Special Session - Monday 31 August, 09:30-10:30H, Auditorium 6
This special session on Monday morning looks a number of issues surrounding innovative cellular therapies, including the European Medicines Agency (EMA) perspective on regulation, transfer of technology from academia to industry, and the reasons for delays in the production of CAR T-cell therapies.
The first talk is by Jordi Llinares Garcia, Head of Scientific and Regulatory Management at EMA. “Healthcare systems are not yet ready to embrace all the advanced products in the pipeline. To promote public health in the current environment, regulators need to become enablers for science to be translated into the reality of healthcare systems,” he explains. “With that objective regulators should be scanning the horizon, identifying the main gaps and connecting the various stakeholders in order to bridge those gaps.”
The EMA regulatory strategy is a plan for the next 5-10 years that identifies key priorities where new or enhanced engagement is essential to the continued success of the agency. The creation of the regulatory science strategy has been a 1.5 year project in which public engagement in the consultation phase and the different workshops with stakeholders have been key elements of its conception. The strategy was adopted and published in March 2020, and this presentation will outline its main goals and recommendations regarding advanced therapies.
Dr Sol Ruiz of the Spanish Medicines Agency (AEMPS) will then discuss the regulatory framework for innovative cellular therapies. She will explain how advanced therapy medicinal products (ATMP) in the European Union (EU) include those based on gene therapy, somatic cell therapy and tissue engineering.
The European legislation requires a centralised marketing authorisation procedure for ATMP (although exceptions are allowed) coordinated by EMA. A specialised and multidisciplinary committee (Committee for Advanced Therapies, CAT) was established in January 2009 within the EMA. The main responsibility of the CAT is to prepare a draft opinion on each ATMP application submitted to the EMA for which the Committee for Medicinal Products for Human Use (CHMP) will adopt a final opinion.
Dr Ruiz says: “So far, 15 ATMP have received approval for marketing authorisation through the centralised procedure; however, some are not available anymore. This shows that, even after overcoming the big challenges that exist for the development of these medicines compared to other biologics (regarding their production, non-clinical and clinical development), there are other post-marketing aspects that need to be considered. The knowledge acquired in this area over several decades offers hope for the future of these types of medicinal products.”
“How are cellular therapies translated from academia to industrial partners: pitfalls and opportunities" is the subject of the third talk by Professor Jürgen Kuball, University Medical Center Utrecht, Netherlands.
Since 2020, the cellular therapy module of the EBMT registry captures data to support post-authorisation studies for market authorisation holders (MAHs) and EMA.
Professor Kuball will explain how the process toward a positive qualification opinion has attracted interest from many other stakeholders, such as scientists and health technology assessment bodies, and was the spin-off for a stimulating development which defined the need for a registry to comply with regulatory requirements, and also inspired ways to deal with CAR-T cell programmes in terms of centre qualifications and educational standards for professionals.
Professor Kuball concludes: “The positive qualified opinion of the EBMT registry by EMA to monitor long-term efficacy and safety of commercial CAR T-cells created opportunities and challenges, and served as a catalyst to launch a novel CAR T-cell community.”
Joaquín Martínez López, of Hospital 12 de Octubre and Complutense University, Madrid, Spain, will then ask where is the access bottleneck for CAR T-cells: centers, industry or authorities?
Access to CAR T therapies has changed the relationship between hospitals, health authorities and pharmaceutical companies. “These changes were essentially that hospitals provide some materials to pharmaceutical companies; then they manipulate this material and the they return this material as a personalised medicine,” explains Dr Martínez López. “The costs and the quality requirements of this process are very high. These situations have created a lot of bottlenecks at different levels to give these new treatments to patients in an adequate time frame .”
He will discuss his view that the pharmaceutical industry would like to control the entire process, saying: “Industry qualifies centers and includes a lot of specific requirements, some of them excessive. Meanwhile regulators want to assure minimum quality standards and health authorities want to control the overall process to avoid inequality and discrimination in access.”
Dr Martínez López will also address the costs involved in CAR-T therapy, including for hospitals, health authorities and insurers, and the issues around toxicity and limited efficacy of the therapy.