Conducting user research with rare disease patient participants: key considerations
- User research
Do you want to create a positive impact for the people who really need it? Well-designed digital products and services can make a big impact for those living with rare diseases, helping them to feel supported and reassured that their unique needs are accounted for. Thomas Michalak writes here for Pharmaphorum.
Each person living with a rare disease has a unique experience of their condition, but all will have faced similar challenges. They have suffered from an overwhelming diagnosis experience that has left them feeling misunderstood, isolated, and desperate for someone to help them connect symptoms and make sense of the sheer volume of information.
Many will also face barriers to care, where access to specialists is limited and often geographically out of reach. That’s where technology coupled with user-centred design can help – bringing solutions and convenience to these pain points and positively impacting people’s well-being. The article was originally published by Pharmaphorum: https://pharmaphorum.com/views....
Consultations and clinical trials are often few and far between. Data points are snapshots months or years apart, captured in an out-of-home location, creating an overly simplified and biased representation of the person’s well-being or reaction to treatment. By including a higher frequency of entries from the person’s day-to-day life, we can correlate behaviours and outcomes with treatment more effectively.
For example, digitally reported patient outcomes can help widen the pool of patients and bring data points closer, accurately representing the patient’s outcome. With such heterogenous experiences in rare diseases, digital solutions can create a more inclusive process and data set. Research in rare diseases will benefit from identifying cause and effect, which consider behaviours, social economics, and the patient’s emotional state.
In the US, 88% of people living with a rare disease would consider investigational treatment, compared to only 62% 30 years ago*. This indicates the openness of rare disease patient communities to new treatments and a higher likelihood of early adoption.
New digital products always rely on early adopters to create a community and improve the product. Patients and their caregivers are often very proactive and passionate about advocacy; they will join charities and online groups, or even take on patient advocacy roles, often out of necessity.
Communities that had to solve major problems because of systemic issues, lack of funding, and disparities often hack solutions together to get by and survive. It is this passion and fundamental need for change that suggests digital products can have a higher success rate when supporting rare disease communities.
Digital products in rare diseases will, by design, be user-centred as they aim to connect the dots between medical and emotional circumstances. By mapping patients’ journeys, a better understanding of people’s daily lives will lead to more relevant and efficient solutions that can positively impact people’s well-being.
This new information can help pharma increase adherence by improving the quality of treatment and creating patient-centred programs and materials. If published, this information will also make the brand more favourable to healthcare providers (HCPs) and patient advocacy groups, demonstrating a more profound empathy for patients and their struggles.
Another way to increase people’s quality of life is by bringing convenience. Online consultations can increase access to specialists, and patient databases can help HCPs make a more accurate diagnosis.
The success of digital products, such as LivingWith, in chronic conditions could be replicated for rare diseases. The app connects people to their clinicians to monitor condition activity, episodes, and medication, encouraging patients to track personal trends, symptoms, and triggers. With 78% of users having experienced some improvement in their condition since using this app†, imagine the impact for a teenager living with Haemophilia B and his consultant when they can access a fuller picture of his well-being and joint pain. This could considerably increase the quality of consultations by allowing patients to consider and communicate with their practitioners, not just about the recent weeks, but about the past months or years.
Those living with rare diseases can often feel socially marginalised and isolated due to their condition, leading to an increased risk of depression‡, self-harm, or drug abuse§. Social media-style sites for rare diseases can connect patient communities globally. Initiatives like Share4Rare are already supporting patients and carers by allowing them to be heard and emphasised.
People newly diagnosed with rare diseases and their families are often overwhelmed with information and the realisation that there isn’t a straightforward treatment to support them.
During your user research, pay close attention to patients’ cognitive load before and after the diagnosis. No matter how great your digital solution is, it will be ignored if the person isn’t in the right mindset. Working closely with HCPs will also help you understand when and how to introduce your solution.
A less crowded digital landscape
Soon, digital therapeutics and digital health solutions will undeniably become standard offerings in our health systems. Initiatives like DiGa‖ in Germany and Norddec in Scandinavia¶ prove that the health community has recognised the benefits of digital solutions in supporting HCPs and empowering patients.
If you are a specialist rare disease pharma company, investing now in digital solutions will help you secure a “first to market” position that will increase your chances of being recognised as the favourable solution for HCPs and patients.
Working in the area of rare diseases, you are undeniably passionate, emotionally engaged and dedicated to helping patients. This passion means you will carry your digital product through storms and fight for its success. You always advocate putting the patient’s needs at the centre of your digital strategy. Armed with a passionate mission and authentic values, you will have higher chances of success, even in uncertain times, by remaining focused on what matters most — the patient.