By Amy Dockser Marcus, Wall Street Journal
Is it time to declare a national “war on rare diseases” comparable to the famous “war on cancer” that began in the 1970s?
The Institute of Medicine report released today stops short of recommending a full martial assault, but it does call for the creation of an aggressive national strategy to accelerate drug development. Policymakers, Congress, the NIH, FDA and patient advocates are all taking a hard look at how to improve research and development for rare diseases, which affect about 25 million people a year.
A number of innovative approaches are already being tried, including a program at NIH to get more involved in the development of drugs for rare diseases and an effort by the FDA to encourage orphan drug filings. The report lauds these new approaches but says that the sheer number of rare diseases, the different priorities of various researchers and advocacy groups and limited available resources require a national, integrated strategy.
One key recommendation is national task force, set up by the head of HHS, to make sure NIH, FDA and patient-advocate efforts are better coordinated and monitored.
Other suggestions include setting up a repository of publicly available animal models for rare disorders and another public repository of biological data on rare diseases. Both could potentially be used by patient groups as well as investigators.
Thomas Boat, the chairman of the IOM committee that wrote the report, tells the Health Blog that up until now, rare-disease care and research has been disorder-specific, so “efforts have really been segmented.” Boat says the recommendations are designed to increase cross-fertilization of ideas and sharing of resources such as repositories and specimens. He adds that committee members briefed NIH and FDA officials on the findings last week and also hope to talk to legislators.
Timothy Coté, director of the FDA’s orphan products development office, tells the Health Blog a congressionally established committee he chairs is assessing the new report. Its recommendations for how FDA ought to tackle rare diseases are due to Congress by next March, he says, and the report’s ideas will be a big part of that strategy.
“They are right. We feel we already launched a war, and now it’s time to escalate,” he says.
Photo: Associated Press