Brussels, 16 February 2017 - The European Biopharmaceutical Enterprises (EBE) and the European Federation of Pharmaceutical Industries and Associations (EFPIA) today express their support for key aspects of the “Position on Hospital Exemption” for Advanced Therapy Medicinal Products (ATMPs), developed by the Alliance for Regenerative Medicines (ARM). ARM, EBE and EFPIA call for greater transparency on all ATMPs available to patients, whether through EU authorisation or national exemptions issued in accordance with Article 28 (2) of the Advanced Therapy Medicinal Product Regulation[1].

“We welcome the initiative of the multi-stakeholder alliance ARM,” said Barbara Freischem, Executive Director of EBE. “Currently, there is no one place that patients can turn to in order to find out whether a product might be available to treat their condition, often a condition with few patients across Europe.”

As we approach the tenth anniversary of the publication of the ATMP regulation, ARM, EBE and EFPIA find that the Member States' interpretation of the provisions of Article 28 (2) – the so-called ‘hospital exemption’ rule - remains varied, without overall transparency as to how each Member State approaches the topic or implements national exemptions. This is particularly relevant in cases where an exemption is granted in a Member State to a product similar to an ATMP that has received EU authorisation, or when a well-structured clinical trial can be conducted instead.  

Intended to ensure access to custom-made therapies for individual patients who have no other treatment option, EBE and EFPIA note the risk of the lack of transparency and collective oversight of hospital exemptions. This leads potentially to a parallel system in which products approved for use in individual patients under the hospital exemption rule in reality may avoid appropriate clinical trials and the scientific rigour of the process leading to a marketing authorisation as mandated by Regulation (EC) No 1394/2007.

Ultimately, this works to the detriment of patients across Europe:

• There is often no transparency with regard to adverse events and efficacy data, which are essential to accumulate knowledge in this new field.
• There is a concern that hospital-exempted products could hinder the inclusion of candidate ATMP products in clinical trials and later filings for European authorisation.
• There is unequal access to treatment for patients in all Member States due to the lack of availability of Europe-wide-authorised treatment options, with exempted products only being available in the Member State granting the exemption.
• There is a lack of harmonisation, which could lead to differences and unequal access for patients between and within Member States.

In the process of developing an industry-specific position, EBE and EFPIA applaud ARM for having taken the initiative on hospital exemptions for ATMPs. EBE and EFPIA support ARM’s call for action and encourages all stakeholders to come together to find appropriate solutions that ensure patient access to much needed therapies in cases where the development of authorised products is not feasible. They should simultaneously ensure that all other ATMPs are assessed and regulated within the system that European legislators envisaged as the appropriate pathway: the central marketing authorisation system.

ARM’s Position is timely in following the publication of an action plan by the European Medicines Agency (EMA) on 6 February 2017, detailing issues identified at a multi-stakeholder workshop hosted by the EMA on 27 May 2016, one of which was the need for greater transparency on hospital exemptions. The ARM Position on Hospital Exemptions is available here.

Notes for editors

European Biopharmaceutical Enterprises (EBE) represents the voice of biopharmaceutical companies of all sizes in Europe and is a specialised group within the European Federation of Pharmaceutical Industries and Associations (EFPIA). Established in 2000, EBE is recognised as the leading biopharmaceutical association in Europe.

Read more about us at www.ebe-biopharma.eu

The European Federation of Pharmaceutical Industries and Associations (EFPIA) represents the pharmaceutical industry operating in Europe. EFPIA is the voice on the EU scene of 1,900 companies committed to researching, developing and bringing to patients new medicines that will improve health and the quality of life around the world.