Both regulatory agencies require the submission of an annual report for orphan designated products. These reports provide information on the status of the development of orphan medical products, including a review and status of ongoing clinical studies, a description of the investigation plan for the coming year, any anticipated or current problems in the process, difficulties in testing, and any potential changes that may impact the product’s designation as an orphan product.

Both regulatory authorities will exchange the annual reports electronically through a secured portal. Both agencies have agreed to accept the reports on World Rare Disease Day, or, if the sponsor chooses, on their normal annual reporting date.

Presently, U.S. regulations require that orphan product sponsors submit an annual report within 14 months after the date of orphan designation and annually thereafter until market approval is obtained. In the European Union, the Regulation on orphan medicinal products requests annual updates on the status of product development after designation. Currently, even if an orphan product was granted designation on the exact same day in both the USA and European Union, companies submit different reports to their respective regulatory agency. This optional process for submission will not introduce any new regulatory requirements.

Each regulatory body will conduct their own review and assessment of the annual report to assure the information meets all the legal and scientific requirements of each agency. The use of one annual report will benefit sponsors by eliminating the duplication of efforts, and by simplifying the process with a single document submission that meets the requirements of both the FDA and EMA. The use of a single annual report will also help regulators better identify and share information throughout the development process of an orphan product.

Outcome:

This one annual report submission to both regulatory agencies is voluntary, and will only be applicable to sponsors who have obtained an orphan designation status for their product both in the EU and US. Since February 28,  2010 sponsors may send the single Orphan Drug Designation Annual Report to each Agency.

Source:

EMA/ Announcement