What does patient-centricity really mean in clinical outcomes research?
Patients regularly invest their time and effort into qualitative patient- interview studies to provide COA researchers with a deep understanding of their disease experiences. The qualitative data collected during these studies provide the evidence needed to develop and assess clinical outcomes assessment (COA) instruments to the standard required by regulators and reimbursement agencies.
The benefits for the researcher and sponsor are clear; to inform the suitability and validity of health-related questionnaires to support clinical trial endpoints, often in the form of patient-reported outcomes (PROs). The qualitative findings are also used to establish appropriate clinical trial endpoint hierarchies.
Patients, however, are rarely provided the opportunity to comment on the study methods or see the results despite their essential contributions to the COA development process. Programs such the US FDA Patient Representative Program℠(1) and the UK Patients Involved in NICE (PIN)(2) are designed to ensure the patient voice is heard in the decision-making process.
In an effort to encourage truly patient-centric research, the DRG Abacus COA team continually evaluates current practices to ensure meaningful and rewarding patients engagement throughout the process. With rigorous focus on identifying best practice approaches, our team has seen the following initiatives successfully implemented:
Recruitment of patients to steering committees
Post-interview feedback from patients on the process
Direct dissemination of study results to the patients
While these methods offer a number of benefits, there are some challenges to consider as well. Our COA team is pleased to share an evaluation of these three initiatives and insights gained from our own implementation.
Methods in Practice
Recruitment of Patients to Steering Committees
Patient representatives are invited to contribute as researchers in the development of research projects as part of the research steering committee.
Involvement of a separate group of patients and/or patient advocates in the research steering committee to those patients taking part in the interviews ensures the research remains patient-focused.
Patient representatives can inform the development of qualitative study protocols. This ensures that any patient-facing material is appropriate in terms of language (e.g., informed consent form) and that the draft interview guide content is relevant for the patient interviews.
The involvement of patient representatives in the research design process increases the credibility of study outcomes, particularly from a regulatory perspective and for peer-reviewed publications.
Patient representatives are uniquely placed to identify potential barriers to recruitment and raise awareness of sensitive issues that may arise during patient interviews.
Patient representatives may also review top-level study findings to help researchers recognise relevant knowledge gaps for further research consideration.
Patient representatives may feel intimidated and wary to share their opinions, especially if they feel they may contradict experts in the field.
Expert clinicians may disregard patient-reported symptoms and impacts based on their clinical experience or scientific evidence.
The DRG Abacus COA team has been successful in mitigating these potential barriers through sensitive management of steering committee meetings and communications, and providing patients with the guidance and support they need to overcome challenging situations.
In practice: We invited a patient representative to review and comment on the symptoms and impacts reported by patients during an interview study. Additional insights offered by the patient representative were used to support the study results in the associated peer-reviewed manuscript.
Post-Interview Feedback from Patient Participants
Requesting post-interview feedback may capture important issues that the patients may not have discussed during the interviews (e.g., topics they were uncomfortable sharing directly with the interviewer)
This post-interview ‘quality check’ can inform content adaptations to improve the interview guide/strategy and issues covered in future interviews.
Post-interview feedback can inform methodological adaptations such as:
Delivery method (e.g. phone, face-to-face)
Interviewer preference (e.g. someone with no disease experience, patient advocate or peer-to-peer)
The overall interview experience may be improved by determining best practices and considertions on engaging patients.
Example: some patients feel more at ease sharing their disease experiences with a trained fellow patient whiole other patients may wish to explore their experiences with an independent third party.
Patient burden should be considered:
will patients be prepared to give up more of their time?
is it appropriate to go back to certain patient groups, especially if their disease state has changed between the interview and requesting feedback (e.g. patients diagnosed with progressive diseases)?
How will the findings be incorporated into the study results? Details of how the feedback data will be collated, analysed and reported would need to be discussed and agreed with the study team, clinicians, sponsor, and clearly described in the study protocol for ethical review board submissions.
Patients’ written consent should be obtained pre-interviews, so it cannot be conducted as an afterthought.
In practice: We have ensured that post-interview patient feedback forms are included in protocols for ongoing studies that specify qualitative patient interviews, and we will continue to do so for similar future studies. Moving forward, we intend to implement any practical feedback from patients to improve the patient interview experience by refining our interviewing methods.
Direct Dissemination of Qualitative Interview Study Results to Patients
Disseminating research results to the participants in a transparent and understandable format is considered good practice in academia.
It is mandatory for certain clinical trial summary results to be made publicly available on the gov(3) database in the US and in the European Clinical Trials Database (EudraCT)(4), in language easily understandable by lay readers
The UK’s National Health Service NHS(5) encourages dissemination of results if patients are involved in the research.
Many COA studies are disseminated in the form of published peer-reviewed journal articles and/or conference posters. However, these publications may not be freely available to the general public, and may not be in patient-friendly language.
Study participants may be unaware of a publication’s existence, as it is not standard practice to inform participants after a journal article/conference poster is published.
Inherent delays between completing interviews and publishing results may impact the feasibility of sharing findings with patient populations, particularly those with a progressive disease.
If patients are not in agreement with the overall study findings, they may feel upset/frustrated/angry that their contributions were under-represented, which can be mitigated by thoughtful summary development.
Sometimes, commercially sensitive information prevents findings from being disseminated immediately. A clear plan for dissemination to patients could be discussed and agreed in advance by the study sponsor, clinicians, and research team, and incorporated into the protocol.
In practice: Following a recent interview project, we provided a top-line summary of the study results to a patient representative group to disseminate to participants. Moving forward, we plan to ensure top-line summaries of all study results are provided directly to participants following completion of interviews.
A Best Practice Approach
These methods can improve the experiences of patients and may also increase their engagement in future COA research studies. This level of patient engagement can also support initiatives made across industry to endorse patient-centricity in the different stages of medical product development and in the decision-making process associated with the approval and reimbursement of these products.
Best practice methods for Clinical Outcomes Assessment research should engage patients wherever possible. Such methods should include:
Patient review of study materials, particularly the interview guide
Post-interview patient feedback to inform the design of future interviews
Where possible, the final research findings should be shared with patients in a transparent and timely manner.