Early engagement of the “patient voice” to inform patient reported outcome measurement in global pivotal trial of Cholangiocarcinoma

Objectives

Regulatory authorities (e.g. FDA, EMA) endorse initiation of partnerships with patients in the evaluation of medicines. Early patient involvement in clinical trials is critical for obtaining feedback on meaningful clinical endpoints and for assessment of study feasibility, which is particularly pertinent for rare diseases. Here, we describe an exercise of patient / carer engagement and opinion-gathering to inform the patient reported outcome (PRO) measurement strategy for a global pivotal treatment trial of a rare disease, cholangiocarcinoma (CCA).

Methods

Patients with CCA and their carers attending an annual patient conference were briefed about a pivotal trial, in its design phase and invited to give opinions, according to an eight-question survey followed by audio-recorded, one-to-one interviews, regarding: 1.) selected PRO instruments (EORTC QLQ-C30 and QLQ-BIL21); 2.) proposed timing of PRO measurement during treatment and follow-up; 3.) whether a qualitative (i.e. exit interview) sub-study as part of the global trial was considered feasible, appropriate and worthwhile.

Results

Four patients and one carer provided feedback. Participants identified a meaningful endpoint, described as “right-shoulder pain”, as missing from the selected PRO instruments. Overall, the PRO items were described as “easy to understand” and relevant to their experience of CCA. Participants were satisfied with the proposed timing and frequency of PRO measurement during and after treatment and reported hypothetical willingness to participate in an exit interview sub-study.

Conclusions

Engaging patients and carers early in the trial design has been valuable for the sponsor, enabling an additional patient-relevant endpoint to be incorporated into the trial protocol, and has confirmed the relevance of selected PRO items and endorsed feasibility of PRO completion at multiple time points and at follow-up. This exercise has allowed for patients with CCA and their carers to contribute to the development of a patient-centric protocol for this rare disease treatment trial.