Scientific publication in Clinical Pharmacology and Therapeutics

Authors from several affiliations primarily Quinten Health offer through this scoping review a description and comparison of nine structured frameworks developed by regulatory authorities and research institutions to support stakeholders in identifying and/or, evaluating, and/or selecting real-world data (RWD) sources—particularly patient registries—fit for regulatory and HTA evidence generation. With publications from bodies such as the European Medicines Agency (EMA), the Food and Drug Administration (FDA), and both academic and private research institutions, the study highlights the growing importance of structured tools to assess the real-world effectiveness of healthcare products.

The article emphasises how regulatory bodies increasingly require high-quality, relevant, and traceable data to support decision-making processes. Each framework is briefly presented, followed by a comparative review of the evaluation criteria they apply, covering dimensions such as study design, data reliability and relevance, ethical considerations, and practical constraints such as cost and feasibility.

Key findings from the review:

• A description of the nine identified frameworks and their scope of use:
  Structured Preapproval and Postapproval Comparative study design (SPACE), Structured Process to Identify Fit-For-Purpose Data (SPIFD) 1/SPIFD2, PICOTS (Populations, Interventions, Comparators, Outcome(s), Timing and Setting), MATURITY, PICOTS – 3-step conceptual mode for oncology, RWD-Cockpit, Registry Evaluation and Quality Standards Tool (REQueST), Authentic Transparent Relevant Accurate Track-Record (ATRaCTR).
• Recognition that each framework has context-specific strengths and limitations depending on its intended use.
• All frameworks cover the dimensions of RWD assessment and selection, but none allows for RWD discoverability.
• A mapping of shared and unique criteria across nine frameworks, covering study design robustness, data reliability and relevance, ethical standards, and the adopted scoring system.

This study advances the understanding of available tools to assess RWD sources, calls for broader, more systematic use of these frameworks to ensure transparency, consistency, and rigor in evidence generation and strengthens the need for a tool combining discoverability and assessment for ‘fit-for-purpose’ RWD ultimately enabling more informed, efficient decision-making in healthcare product development.

The full paper can be found via the following link: Navigating the Real World: A Scoping Review of Structured Frameworks to Effectively Identify, Evaluate, and Select Real-World Data Sources for Fit-for-Purpose Studies - PubMed