The US Orphan Drug Act (ODA) was passed in 1983 with the aim of stimulating the development of drugs for rare diseases, defined as those affecting fewer than 200,000 people in the United States. The law provides 7-year marketing exclusivity to sponsors of approved orphan products and a tax credit of 50 percent of the cost of conducting human clinical testing. Exclusive marketing rights begin on the date that the marketing application is approved by FDA for the designated orphan drug, and applies only to the indication for which the drug has been designated and approved. Other companies must prove clinical superiority before they are allowed to market another version of the same drug. Since 1997, companies developing orphan products also benefit from an exemption from the usual user fees charged by the Food and Drug Administration (FDA). Applications for orphan drug designation are handled by the Office for Orphan Product Development (OOPD), part of the FDA.
The OOPD also provides research grants for phase 1, 2, or 3 clinical testing of new therapies to treat orphan diseases prior to approval. In financial year 2006, over 14 million dollars will be made available for this programme. So far 39 orphan products have been granted marketing approval following funding of clinical studies through the scheme.
The US ODA in figures:
Since 1983:
2175 applications for orphan drug designation have been received by the OOPD 1024 orphan products have been give orphan drug designation 272 orphan products have been given marketing authorisation 224 products were withdrawn by sponsors before marketing authorisation was granted Transatlantic cooperation Since the beginning of this year, companies wishing to apply for orphan designation in both Europe and the USA have the possibility of applying for parallel scientific advice from the European Medicines Agency (EMEA) and the FDA through a
pilot programme initiated as part of a confidentiality agreement between the two agencies. The goal of this pilot is to provide a mechanism for EMEA and FDA assessors and sponsors to exchange their views on scientific issues during the development phase of new medicinal products with the expected advantage of increased dialogue between the two agencies and sponsors from the beginning of the lifecycle of a new product, a deeper understanding of the basis of scientific advice, and the opportunity to optimise product development and avoid unnecessary replication of testing or unnecessary diversity of testing methodologies.
The confidentiality arrangement between the two agencies has been
extended for five more years, following the positive experience gained since the
initial arrangements were signed in September 2003. As well as covering parallel scientific advice, the confidentiality arrangements allow the agencies to exchange other types of information as part of their regulatory processes, including legal and regulatory issues, scientific advice, orphan drug designation, inspection reports, marketing authorisation procedures, and post-marketing surveillance. An exchange programme for staff of both agencies is also envisaged as part of the arrangement, with the aim of installing a culture of increased staff awareness.