The IRDiRC Patient-Centered Outcome Measures (PCOM) Task Force has published its post-workshop report, which includes recommendations and guidelines for the development of PCOM. The report and its recommendations have resulted from the IRDiRC PCOM workshop that was organized on November 30, 2015 in Paris, France.
The recommendations cover a large spectrum, and start with recommendations to respect core principles, e.g. clearly defining what, why, how and where to measure, and who is qualified to measure, before the actual measurement. Guidance is provided on the information of what to measure, based on qualitative interviews with stakeholder groups, informing both the benefits and the harms which patients experience.
The recommendations continue with guidance on how to transfer available knowledge on PCOM to the rare disease field, including the preference of adaptation of existing tools to the specificities of a rare diseases. Guidance is given about the search for existing PCOMs, and the assessment of available instruments.
Furthermore, recommendations are outlined to cover the development of new tools as a non-competitive activity. It needs to be taken into account that most rare diseases evolve rapidly over time and therefore require a dynamic model of PCOM. Qualitative research could address critical challenges of outcome measurement in rare diseases, among which: complexity, variability, individualization, lack of background knowledge and small sample sizes.
The recommendations end with guidelines on the sharing of PCOM data and the development of training tools to further inform key actors in the rare disease field.
More information about this Task Force can be found here.