Studying the scientific validity of RWE study designs and analytical approaches, to better inform pharmaceutical R&D and healthcare decision makers on their potential for use in assessment of effectiveness.
Healthcare decision makers need to be confident in the precision and scientific validity of innovative solutions, which use RWE to estimate the effectiveness of medicines.
WP2 has two main goals. First, to explore how an efficacy-effectiveness gap may be anticipated earlier and how this may be addressed during the drug development process. Second, to investigate which scientifically valid and relevant elements of pre-launch trial design can be used to assess the effectiveness of medicines.
The key activities of WP2 are:
- To identify the key drivers of the efficacy-effectiveness gap in relevant drug development case studies, using literature reviews and analysis of patient-level data in:
- Hodgkin’s Lymphoma
- Cardiovascular diseases
- To test several design options and analytical tools through simulation studies.
The main output for WP2 is the development of precise and scientifically valid tools and guidance for design of trials which use RWE, to estimate the effectiveness of medicines.