the personal views of a doctor in industry

NIH to kick-start research into rare and neglected diseases

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“The National Institutes of Health is launching the first integrated, drug development pipeline to produce new treatments for rare and neglected diseases. The $24 million program jumpstarts a trans-NIH initiative called the Therapeutics for Rare and Neglected Diseases program, or TRND.” ( NIH Announces New Program to Develop Therapeutics for Rare and Neglected Diseases, NIH News)


Rare diseases are those that affect less than 200,000 Americans, and neglected ones are those that affect the global poor disproportionately.


The program will identify suitable compounds for research in these disease areas and NIH will take them through pre-clinical development themselves. If they manage to get one through that far then they will look for external partners to take the compound on to phase 1 trials in humans.


Why do they feel the need to step in? According to the NIH press release, “Private companies seldom pursue new therapies for these types of illnesses because of high costs and failure rates and the low likelihood of recovering investments or making a profit”. As acting Director Raynard S. Kington notes, “The federal government may be the only institution that can take the financial risks needed to jumpstart the development of treatments for these diseases, and NIH clearly has the scientific capability to do the work.”


This is good news. Governments have a duty to to use some of our tax dollars to look after the less fortunate members of society. Some commenters are missing this point. The WSJ health blog for example: “It’s tough to get drug companies to work hard on drugs for diseases that are either rare altogether or common only in parts of the world where people can’t afford to pay much for treatment” (NIH Treads Where Drug Industry Rarely Goes). The point is that the pharmaceutical industry has no duty to be in unprofitable areas, and if it does venture there then it should be recognized for what it is: philanthropy.



Written by Pillhead

May 22, 2009 at 1:49 pm

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