Charities Try to Fill Research Gaps to Combat Rare Diseases

As health charities seek to develop drugs, many are focusing their money on the crucial first stages of devising new treatments. But some groups are also trying to fill other gaps that impede progress toward new treatments.

Finding enough patients to test new therapies can be difficult when dealing with diseases that affect only a small number of people, says Robert J. Beall, chief executive of the Cystic Fibrosis Foundation, in Bethesda, Md.

“There are a lot of rare diseases that have so few patients you can’t do the clinical trials, or it’s just very difficult,” he says.

To avert such a problem, his charity set up a network to help companies design and conduct clinical trials. It also ran a campaign to educate its members about the importance of clinical trials in developing new treatments.

In addition, the Cystic Fibrosis Foundation’s patient registry tracks information on the more than 24,000 people who are treated at its 115 cystic-fibrosis care centers across the country and keeps it anonymous to protect patient privacy. Among the many uses for the data: helping researchers design clinical trials for potential therapies.

Models for Research

Developing disease-specific research tools is another way health charities seek to speed new treatments.

The Michael J. Fox Foundation for Parkinson’s Research, in New York, has awarded more than $8-million for research to help develop animal models of Parkinson’s disease. The dearth of such models has been a roadblock in the development of treatments that tackle the disease itself, rather than just its symptoms.

Because drug companies cannot earn money by developing the models, they have no incentive to develop them, says Katie Hood, the charity’s chief executive.

“All of the other players wish that these tools existed, but none of them really wants to invest that much money in getting them done,” she says.

Educating Doctors

Approval of a new treatment doesn’t necessarily end charities’ involvement.

In addition to paying for research for new treatments, the Juvenile Diabetes Research Foundation, in New York, has turned its attention to educating doctors about new developments and convincing insurance companies to pay for them.

When implantable glucose-monitoring devices were first introduced, insurance companies said the devices were a nice idea, but they wanted proof that they worked better than the common finger-prick test, says Peter T. Lomedico, director of industry partnerships at the Juvenile Diabetes Research Foundation.

So the diabetes organization ran a clinical trial with more than 300 participants comparing the two approaches. The study found that the devices provided for better blood-sugar control and that the people using them had fewer hypoglycemic episodes. As a result, says Mr. Lomedico, insurance companies are slowly expanding coverage for the devices.

“We came to realize that simply having regulatory approval sometimes is not enough,” he says. “Sometimes you need to deal with issues like reimbursement and physician acceptance in order to have these therapeutic options really available to patients.”