Guidance on Validity of Clinical Studies

This guide outlines essential guidelines for assessing the validity of clinical studies within the framework of Health Technology Assessment (HTA). Here are the main points covered:

1. Importance of Validity: The guide emphasizes the need to evaluate both internal and external validity of clinical studies. Internal validity ensures that the study accurately measures what it intends to, while external validity assesses the applicability of the findings to broader populations.
2. Statistical Precision: The guide highlights the importance of statistical precision in interpreting study results, focusing on how well the study can estimate the effect of an intervention or treatment.
3. Study Designs: It provides an overview of various clinical study designs, including:

• Interventional Studies: Such as randomized clinical trials (RCTs), which are considered the gold standard for establishing causal relationships.
• Observational Studies: Including cohort studies, case-control studies, and cross-sectional studies, which offer insights into real-world settings but may have limitations in establishing causality.

1. Strengths and Weaknesses: Each study design is evaluated for its strengths and weaknesses, with recommendations provided to enhance the quality of research. For example, while RCTs can minimize bias, they may not always reflect real-world scenarios.
2. Real-World Data (RWD) and Real-World Evidence (RWE): The guide discusses the increasing relevance of RWD and RWE in clinical studies, highlighting how they can complement traditional clinical trial data by providing insights into treatment effectiveness in everyday practice.
3. Role of Registries: The use of registries in clinical research is addressed, emphasizing their role in collecting long-term data on patient outcomes, safety, and effectiveness, thereby contributing to the body of evidence supporting HTA.

Overall, this guide serves as a comprehensive resource for stakeholders involved in HTA, facilitating informed decision-making by ensuring that clinical study results are robust, reliable, and applicable to real-world contexts.

Source: https://health.ec.europa.eu/document/download/9f9dbfe4-078b-4959-9a07-df9167258772_en?filename=hta_clinical-studies-validity_guidance_en.pdf

Region: EU Harmonized (27 Markets)