The aim of clinical trials is to evaluate the effectiveness and safety for patients of a medical intervention, based on comparable results: the outcome measures. Despite guidance documents on outcomes, many trials, on rare diseases in particular, still do not include standardized outcomes. This situation leads to failures in late-stage of orphan drug development or simply difficulties to prove their clinical effectiveness.
The paper published in the Orphanet Journal of Rare Diseases is a position statement based on the work and discussions done by the IRDiRC Task Force on Patient-Centered Outcome Measures (PCOM). The authors, Thomas Morel and Stefan Cano, emphasize on PCOMs being the best opportunity to obtain a meaningful and interpretable measure of the patient benefit. While PCOMs are still largely omitted across the medical and research community, in particular for rare diseases, fully embracing PCOMS would lead into a win-win scenario for patients, clinicians, researchers, regulators, health technology assessment and payers. The authors described the benefits brought by PCOMs as following:
- Measurement of what matters to patients
- Guidance of treatment decisions
- Improvement of drug development and reimbursement outcomes
- Contribution to understanding natural history
- Demonstration of clinical effectiveness
- Interpretation of surrogate or composite endpoints
The authors describe methods used in PCOM research with their advantages and constraints as well as the key role often played by patients’ organizations in leading patient outcome measurement initiatives. They also discuss how PCOMs can be applied to rare diseases. While it seems unrealistic to develop PCOM specifically to all rare diseases, the careful examination of existing tools and their (re)use is worth considering. Finally, the authors defined 5 principles that should guide the development meaningful PCOMs for rare diseases: Collaboration, Alignment, Integration, Innovation and Communication.