According to a story from Federal Telemedicine News, there are around 7,000 diseases known to science that can be considered a rare disease. When we consider these diseases in the aggregate, that means that a large number of people around the world are directly affected by them, even if the patient population for a given rare disease is fairly small. As an example, there may be as many as 30 million people in the US that are rare disease patients.
The Rare Diseases Clinical Research Network
In 2002, Congress established the Rare Diseases Clinical Research Network. This network was designed to make these illnesses easier for scientists to study. Part of this network is the Consortium of Eosinophilic Gastrointestinal Disease Researchers, which is stationed out of Cincinnati Children’s. The National Center for Advancing Translational Sciences (part of the National Institutes of Health) recently awarded the hospital a $28 million dollar five year grant designed to support its new role as the coordinating and data management center.
This entails the largest grant that Cincinnati Children’s has ever received. Additionally, the hospital will play a role as a center of learning for scientists to share best practices related to conducting research on rare diseases. Ultimately, the hospital aims to be the primary source of network research data for a variety of relevant stakeholders, from the scientists, patients and their families, the US Food and Drug Administration (FDA), and drug companies.
The Brittle Bone Disorders Consortium
The Baylor College of Medicine has also served as the leader of the Brittle Bone Disorders Consortium, using $7 million in grants from the NIH over the last five years. This consortium primarily focuses on osteogenesis imperfecta, which causes unusually brittle bones. The efforts of the consortium have successfully led to the development of a treatment that can impact the disease mechanism of action.
The FDA’s Orphan Products Clinical Trials Grants Program has been used to 12 grants to the tune of $15 million that are intended to fund the implementation of clinical trials for rare disease treatments for the next four years.
