Case Overview, Funding for Cystic Fibrosis Research

 

           Thirty thousand Americans suffer from cystic fibrosis, a terrible disease that cuts short the lives of its victims. Indeed, those afflicted with cystic fibrosis have an average life expectancy of just 32 years. The culprit is a defective gene that causes the body to produce a thick mucus that compromises the lungs and damages the pancreas. The primary symptoms include a persistent cough and shortness of breath.

          Although a cure does not appear to be close, there is some good news as better care and new drugs have lengthened the lives of those who suffer from cystic fibrosis. There is a great deal of research on the disease underway and there's optimism that new treatments are on the horizon. A network of eight leading hospitals spearheads research on cystic fibrosis and pharmacological trials can be expedited through this set of research centers. Working together these research institutions form a unique clinical trials network - the Therapeutics Development Network - that shares data efficiently and maintains clinical trials infrastructure so that new experimental drugs can be put into the trial phase quickly. This structure is quite different from standard routine, in which running trials on a new drug requires building the infrastructure from scratch, substantially lengthening the trial process, and thus delaying the approval of potentially useful drugs.

          Research costs money and lots of research costs lots of money. The National Institutes for Health (NIH) is the federal government's primary source of support for medical research. The NIH is a huge enterprise composed of 27 different institutes and research centers. It has its own staff of physicians and scientists and conducts research in its own labs. NIH also funds research at universities and hospitals around the country. NIH receives budgetary substantial support from the Congress and its appropriation for 2002 was $23.4 billion.

          That 23.4 billion is allocated among a large number of diseases and there's probably not a single organization working on behalf of those with a particular disease that feels that enough research is being done on that malady. Congress does not set the relative allocations among diseases, though legislators are certainly not hesitant to encourage the NIH to do more in a particular area. The reality is that the disease groups are in competition with each other. Although its fortunate that cystic fibrosis affects only a small number of people, there's concern among those who work on behalf of that constituency that they're at a disadvantage in comparison to more common diseases. An advocate on behalf of cystic fibrosis said, "For example, diabetes-there are so many people in this country that have diabetes and congressmen that are affected personally that it is easier for them to recognize the importance of getting involved."

          The Cystic Fibrosis Foundation is the primary voice for those who have the disease and their families. Despite the competition among the groups in Washington working on behalf of various illnesses, these organizations recognize that they have common cause in trying to expand the "pie"-the overall budget for NIH. The Ad Hoc Group for Medical Research is the coordinating body for this coalition. When advocates for cystic fibrosis suffers go to Capitol Hill, their message is simple: "the value in investing in research and that's not a hard sell. Most people are supportive of research, and specifically, our mission [is] the fact that we have been successful so far."

          In 2002 the Cystic Fibrosis Foundation was working with the other disease groups for an NIH budget of 27.3 billion. When the final numbers were approved by Congress the disease groups had gotten an increase in NIH funding. The other major goal in 2002 was to gain more funding for the unique Therapeutics Development Network, while advocates were disappointed that NIH didn't move to direct funding of the clinical trials network, their push for support continued into 2003 during the 108th Congress.