A new programme to develop cures for rare or ‘orphan’ diseases has been unveiled in the US. The National Institutes of Health is putting $24 million up for work on some of the 6,600 rare diseases that impact 25 million Americans and which currently have no effective treatments.

“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,” says NIH Acting Director Raynard Kington (press release).

NIH’s definition of a rare disease is “one that affects fewer than 200,000 Americans”. It also notes that it can cost $10 million to get a treatment through the pre-clinical drug trial process. Between 80 and 90% of drugs fail, leading this stage to be dubbed the ‘valley of death’.

Obviously $24 million isn’t going to go far with costs of up to $10 million per drug, but NIH’s new Therapeutics for Rare and Neglected Diseases programme will aim to improve the drug development process itself, as well as coming up with its own treatments.

”Preclinical work is hard and our resources will be limited,” Stephen Groft, director of the NHI rare diseases offices acknowledges (WSJ).

Reuters notes the TRND programme will also be reporting its failures, something not widely practised in drug development. “We are going to tell everyone what we are doing,” says Christopher Austin of the NIH Chemical Genomics Center. “That alone will be revolutionary.”

Derek Lowe, on the In the Pipeline blog, adds:

Treating rare diseases can be quite profitable in the industrialized world (ask Genzyme, among other companies), but if the conditions are localized in poorer areas no one’s likely to take a crack at them. So my first reaction is ‘Good, and the best of luck to you’.

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