OOPD provides incentives for sponsors to develop products for rare diseases program enables the development and marketing of drugs, biologics, and medical devices for rare diseases Orphan Drug Designation Program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.
https://www.gilead.com/news-and-pre...cess-to-remdesivir-outside-of-clinical-trials Gilead Sciences Statement on Access to Remdesivir Outside of Clinical Trials Gilead is working to rapidly assess the safety and efficacy of remdesivir as a potential treatment for COVID-19 through multiple ongoing clinical trials. Enrollment in clinical trials is the primary way to access remdesivir to generate critical data that inform the appropriate use of this investigational medicine. We recognize that there are severely ill patients who are unable to enroll in clinical trials and for whom no approved treatment options are effective. Gilead has been working with regulatory agencies to provide remdesivir to these patients where feasible. To date, we have provided emergency access to remdesivir for several hundred patients in the United States, Europe and Japan. In recent weeks, there has been an exponential increase in compassionate use requests for emergency access to remdesivir, related to the spread of the coronavirus in Europe and the United States. This has flooded an emergency treatment access system that was set up for very limited access to investigational medicines and never intended for use in response to a pandemic. To streamline the emergency access process, Gilead is currently in the process of transitioning from individual compassionate use requests to expanded access programs. This approach will both accelerate access to remdesivir for severely ill patients and enable the collection of data from all participating patients. These programs are currently under rapid development in conjunction with national regulatory authorities worldwide, and may vary by region based on local laws and regulations. During this transition period, we are unable to accept new individual compassionate use requests due to an overwhelming demand over the last several days. We are focused now on processing previously approved requests and anticipate the expanded access programs will initiate in a similar expected timeframe that any new requests for compassionate use would have been processed. As an exception, compassionate use requests may still be made for pregnant women and children less than 18 years of age with confirmed COVID-19 and severe manifestations of disease. We recognize the urgent need and are working to implement expanded access programs as quickly as possible, with the continued support and collaboration of regulatory agencies.