Patient-Centered Outcome Measures


As clinical trial outcome measures and reports are vital for decision and policy makers to introduce appropriate recommendations, regulatory agencies, standards organizations and international societies have issued a number of guidance documents on outcomes. However, many trials, particularly on rare diseases, do not yet include standardized outcomes in clinical data and as such insufficient attention to the selection of outcomes has often led to a waste of generated data, research efforts, and inefficiencies in drug development and in regulatory review processes.

As a relatively new concept, Patient-Centered Outcome Measures (PCOM) aims to place patients, their families and carers at the heart of decisions concerning the most valuable criteria in health assessment, rather than leaving assessments solely to clinicians. PCOM should provide evidence on the impact of the disease and treatment on patients. Their identification and validation require rigorous planning, methodology, and partnership between investigators and patient organizations.


PCOM can provide evidence on the impact of the disease and treatment on patients. The objective of the PCOM Task Force is to identify what should and can be done to support the development of patient-relevant outcome measures for rare diseases in order to improve the feasibility and quality of forthcoming trials and to provide data of relevance to the patient community and other decision makers. In order to reach these objectives, the Scientific Committees of the IRDiRC set up a Task Force on PCOM initiatives to discuss actions to improve clinical research in the field of rare diseases.

Steering Committee (6)

The Patient-Centered Outcome Measures Task Force Steering Committee members are:

  • Steven Hass – Genzyme, USA
  • Thomas Kelley – UK Foundation Program Office, member of ICHOM, UK
  • Marshall Summar – Children’s National Medical Center, member of PCORI, USA
  • Sharon Terry – Genetic Alliance, USA
  • Margaret Vernon – ISPOR, UK
  • Paula Williamson – Liverpool University/ member of COMET, UK


Together with the IRDiRC Scientific Secretariat, the Task Force has published the post-workshop report and its recommendations. This report and its annexes are available for consultation.


Measuring what Matters to Rare Disease Patients – Reflections on the work by the IRDiRC taskforce on Patient-Centered Outcome Measures
Thomas Morel and Stefan J Cano
Orphanet Journal of Rare Diseases, November 2017, doi: 10.1186/s13023-017-0718-x, access the abstract here