How to collaborate with patients to create a real-world study

White paper

How to collaborate with patients to develop a patient-centered study

Emma Bagshaw, Catherine Bottomley, Vitaccess Ltd

Tom Smith, independent Patient Engagement Consultant

Introduction

Vitaccess is a patient-centered research organization. We know that the best way to design and run patient-centered research is to work closely with the people who know betterthan we do: patients themselves. In this white paper, co-authored with independent Patient Engagement Consultant, Tom Smith, we discuss how to approach patient collaboration in patient-centered study development.

Why collaborate with patients when developing a patient-centered study?

Patient-centered studies put patients at the heart ofthe research. They are designed around patients’ experiences, needs, and preferences, and often collect data directly from patients orthose closest to them, through questionnaire-based methods, interviews, orfocus groups.

The ultimate goal of patient-centered research is to improve patients’ lives. By gaining a deep understanding of patients’ lived experiences, researchers, healthcare providers, and manufacturers are equipped with better information from which to make decisions and developments that enhance patient outcomes.

When developing such a study, collaborating with patients makes intuitive sense: ifthe aim ofthe study is to learn more about a particular group of people, it is a good idea to seek input from those people when building the study.

In this white paper, we discuss how to approach patient collaboration when planning a patient-centered study and the benefits it can bring.

There are three vital reasons for study sponsors and research organizations to work with patients when developing a patient-centered study.

To improve data quality. Patient input during study design helps to ensure that the data collection process is user-friendly, and that data entry requirements or interview questions are understood by participants. This reduces the risk of missing and erroneous data and therefore improves the overall quality ofthe study dataset.

To improve data quantity. When talking to your target population, you can explore motivations and barriers around study participation, devise ways to encourage recruitment and engagement, and thus increase the volume and completeness ofthe study dataset.

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To improve data relevance. Speaking with patients helps you to understand which aspects oftheir condition are most bothersome and which outcomes may have the biggest impact on their lives. You can then build a study that collects meaningful, patient-relevant data.

As a patient-centered research organization, we understand that there are myriad other benefits to working with patients; however, one that is especially valuable is fostering good relationships with patients. Respectful, ambitious, and meaningful collaborations with patients and advocacy groups, which ensure that patients feel empowered, valued, and heard, help to build long-term relationships for ongoing discussions and future work. They reinforce a positive brand image among yourtarget market and differentiate you from competitors. They also promote a sense oftransparency, cooperation, and humanity across the healthcare industry and clinical research; this improves patient trust, boosts study participation, and contributes to improved healthcare confidence. Increasing transparency between industry and patients benefits everyone.

Designing a study with patients will always return better data, improve the interpretation of that data, and, ultimately, yield better outcomes for patients.

When should you collaborate with patients?

Patient input should be sought during the development of all patient-centered research, but it is invaluable when designing a study that collects self-reported data. If patients are self-reporting – and the burden of providing study data is placed on them ratherthan a professional – you should do all you can to make study participation a user-friendly experience. Participation needs to fit in with patients’ day-to-day lives as seamlessly as possible. Ifwhat you are asking of patients is cumbersome, intrusive, or incongruent, you increase the dropout risk dramatically. A study collecting patient-reported data needs to be intuitive, engaging, and meaningful, so that participants feel motivated to provide data and so it is easy forthem to do so correctly. Patient involvement in study design helps to achieve these aims.

Patients can, and should, be involved at all stages of study development, from initial concept design, protocol development, and selection of outcomes, through to analysis and dissemination of results. The figure below presents contributions that patients can make at key study stages, with a focus on studies that collect patient-reported data.

Patient involvement in the study development pathway

Study advisory board Protocol and study design

Study design and protocol development Materials development

● Insights into the condition; for example, symptoms, outcomes, issues of importance, and emerging themes from within the patient community.

● Input into overarching study plan, to ensure that the overall aims and approach are right.

● Feedback on data collection tools, including relevance, clarity, tone of voice, response options, and visual presentation. Tools should also be validated by testing them with patients, to confirm that they capture the data of interest, particularly when using novel bespoke questions.

● Feedback on assessment method and schedule, especially the associated burden on study participants, and any accessibility requirements or limitations.

● Review of planned analyses, to ensure patient-relevancy of data output.

● Input into recruitment strategy, including motivations, barriers, and recruitment channels.

