Improve evidence and overcome the barriers in clinical practice for rare diseases

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.

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(source: OrphaNews Dec 19, 2018)