The PACIFIC Study: A Patient-Centred Approach to Designing Rare Epilepsy Clinical Trials

About this study

People with rare epilepsy syndromes, including those associated with Tuberous Sclerosis Complex (TSC), are often under-represented in clinical trials. Traditional clinical trials can be difficult to join because they usually require frequent hospital visits, focus mainly on seizure counts, and are often designed with limited input from patients and families.

This Australian study explored a different approach. Instead of designing a clinical trial only through researchers and clinicians, the researchers worked alongside people with lived experience, caregivers, health care professionals, researchers and industry partners to co-design a more patient-centred clinical trial model. The aim was to create a trial that is practical, inclusive and measures outcomes that matter most to people living with rare epilepsy.

How the study was conducted

The researchers used a three-stage co-design process involving 40 participants from 4 equal stakeholder groups: patients and caregivers, clinicians, researchers, and industry representatives. The process involved the completion of surveys, participation in in-depth interviews and a series of workshops where stakeholders worked together to develop recommendations for a future rare epilepsy clinical trial. The final protocol was built through consensus rather than being designed solely by researchers.

Outcomes of the study

The study identified 4 key priorities for improving clinical trials for rare epilepsy.

1. Make clinical trials easier to access

Participants strongly supported more flexible trial designs that reduce the need to travel to major hospitals. Suggestions included:

• home-based nursing visits
• telehealth appointments
• local pathology and pharmacy services
• home delivery of study medication

These changes could make participation easier for families living in rural and regional areas or those caring for children with complex medical needs.

2. Improve inclusion and cultural safety

Participants said trials should be designed so that people from different cultural, language and socioeconomic backgrounds can participate more easily. They recommended:

• information written in plain language
• translated resources where needed
• culturally appropriate communication
• better co-ordination between hospitals, primary care and community services

3. Measure outcomes that matter to families

While reducing seizures remains important, participants felt this should not be the only measure of success.

Families wanted trials to also assess improvements in:

• quality of life
• sleep
• behaviour
• daily functioning
• participation in school and community activities
• caregiver wellbeing
• seizure-free days rather than seizure counts alone

These broader outcomes better reflect the everyday impact of treatment.

4. Improve communication using digital tools

Participants supported using technology to make trial participation simpler and more accessible, including:

• mobile apps
• secure messaging
• electronic symptom diaries
• educational videos
• chat-based support
• easier ways to communicate with the research team

They also emphasised that digital tools should be simple, trustworthy and designed with input from patients and families.

Conclusions of the study

This study showed that patients, families and health care professionals can successfully work together to design clinical trials that better reflect the needs of people living with rare epilepsy.

Rather than focusing only on scientific outcomes, the proposed trial model combines scientific rigour with patient priorities. The researchers concluded that involving people with lived experience from the beginning of trial design can produce studies that are more practical, inclusive and relevant to everyday life.

The study also demonstrated that decentralised and patient-centred clinical trials are feasible and may improve participation while maintaining research quality.

Implications of the study

Although this research did not test a treatment, it provides an important framework for how future clinical trials should be designed.

For the TSC Community, the findings suggest that future epilepsy trials could:

• involve patients and families as equal partners in research design
• reduce the burden of travel through home-based and virtual participation
• include outcomes that reflect everyday quality of life, not just seizure reduction
• improve access for people living in regional or remote communities
• make research more inclusive for culturally and linguistically diverse families
• use digital technologies to improve communication and support throughout the trial

These recommendations may help create future epilepsy clinical trials that are more accessible, meaningful and responsive to the needs of people living with TSC and other rare epilepsy syndromes.

DISCLAIMER

This information is intended to provide some insights into recent TSC-related research.  It is not intended to, and it should not, constitute medical or other advice.  Readers are warned not to take any action without first seeking medical advice.

Truong, L., Stettaford, T., Watt, K. et al. 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. BMC Med Res Methodol 26, 32 (2026). https://doi.org/10.1186/s12874-025-02757-1