Take action to support the OPEN ACT, HR 971 (Orphan Product Extensions Now, Accelerating Cures & Treatments), which helps remove drug development roadblocks and incentivizes the development of treatments for the benefit of all rare disease patients. Biopharmaceutical companies possess a wealth of scientific knowledge from drugs that have already been approved for common diseases, some of which may have the ability to treat rare diseases.
However, there are inadequate incentives in place for companies to study potential impact for rare disease patients. Some patients may elect to take an approved drug for off-label use, in the hopes that it could treat a rare disease. Off-label usage, however, is typically not studied or tracked for clinical effectiveness. In addition, off-label use is not reimbursed by insurance plans, creating undue and unnecessary financial burdens on patients.
But Congress can fix this situation. Simple legislative changes can provide incentives for industry to invest in re-purposing existing drugs for potential use in the rare disease community. Modeled on the incentive programs in the Best Pharmaceuticals for Children Act (BPCA), the OPEN ACT would make available to drug companies an “Orphan Product Exclusivity Extension,” which would provide an additional six months of exclusivity to the patent life of the major market drug being repurposed for rare disease treatment so long as the sponsor company establishes that the therapy: (1) is designated to treat a rare disease, (2) obtains a rare disease indication from FDA on the drug label. These simple legislative changes could result in hundreds of new treatment options for rare disease patients who cannot afford to wait any longer for the therapies they need.