Achieving more meaningful patient engagement in rare disease trials

Patient identification remains the biggest challenge in rare disease trials. However, patient engagement is seeing vast improvements due to advances in technology and its application within the data collection process.

Bruce Hellman at uMotif

Drug development and research into rare diseases is a fast-growing area for the pharmaceutical industry. It is also one that contributes a multitude of complexities in managing clinical trials. These challenges go beyond those experienced in the study of more common conditions, with patient enrolment, engagement, and retention all being more difficult due to smaller and more geographically dispersed study populations. Collecting representative data, often across a broad spectrum of symptoms, calls for improved ways of engaging patients so that partaking in a trial becomes seamless with their everyday life and habits.

The unique demands of rare disease studies have opened the door for technology to change the way that investigators and study sponsors interact with patients in these trials. Never has the focus on using technology to achieve meaningful patient engagement been more crucial.

Rare Disease Clinical Trials

In the UK, 3.5 million people are affected by a rare disease, with many conditions having no effective treatment (1). To be defined as a rare disease, a condition must affect fewer than one in 2,000 people. Diseases that fall into this category include conditions across oncology,
immunology, and haematology, as well as many rare central nervous system (CNS) conditions. One of the main challenges in recruiting patients into clinical trials for rare disease is identifying the relatively small number of individuals with any one illness. Pharmaceutical companies may spend as much as $100,000 to recruit a single patient. This means that keeping every enrolled patient engaged in a trial is critical.

For many patients, staying engaged is something that they will do without issue. They may have exhausted all treatment options, for example, and, understandably, have become willing to go to any length to learn more about their condition, improve their quality of life and to influence future treatment options. In contrast, other groups can be difficult to find and may not be as dialled in to their condition, making the engagement element of the clinical trial paramount.

Making the Use of Technology Meaningful

Technology in rare disease clinical trials is not new, but there is good reason to question whether data collection in these trials is really working for patients and whether it is
contributing to a positive experience. Usability – especially if a particular eClinical solution is compelling enough for a patient to use routinely – should be front of mind when sponsors are assessing the benefits for their study. If patients are engaged with the trial experience, then they are much more likely to remain in a study and more complete, consistent, and insightful data will follow for the sponsor. Furthermore, with a higher rate of trial completion, the substantial costs of patient recruitment are minimised.

Emerging technologies have been designed with a better sense of the need for intuitive user interfaces that patients both understand and enjoy using. There is also more awareness that patients themselves have an interest in the data and insights that they are providing. With new technology there is the opportunity to put a tool in patients’ hands that can help them focus and engage on the most important aspects of their health. Studies have shown that when using electronic clinical outcome assessments (eCOAs) or electronic patient-reported outcome (ePRO) solutions, many patients go beyond the minimum requirements for data input. For example, where patients are obligated to supply data on a monthly basis, it is often seen that they choose to use these solutions daily, resulting in much more granular data being collected than what was originally anticipated. It is this ability to engage patients in a way that becomes synonymous with their daily behaviour that is so important for eClinical technology today.

Additional capabilities of new technologies – for example the use of body mapping to track the location of pain across a variety of disease areas – are also helping to build a better picture of conditions that may, overall, not be very well understood by medical professionals.

A further area where advancements in technology are making a difference is in the study of
paediatric rare disease. Technology use here can be challenging because a single caregiver may not always be responsible for the patient. Care clusters are a new way that platforms
are allowing more than one family member or carer to enter data about a single patient. Capturing multiple perspectives in this way is enabling paediatric trials to gain more reliable and less subjective data.

Technology as an Answer to Patient Needs

Through modern technology there is an opportunity for patients to give more insight into their conditions. An example of how technology has enabled patients to shed further light on their disease is the story of Jessica Morris, who was diagnosed with a rare brain cancer, glioblastoma. As one of the most aggressive and hard to treat
cancers, glioblastoma has a five-year survival rate of just 5%. Due to relatively small patient numbers (18,000 people are diagnosed each year in the US) it can be hard for patients, clinicians, and researchers to make new breakthroughs.

As with many other conditions, patients with glioblastoma are taking an increasingly active and engaged approach to drive forward treatment and understanding of their cancer. Jessica and her neuro-oncologist, Dr Fabio Iwamoto of Columbia University, launched a
patient-powered movement for brain cancer. Using an eClinical platform, patients with the disease were able to track their symptoms, outcomes, experiences, and health data to
drive new insights and support similar patients with their journey.

Final Thought

Finding patients that are suitable and fit the criteria of a protocol is the greatest challenge for rare disease trials. If studies are to keep patients engaged, they need to be given an experience with technology that is fulfilling, enriching, and rewarding to the individual. This means giving them more content, more feedback, and more updates to ensure they remain an active participant in a study for as long as possible.

Technology that puts patients at the heart of these trials and focuses on the need to achieve
long-term engagement, will have a considerable impact on the ability to keep populations on study and maintain the collection of high-fidelity data. In adopting technology that makes the patient experience a priority, sponsors can put strategies in place that will ensure happier
patients, better quality data, more successful trials and, as a result, more new medicines for uncommon diseases reaching the market.


About uMotif

uMotif’s mission is to put patients at the centre of clinical research. Designed with patients for patients, the uMotif patient-centric eClinical platform powers site-based to fully decentralised clinical, real-world, and post-marketing research. By engaging patients and healthcare professionals, uMotif is trusted by global pharmaceutical companies, biotechs, CROs, and academic institutions to capture eConsent and large volumes of ePRO, symptom, and wearable device data. Visit