● Input into engagement and retention strategy, including codesigning any incentives, reminders, or within-study communications.

● Feedback on promotional and recruitment material, including likely appeal ofthe study, clarity and completeness of content, and distribution channels.

● Review of patient information form and informed consent process for comprehension, completeness of content, and ease of completion.

● Usertesting of data collection process; for example, testing digital tools for user experience and ease of data input.

● Feedback on local versions of study materials, such as accuracy oftranslation and cultural applicability.

During the study Results dissemination

● Assisting with recruitment and engagement, via emails, social media, or word of mouth to patient networks. Patient-voiced study updates – for example, recruitment numbers in their region – help participants to feel that they are part of a community.

● Sharing insights into challenges and potential solutions; for example, any issues with recruitment or engagement, or amending data collection to improve data quality.

● Providing feedback on howto share results to maximize relevance, understanding, and reach of the data among the target population.

● Ensuring that results-sharing makes patients feel recognized and valued for their contributions.

● Co-authoring publications or reviewing them from a patient perspective. Having a respected member of the patient community as a co-author increases patient trust in the intentions and accuracy of publications.

● Actively sharing results; for example, through patient advocacy group communications, or appearing in a webinar.

Involving patients early helps them understand they are part of the team

If you only involve patients at one stage, seek their early input into study design, before the protocol is finalized and goes through ethics review. This provides an opportunity to make sure that the right data are being collected in the right way, while there is still chance to make any necessary changes. It also demonstrates to an ethics committee that you are serious about putting patients’ needs and preferences first, which will make it far more likely to secure ethical approval the first time around. Increasingly, ethics committees are requesting evidence of patient input into submitted documents, such as protocols and participant-facing material. It is only a matter oftime before patient co-creation is mandatory.

By involving patients early, you are also building a connection and sense of collaboration, meaning that the patient community is then more likely to work with you in later stages, for example in study promotion or publications co-authorship.

To identify key opportunities for patient involvement, we strongly recommend developing a patient involvement plan at the start of a project, setting out what involvement is anticipated, when, and why. This ensures that patient input is incorporated systematically at key stages,and consistently across all studies and therapy areas, as well as safeguarding against missed opportunities in the race to reach study launch. A plan is also a useful way to identify and schedule the time needed to establish contact with patient partners and navigate onboarding processes, so that these potentially time-consuming activities do not unduly delay the project.

How to identify patients

In many circumstances, it makes sense for patient collaborators to closely represent the target study population, so that their input is as relevant as possible. Consider the demographic profiles that you hope to include in the study itself and, where possible, seek to collaborate with patients who have these characteristics. If there are discrepancies between the broader population of interest and those who are likely to either provide input on study design or take part in research, that itself is useful information, and is food for thought in terms of barriers to participation.

That said, many experiences of disease or ill health are universal, so you do not always need to partner with patients who have a specific indication in order to unlock the full potential of a study.

It is also valuable to consider patient collaborators who possess skills and experiences that are relevant to the study creation process, but who do not necessarily have the target condition. Many technical aspects of study development, such as dissemination plans, are relatively similar across studies, meaning that a patient who has experience of these may be more suitable than someone with the target condition who lacks experience.

In some cases, an expert perspective is necessary. This is where patient advocates and patient engagement consultants come in.

● Patient advocates are volunteer or professional patient representatives whose mission is to promote the rights and needs of their community.

● Patient engagement consultants partner with patients, the healthcare industry, and professionals to support patient interests.

In fact, there are advantages to collaborating with people who have the target condition as well as those with above- or across-disease level experience

These experts are often patients themselves, or supporters or carers of people who are. Patient advocates and engagement consultants tend to have condition-specific or general healthcare knowledge that extends beyond their own experiences, meaning they can, as individuals, speak broadly about the likely experiences and opinions of a target population. They can also draw on past experiences of collaborating with commercial organizations, regulators, and patient groups.

From a practical perspective, especially for time-constrained projects, expert patients can be desirable partners because their familiarity with the healthcare research process means that they are able to get up to speed quickly, and their expectations of the collaboration process are more likely to be aligned with those of researchers.

That said, expert patients may not be representative of the target patient population as a whole.

● They are likely to be more knowledgeable about their condition and its management than is typical.

● They may be better able to navigate the healthcare system.

