Patient-Centred Research (WP3)

The general framework of the ERICA project’ WP3 is Patient Centered Research for Rare diseases. To facilitate the Europe-wide implementation of standardized Patient-Centred Outcome Measures (PCOMs) and Patient Reported Outcome Measures (PROMs) for rare diseases while ensuring the involvement of the patient community in their development and validation process.

WP3 is led by Ana Rath and María del Mar Mañú Pereira, respectively from Orphanet and ERN-EuroBloodNet, in collaboration with MAPI Research Trust.

Specifically, the WP3’s key goal is to promote and disseminate in Europe the adoption of standardized Patient Reported Outcome Measures (PROMs) for rare diseases while ensuring the involvement of the ERNs Health care providers and RDs patients organizations in this process.

WP3 aims concretely to facilitate via the creation of the repository of existing PRO and ObsRO for rare disease the use of those standardized tools that consider patients and caregivers’ perspectives in reporting health outcome measures and in the health assessment decision-making process for rare diseases.

Accordingly, the WP3 team would like to reach the following achievements:

  1. to create a central repository of (validated) common and domain-specific RD PROMs for ERNs

First version launched! PROMs Repository

  1. to define priority areas for future PROMs development
  2. to support ERNs in the adoption of validated instruments for PROMs

During the ERICA WP3 ‘Patient centred research’, the Expert Working Group Kick-off Meeting was presented during the first part, the action plan to the Expert Working Group and participants. Specifically, it has been described the Questionnaire Database Presentation – PROQOLID™, the Orphanet Disability questionnaire (ODQ), and the strategy for the RD Clustering, Questionnaire Coding and PROs selection.

Whereas during the second part of the meeting, an interactive exercise has allowed to tackle some needs for the use of PROMS repository, the ideal context of use of RDs PROMs, the expected search engine terms to be inserted in the repository and the expected results.

Results of this exercise are under analysis and will be used for customizing the repository of existing PRO and ObsRO for Rae Disease adapting it as much as possible on expressed needs and expectations.

The Kick-off meeting of WP3 Patient Centred Expert Working Group (PCEWG) was held in two consecutive sessions:

Session I was dedicated to present the work done so far for the identification and codification of instruments for the assessment of Patient Reported Outcomes (PRO) in rare diseases (RD). Session I gave the frame for an open discussion in Session II to better understand and define the RDs community needs in terms of building a repository of PROMs for RD.

Videos available.

See also updates presented during the ERICA 2nd GA in Bologna: