The expanding universe of orphan drug regulation: transatlantic collaboration now in place
The European Medicines Agency (EMEA) announced it has just passed a milestone with the recent recommendation of marketing authorisation for its 40th orphan medicinal product. Things are truly moving forward in the field of rare disease medicinal products and collaboration is a part of this forward motion. The US Food and Drug Administration (FDA), the European Commission, and the EMEA have decided to expand many of their cooperative activities - including the area of medicinal products for rare diseases - in order to better protect public health, reduce regulatory burden and costs, and bring "innovative products to patients in a timely manner".
The call for further collaboration also extends to paediatric medicines, working within the framework of the recently-implemented Paediatric Regulation in the EU. It has been estimated that up to 80% of rare diseases affect children. The EMEA and the FDA are committed to developing a foundation to facilitate the regular exchange of information on scientific and ethical issues to minimise the exposure of children to unnecessary trials. The Principles of Interactions document has been created to achieve this goal and to better coordinate paediatric medicinal product development on both sides of the Atlantic. The document delineates several goals, including the development of global scientific paediatric plans compatible to both agencies. Information to be shared includes material referring to trial design issues, draft guidance documents, and safety issues such as adverse drug reaction reporting and database statistics. The two groups will be granted observer status for each others' Paediatric Committee meetings to be better kept abreast of "optimal mechanism and timing of exchanges". Monthly conferences and listings of applications and decisions are also proposed. A secure link, such as Eudralink, will be employed for the exchange of information.
It was in September 2003 that Confidentiality Arrangements between the EU and the FDA were reached in the context of regulatory cooperation and transparency, establishing a framework for "upstream regulatory cooperation including the possible exchange of information on advance drafts of legislation and regulatory guidance documents as well as non-public information" relating to the assurance of "quality, safety and efficacy of medicinal products - including orphan medicinal products authorised or under review both in the USA and the EU." To facilitate this exchange of documents and information, an Implementation Plan describing the processes of information and document exchange was finalised in September 2004. A year later, a five-year extension of the plan was signed into effect. Within this plan, article 2.1.4 calls for a regular exchange of information in the area of orphan medicines, among others, on topics including marketing authorisation applications, extensions of indications, and risk management plans.
A Transatlantic Workshop on Administrative Simplification in Medicines Regulation is being planned for late November in Brussels, Belgium.
The extension of existing collaboration can only mean good news for orphan drug development, often hindered by small clinical trial sizes and limited potential customer bases, resulting in lengthy delays and high costs. Rare disease patients, their families and stakeholders on both sides of the pond have reason to celebrate with the deepening of international collaboration.
