Now more than ever, patients are increasingly taking part in shared healthcare decisions with their physicians. Information from clinical research, on which evidence-based medicine draws upon, ideally needs to make sense to patients and not only physicians.
Representativeness of the patients' perspectives in the final results of clinical research is therefore critical for shaping the content and quality of medical knowledge and for shared clinical decision-making. As a result there is increased emphasis and even requirement from organizations around the world to include patient research partners (PRPs) as equal members in the medical research team. PRP inclusion means participation in research question generation, study design, data analysis/interpretation, authorship and results dissemination. Several questions remain: 1) How can we maximize the impact of the perspective patients bring to the table? 2) How can we ensure we capture the patients' perspective accurately and carry it forward into the final product of research? We can maximize impact and reach by including the patients' perspective in research that defines the way we assess disease and disease targeted interventions. Randomized controlled trials (RCTs) and longitudinal observational studies (LOS) are currently our main source of clinical information regarding efficacy of disease targeted interventions. Outcomes assessed in RCTs and LOS, as well as the health measurement tools used to assess these outcomes need to include the patient perspective in order for the information generated from these studies to be valid and usable. A conceptual diagram to illustrate how the patients' perspective is critical in generating health information for patient and physician shared decision making is represented in the Figure.
Case study: the updated Psoriatic Arthritis (PsA) Core Domain Set patient domains . A core domain set is the minimum set of outcomes that need to be assessed to evaluate the effect of disease targeted interventions. The first PsA core domain set was developed in 2006 by physicians and methodologists. The core set update study identified clinical trial outcomes important for patients as a group and for physicians as a group and reconciled both perspectives to recommend an updated PsA core domain set that represents the perspective of both patients and physicians. Five PRPs were members of the working group and contributed during all stages of the project. The perspective of patients was included as follows: a) domain generation through qualitative data collection and analysis from international focus groups; b) first domain prioritization exercise in electronic surveys with patients and physicians; c) a face to face nominal group technique meeting with 12 patients and 12 physicians to prioritize domains; d) second domain prioritization exercise through electronic surveys with patients and physicians; e) discussion and voting at the 2016 Outcome Measures in Rheumatology conference. During the process of achieving consensus we observed the following dynamic for patient prioritized domains (percentages represent proportions of patients ranking each domain important in the first survey and then the second survey): 1) Pain was rated important by 76% of patients in the first survey and 82% in the second survey and remained a core domain; 2) Fatigue 78% then 71% and became core domain; 3) Physical function 72% then 80% and remained a core domain; 4) Participation (daily activities and employment/work) was important to 72–76% of patients in the first survey and to 78% in the second survey and became a middle circle domain (important, not required); 5) Emotional well-being 60% then 57% and was placed in the middle circle; 6) Independence 82% then 63% and placed on the research agenda. In each of these situations the trend in the patient's vote as a group followed the physician's vote with one exception: the domain participation.
Summary of challenges and solutions for ensuring representativeness of the patients' perspective in the final results Patients may change their views and align with physicians during the course of a research project. PRPs can help increase awareness of the patients' perspective throughout all stages of the research process. Special attention should be given to: 1) use of methods that adequately capture the patient perspective in the initial stages of a research project, and 2) allowing a robust patient perspective to take shape from patients as a group, before the consensus process with additional stakeholders begins. In addition, supporting patients with adequate information and materials and involving PRPs in their development will maximize understanding of the task for patients and their full participation.
Disclosure of Interest None declared