To see criticism about Reckitt Benckiser’s buprenorphine’s orphan drug status, see this news article: http://www.baltimoresun.com/news/nation-world/bal-te.orphan18dec18,0,7085040.story . The orphan drug act offers tax breaks and a seven-year exclusivity to encourages companies to pursue treatments for diseases afflicting small numbers of patients. To see criticism about misuse of exclusivity, click http://www.cptech.org/ip/health/orphan/

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