Value for both Decision-Makers and Researchers

Potential Benefits for Groups conducting research for regulatory and HTA activities

• Define and Align Research Questions Early: 
  Allocate adequate time and resources to develop a study protocol with clearly defined research questions that are relevant to regulatory and/or HTA decision-making. 
• Engage Decision-Makers Proactively and Iteratively: 
  Plan for and invest in sustained engagement with regulatory and HTA authorities throughout the study lifecycle. This enhances alignment of methodological expectations, facilitates timely feedback, and improves the overall credibility and relevance of the evidence.
• Design Fit-for-Purpose Studies with Stakeholder Needs in Mind: 
  Tailor study design, data selection, and analytical methods to address specific regulatory or HTA contexts. Consider the decision-makers' evidentiary standards, transparency requirements, and the level of uncertainty they are willing to accept.
• Adopt and Follow Established Methodological Frameworks: 
  Utilise recognised frameworks and guidelines (e.g. from EMA, FDA, NICE, CIHR, ICH E6/E8) to ensure transparency, reproducibility, and methodological rigor. Where appropriate, register relevant details of study protocols in advance.
• Ensure Data Quality and Relevance: 
  Use data sources that are fit for purpose (reliable and relevant). Clearly document data limitations, and how these are addressed in the analysis.
• Promote Transparency and Documentation: 
  Maintain comprehensive documentation of study design decisions, protocol deviations, and analytical choices. Share results and interpretations with external reviewers, including sensitivity analyses and uncertainty assessments, to facilitate robust decision making.

Potential Benefits for Regulators and HTA bodies using research results for decision-making

• Issue Clear and Transparent Guidance on RWE Acceptability: 
  Provide examples of the specific types and sources of RWE that are acceptable or not acceptable for regulatory and/or HTA submissions. Clearly outline methodological standards, data quality expectations, and examples of contexts in which different RWE approaches may be considered valid.
• Enable Early Scientific Engagement on RWE Study Design: 
  Allocate dedicated resources to support early and meaningful engagement with sponsors designing RWE studies. Early dialogue should focus on key aspects such as study objectives, design, data sources, endpoints, and analytical methods to ensure alignment with decision-making requirements.
• Build Internal Capacity to Evaluate RWE Protocols: 
  Ensure sufficient internal expertise and capacity to review and critically assess pre-specified RWE protocols. This includes the ability to evaluate scientific rigor, methodological appropriateness, and relevance of the study design to the intended regulatory or HTA purpose.