Regulatory and health technology assessment advice on postlicensing and postlaunch evidence generation is a foundation for lifecycle data collection for medicines

Abstract

The understanding of the benefit risk profile, and relative effectiveness of a new medicinal product, are initially established in a circumscribed patient population through clinical trials. There may be uncertainties associated with the new medicinal product that cannot be, or do not need to be resolved before launch. Postlicensing or postlaunch evidence generation (PLEG) is a term for evidence generated after the licensure or launch of a medicinal product to address these remaining uncertainties. PLEG is thus part of the continuum of evidence development for a medicinal product, complementing earlier evidence, facilitating further elucidation of a product's benefit/risk profile, value proposition, and/or exploring broader aspects of disease management and provision of healthcare. PLEG plays a role in regulatory decision making, not only in the European Union but also in other jurisdictions including the USA and Japan. PLEG is also relevant for downstream decision‐making by health technology assessment bodies and payers. PLEG comprises studies of different designs, based on data collected in observational or experimental settings. Experience to date in the European Union has indicated a need for improvements in PLEG. Improvements in design and research efficiency of PLEG could be addressed through more systematic pursuance of Scientific Advice on PLEG with single or multiple decision makers. To date, limited information has been available on the rationale, process or timing for seeking PLEG advice from regulators or health technology assessment bodies. This article sets out to address these issues and to encourage further uptake of PLEG advice.

Real-world evidence: perspectives on challenges, value, and alignment of regulatory and national health technology assessment data collection requirements

“…Pharmaceutical companies should be actively engaged in discussions and come forward with proposals early on to determine which type of real-world studies or data sources could be suitable for the required PLEG plan. This is an observation also made in a recently published review on PLEG advice (22), suggesting that common PLEG programs could help improve market and patient access due to the streamlined evidence development.…”

“…Moreover, the willingness to accept RWE for decision making varies between regulatory authorities and HTAbs. The EMA has experience with RWD collection used to monitor the long-term safety of drugs in practice and to support lifecycle benefit–risk evaluations (13;22). In the German HTA context, stakeholders still perceive hurdles with the usage of RWE in terms of quality and privacy of RWD (21).…”

Towards a better use of scientific advice for developers of advanced therapies

“…During initial assessment of a marketing authorisation application, there are often efficacy/safety gaps and having plans for Post‐Licencing Evidence Generation timely might be instrumental in allowing approval in the presence of uncertainty once a positive benefit‐risk balance is demonstrated. Product‐specific proposals for postlaunch evidence generation advice early in the development are welcomed both by regulators and Health Technology Assessment bodies 17 …”

Postlaunch evidence-generation studies for medical devices in Spain: the RedETS approach to integrate real-world evidence into decision making

“…Regardless of the hierarchy of evidence of observational MS, these are intended to supplement the insufficient evidence provided from available clinical trials, either when the evidence base to support decisions is immature or when there is substantial uncertainty surrounding the effectiveness, safety, or cost-effectiveness, by measuring relevant outcomes for patients under real-world conditions (27). In addition, information about uncertainties stemming from surrogate end points, long-term efficacy, effects in specific subgroups, a product's use in clinical practice (e.g., its place in the treatment algorithm; treatment duration and adherence), or changes in standard of care, could be also provided (23).…”