Practical guidance for engaging patients in health research, treatment guidelines and regulatory processes: results of an expert group meeting organized by the World Health Organization (WHO) and the European Society for Clinical and Economic Aspects of Osteoporosis, Osteoarthritis and Musculoskeletal Diseases (ESCEO)

Abstract

There is increasing emphasis on patient-centred research to support the development, approval and reimbursement of health interventions that best meet patients’ needs. However, there is currently little guidance on how meaningful patient engagement may be achieved. An expert working group, representing a wide range of stakeholders and disciplines, was convened by the European Society for Clinical and Economic Aspects of Osteoporosis, Osteoarthritis and Musculoskeletal Diseases (ESCEO) and the World Health Organization (WHO). Through a structured, collaborative process the group generated practical guidance to facilitate optimal patient engagement in clinical development and regulatory decisions. Patient engagement is a relational process. The principles outlined in this report were based on lessons learned through applied experience and on an extensive dialogue among the expert participants. This practice guidance forms a starting point from which tailoring of the approach to suit different chronic diseases may be undertaken.

Involving patients and nurses in choosing between two validated questionnaires to identify chemotherapy‐induced peripheral neuropathy before implementing in clinical practice—A qualitative study

“…However, it may be discussed on which level the patients were involved. De Wit et al 2019 where patients contribute with their individual experience and the information flows from patient to the researcher often by means of a kind of interviewing (de Wit et al, 2019). Thus, the consulting role was evident in our study.…”

“…Even though the patients were experts in their disease and experiences of CIPN, they were not involved as collaborators, which would have meant participation in the planning and executing of the study (de Wit et al, 2019). Anticipated to take an essential role in the subsequent implementation process, the nurses were involved as well as consultants and advisors.…”

Patient engagement in clinical guidelines development: input from > 1000 members of the Canadian Osteoporosis Patient Network

“…The patient perspective is now recognized as fundamental in health research, guideline development, and treatment regulatory processes and recommended by many organizations [26]. Growing recognition that people with lived experience can make important contributions to the study of health and healthcare has resulted in increasing participation of patients in different levels of involvement from consultation such as surveys and interviews to active engagement like shaping research questions.…”

Update on the ESCEO recommendation for the conduct of clinical trials for drugs aiming at the treatment of sarcopenia in older adults

“…With this proposal, we ensure to capture first an objective endpoint, by recommending a physical performance measure, for which a surrogate value for hard clinical endpoints such as mortality, hospitalisation, fractures and falls is already well established [33]. Second, the use of a PROM will allow to capture more subjective but equally important aspects of patient-relevant efficacy since the patient perspective is now recognized as key parameters in the evaluation of health interventions [34,35]. There is an increasing emphasis on patient-centred research and PROMs are increasingly recognized by government regulatory agencies (e.g., FDA, EMA, etc.…”