Table 1

Opportunities for patient engagement in different phases of research

Research issue
Research phaseMethodPutative result
Identify knowledge gaps and clinical needsDefine research questionDirect contact with patients;
Consultation of patient support groups
Define most urgent and most relevant research questions
Explore grant opportunitiesResearch fundingCollaborate with a patient organization;
Mobilize ‘influencers’ among the patients
Apply for funding sources likely to fund your (rare disease) research
Review of study design and study procedures by patientsDesign studyCollaborate with a patient organization;
Test if the patient information folder is clear and if patients are willing to undergo the procedures
If patients approve study procedures and information, more patients are likely to participate
Update patients on the research progress and share novel insightsDuring the studyMeetings, newslettersHelp patients to better understand their disease;
They can ask their physicians about novel treatment or diagnostic strategies
Receive feedback on study resultsDuring the studyMeetings, questionnairesPatients can drive the implementation of research findings into the clinic
Show results of research on a group level and brainstorm on future directionsPublication of resultsConfer with private sponsors and patient organizationsPatients can help to prioritize the research agenda