Leah Burns1, Nadege Le Roux2, Robert Kalesnik-Orszulak3, Jennifer Christian4, Mathias Hukkelhoven3, Frank Rockhold5, John O'Donnell6. 1. Worldwide Health Economics and Outcomes Research, Bristol Myers Squibb, Princeton, New Jersey. Electronic address: leah.burns@bms.com. 2. Regulatory Intelligence, Bristol Myers-Squibb, Boudry, Switzerland. 3. Global Regulatory Strategy and Policy, Bristol Myers-Squibb, Princeton, NJ. 4. IQVIA, Durham, North Carolina. 5. Department of Biostatistics and Bioinformatics, Duke Clinical Research Institute, Durham, North Carolina. 6. Worldwide Health Economics and Outcomes Research, Bristol Myers Squibb, Princeton, New Jersey.
Abstract
PURPOSE: Interest in leveraging real-world evidence (RWE) to support regulatory decision making for product effectiveness has been increasing globally as evident by the increasing number of regulatory frameworks and guidance documents. However, acceptance of RWE, especially before marketing for regulatory approval, differs across countries. In addition, guidance on the design and conduct of innovative clinical trials, such as randomized controlled registry studies, pragmatic trials, and other hybrid studies, is lacking. METHODS: We assessed the global regulatory environment with regard to RWE based on regional availability of the following 3 key regulatory elements: (1) RWE regulatory framework, (2) data quality and standards guidance. and (3) study methods guidance. FINDINGS: This article reviews the available frameworks and existing guidance from across the globe and discusses the observed gaps and opportunities for further development and harmonization. IMPLICATIONS: Cross-country collaborations are encouraged to further shape and align RWE policies and help establish frameworks in countries without current policies with the goal of creating efficiencies when considering RWE to support regulatory decision-making globally.
PURPOSE: Interest in leveraging real-world evidence (RWE) to support regulatory decision making for product effectiveness has been increasing globally as evident by the increasing number of regulatory frameworks and guidance documents. However, acceptance of RWE, especially before marketing for regulatory approval, differs across countries. In addition, guidance on the design and conduct of innovative clinical trials, such as randomized controlled registry studies, pragmatic trials, and other hybrid studies, is lacking. METHODS: We assessed the global regulatory environment with regard to RWE based on regional availability of the following 3 key regulatory elements: (1) RWE regulatory framework, (2) data quality and standards guidance. and (3) study methods guidance. FINDINGS: This article reviews the available frameworks and existing guidance from across the globe and discusses the observed gaps and opportunities for further development and harmonization. IMPLICATIONS: Cross-country collaborations are encouraged to further shape and align RWE policies and help establish frameworks in countries without current policies with the goal of creating efficiencies when considering RWE to support regulatory decision-making globally.