The Alliance for Regenerative Medicine Publishes Position on Hospital Exemption of Advanced Therapy Medicinal Products in Europe

BRUSSELS, BELGIUM and WASHINGTON, DC--(Marketwired - Feb 16, 2017) - The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader regenerative medicine and advanced therapies sector, today announced it has published its position on Hospital Exemption (HE) in response to vastly different interpretations and implementations across the European Union of Article 3(7) of Directive 2001/83/EC within article 28 (2) of the Advanced Therapy Medicinal Products (ATMP) Regulation.

ARM believes that HE is a useful pathway to enable patients to receive an ATMP under controlled conditions in cases where no authorised medicinal product is available for an indication with a high unmet medical need. However, varying interpretations and implementations of HE among European Member States may lead to the supply of a medicine under HE when there is an existing licensed alternative that has undergone rigorous quality testing and demonstrated proven safety and efficacy. This undermines the case for investment in the licensing of ATMP, and as such, may act as a deterrent to developers and could ultimately limit the supply of products for European patients.

"Throughout our efforts to advocate for the consistent interpretation and implementation of HE across the EU -- a topic that has been widely debated for several years -- we have been focused on understanding and defining an approach that will generate optimal outcomes for patients," said Jacqueline Barry, Chief Clinical Officer at Cell & Gene Therapy Catapult and chair of ARM EU Regulatory Committee. "This includes fostering a regulatory pathway for the development and widespread availability of innovative, disruptive medicines across the European community."

In its position paper, ARM has outlined a series of proposals aimed at creating consistent implementation of HE across European Member States, including:

• Issue guidelines to more specifically define the scope of HE to situations of high unmet medical need and no treatment alternatives. Such guidelines should state clearly that when patients have access to an ATMP with a Marketing Authorization, Member States should not authorize HE for the same medical indication. The guidelines should also address the possible interference of HE with recruitment of patients in clinical trials for the same indication.