On behalf of the rare disease community, we are respectfully requesting that just one dollar for each and every person suffering with a rare disease be appropriated by this Subcommittee for the FDA S Orphan Products Research Grant Program. Twenty-five million dollars would represent a minimal investment by the federal government in the development of lifesaving treatments that the private sector is not interested in. But the return on investment could be phenomenal if only a few new orphan drugs or devices are developed to reduce the burden of disease and death for thousands of patients with rare disorders. As you can imagine, appropriating just one dollar for each rare disease patient in America, rather than the current funding level of a mere fifty cents per patient, is a win-win situation. Patients win when their symptoms are alleviated or cured.
Families win when their loved ones no longer suffer.
Society, as a whole, wins when patients are able to return to school or work to become productive tax-paying citizens. Pharmaceutical and biotechnology companies win when they are able to market new therapeutic products when part of the development costs are subsidized. The scientific community wins when the knowledge they gain can be applied to more prevalent diseases. And, finally, the government wins when the drain on healthcare dollars is minimized. FDA Orphan Products Research Grants Program Congress created the research grants program in FY 1983 to provide funding for pivotal clinical trials on new orphan drugs, medical devices, and medical foods for rare diseases.