In this blog, Chris Moore, Senior Clinical Project Manager at Boyds, explores the role of patient and public advocacy and engagement in the clinical trial planning process.
In the ever-evolving drug development landscape, patient advocacy and engagement have become key components of the clinical trial planning process, with the ultimate goal of improving outcomes for patients. Regulatory bodies and ethics committees in the UK – and Institutional Review Boards (IRBs) in the US – are placing increasing emphasis on the importance of involving patients in clinical trial set-up and design, recognising them not just as participants but as partners. In order to produce more patient centric trials and trial documentation, sponsor collaboration with Patient and Public Involvement Groups (PPIE) is happening earlier in the trial design process than ever before, with the aim of enhancing patient recruitment, retention and data quality, as well as helping to ensure an ethically planned trial.
Patient-centred trial design
Patients are increasingly being seen as key stakeholders in clinical trial planning, with their perspectives offering valuable insights that can improve trial design and outcomes. The goal for drug developers is clear – to learn as much as possible about the disease and – specifically from the patient groups – what it is like to live with the disease to help researchers tailor clinical trials to the needs of the participants.
The importance of patient engagement
Engaging patients in the clinical trial planning process assists sponsors in creating therapies that are going to have a meaningful impact for the patients and design trials that are well-received by the potential participants. Beyond the ethical imperative, patient engagement can significantly improve trial outcomes, including:
- Better recruitment and retention: When patients are involved in shaping the design of a study, they can ensure that their needs are considered, and changes are made where needed to make the participation easier for future participants. For example, if a symptom of an indication was hand tremors, it may be harder for these participants to collect samples into small tubes at home and these samples may have to be collected at site.
- Improved data quality: By incorporating patient feedback, sponsors can adjust study protocols to address real-world challenges. For example, studies capture more accurate ePRO (electronic Patient Reported Outcomes) data when the participants know the reason why the data is being collected and if the data being requested is relevant to their day-to-day management of the condition.
- Enhanced ethics and approvals: Ethics committees, IRBs, and regulators know the value that patients add to the design and planning of clinical trials and for that reason, are requiring evidence of patient engagement from sponsors. Upon review of trial applications, the agencies may consider if participants would understand specific terminology in documents or agree to certain assessments in the trial schedule. If patient engagement has been adequately completed and documented in the application, it can go a long way to alleviating any concerns the agencies may have on behalf of the patient population.
Moreover, patient engagement with a diverse patient group can help identify and reduce specific barriers to participation for particular groups, ensuring a more inclusive recruitment process and increasing diversity within subsequent clinical trials.
Practical approaches to patient engagement
There are two primary approaches to engaging patients. Sponsors may take a study-specific approach involving a more targeted, short-term engagement where patient groups review specific trial documents, such as protocols or informed consent forms (ICFs). This approach is typically used when time is of the essence, as would be the case when patient engagement is included to meet a specific study timeline.
Sponsors may wish to take a programme-level approach, involving more long-term engagement, where patient advocates are involved in multiple studies or phases of development. This allows for deeper relationships between the sponsor and patient group, allowing the patient group to gain a broader understanding of the sponsor’s goals. This can lead to more impactful contributions over time, as the patient group can add value to nuanced discussions in-between the larger study projects.
Regulatory guidance
In many regions, including the UK, regulators are beginning to ask sponsors to demonstrate their efforts in engaging patients. For instance, as part of the combined review submission in the UK, sponsors must outline their patient engagement strategies and explain how engagement to-date has specifically influenced the trial. If patient engagement has not been incorporated, sponsors are required to justify the rationale for not performing patient engagement.
Although patient engagement is not yet mandatory, it is highly encouraged and becoming increasingly important. If the ethics committee or IRB reviewing a submission believe that the study document’s language was too technical or the design did not consider the restrictions of the patient population, the committees can request that patient engagement is performed. The landscape is shifting towards making patient engagement an essential part of the approval process. Proactively engaging patients early in the trial planning process, rather than scrambling to include patients later in the process, could be crucial for a smoother approvals application.
Patient feedback framework
In the UK, the Health Research Authority (HRA) outlines four key principles for involving patient feedback in clinical trials. These principles provide a framework for sponsors to ensure that their patient engagement efforts are meaningful and effective:
- Involve the right people
Ensure the feedback you gather comes from individuals who are representative of the patient population in your proposed trial. This would include input from parents and/or legal guardians if the trial involves children or caregivers and/or care providers if the trial involves adults with applicable disability or illness. - Involve enough people
Whilst it would be easier to gather feedback from a small group of patients, a broader spectrum is necessary to capture diverse experiences and symptoms. The patient population may experience a condition in varying ways, and it’s important to account for these differences. There are notable exceptions, such as specific rare diseases where the patient population may be too small to reasonably engage with a broad spectrum of patients. - Involve people adequately
It is also important to ensure the engagement process is meaningful. Provide sufficient time for patient groups to review study materials, participate in meetings, and provide feedback. This could involve multiple touchpoints, such as email exchanges, phone calls, or regular meetings. Understand that patient groups do not generally work at the same pace of people in the industry and are usually volunteering their free time to assist sponsors. - Demonstrate how feedback has shaped the trial
It’s crucial to show agencies specifically how the patient feedback has influenced the design and/or management of the trial. Whether it is adjusting patient information sheets, modifying visit schedules, or refining protocols, documenting the impact the patient input has made to the trial ensures that the engagement process is transparent to the agencies and highlights the changes that were made by the sponsor for the benefit of the participants.
Challenges and opportunities
Despite the growing emphasis on patient engagement by all industry stakeholders, there are still challenges to overcome, one of which is the issue of diversity. Whilst there are many patient advocacy groups doing great work to involve patients in research, there remains a lack of representation from specific demographics historically under-represented in research. In some cases, patient groups may consist of a narrow subset of the patient population, such as predominantly white or affluent individuals, which can lead to feedback that doesn’t fully capture the diversity of the patient community. This is especially problematic in larger trials and those for more common conditions where the patient pool is diverse.
Another area for improvement is the inclusion of vulnerable populations, such as those with disabilities. In clinical trials, sponsors should ensure that all participants, including those with physical or sensory impairments, have equal opportunities to engage in their trials. This might involve providing accommodations such as accessible advertising materials or ensuring on-site support for deaf or blind participants.
Looking to the future
As patient engagement becomes more embedded in the clinical trial process, there is a real opportunity for the drug development industry to partner with patient populations to significantly benefit both parties, with better data and improved trials. Ultimately, patient engagement isn’t just good practice; it’s the right thing to do. By ensuring that patients are considered partners in the process, sponsors can streamline the development of future therapies and ultimately achieve the shared goal of improving patient outcomes.
To find out more about this topic, listen to our Conversations in Drug Development podcast episode: Patients as Partners: Advocacy’s Role in Shaping Drug Development here.
