Conference on Rare Disease Research Organized by FEDER

The Importance of Patient Participation in Rare Disease Research

This event, organized by FEDER (Spanish Federation of Rare Diseases), focused on the crucial role of patient participation in research. The roundtable discussion, “Patient Participation in the Research Process,” featured Dr. Eva Bermejo, Director of the Institute for Research on Rare Diseases at the Carlos III Health Institute; Nazaret Pareja, psychologist at the Neuromuscular Diseases Association of Andalusia; and Noelia Canela, President of Fundación Noelia.

From the outset, the debate highlighted the fundamental role of patients in generating research questions. Eva Bermejo emphasized that the initiatives of the Carlos III Health Institute are guided by patient needs and their involvement in committees such as the Research Ethics Committee.

Nazaret Pareja explained how associations aim to improve the quality of life for people with neuromuscular diseases, stressing that research is the primary reason patients turn to these organizations. However, they face challenges such as lack of funding and the geographical dispersion of affected individuals.

For her part, Noelia Canela shared the experience of Fundación Noelia, which was established nine years ago following her son’s diagnosis. She emphasized the importance of optimizing resources and fostering collaboration among researchers. The foundation has successfully supported research with €800,000, promoting the development of three different therapeutic approaches.

A key aspect addressed in the discussion was the need for patient registries. Noelia Canela highlighted that without registries, affected individuals do not exist for research or the pharmaceutical industry. Additionally, the creation of an international consortium was emphasized, bringing together various research groups to advance effective therapy development.

The debate concluded with a unanimous message: research is hope, and only through collaboration between patients, researchers, and administrations can a better future be achieved for individuals with rare diseases.