Working Group XVII – Long-term safety of medicinal products

Background

Despite the large amount of preclinical and clinical safety data collected during its development, the safety profile of a new medicinal product is only partially known at the time of its approval. Indeed, the clinical trial process involves studying these medicinal products in a relatively small number of selected individuals for a short period of time. Certain side effects may only emerge once these products have been used by a more heterogenous population (specific vulnerable subgroups such as paediatrics, pregnant women, elderly) that might not be well-represented in pre-approval trials but are crucial for post marketing surveillance, including people with other concurrent diseases, and over a long period of time. That is the reason why post-marketing pharmacovigilance plays a crucial role in individuals receiving the medicinal product as it allows the identification of adverse reactions that could not be observed in clinical trials.
The ICH E1 guideline, initially developed in 1994, provides recommendations on the extent of population exposure needed to adequately assess the clinical safety of drugs intended for long-term treatment of non-life-threatening conditions. However, the medicinal product landscape has tremendously evolved since then and is constantly changing with the emergence of new therapies such as advanced therapy medicinal products (ATMPs), individualized cancer medicines or treatment for rare diseases.
An updated guideline is necessary to accurately reflect current and future therapeutic landscapes and provide guidance and methodology to determine whether the benefit-risk balance of a medicinal product remains favourable across lifecycle, or if the short-term established efficacy is outweighed by long-term adverse effects. A new guideline would also significantly benefit the field by providing greater clarity, flexibility, methodological rigor, and adapted monitoring tools facilitating the timely approval and effective long-term safety monitoring of novel therapies.

CIOMS full concept note on Long-term safety of medicinal products

Working Group objectives

It is anticipated that CIOMS Working Group XVII (WG) will develop a realistic scope based on aspects of medicinal product and potential long term safety concern. While therapeutic indication and class could be a vast scope for any detailed consideration, it is likely that the WG will develop several case study examples, including a range of these parameters. These will make the WG output as widely applicable as can be achieved within the time constraints. The WG will then aim to create a CIOMS Framework for addressing long term safety aspects in relation to medicinal products throughout their lifecycle. The CIOMs Framework will aim to provide guidance on criteria for determining the need for pharmacovigilance and risk management measures to address long term safety concerns for medicinal products.

Progress

• First meeting will be held 2-3 December 2025 (Geneva)

CIOMS Working Groups usually take around 3 years to finalize their consensus document and recommendations. Most groups hold one or two in-person meetings per year, with virtual meetings in between. The groups make use of collaborative efforts and capitalize on existing initiatives in order to provide output that is as comprehensive as possible, does not duplicate other efforts and has added value.