Even in the best of circumstances, clinical research is a complex and challenging endeavour. In recent years, however, the pharmaceutical industry has realised that the unique insight only a patient can provide is crucial to successful trial design.

The logistical realities of this are being addressed in a forthcoming Patient Engagement How-to Module for the Clinical Trials Phases from the PFMD-led working group 2a, one of two focused on this stage. These practical guides will aim to iron out the natural teething problems for an industry still getting to grips with the practicalities of patient involvement in the early stages of medicines development. 

This particular module aims to offer a step-by-step guide to engaging patients and caregivers in the selection, development and interpretation of clinical outcome assessment (COA) instruments. A COA is a measure that describes or reflects how a patient feels, functions, or survives, such as patient-reported outcome (PRO) measures, observer-reported outcome (ObsRO) measures, or clinician-reported outcome (ClinRO) measures.

Ashley Duenas is a Research Associate III with the Patient-Centered Research team at Evidera/PPD, a contract research organisation (CRO) that was ahead of the curve on patient engagement in research at all stages. Her day-to-day work involves working with and communicating with patients on a range of studies and disease indications; she says patient involvement is fundamental to their research.

“Implementing patient engagement might appear overwhelming if it is a new process for organisations and the barriers and facilitators are unclear. There are many ways to involve patients in research at different timepoints – what is important is understanding how to implement this well and also being able to anticipate some of the challenges,” she comments.

Duenas’ experience at Evidera/PPD lies specifically in integrating the patient perspective in research when developing validated COAs for regulatory approval. CROs are in a unique position to share the benefit of experience working across a number of different studies, Duenas points out.

“This can encompass interviewing patients to understand their experiences of a disease prior to developing or revising a COA instrument, in addition to gathering patient input to validate relevant attributes for use within surveys aimed at understanding patient preferences on the benefit/risk of treatments,” she explains. 

Evidera/PPD is also involved in efforts related to patient input on protocol design and supporting innovative methods to make clinical trials more patient centered, such as home health visits (so patients don’t have to visit clinical sites), remote/digital data collection (such as electronic consent), or other support services that bring the trial to the patient.

Duenas was, therefore, a natural choice to collaborate on this module. She says patients are necessary to identify relevant endpoints within a COA and explains there are many ways in which this can happen: as a patient participating in an interview or survey, or as a patient advisor who might be more involved in providing feedback throughout the study.

“If we’re not measuring what matters most to patients and capturing those concepts early on in the study design phase, then we can miss out on capturing some very important aspects that a treatment can have on a patient’s quality of life. In a trial you want to understand how treatment can impact not only the clinical aspects of the disease but also whether there are any changes in their day-to-day life – whether that’s being able to do more physical activities, or there are emotional, social, or other aspects that change as a result of treatment,” she explains.

Working with an array of patients, advocates and industry thought leaders means the module has benefitted from a range of different perspectives, Duenas says.

“We’ve been fortunate to have a few in-person workshops which have been very beneficial to understand each other’s experience and shared purpose for this work. Initially, there were only three of us who strategized and outlined our How-to Module and this has evolved to include additional patient and other stakeholder perspectives. It’s been a very positive experience working with a range of individuals cross-nationally who are committed to moving patient engagement efforts forward so that it becomes a standard process in the drug development continuum.”

Duenas and her colleagues in the working group received feedback on their initial draft of the module at the 2019 Patient Engagement Open Forum. Following this, patient feedback was incorporated into the module, as was the PE Quality Guidance.

“I’ve been involved in each step of the development, from the conceptualization to writing up the module. There is a standard scientific process for developing COAs so it’s very important that our module brings together much of the existing work out there including the latest FDA guidance, ISPOR guidance, ISOQOL guidance, and other relevant resources,” she explains. 

She reiterates that the multi-stakeholder perspective makes the module “unique”; “it’s driven by feedback from patients who want to be involved, as well as a scientific understanding of what’s required for validating COAs.” The module will hopefully be finalised by the autumn.

According to Duenas, it’s about moving the needle forward to ensure the patients’ voice becomes the norm in drug development. “We are working hard to build an evidence base to better understand and document the value of patient engagement in our work.”