Advanced Therapy Medicinal Products (ATMPs) receive specific consideration during the marketing authorisation process due to their novelty and innovation. Advanced therapies include gene therapeutics, somatic cell therapeutics and tissue engineered products. ATMPs must undergo a centralised marketing authorisation procedure at the European Medicines Agency (EMA). The assessment of an ATMP is carried out by experts from the national medicines authorities of the EU Member States in various European committees. The Committee for Advanced Therapies (CAT), created specifically for the ATMP product group in 2009, is responsible for the scientific evaluation of quality, safety – including environmental compatibility – and efficacy of ATMPs. The Paul-Ehrlich-Institut has been represented by experts on the CAT since its founding. Dr Martina Schüßler-Lenz has served as the elected Chair of the CAT since her appointment by the Paul-Ehrlich-Institut in February 2017. In accordance with the requirements of the European Commission, her mandate will end on 14 February 2023 after two terms of office. As Dr Schüßler-Lenz's mandate comes to an end, the Paul-Ehrlich-Institut would like to take the opportunity to highlight its commitment to the CAT.
ATMPs are now widely available as an option for patients. With their special mode of action, ATMPs offer new treatments for patients who have often exhausted "conventional" therapies. These treatment options are typically aimed at the causes of disease. They also open up possibilities for patients, predominantly those with rare diseases, for which there are currently no treatment options.
From 2017 to 2020, the number of marketing authorisations submitted to the EMA and evaluated by the CAT increased significantly compared to previous years. The first CAR T-cell drugs were followed by the first gene therapy for the treatment of spinal muscular atrophy (SMA) and other gene therapeutics for the treatment of seriously ill children and adults. The focus in recent years has also been on strengthening clinical expertise in the CAT; ensuring consistency in scientific evaluation; intensifying cooperation with other EMA committees, especially the Committee for Medicinal Products for Human Use (CHMP) and the Pharmacovigilance Risk Assessment Committee (PRAC); strengthening exchanges with Health Technology Assessment (HTA) officers in the healthcare sector, the pharmaceutical industry and patient representatives; as well as providing more information on the expertise and scope of the CAT.
How do we ensure the safe use of highly innovative ATMPs in the EU under everyday conditions? How readily can patients access ATMPs in the EU? What are the challenges and obstacles? How can we reduce administrative hurdles when it comes to marketing authorisation? Are our requirements for the marketing authorisation holders in the post-authorisation phase reasonable and data-driven? Under the auspices of the Paul-Ehrlich-Institut, answering these questions in conjunction with the CAT's regulatory tasks was a high priority.
"Martina Schüßler-Lenz, in her position as CAT Chair, has brought her expertise as a representative of the Paul-Ehrlich-Institut to the European level. Through her personal commitment and comprehensive specialist knowledge, she has raised the proper application of scientific benefit-risk assessments of the completely new and particularly complex advanced therapy medicinal products to a higher level,"
stated Professor Klaus Cichutek, President of the Paul-Ehrlich-Institut.
The Paul-Ehrlich-Institut will continue to contribute its expertise to the CAT and advance its work in the committee by preparing guidelines, participating in cross-committee projects, working on the simplification of procedures and requirements for ATMPs, training experts and organising scientific workshops. The aim is to make innovative, safe and effective medicines available to patients in the EU.
Dr Martina Schüßler-Lenz joined the Paul-Ehrlich-Institut in 2005 as a specialist in internal medicine. After clinical and scientific work conducted in Berlin, New York and Barcelona, she completed her specialist training in internal medicine with Professor Christoph Huber at the University Hospital in Mainz. Dr Schüßler-Lenz acquired profound knowledge of the drug development process in her time working in the pharmaceutical industry, during which she supervised and led the clinical development of haemato-oncological test substances from 1992 to 2005.
Dr Schüßler-Lenz has been familiar with the tasks and working methods of the CAT since the entry into force of the European ATMP Regulation and the establishment of the CAT in 2009. Serving initially as a delegate, then as vice-chair beginning in 2014, and then as chair since February 2017, she has decisively shaped the direction of the CAT.
As a clinical evaluator of advanced therapy medicinal products and coordinator of the research program of the Paul-Ehrlich-Institut and the German Consortium for Translational Cancer Research (DKTK), Dr Schüßler-Lenz has always been particularly concerned with the regulatory support of academic developers. Now she will have the chance to refocus on this and other topics in her work at the Paul-Ehrlich-Institut.
"Advanced therapy medicinal products are now widely available to patients. In the age of gene therapy, leading the Committee for Advanced Therapies and helping to shape the marketing authorisation of safe and effective gene and cell therapies was both a challenge and rewarding experience at the same time. The fact that we have been evaluating first-in-class drugs in the CAT since 2009 – from the first tissue engineered product in 2009, to the first gene therapy product in 2012, to the first CAR T-cell products in 2018 and the first gene-edited ATMP in 2023 – this distinguishes the CAT from other committees at the EMA and makes our work so exciting. I would like to thank the management of the Paul-Ehrlich-Institut, the experts on the Paul-Ehrlich-Institut's ATMP team, the colleagues at the CAT and the European Medicines Agency for their outstanding support over the past six years,"
said Dr Martina Schüßler-Lenz.
Updated: 20.01.2023
