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Review
. 2020 Jun;86(6):1034-1051.
doi: 10.1111/bcp.14279. Epub 2020 Apr 24.

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

Jane Moseley  1 , Spiros Vamvakas  1 , Michael Berntgen  1 , Alison Cave  1 , Xavier Kurz  1 , Peter Arlett  1 , Virginia Acha  2   3 , Simon Bennett  3   4 , Catherine Cohet  3   5 , Solange Corriol-Rohou  3   6 , Emma Du Four  3   7 , Christelle Lamoril  3   8 , Anja Langeneckert  3   9 , Maren Koban  3   10 , Muriel Pasté  3   5 , Susan Sandler  3   11 , Karin Van Baelen  3   11 , Agnese Cangini  12   13 , Sonia García  13   14 , Mercè Obach  13   15   16 , Emmanuel Gimenez Garcia  13   15 , Leonor Varela Lema  13   17 , Hanna-Mari Jauhonen  13   18 , Piia Rannanheimo  13   18 , Deborah Morrison  13   19 , Marc Van De Casteele  13   20 , Anna Strömgren  13   21 , Anders Viberg  13   21 , Amr Makady  13   22 , Chantal Guilhaume  13   23
Affiliations
Review

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

Jane Moseley et al. Br J Clin Pharmacol. 2020 Jun.

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.

Keywords: drug development; effectiveness; health economics; medicines regulation; safety.

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Conflict of interest statement

No external funding has been used in the preparation of this manuscript.

Figures

FIGURE 1
FIGURE 1
Status, objectives and design of studies imposed by regulators in conditional marketing authorisations from the report on 10 years of conditional marketing authorisation experience (n = 77). 20 MAA, marketing authorisation application; PK, pharmacokinetics
FIGURE 2
FIGURE 2
Options for seeking European regulatory or HTA body advice on PLEG according to stage of development, which are discussed in this paper. Solid line: Subject to validation/prioritisation criteria. Broken line: Case by case justification. EMA: European medicine agency; EUnetHTA: European network of health technology assessment; PLEG postlicensing or postlaunch evidence generation. MA: Marketing authorisation; NCA: National Competent Authority; HTA: Health technology assessment
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References

    1. Bec G, Stracenski I, Castelnovo T. MAH compliance with post‐authorisation: obligations in the EU: a five‐year review. Regul Rapp. 2018;15(11):29‐33.
    1. Santoro A, Genov G, Spooner A, Raine J, Arlett P. Promoting and protecting public health: how the European Union pharmacovigilance system works. Drug Saf. 2017;40(10):855‐869. - PMC - PubMed
    1. Arlett P, Portier G, de Lisa R, et al. Proactively managing the risk of marketed drugs: experience with the EMA pharmacovigilance risk assessment committee. Nat Rev Drug Discov. 2014;13(5):395‐397. - PubMed
    1. Highlight report of the 4th industry stakeholder platform on research and development support. 2019; https://www.ema.europa.eu/en/documents/report/highlight-report-fourth-in.... Accessed December 16, 2019.
    1. European Medicines Agency . Scientific guidance on post‐authorisation efficacy studies. 2016; https://www.ema.europa.eu/documents/scientific-guideline/scientific-guid.... Accessed March 29, 2020.