Conducting research with rare disease patient participants: key considerations

Often in healthcare, the individuals that we seek to understand and learn from can be vulnerable both physically and mentally. Episodes of ill-health or serious injury are traumatic for many individuals, and those that live with chronic conditions are often at a far-increased risk of developing a mental health disorder. 

Vulnerable individuals are also often heavily burdened by widespread misunderstanding of their condition by healthcare professionals. Patients with rare diseases, for example, often face difficulties in clinical care due to the low prevalence of their diseases and the resulting healthcare professionals' lack of expertise. 

Many patients living with rare or ultra-rare diseases experience a drawn-out and uncertain diagnosis journey spanning years and across multiple phases of their development throughout childhood and early adulthood. 

Patients may have experienced feeling ‘different’ from everyone else, as well as frustrated and misunderstood until the point of their diagnosis. They may have experienced difficulty in forming their identity, often reporting feeling “defined by their condition”. Physical symptoms can further impair agency. This can lead to mental vulnerability and predisposition to conditions such as depression and anxiety. 

Conducting user research with vulnerable populations, such as rare disease patients, is crucial to understand how their physical and mental health needs can be met through digital tools, and how their overall quality of life could subsequently be improved. 

The first challenge before any research project begins is recruitment. There is naturally a diminished participant pool when recruiting from vulnerable patient populations and sometimes these patients can be few and far between geographically, making face-to-face sessions logistically difficult - or impossible. 

Vulnerable patients are less likely to volunteer for research due to barriers at the individual level: distrust of research, lack of confidentiality, fear of safety, schedule conflicts, lack of knowledge about research. 

Conducting user research with vulnerable participants

When conducting user research with vulnerable patients, qualitative methods are favoured. It’s essential that participants feel they are being treated as human beings, whose opinions are valued and important, and not as subjects. 

Qualitative methods, such as user interviews and field studies, are best used to understand human experience in the context of the individual's environment. They can build a wealth of knowledge about the patients' lived experience and build insight that cannot be achieved with quantitative methods alone, which often consider each participant in a research condition as equal subjects. 

Vulnerable participants often experience the world significantly differently.  Somebody who experiences chronic pain, for example, may have developed a sense of learned helplessness over time and believe that there is nothing they can do to improve their quality of life. 

User researchers should utilise qualitative methods to understand as closely as possible the issues faced by patients on a daily basis. By attempting to understand everything — their daily schedules, physical capabilities, interpersonal relationships, mental health, perceptions of HCPs, treatment pathways — researchers can begin to identify core opportunity areas to meet underserved needs with digital solutions. 

In qualitative research such as interviewing, user researchers need to build rapport with their participants to enable them to feel comfortable sharing their experiences. This is especially important when interviewing vulnerable patients who may view the researcher as ‘just another HCP’ or ‘just someone else who wants data from me’. 

Observing and reacting to nuances in behaviour, language, body language, and tone of voice in these sessions is just as crucial as the questions themselves. Probing participants in the wrong ways may cause them to shut down and close off from you as a defence mechanism. The golden rule of silence is a great tool to use; vulnerable participants may be used to being told what to do and how to act by HCPs and carers, so giving them a chance to speak freely and facilitating open conversation is key.  

A user researcher has as much responsibility to empower the participant as they do to collect data and insights. As previously mentioned, vulnerable patient participants may have experienced a lifetime of being instructed on how to treat themselves and how to behave. 

In cases of rare diseases, the patient and HCP relationship may not fit with the traditional assumptions of medical care. A lack of autonomy can lead to patients feeling helpless in decision making, potentially leading to mental disorders such as depression. Vulnerable patients can feel like slaves to their own condition and can become isolated from the rest of society, feeling as though nobody truly understands them or wants to hear their story 

User researchers can offer the opportunity to give the patient a voice. By listening and attempting to truly understand life from the patient's perspective, this can empower patients to feel seen and heard, and to feel that their needs are being cared for. Patients can feel as though they are involved in decision-making and regain a sense of control and confidence in their care.

Co-designing digital solutions with patients, as well as letting patients see how the outcomes of their research are being practically applied, can help ensure they feel valued and listened to. 

Reporting and safeguarding protocols: Ethical concerns

During the user research process, vulnerable patients may share sensitive information about their physical or mental wellbeing, or regarding relationships with HCPs and carers that need reporting. 

Whilst the research session should be a safe space for patients to discuss their experiences openly, protocols need to be in place to ensure that the appropriate reporting is done to protect patients from harm. Researchers need to establish these protocols for protecting participants with the relevant care bodies before data collection begins. 

Researchers: Checking in with yourself

Researching vulnerable patient populations can be mentally taxing. Stories and perspectives from participants can be emotionally challenging and will require a degree of resilience to remain composed and focused on the interview. 

Particularly for longer and more emotionally demanding projects, the burden can wear heavy over time and take a toll on your own mental and physical health.

It’s important that those conducting research take the time to check in on themselves and understand how they are feeling. If needed, speak to somebody — such as a peer, friend or colleague — to share how you’re feeling. If you can't maintain your own wellbeing, you won’t be able to maintain a good standard of research or do right by your research participants.

Conducting research with vulnerable patient participants in healthcare can be challenging, and must be approached with the utmost caution and care. But, when done correctly, the impact of undertaking research in order to make informed decisions about your digital solutions can be life-changing for these patients. 

We collaborate with healthcare and pharmaceutical organisations around the world to deepen customer connection by creating meaningful digital experiences. Our team take a truly user-centric approach to all that we do, utilising our in-house clinical UX research team to conduct research with patients and HCPs on behalf of our clients. 

To find out more about our experience within the rare diseases space, or how we can work together to enhance your digital solutions, get in touch.