b'Orphan Drug Research Because a single autoimmune condition usually affects only a small percentage of the population, at times it may not seem like a wise nancial investment for drug developers to pursue an experimental treatment. Even a more common autoimmune disease such as MS, which affects one million Americans, is small in comparison to conditions such as high cholesterol, for which 47 million Americans are treated every year, or the 40 million being treated for migraine. Nearly 95 million Americans are treated for high blood pressure. Yet some of the 100 autoimmune diseases, affecting 200,000 Americans or less, are classified as "rare diseases". For these conditions, there would seem to be little incentive for companies to invest millions or billions of dollars in research. However, the government addressed this issue with the Orphan Drug Act in 1983. An orphan drug is a drug for a rare disease or condition. Some rare disease treatments have been orphaned or discontinued because there was not enough financial incentive to continue development or production. The Orphan Drug Act incentivizes drug development for rare diseases. Understanding the Orphan Drug Act Here are the five key provisions of the Orphan Drug Act that all lead to encouragement and incentives for pharmaceutical companies to invest in further research.1. Orphan Drug Designation: The ODA allows drugs intended for the treatment, diagnosis, or prevention of rare diseases to be designated as orphan drugs by the FDA. This gives many incentives to sponsors, such as tax credits for qualied clinical testing expenses, eligibility for orphan drug grants, and exclusivity in the market upon approval. 2. Market Exclusivity: Orphan drug designation grants the sponsor of the designated drug a period of market exclusivity. This means during the time an orphan drug is approved and being sold in the market, no other drug with the same active ingredients and indication for the same disease state can be approved by the FDA. This period of exclusiveness is generally seven years and will begin on the rst day of FDA approval of the drug.3. Tax Incentives: Sponsors of orphan drugs are eligible for tax credits. The tax credits can be put towards qualied clinical testing expenses that occur during the development of the designated drug.4. Grants: The ODA authorizes the FDA to award grants that support clinical testing of orphan drugs. These grants can help with covering the costs linked to conducting clinical trials and further contribute to the betterment of treatment for diseases such as MS. 5. FDA Assistance: The FDA helps sponsors pursuing orphan drug generation and approval, including guidance on regulatory requirements, an expedited review processes, and access to scientic expertise on related information.17'