For decades, real-world data (RWD) has supplemented findings from clinical trials. It’s helped us understand drug utilization, efficacy, and adverse events that may not have been apparent during initial asset testing and approval.
In some ways, however, traditional RWD isn’t really from the real world — it’s from the clinical world, mostly from claims and electronic health records. This is particularly true for neurological conditions, where so many significant everyday health experiences are invisible in current data sets.
How can we understand the lived experience of a migraine patient’s day-to-day sleep issues, or a multiple sclerosis patient’s mobility loss, to close gaps in our knowledge and support better outcomes?
The next step in the evolution of RWD is a major advancement in patient-generated health data (PGHD), which the FDA defines as “created, recorded, or gathered by or from patients,” such as health history narratives, symptom progression, and lifestyle behaviors. By leveraging this data to create a more complete picture of patient health, companies can mitigate diagnosis, onboarding, and adherence issues; see the impact of treatments on everyday health behavior; and identify and intervene based on granular, “real-world” segmentation and triggers.
Traditional RWD alone falls short
Key neurologic treatment areas share critical challenges in understanding the patient experience.
Incomplete picture of the patient journey: Many central nervous system (CNS) patients go months between visits with providers, so claims and EHR data suffer from large gaps in the patient journey. These sources don’t paint a complete picture of overall health, symptom and disease severity, or when and how patients are benefiting from their treatment. This can lead to many CNS patients cycling through multiple therapies before they achieve adequate disease management.
Subjective endpoints: CNS disease experience can be highly subjective without easily discernible biomarkers and clinically ambiguous patient-reported symptoms. It’s hard to quantify how someone lives and copes with a condition and how these things change over time. Lab values and imaging don’t qualify these experiences; amyloid scans alone, for example, aren’t enough to assess the degree of Alzheimer’s progression. This can lead to diagnostic errors and missed treatment opportunities.
Reliance on recall: Headache diaries are the gold standard for migraine symptom history, but they require months of accurate and time-intensive capture by patients in the midst of work, family, pain, disability, and other everyday hurdles. Many patients simply complete diaries from memory. Without reliable, objective symptom records, clinicians and researchers assess the impact of therapeutics without the concrete information they need.
Patient-generated data fills in the gaps
Off-the-shelf devices – such as computers, mobile devices, wearables, and other biosensors – can offer inexpensive, objective data that quantify disease burden and add much-needed context to the complex, longitudinal CNS patient health journey.
A more complete health picture
Multimodal sensor streams capture granular indicators of disease status, severity, progression, treatment efficacy, and outcomes. They can quantify physical characteristics as well psychosocial factors, all while helping to fulfill requirements by payers for clinical data.
For example, mobility data might serve as a proxy for disease state and progression in patients living with MS. For migraine patients, we might explore the association between passively collected activity data, headache burden, and quality of life. We could integrate patient streams with other data sources, such as weather, to look for trigger patterns in individuals and across populations. Patients could have more productive conversations with their providers and accelerate timelines for diagnosis and treatment.
Direct patient data collection is non-invasive and isn’t interrupted if patients move between providers or insurers. When mapped to traditional “real-world” clinical and claims data, it provides a more comprehensive view of people’s lives.
Enhanced patient segmentation
Patient-generated data can help pharma segment and identify populations by neurologic phenotype, demographics, geography, and more at a scale that’s impossible, or just financially impractical, in clinical trials.
At the population level, it’s much easier to see phenotypic differences in things like migraine frequency. Patient-generated data can help pharmaceutical developers and manufacturers track and characterize frequency, gather efficacy data, and use that information to improve migraine treatments as well as reimbursement rates.
With significantly more volume and diversity across the patient population, companies can utilize social determinants of health (SDoH) inputs, diversify research populations, and adopt more inclusive societal perspectives for decision-making.
Having more-relevant CNS measures also enables more personalized patient engagement and support to optimize adherence and, thus, the benefits of treatment. Longitudinal patient-generated data can help find the right patient at the right time to make the right intervention, independent of their engagement with the traditional healthcare system.
The next step to get closer to patients
Patients’ lived experiences fill the most glaring gaps in RWD by offering a more complete and insightful picture of their health. As we establish benchmarks, combine data sources, implement analytics, and explore the full potential of increasingly rich data, we’ll build new ways of measuring the state and trajectory of disease.
Closing these gaps aligns incentives across the health ecosystem: Providers prescribe more personalized, effective treatments; patients feel better; and costs go down. Pharma can discover more meaningful, actionable patient insights to drive biomedical innovation and increase market value and differentiation.
If we trust patients and empower them with better tools to share their health experiences, we not only get better data, but also place patients at the true center of the highest quality care.
To see examples of real RWD in action, visit Evidation’s case study library.
The editorial staff had no role in this post's creation.