Recently, the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) has published its 2023 Work Plan. In this report the Committee highlights the actions and activities on the regulation of Advance Therapy Medicinal Products (ATMPs) that will take place during the year. The activities outlined in the CAT work plan for 2023 have been agreed taking into consideration the Agency’s prioritization set forth in the EMA multi-annual work programme 2022-2024. Some of these actions are:
Guideline on requirements for investigational ATMPs in clinical trials
This guideline provides guidance to ATMP developers to understand the requirements for ATMPs during clinical development. The aim of this guideline is to facilitate the development of ATMPs and the preparation of EU clinical trial application.
A draft guideline was published in 2019, and now the CAT’s goal in 2023 will be to finalize the guideline after an external consultation and publish the final version. If you are interested, you can find this guideline here.
New Active Substance (NAS) status of ATMPs
Compared to chemical or biological substances, specific considerations are needed to establish the NAS status of ATMPs. All things considered, the reflection paper on criteria to be considered for the evaluation of the NAS status of biological substances encompasses ATMPs and provides guidance on the elements required to be submitted by applicants to substantiate a NAS claim and describes the criteria that should be applied during NAS assessment.
Objective of the Committee is to provide guidance on the criteria to be considered for the evaluation of the NAS status of ATMPs, for this they will review, analyze and address where appropriate the comments (related to ATMPs) received during the public consultation of the draft reflection paper for the NAS status of biological substances, and the CAT will contribute to the finalization of the reflection paper.
We at Revio reviewed this reflection paper on the NAS status after its publication, you can check it here
Benefit/Risk methodology and communication
Benefits and risks require continuous evaluation throughout the lifecycle of a medicine. The objective is to balance benefits and risks in a way that is as robust, consistent and as transparent as possible.
Key objective is the continued overview of developments in assessing and communicating the benefits and risks and to pilot the feasibility of using a more explicit approach in describing value-judgments in the current benefit/risk assessment framework/template.
This year, CAT will contribute to the finalization of the reflection paper on single-arm trials that are submitted as pivotal evidence in marketing authorisation dossiers and will initiate a pilot in collaboration with CHMP to quantify impact of expert elicitations in the context of hypothetical Scientific Advisory Groups.
Real World Data (RWD) in regulatory decision making of ATMPs
RWD are used in the development, authorisation, safety and efficacy follow-up and monitoring of ATMPs. Analysis of RWD could further support regulatory decision-making and provide an additional perspective on the use and performance of clinical drugs.
This way, the objective is to further the understanding of the use of RWD including natural history data, retrospective patient level treatment data and registry-based data in regulatory decision making pre-and post-authorisation and in-patient access to ATMPs.
Post-authorisation safety and efficacy follow-up and RMP
Post-authorisation follow-up of patients treated with ATMPs is essential to collect data on long-term safety and efficacy of the authorised ATMP. Thus, it is essential that appropriate guidance is available to ATMP developers in order that appropriate post-authorisation studies can be planned that allow the generation of information.
The committee’s objective will be to review and analyse the comments related to ATMPs on the guideline on safety and efficacy follow-up and risk management of ATMPs and prepare scientific supplication on the follow-up of patients treated with AAV-based gene therapies and CAR-T cells.
We hope you found this information interesting and useful, if you would like to read the complete report you can find it here.
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