Work

PAtient-Centric epilepsy clinical trIal model For Improved health outcomes using Cannabidiol (PACIFIC study)—a methodology for developing patient-centred clinical trials in rare epilepsy syndromes

Year: 2026

Type: article

Abstract: Clinical trials for rare epilepsies face substantial methodological and ethical challenges. Small and heterogeneous populations, coupled with limited validated outcome measures, often render traditional designs underpowered and unable to capture outcomes that are meaningful to patients and their families. Innovative approaches—such as decentralised, adaptive, and participatory designs—offer potential solutions but have rarely been systematically applied in this context. We employed an exploratory three-phase sequential mixed-methods design to co-design a patient-centred clinical trial protocol for rare epilepsies in Australia. An expression-of-interest process recruited 40 participants, equally distributed across four advisory groups: patients, clinicians, researchers, and industry representatives. Phase 1 surveys collected demographic information, trial preferences, digital literacy, and perspectives on participation. Phase 2 comprised semi-structured interviews, analysed using reflexive thematic analysis, to identify themes relevant to trial design. In Phase 3, a consensus-driven process involving four structured online workshops with a multidisciplinary subcommittee translated these findings into a trial protocol recommendation. Four priorities emerged: (1) decentralised trial models to improve access and inclusion, particularly through home-based care; (2) embedding cultural safety and systems integration to support diversity; (3) prioritising outcomes beyond seizure reduction, including quality of life and patient-reported measures; and (4) improving communication and accessibility through digital innovation. These insights informed recommendations for an ethics-approved protocol emphasising inclusivity, feasibility, and real-world relevance. This study demonstrates the feasibility of participatory co-design in developing rare epilepsy trial protocols. Embedding patient perspectives and adopting innovative methodologies can enhance scientific rigour, build trust, and strengthen the clinical and policy impact of rare disease research. What is this study about? Clinical trials are used to test new treatments, but they can be very hard to run for rare epilepsies. The small number of patients, individual differences, and lack of clear outcome measures often make traditional trials challenging. This study aimed to design a new approach to clinical trials that puts patients and families at the centre. What did we do? We carried out the study in three steps. First, 40 people (families, clinicians, researchers, and industry partners) completed surveys about their demographics and experiences with trials, their preferences, and their use of digital tools. Second, we interviewed all 40 people to learn what matters most to them in trial design. Finally, we brought together patients, families, clinicians, researchers, and industry partners in four workshops to identify indications for trial design. What did we find? Four main priorities were identified: making trials easier to join through home-based options; ensuring cultural safety and better coordination across services; measuring outcomes that go beyond seizures, such as quality of life; and improving communication through clear information and digital tools.Why is this important? The study shows that patients and families can play a central role in designing clinical trials. By listening to their needs and experiences, trials can become more inclusive, trustworthy, and relevant to everyday life. This approach could make research in rare epilepsies—and other rare diseases—more effective and meaningful.

Source: BMC Medical Research Methodology

DOI: 10.1186/s12874-025-02757-1