Patient-centered real-world research should be an essential part of the evidence development strategy for an immunology portfolio.
Here, we discuss how this type of research can add particular value in autoimmune and immune-mediated diseases.
Accurate measurement of fluctuating symptom burden
Immunological diseases are typically chronic and are often characterized by periods of milder symptom severity interspersed with acute symptom worsening or âflaresâ â consider asthma attacks, for example.
These symptom fluctuations can be difficult to capture accurately, ideally requiring detailed data collection over a long time frame. One way to address this challenge is to collect longitudinal patient-reported data via a real-world study. Such studies can allow patients to conveniently report symptom changes as they occur, via an app on their phone, thus providing extremely granular data, unaffected by recall bias and at low patient burden.
We implemented this approach recently in a peanut allergy study that we ran for a client. Patients received a weekly text message asking whether they had been exposed to the peanut allergen in the past week. Only if the participant replied âYâ (i.e., âyesâ) were they presented with a link to a follow-up survey. This approach yielded detailed information about potential changes in the patientâs status while imposing minimal burden. Indeed, the ease of providing data in this way drove excellent longitudinal engagement, with 100% completion of follow-up surveys at three months, thereby generating an invaluable dataset.
Comprehensive understanding of patientsâ experiences
A challenge inherent to many immune-related diseases is that some symptoms are difficult to measure objectively, but are nonetheless extremely debilitating. A good example is the adverse mental health impact of a visible skin condition like atopic dermatitis or psoriasis. Consequently, the full patient burden of such conditions may be underestimated. This is where patient-centered research plays an important role. Patient-reported data about symptoms, or the impact of a condition on daily life, productivity, or mental health, can be an important complement to clinical data, thus helping to establish a comprehensive understanding of disease burden.
Efficient identification of large-scale, cross-disease patterns
Many immune-mediated diseases appear clinically unrelated but share similar underlying immune dysregulation, meaning that they can be targeted by the same treatment: a single therapy may be effective across immune-mediated conditions in dermatology, gastroenterology, and also rheumatology. Multiple immunological diseases can also co-exist in the same patient or family. It can therefore be useful to study such interrelated diseases together, to identify patterns and common characteristics. A real-world registry is sensible way to do this, enabling data generation at a scale large enough to detect trends, while capitalizing on the cost- and time-efficiencies afforded by the âmulti-disease, one studyâ approach. Such registries can add value at multiple stages of portfolio development, from building an early stage understanding of disease mechanisms, through to post-launch assessment of real-world effectiveness, identification of treatment response predictors, and long-term monitoring of safety.
To move beyond symptom management to disease modification, remission, and cure, a deeper understanding of immunological diseases is necessary. Real-world research provides opportunities to gather a wealth of detailed information by linking patient data from multiple sources. Genetic and molecular data, clinical data from medical records, and patient-reported data about symptoms and adverse events can all be linked, thereby permitting analysis of, for example, underlying drivers of disease severity or predictors of treatment response. At Vitaccess, we specialize in this type of research, with many of our projects involving the prospective longitudinal collection of both clinical and patient-reported data.
Conclusion
Patient-centered real-world research is invaluable in immunology, to gain a detailed understanding of these diseases and learn how to target treatments appropriately.
At Vitaccess, we have a wealth of experience in patient-centered real-world studies, including in chronic and immunological conditions. Please contact us if you are interested in learning more.
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