In the field of rare diseases, patients or parents usually know more about their (child’s) disease than most medical professionals, and they may have an explicit opinion about the type of research that should be done in this particular field. Hence, their involvement in clinical trial design (and execution) may lead to better, and better accepted, trial designs and derived results.
The combination of patient involvement in trial design and increasing their knowledge on these aspects of trial designs, would in turn allow for a better motivation for patients (not) to participate in these trials. Also, patient recruitment and adherence to protocol will be enhanced and drop-outs reduced, resulting in a more cost-effective way to perform these trials.
Patient perspective and involvement in trial design
Many initiatives have increased awareness to include patients and their perspectives in clinical research. However, to date no systematic approach exists to include patients' perspectives in clinical trial design. This is particular relevant in rare diseases, since due to the small populations there is limited opportunity for replication and extension of research to separately address these perspectives. Additionally, the acceptability for patients of particular trial design aspects will improve the feasibility of conducting clinical trials in rare diseases.
A key area of improvement that will typically fit rare diseases is explicit use of information and data from patient registries. Researchers of the ASTERIX project will explore what type of information should be included in these registries to make them most useful for novel trial designs.
Patient focused outcomes
The use of valid, reproducible (reliable) and responsive (sensitive to change) outcomes is a big challenge in rare diseases and developing new disease-specific outcome measures in rare diseases is usually not feasible.
Patient Think Tank
Early in the project, a Patient Think Tank will be set up in which patient representatives collaborate with the researchers across the project to optimize the methods of information gathering and ensure a process of constant feedback. This Patient Think Tank will be active during the entire project, and will also be consulted with regard to the knowledge translation and dissemination of the methodology developed in WP2 and WP3.
Through the multidisciplinary composition of the research team, including patient, academic, regulatory and industry perspectives, this think tank will function as an innovative and creative platform to develop new methodology.
A patient survey will be conducted as part of WP4. Translation of ASTERIX results in proper layman language will ensure adequate dissemination of scientific results to patients, patient organizations and other non-academic target groups.