“Scientific opportunity has to be a big part of it,” says Francis Collins. Sometimes when a rare disease hits that moment of scientific opportunity it can reveal things about common illnesses as well.

Question: How does the NIH decide which medical research to fund?

Francis Collins: The process that NIH goes through to decide which research to fund is complex; there’s a lot of factors.  Certainly the burden of the disease has to be one of those, but if that was all you paid attention to, rare diseases would get neglected so that can’t be the only story.  

Scientific opportunity has to be a big part of it.  There’s no point throwing money at a problem if nobody had any ideas about how to move the ball forward.  And you can see then sometimes when a rare disease which may not affect that many people hits that moment of scientific opportunity, and oftentimes rare diseases teach you a thing about common diseases as well.  So, it’s a complicated mix.  

NIH depends very heavily on the scientific community to come forward with their best and brightest ideas, and they send us their grant proposals in an unsolicited way and that’s where the majority of our money goes.  But we also identify areas which are ripe for exploration, where something is really starting to go great guns and we don’t want to slow that down, in fact, we want to speed it up.  So in that situation, NIH would issue what’s called a "Request for Applications" saying, we think there’s opportunity here scientifically so we’re gonna set aside some money and we want people who have skills and interest in that area to come forward with some ideas and we’ll pay for the best in the group.  

Question: Since becoming director, have you put a specific emphasis on addressing specific diseases?

Francis Collins: I think it’s hard to pick out individual diseases and say, "Well those are more important that the others." But I think one can look at circumstances where there are especially ripe opportunity.  Cancer certainly would be on that list because we are beginning to understand cancer on a detailed molecular level, in ways that we never dreamed possible.  So there’s a real potential there for moving forward in a new quantum leap into understanding.  

Autism is certainly something many people are now focused on as an area of very high priority disease that affects now one in 100 kids.  This seems to be more common all the time and we don’t understand it very well—a very high sort of public health significance.  But many other things would also sort of fit on my list, diabetes, heart disease. Alzheimer’s disease—good heavens, when you look at the burden that is going to place on individual’s families and our economy.  

It’s all a mix though, and frankly what I’ve tried to do since I became Director of NIH in August of 2009, is to identify specific areas that actually touch on multiple diseases that are ripe for investment, so I came up with a series of five themes that, if pursued vigorously, could really change the landscape, but they would really do that for lots of diseases not just a few that are specifically targeted.

Recorded September 13, 2010
Interviewed by David Hirschman