Patient Involvement In Design Research Articles

Patient Involvement in the Design of an Innovative Clinical Study to Compare the Palatability of Anti-Hyperkalemia Medications.

The inclusion of patient representatives as study consultants brings diverse perspectives, insights, and experiences to clinical trial design and execution, and their role in the clinical trial development process is being increasingly recognized and valued. The APPETIZE study evaluated the palatability of, and preference for, three potassium binders for treating hyperkalemia in patients with chronic kidney disease. A core aspect of the development of this study was the inclusion of a patient representative during the design stage. Here, I describe the process of patient involvement in the APPETIZE study design (ClinicalTrials.gov Identifier: NCT04566653), the resultant positive impacts, and key learnings. A patient with chronic kidney disease was invited to be a member of the APPETIZE trial design team. This patient representative attended study team meetings and provided invaluable input into protocol development, questionnaire selection, design of patient information sheets and consent forms, and primary manuscript structure. These critical insights resulted in an enhanced trial design and generation of high-quality, patient-relevant data. APPETIZE provides an excellent example of a patient preference study that relied on input from multiple stakeholder groups, including, most notably, the patients themselves. This approach may serve as a model for early and deep patient engagement in the design and interpretation of clinical trials.

Challenges and key lessons from the design and implementation of an international haemophilia registry supported by a pharmaceutical company.

IntroductionReal‐world data are lacking regarding the relationship between prospectively collected patient‐reported outcomes (PROs), clinical outcomes and treatment in people with haemophilia (PWH). The Expanding Communications on Hemophilia A Outcomes (ECHO) registry was designed to address this data gap, but a range of difficulties led to early study closure.AimTo describe the challenges faced and lessons learned from implementing a multinational haemophilia registry.MethodsThe Expanding Communications on Hemophilia A Outcomes was planned as a five‐year observational cohort study to collect data from 2000 patients in nine countries. Based on direct observations, feedback from patients enrolled in ECHO, challenges of the study design and input from study‐sponsor representatives, the ECHO Steering Committee systematically identified the challenges faced and developed recommendations for overcoming or avoiding them in future studies.ResultsThe study closed after two years because few countries were activated and patient recruitment was low. This was related to multiple challenges including delayed implementation, stringent pharmacovigilance requirements, objections of investigators and patients to the burden of multiple PROs, data collection issues, lack of resources at study sites, little engagement of patients and competing clinical trials, which further limited recruitment. At study closure, 269 patients had been enrolled in four of nine participating countries.ConclusionsResearchers planning studies similar to ECHO may want to consider the barriers identified in this global registry of PWH and suggestions to mitigate these limitations, such as greater patient involvement in design and analysis, clearer assessment and understanding of local infrastructure and potential changes to the administration of the study.

Running the Numbers

### Background Patient research partners (PRPs) are increasingly included in research as well as guideline development task forces in rheumatology. In observational and registry studies the role and involvement of PRPs is not clear. ### Objectives We aimed to evaluate current PRP involvement in registry research as well as benefits and barriers of implementation in practice. ### Methods Based on a round table discussion during the EULAR Registries and observational drug studies meeting (RODS) in 2019 we used mixed methods methodology (survey and focus groups) to study the role of PRPs and involvement in research. The survey was sent to investigators of registries across Europe, participants of RODS meetings and members of the EULAR PARE (People with Arthritis / Rheumatism across Europe) network. Interested survey participants were further invited to one of three focus groups. The conduction of the focus groups (FG) was guided by a semi-structured guide based on the survey results. An inductive thematic analysis approach was applied to qualitative data. ### Results We received 45 survey responses with around half of them from patients. The mean importance of PRP involvement was scored VAS±SD: 75±24mm (on a 100mm VAS), but the actual involvement was rather low (VAS: 32±20mm) and 12 participants had no experience with PRPs. The most common current practice was patient involvement in design of studies (32%), cooperation with patient organisation for recruitment (32%) and involvement of PRPs in the management board (32%). The most important need of PRP integration was attributed in the stages of design and preparation before recruitment. 11 survey participants even indicated that PRPs should be involved in all stages/tasks of research. During the focus groups 5 main themes (Table 1) could be identified. Complementary perspectives lead to greater synergy between researchers and patients and further the aim of deriving more valid outcomes. For successful involvement of PRPs, ideally PRPs should be heterogeneous in regard to their lived experience, education ought to be provided and researchers are responsible to offer an environment where PRPs feel comfortable. All FGs elaborated on different roles that PRPs can take on, although the researcher FGs outlined more different tasks of involvement than the PRP group. One theme summarised different barriers for PRP involvement, which have not been a topic of discussion in the FG composed of PRPs only. ### Conclusion There is room and much appreciation of PRP involvement in registry research. Researchers and PRPs could use the strategies proposed in this study to define PRP roles and plans of ongoing and future studies. ### Disclosure of Interests Paul Studenic: None declared, Mandeep Sekhon: None declared, Loreto Carmona: None declared, Maarten de Wit: None declared, Elena Nikiphorou Speakers bureau: Celltrion, Pfizer, Sanofi, Gilead, Galpagos, Abbvie, Eli Lilly, Grant/research support from: Pfizer, Eli Lilly