● Their condition may be both less severe – meaning that they are more able than most to participate in advocacy or collaborative work – and better managed.

Expectations for the study population may therefore need to be adjusted accordingly.

It is sensible to work with different types of people at different stages of a study – i.e., a small group of expert patients during protocol development, a broader pool for user testing of data collection tools, and a plain language specialist to review supporting materials.

Where to find patients

Finding patients to work with can be challenging. To do so efficiently, especially in rare diseases where patients are likely to be geographically dispersed, it helps to identify places where patients naturally interact with each other and discuss their condition:

● in person - such as at a specialist clinic or patient conference

● virtually - perhaps in social media support groups orvia reputable patient influencers and theirfollowers.

When looking specifically for expert patients, search for patient education or support groups in the therapy area in orderto identify contributors to their blogs, webinars, and social media.

Be aware that the people you find most easily and who are comfortable working with you – expert patients or otherwise – are likely to have greater healthcare knowledge and confidence than the average patient, so do have that in mind when interpreting their input.

The figure below presents several avenues to explore.

Where to find patient collaborators

Patient education groups

Online communities (e.g., Facebook groups)

Patient support groups

Existing study sponsor relationships

Patient authors &presenters

A dedicated recruiter

Leaflets &posters in healthcare settings

Patient conferences

How to approach patients

When approaching patients from a position of relative expertise, as with approaching any non-expert member ofthe public, two points are particularly important: first, communicating in plain English, and second, being transparent. Many patients will be unfamiliarwith the drug development and research process, and will therefore value layfriendly communication. Among the general public and in patient communities, there is often some suspicion surrounding the pharmaceutical industry, so it is important to build a true sense oftrust by being as transparent as possible from the start.

Be sure to share the following information upfront, so that potential collaborators can make an informed decision about whetherto work with you.

● Study aims and purpose.

● The benefits - ofthe study itself and of contributing to its development - to them, to other patients, and to commercial or academic stakeholders.

● Contracting and confidentiality requirements. Many patients will be new to contracting and may find it worrying - care should therefore be taken when presenting and explaining contracts.

● Remuneration and management of expenses.

● The level and type of commitment that is required from them at each stage.

● Expected timelines.

How to work with patients

Across all of your interactions with patient collaborators, there are some general principles to have in mind to ensure a positive and fruitful experience for everyone involved.

● First, consider how you would work with a lay person who does not have experience ofthe healthcare industry. You can then assess whetherthere are disease-specific factors to consider and adjust forwhere necessary, such as accessibility issues, cognitive difficulties, or limitations on travel and/or in-person interactions.

● Budget appropriately. Research fair payment rates priorto discussing the terms of the collaboration, and never expect patients to volunteertheirtime forfree. Do not offer exposure in lieu of payment. Collaborators should never be out of pocket as a consequence of contributing to a project or participating in a study.

● Make patients’ involvement straightforward, and give them as much choice as possible about how they wish to contribute. Considertime commitments, travel requirements, and how their contribution will fit in with their lives, work, and own medical needs. Be prepared to be flexible and make accommodations where necessary; for example, longer review times, bringing a caregiver ortranslatorto meetings, and holding meetings at convenient times. Rememberthat forthe vast majority of patients this is not their job, so adjust your approach and expectations accordingly.

● Communication is crucial. Set out clear expectations at the start, and keep patients informed with progress and decisions made; share any resulting publications or outputs.

● For best results, especially for longer-term collaborations, approach patient involvement as a respectful collaboration between members ofthe same team, ratherthan treating patients as an external resource or consultant. Maintain a two-way flow of information, so that it is not just the researcher mining information with nothing given back to the patient.

● Once patients’ involvement has ended, thank them, and consider seeking their feedback about how the collaboration went, to inform your approach to patient involvement in the future. Where appropriate, ask ifthey consent to you retaining their contact details forfollow-up questions or other collaboration opportunities.

What is the best methodology for patient collaboration?

There are various ways to collaborate with and seek input from patients during study development. Here, we describe common approaches and discuss when each is appropriate.

One-off or short-term consultation

A one-off or short-term consultation is an opportunity to receive input from patients through a predefined interaction. It is useful when seeking feedback or insights on a specific element of a study, such as instrument selection or choice of outcomes. As a non-recurring interaction, it is suited to a cross-sectional or short longitudinal study, where, for instance, there is little opportunity to benefit from patient feedback on midstudy design amendments.

