The Value of Early Patient & Public Involvement in the Clinical Development Pathways

Case Study for a Cell Therapy in Hearing Loss

Context

Rinri Therapeutics is pioneering cell therapies to restore hearing loss caused by the loss of auditory neurons for which no treatments exist. Unlike other therapeutic areas in which cell and gene therapies are already established, often for life‑threatening conditions, hearing loss is still managed primarily through device‑based interventions. These approaches are palliative: they address symptoms rather than the underlying cause, and are unable to restore normal hearing, leaving patients with a continued and often profound impact on their quality of life.

Recognising this early, the company understood that meaningful patient involvement, particularly in identifying unmet needs and gaps in existing treatments, would be critical to the therapy’s success. As a result, Patient and Public Involvement and Engagement (PPIE) was embedded in the drug development process from the outset to explore patients’ perceived the value of the therapy and whether they were willing to accept its associated risks and burdens.

PPIE Activities

The first phase of Rinri’s PPIE activity aimed to understand the need for the therapy, key concerns and assess its acceptability. Rinri conducted a large survey of 230 people, involving individuals with various types and degrees of hearing loss, their family members, and professionals who cared for them. Participants were asked what factors would encourage or discourage them from joining a clinical trial of a cell therapy for hearing loss. The findings were then explored in greater depth through focus groups involving each stakeholder group.

As development progressed, Rinri established a participant advisory group to provide ongoing input into the development of the study protocol and patient-facing materials. The group comprised predominantly lay members with lived experience of hearing loss, alongside a smaller number of contributors with PPIE experience. Their feedback informed both strategic and operational aspects of the proposed trial by identifying barriers and facilitators, and by exploring patients’ and carers’ attitudes towards issues such as trial design (whether blinded or randomised) and the acceptability of immunosuppressants as part of the treatment.

The participant advisory group also played a major role in shaping materials for participants. Rather than adapting standard templates, the group and Rinri co-developed over 20 new documents. These included participant-facing information such as the participant information sheet, a family and friends information sheet, and a trial decision guide, as well as practical tools for the trial site teams, including the informed consent checklist, deaf awareness resources and a recruitment conversation guide.

Overall, the development journey demonstrated how PPIE was integrated from the outset to support the design of a clinically feasible, participant-centred trial.

Commercial benefits of PPIE

Improved protocol and trial design: PPIE identified participants’ key priorities, including safety, speech comprehension, practical burden, and support needs. These insights informed the trial design, guided the selection of outcomes and improved its feasibility and efficiency, while reducing the risk of trial failure resulting from poor design.

Regulatory benefit: The collaborative work with the advisory group added tangible value by improving the clarity and accessibility of participant-facing materials and aligning them more closely with participants’ information needs. The fact that the materials received few comments from the Medicines and Healthcare products Regulatory Agency (MHRA) and the Research Ethics Committee (REC) suggested that the PPIE input strengthened confidence in regulatory reviews, thereby helping to minimise delays to trial timelines caused by major revisions.

Time and cost savings: Early PPIE reduced the likelihood of rework, protocol amendments, delays in regulatory approval, and may also have mitigated future recruitment and retention challenges by designing a trial in which patients were willing to participate. Integrating PPIE into clinical development was more cost-effective than conducting it separately, as Rinri’s early involvement activities additionally served as a market research activity for their product.

Learn more about the commercial benefits of PPIE in PPIE Beyond Ethics: A Business Case for ATMP Developers

Key learning

Implementing PPIE early and treating it as part of therapy development: PPIE needed to begin at the earliest stages of therapy development and be embedded into day-to-day decision-making alongside the scientific, strategic and operational planning, rather than being treated as a ‘tick-box’ compliance exercise added later or limited to participant‑facing documentation

Building trust through meaningful involvement: Trust with patients was strengthened through active listening, openness to critique, and transparent communication about why certain suggestions could not be implemented. This helped sustain meaningful involvement among the focus group and advisory group members. PPIE also built trust with trial site teams, who particularly valued the practical tools that were developed to support them. This greater trust may in turn improve future uptake and engagement, supporting Rincell-1’s continued development plan.

Keeping information practical and relevant: Information provided to patients needed to be tailored to reflect their priorities, recognising that these may differ from those of the clinical and scientific team. Working with people with lived experience was therefore essential to gaining this insight. In Rinri’s case, many were primarily concerned with whether the cell therapy was safe and unlikely to cause harm, rather than with detailed scientific explanations of how it worked.

Organisational culture matters: Rinri’s academic and clinical roots, small team structure, and leadership support made it easier to integrate PPIE directly into clinical development.

Infographic Summary

This infographic can be downloaded from the attachments section below.

Acknowledgements

Author

Dr Hsin-Yu Kuo, EuroGCT Project Manager – Research Information and Networks.

Reviewed by

Emily Merrell, Director, ATTC network Coordination

Hannah Ekbatani, Programme Manager, ATTC network

Dr Efstratia Papoutselou, Clinical Development Scientist and Patient and Public Involvement Lead at Rinri Therapeutics

This case study was developed on the basis of an interview with Dr Efstratia Papoutselou. We thank Dr Papoutselou for her invaluable insights and contributions to this case study.