Achieving effective patient and public involvement in international clinical trials in neurology

Abstract

There is a growing need for patient and public involvement (PPI) to inform the way that research is developed and performed. International randomized controlled trials are particularly likely to benefit from PPI, but guidance is lacking on how or when it should be incorporated. In this article, we describe the PPI process that occurred during the design and initiation of an international treatment clinical trial in MS. PPI was incorporated using a structured approach, aiming to minimize bias and achieve equivalence in study design, implementation, and interpretation. Methods included PPI representation within the study research team, and the use of focus groups, analyzed using thematic framework analysis. We report the outcomes of PPI and make recommendations on its use in other neurology clinical trials. By sharing our model for PPI, we aim to maximize effectiveness of future public involvement and to allow its effect to be better evaluated.

Determining the effectiveness of early intensive versus escalation approaches for the treatment of relapsing-remitting multiple sclerosis: The DELIVER-MS study protocol

“…The current study will be conducted in adherence with engagement principles based on trust, respect, and transparency. [20] Patients with MS will be integral members of the research team and compensated for their time at fair market value.…”

“…The recruitment flow diagram is presented in Figure 1. The study steering committee and study advisory committee comprised of investigators, patients, patient advocacy agencies, and third-party payer representatives developed and finalized the study protocol [20].…”

“…The emphasis of the study is to conduct a pragmatic randomized trial. Preparatory data collected for the DELIVER-MS study, previously published [20], indicated that a sizeable proportion of individuals would not be willing to be randomized. To provide additional data regarding the primary research question, participants who do not agree to randomization, or who agree to randomization but are not approved by insurance for coverage for a medication in the arm to which they were randomized despite appeal, are invited to participate in the observational cohort of the study.…”

“…Brain volume loss over three years was selected as the primary outcome for the study because it is meaningful to patients (as demonstrated in preparatory focus groups), [20] is feasible to be conducted in a 3-year multicenter study, and is the currently available measure most predictive of future MS-related disability. Volume loss shows good correlations with physical disability over both the short term [33] and long-term (Table 3).…”