Group workshop

Group workshops are a time-efficient way to gather a lot of information, and are a great stimulator of conversation, brainstorming, and creativity. This makes them brilliant for generating ideas. Sharing and comparing thoughts and experiences with others helps patient participants to solidify their own opinions and contextualize their experiences. Facilitators can easily probe for detailed information and follow interesting lines of conversation.

However, more confident individuals can drown out others, so care should be taken to moderate discussions, particularly ifthe desired outcome is to reach consensus or reveal insights from a broad spectrum of experiences. Group workshops can also be logistically challenging and time-consuming to organize, as well as being potentially less convenient than other options for patient participants, especially if conducted in person. For some patients, in-person group meetings are simply impossible – for example, in the UK, people with cystic fibrosis are advised to avoid being in close proximity with each otherto minimize the risk of cross-infection.

One-to-one discussions

One-to-one discussions, or more formal structured interviews, are perfect for capturing detailed perspectives from individual patients. Being able to do these overthe phone or online makes it easierfor patients to participate, and the privacy of sharing experiences this way may be more attractive to some patients than a room full of people in a workshop. One-to-one discussions can be useful for gathering detailed, contextualized data, because the interviewer can build up a profile of a single person.

However, in contrast with a workshop, the conversation may be more structured, with less bouncing of ideas, so they are often not suited to creative tasks. People may also feel anxious in a one-to-one situation when answering revealing or intimate questions, so special attention should be paid to not asking about anything that is not essential. The process of scheduling and conducting a series of individual discussions can also take a long time.

Written survey

Sometimes, all that is needed is a written survey that can be distributed to a group of patients to complete at their convenience. This could be in a paper, online, or app-based format - whichever yourtarget respondents are comfortable with and will find most convenient. Surveys are relatively time- and costefficient forthe study sponsor, and are often more straightforward than open discussions in terms of analysis, especially iffree-text response options are restricted. The convenience and anonymity ofthis approach are appealing to many patients, meaning that a broad range of perspectives can be gathered.

Drawbacks of surveys include the fact that the defined question structure sets bounds on the information that can be collected, the limited opportunity for clarification between participant and researcher, and the lowervalue that participants may place on a survey relative to an in-person interaction, which can lead to poorer quality responses.

Long-term collaboration

A long-term collaboration is the perfect way to seek regular input from a pool of people as needed overthe duration of a longer study. The same group can meet regularly, building confidence and trust, which yields in-depth and highly relevant insights.

The commitment of a long-term collaboration can be substantial, so this method does tend to attract, and be better suited to, expert patients, rather than general members ofthe target population. Ongoing effort is necessary to maintain group member engagement in the process and reduce the risk of dropouts or “silent” members. That said, to reduce overall burden, the level of patient involvement can be tailored to the needs of specific study stages: patient input is typically heaviest in the early stages of study development, with burden reducing considerably once the protocol has been approved and fewer decisions need to be made.

At Vitaccess, for our longitudinal studies, we recommend establishing a “scientific advisory board”, comprising sponsor, clinical expert, and patient representatives, with a member ofthe Vitaccess study team acting as moderator. Such groups meet regularly overthe course of a study, with interactions governed by a charterthat is drafted collaboratively during the first meeting. Roles can be assigned to group members based on interest and expertise, meaning that the wider group may only need meet once or twice a yearto discuss overarching study considerations, while smaller groups can meet more regularly at relevant time points to discuss specific topics, such as publications or participant communication strategies.

There are many practical considerations forthis type of collaboration, chief among them: how can you regularly get a group of people in a room together, even ifvirtually? Tech-related issues can present further hurdles, as can time-zone differences and language barriers in global studies. These challenges are, however, worth the effort when the result is a group of engaged collaborators who are invested in the study and are working togetherto achieve its objectives.

Collaborating with patients to develop a patient-centered study improves its design, data quality, and interpretation. This equips healthcare decision-makers and developers with robust, patient-relevant information from which to make improvements to patient outcomes. Open communications between the healthcare industry and patients also builds transparency, trust, and patient empowerment.

Ultimately, patient involvement in research improves patients’ lives

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About Vitaccess

Vitaccess is a patient-centric research organization. The company supports you with the generation of real-world evidence and insights through creative patient-centric solutions. To learn more, visit vitaccess.com