WP4: Improved use of patient level information and perspectives
In WP4, researchers will ensure that patients are directly involved in the research process and their perspective is taken into account in the novel methodology for design and analysis of clinical studies to be developed in WP2 and WP3. This way, the final recommendations of ASTERIX will be optimised and validated through consulting patients to accommodate their perspectives. In addition, in WP4 an ethical framework for evaluating innovative trial designs for small populations will be developed.
In particular, researchers in WP4 will aim to provide:
- Guidelines for optimal use of patient registries to inform design for clinical trials for small populations;
- Methodology weighting outcomes based on patient level information;
- Recommendations value of Goal Attainment Scaling as a patient relevant outcome measure;
- Importance and acceptability for patients of particular trial design aspects important in clinical trials for small populations.
Leader WP4: Dr. Hanneke van der Lee, Academic Medical Center Amsterdam