In the EU, as stated in Article 3 of Regulation (EC) No 141/2000 on orphan medicinal products, the criteria for orphan drug designation are as follows:Ī) that it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting no more than 5 in 10,000 persons in the Community when the application is made, The definition of a rare disease varies across jurisdictions. OOPD also maintains a database of products with orphan drug designation. The FDA’s Office of Orphan Products Development (OOPD) is responsible for evaluating requests for orphan drug designation as well as other duties related to the development of products for the diagnosis and/or treatment of rare diseases.
In the US, the Orphan Drug Act, codified in 21 CFR Part 316 provides procedures to encourage and facilitate the development of drugs for rare diseases, including the process for requesting orphan drug designation.
All medicines that gain a GB orphan marketing authorisation will be listed on the Great Britain Orphan Register. However, in contrast to the EU, the MHRA will not review applications for orphan designation until the time of a marketing authorisation application (MAA) submission there will be no pre-marketing authorisation orphan designation. Since the end of the Brexit transition period on 1st January 2021, Great Britain (GB) has its own orphan designation procedure which mirrors that outlined in Regulation (EC) No 141/2000. The final decisions on orphan designation are issued by the European Commission (EC), who also publish a register of designated orphan medicinal products. The Orphan Regulation also established the Committee for Orphan Medicinal Products (COMP), which is responsible for evaluating applications, and recommending orphan designation. In the EU, Regulation (EC) No 141/2000 (the Orphan Regulation), defines the procedure for designation of orphan medicinal products, and defines the incentives for development and marketing of orphan designated medicines. The recognition of this by competent authorities has led to the creation of new regulations and policies to encourage and incentivise orphan drug research and development, including licensing, pricing and reimbursement. Historically, rare disease drug development was often limited by the prohibitive cost of the pharmaceutical program and the perceived low probability of successfully recouping the drug development costs. As such, there is a significant need to develop therapies for these patients. There are between 50 rare diseases2 however only approximately 130 in the EU3, and 500 in the US4 have approved treatments. This represents 400 million people worldwide, of which 30 million Europeans and 25 million Americans, and that makes the diseases and finding treatments for them a serious public health concern2. While each disease is rare, collectively, these disorders affect 6–7% of the population in the developed world1. A rare disease is any disease that affects a small percentage of the population, although the exact definition varies across jurisdictions.
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