RWE 101 – Benefits of RWE in the Context of Rare Diseases


Real-world evidence (RWE) refers to information on health care that comes from real-world settings, such as electronic health records (EHRs), insurance claims, product and disease registries, patient-generated data, and emerging sources like wearables and social media.

RWE is becoming increasingly important in the management of rare diseases like Addison’s disease, which is characterized by insufficient hormone production from the adrenal glands. Here are some benefits RWE can provide:

[1] Improved Understanding of Natural Disease Progression: Due to the low prevalence of rare diseases, our understanding of them often lacks detail. Real-world data can help fill these knowledge gaps by offering insights into the disease’s natural history and progression.

[2] Efficacy and Safety of Treatments: Clinical trials are the gold standard for assessing the efficacy and safety of new treatments, but they may not represent the broader patient population due to their strict inclusion and exclusion criteria. RWE, being derived from a diverse population, can provide insight into how a treatment works in a broader range of patients.

[3] Patient-focused Drug Development: With RWE, researchers can understand the burden of disease and the benefits patients value most, which can guide patient-focused drug development.

[4] Health Economics and Outcomes Research (HEOR): RWE can provide critical data for health economic evaluations, cost-effectiveness studies, and budget impact analyses.

[5] Early Identification and Diagnosis: Since many rare diseases often go undiagnosed due to their rarity and a lack of awareness among healthcare providers, RWE can help in early identification and diagnosis by revealing patterns or correlations that might not be apparent in smaller datasets.

[6] Post-marketing Surveillance: After a drug has been approved, RWE can be used to monitor its long-term effectiveness and side effects in a larger population.

[7] Guiding Clinical Practice: By understanding how treatments work in the real world, doctors can be better informed about the likely benefits and risks for individual patients, thus guiding personalized treatment.

However, it’s important to note that while RWE provides numerous benefits, it also comes with challenges, including data quality and integrity, privacy and security concerns, and the need for sophisticated analytical methods to derive meaningful conclusions from complex and heterogenous datasets.

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