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U.S. Food and Drug Administration	European Commission	European Agency for the Evaluation of Medicinal Products

Project Outline: A common application format for orphan medicinal product
designation in the European Union and the United States
June, 2007

Background

There are many similar requirements that a sponsor has to fulfil for the European Medicines Agency (EMEA) and the United States (US) Food and Drug Administration (FDA) to accept an application for orphan medicinal product designation. Sponsors usually seek orphan designation in more than one territory before applying for marketing authorisation, and often the European Union (EU) and the US are initially included in the sponsor’s plans.

Although applying for orphan designation is not intended to be a highly resource-intensive procedure and is kept as simple as possible from the regulatory point of view, submitting differing application formats to the EMEA and US can represent a substantial burden for sponsors, particularly for small and medium size companies

Despite the fact that the US and the EU have different legislations regarding orphan designation and therefore different designation criteria, a common application format for information required by both Agencies, that is appended by information uniquely required by either Agency, could simplify the sponsor’s task. This common application format may allow more sponsors to apply to both territories at the same time, which could benefit both sponsors and patients with rare diseases through earlier designation. In addition, a common format may set the favourable environment for the FDA and the EMEA to share common experiences and gain understanding of the similarities and differences of the process of medicinal product designation in the two regulatory systems. Finally, this project would fulfil the objective of simplifying administrative procedures in the framework of the Transatlantic Business Dialogue exercise.

Aims

• To lessen sponsor’s regulatory burden by creating a common application format option for orphan designation requests in the EU and the US, without increasing the review burden on either Agency.
• To simplify, within regulatory limits, the administrative requirements for orphan medicinal designation, thus possibly facilitating parallel submissions and future EMEA and US cooperation.
• To fulfil the objectives of the transatlantic simplification process in the framework of the Transatlantic Business Dialogue exercise.

Objectives

• To design a common format/template option for orphan medicinal product designation requests.
• To harmonise, within current regulatory limitations, the administrative requirements sections of orphan designation requests that are common to the EMEA and FDA.
• To clarify, as appropriate, the sections that are specific to different territories, e.g., significant benefit.

Method

A working group comprised of EU and US members will draft a document that fulfils the requirements for a request for orphan medicinal designation according to FDA and EMEA criteria and the aims above. This document will describe and outline a format option consisting of a Common Section with subsections that are shared by the EMEA and FDA plus a Non-Common Section for the requirements not shared by the Agencies. Each section heading will include a brief description of its expected content (e.g., definition of a condition may include the definition of the condition applied for, a summary of the aetiology, diagnostic criteria, pathophysiology, and prognosis. The section should be written in a “medical textbook” style and the information included should be appended with relevant references). This template is proposed to be done as the “notes” that currently appear in the application for orphan designation in the EU, but is not intended to set the criteria for the section content nor to guide sponsors on the different aspects of the application. For instance, if a subset designation is requested, the application format could include a note saying “critical justification of the subset is requested in this section” without entering into the discussion of what would be acceptable for either Agency.

• This draft project outline to be completed by 15 June 2007.
• A first draft application format document should be prepared by the end of the first week of July 2007.
• The final document will be cleared through the EU and FDAprocesses. With no unforeseen problems, the final application format document should be ready by end first week of October.

The final Application Form can be found here

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