Effectively Leveraging RWD for External Controls: A Systematic Literature Review of Regulatory and HTA Decisions.

Autor: Sola-Morales O; Fundació Health Innovation Technology Transfer and International, University of Catalonia, Barcelona, Spain., Curtis LH; Duke Clinical Research Institute, Durham, North Carolina, USA., Heidt J; IQVIA, Regulatory Science and Strategy, Falls Church, Virginia, USA., Walsh L; IQVIA, Epidemiology and Drug Safety Practice, Boston, Massachusetts, USA., Casso D; IQVIA, Epidemiology and Drug Safety Practice, Seattle, Washington, USA., Oliveria S; IQVIA, Epidemiology and Drug Safety Practice, New York, New York, USA., Saunders-Hastings P; Epi Excellence LLC, Garnet Valley, Pennsylvania, USA., Song Y; IQVIA, Epidemiology and Drug Safety Practice, Beijing, China., Mercado T; IQVIA, Regulatory Science and Strategy, Falls Church, Virginia, USA., Zusterzeel R; IQVIA, Regulatory Science and Strategy, Falls Church, Virginia, USA., Mastey V; Regeneron Pharmaceuticals Inc., Health Economics & Outcomes Research, Tarrytown, New York, USA., Harnett J; Regeneron Pharmaceuticals Inc., Health Economics & Outcomes Research, Tarrytown, New York, USA., Quek RGW; Regeneron Pharmaceuticals Inc., Health Economics & Outcomes Research, Tarrytown, New York, USA.
Jazyk: angličtina
Zdroj: Clinical pharmacology and therapeutics [Clin Pharmacol Ther] 2023 Aug; Vol. 114 (2), pp. 325-355. Date of Electronic Publication: 2023 Jun 09.
DOI: 10.1002/cpt.2914
Abstrakt: Real-world data (RWD)-derived external controls can be used to contextualize efficacy findings for investigational therapies evaluated in uncontrolled trials. As the number of submissions to regulatory and health technology assessment (HTA) bodies using external controls rises, and in light of recent regulatory and HTA guidance on the appropriate use of RWD, there is a need to address the operational and methodological challenges impeding the quality of real-world evidence (RWE) generation and the consistency in evaluation of RWE across agencies. This systematic review summarizes publicly available information on the use of external controls to contextualize outcomes from uncontrolled trials for all indications from January 1, 2015, through August 20, 2021, that were submitted to the European Medicines Agency, the US Food and Drug Administration, and/or select major HTA bodies (National Institute for Health and Care Excellence (NICE), Haute Autorité de Santé (HAS), Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG), and Gemeinsamer Bundesausschuss (G-BA)). By systematically reviewing submissions to regulatory and HTA bodies in the context of recent guidance, this study provides quantitative and qualitative insights into how external control design and analytic choices may be viewed by different agencies in practice. The primary operational and methodological aspects identified for discussion include, but are not limited to, engagement of regulators and HTA bodies, approaches to handling missing data (a component of data quality), and selection of real-world endpoints. Continued collaboration and guidance to address these and other aspects will inform and assist stakeholders attempting to generate evidence using external controls.
(© 2023 Regeneron Pharmaceuticals Inc and The Authors. Clinical Pharmacology & Therapeutics published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics.)
Databáze: MEDLINE
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