A study published in Trials highlights the applicable opportunities to overcome the main barriers seen in the field of rare diseases. The study aims to improve evidence-based clinical practice for rare diseases by notably:
- Collecting clinical data in databases and registries.
- Defining measurable patient-centred outcomes.
- Selecting appropriate study designs adapted to small study populations.
- Establishing a multi-stakeholder engagement from patients.
- Implementing educational training on clinical research for clinicians.
- Increasing transparency.
- Simplifying monitoring.
The study also underlines the need for solid evidence before offering innovative treatments to patients, and the need for a multi-stakeholder approach from the early stage of creating evidence, before any clinical research starts. European Reference Networks can provide clinical effectiveness research (CER) with a clear focus on including stakeholder in future activities. The study highlights the fact that the European Union Framework Programme 7 funded project ‘ECRIN-IA’, now an ERIC, can provide the tools and the support needed to help overcome barriers clinical research in rare diseases.
(source: OrphaNews Dec 19, 2018)