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

Author:

Sola‐Morales Oriol1ORCID,Curtis Lesley H.2,Heidt Julien3ORCID,Walsh Laura4,Casso Deborah5ORCID,Oliveria Susan6,Saunders‐Hastings Patrick7,Song Yufei8ORCID,Mercado Tiffany3,Zusterzeel Robbert3,Mastey Vera9,Harnett James9,Quek Ruben G. W.9

Affiliation:

1. Fundació Health Innovation Technology Transfer and International University of Catalonia Barcelona Spain

2. Duke Clinical Research Institute Durham North Carolina USA

3. IQVIA, Regulatory Science and Strategy Falls Church Virginia USA

4. IQVIA, Epidemiology and Drug Safety Practice Boston Massachusetts USA

5. IQVIA, Epidemiology and Drug Safety Practice Seattle Washington USA

6. IQVIA Epidemiology and Drug Safety Practice New York New York USA

7. Epi Excellence LLC Garnet Valley Pennsylvania USA

8. IQVIA Epidemiology and Drug Safety Practice Beijing China

9. Regeneron Pharmaceuticals Inc. Health Economics & Outcomes Research Tarrytown New York USA

Abstract

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.

Publisher

Wiley

Subject

Pharmacology (medical),Pharmacology

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