The US National Institutes of Health (NIH) and the Food and Drug Administration (FDA) should work together to bring products to market (Nature 471, 135; 2011). A telling example reveals how well this can work.

In the early 2000s, the National Institute of Child Health and Human Development conducted clinical trials of 17P, a drug that successfully reduced pre-term birth by more than 30% in women who had experienced it previously (P. J. Meis et al. N. Engl. J. Med. 348, 2379–2385; 2003). The FDA granted the drug orphan status in 2003 to motivate the pharmaceutical company behind 17P to apply for FDA approval, which was granted this year after using the same NIH trial for the approval process.

In the interim, compounding pharmacies sold the drug at about US$400 per course of treatment. Some insurance companies chose not to pay for it because the drug was not FDA approved, which increased their later spend on complications from preterm births that could have been avoided.

With 17P approved and protected from competition for 7 years, the pharmaceutical company raised the drug's price to $30,000 and sent stern letters to pharmacies. In a later announcement of FDA drug-enforcement priorities, 17P was low on the list because no safety issues had been reported regarding pharmacies.

This breakthrough would not have occurred had the NIH not focused on solving the costly and complex issue of preterm birth, or without the FDA having granted orphan status and publicly announcing its enforcement priorities.