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EBMT Legal and Regulatory Affairs Committee

by Eoin McGrath

Inventing our future health care systems starts by shaping our legal and regulatory environment

Our vision: influence decision making

In recent years, the EBMT has increasingly engaged with stakeholders from outside the clinical and scientific setting including regulators and payers. While historically such interactions were ad hoc, it has become clear the need to put in place a structure to maintain these relationships and interactions. Prof. Kröger’s manifesto for his candidacy as EBMT President referred to “Increasing visibility especially on the public policy forum to influence decision making at EU level … in the interest of our patients”.

The kick off

In 2017, the EBMT Board approved the creation of the Legal and Regulatory Affairs Committee (LRAC). The LRAC’s task is to act as the association’s ‘radar’ in terms of monitoring regulatory initiatives coming out of European bodies such as the Commission or the European Medicines Agency and to devise responses on behalf of the members. The LRAC should also identify areas in which EBMT needs to be proactive and ensuring that the professionals’’ point of view is heard.

Past achievements

The LRAC has the advantage of being able to build upon the work already completed over the years by dedicated volunteers and the connections made and sustained with the different European Union agencies. EBMT was invited to contribute to several EU Joint Actions in the area of tissues and cells (ARTHIQS), VISTART, GAPP, EuroGTPII where expertise on hematopoietic stem cells was required. The EBMT responded in 2016 to the IMI consultation on Advanced Therapies Concept Paper (Press Release). A recent major success was achieved with the EMA qualification opinion on the EBMT registry issued in March 2019[Press Release]. A current project sees the EBMT collaborating with the EUnetHTA Joint Action on use of registry data for assessment of new therapies. EBMT is a member of the Common Representation on Substance of Human Origin (CoRe SoHO) and is registered with the European Union Transparency Registry. EBMT participated in the public consultation on the revision of the cell and tissue directives.

New endeavours

These interactions have opened EBMT up to new terminology and concepts such Health Technology Assessment (HTA) and the regulatory framework governing Post-Authorisation Safety Studies (PASS) for authorised products. Happily, EBMT has proven to be sufficiently resourceful to be able to incorporate these new concepts although there is still room for increasing our capacity. Professionalisation and alliance-building should form part of any future strategy in these areas.

As ever, the bulk of the work will need to continue to be undertaken by volunteer experts from the field as they are the ones who understand the science and whose work ultimately will be affected by regulations and directives. More back-office support for their efforts will be put in place in the medium-term with the eventual incorporation of specialised knowledge around advocacy. The Patient Advocacy Committee will be an essential partner in this journey.

The Impact

Legal and regulatory affairs may seem far away from the day-to-day work of healthcare professionals. However, regulations will dictate how we work and interact with other categories of healthcare providers as well as with the patients and families, and so the LRAC provides a new platform to channel the voice of health care professionals to engage with the decision-making processes and make the voice of health care professionals and patient organizations heard. Future ambitions include engaging on all regulatory aspects covering access to human cells and tissues for therapeutic purposes, hospital exemption procedure, the implementation of ATMPs as on site production for CAR T as well as how to deal with legal aspects of patient reported outcomes within the framework of a registry.

If you are interested in participating in the LRAC, please contact Eoin McGrath [] explaining your motivation and any relevant experience.