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

– February 16, 2017

BRUSSELS, Belgium and WASHINGTON, DC – February 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.
• Create a publicly available registry of all sites using ATMP under HE and their therapeutic indications in all Member States.
• Encourage Member States to make HE subject to the approval of an ethical committee review on a case-by-case basis.
• Develop tutorials to educate the medical community and research institutions on requirements for ATMPs, clinical studies and marketing authorization.
• Offer incentives at the EU and/or Member States level to stimulate academic/industry collaborations.

“ARM has taken an important leadership role in advocating for a consistent interpretation and implementation of HE among European Union Member States,” said Eduardo Bravo, Managing Director and CEO of TiGenix, member of the ARM Executive Committee. “We believe that ARM’s initiative for greater clarity on HE addresses the hurdle to patients’ access to effective treatments that the current divergent implementation of HE across the EU poses. Important for companies in our sector, clearly, is market access, and the position proposes a way forward to ensure HE is not misused to circumvent the applicable legal instruments for the authorisation of safe and effective medicinal products in Europe.”

ARM’s official position on HE is available here.

“We are very pleased that a series of planned activities, published by the EMA on 2nd February 2017 to foster ATMP development and enable expanded patient access in the EU, includes support of more transparency in HE, as proposed in our position paper. We encourage the stakeholders involved in the elaboration of the EU plan seeking to optimize the current regulatory framework to consider the other recommendations proposed in ARM’s position on HE,” concludes Annie Hubert, ARM’s director of European public policy.

About The Alliance for Regenerative Medicine
The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine and advanced therapies worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Today, ARM has more than 250 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit