Current Edition

Why is Real World Data the Key to Rare Disease Success?

The quest to understand and treat rare diseases is among the most challenging and vital missions in healthcare today. As the industry seeks innovative solutions, Real World Evidence (RWE) studies utilising Real World Data (RWD) have grown in acceptance. It’s been argued by many that RWD provides valuable insight into how an Investigational Medicinal Product (IMP) performs in the real world, whereas a Randomised Clinical Trial (RCT) setting is heavily regulated and robustly follows the patient inclusion and exclusion criteria defined in a protocol and trial settings. RWD can provide insight that you simply would not get in the traditional sense, and this insight is worth reviewing and not ignoring, as well as opening you up to other patient populations that may not have been looked at previously. This paradigm shift from traditional clinical data to real-world insights marks a new era for researchers, physicians, and patients alike. As the industry adapts, the implications of RWD are revealed, shaping the future of diagnosis, treatment, and patient care.

The Emergence of Real-World Data

Rare diseases, characterised by their low prevalence and complex nature, have long presented unique challenges to researchers. With approximately 7,000 rare diseases affecting over 350 million people worldwide, the research community faces significant hurdles. Traditional clinical trials often fall short due to small patient populations, heterogeneous symptoms, and a lack of historical data, often leading to significant delays in drug development. In this context, Real World Data (RWD) emerges as a critical tool, by tapping into a diverse array of sources like electronic health records (EHRs), insurance claims, patient registries, and direct patient inputs, offering a more comprehensive understanding of rare diseases.

Unlike the structured environment of traditional clinical trials, RWD reflects the complexities of everyday clinical practice and offers a broader, more diverse patient population. It provides insights into disease progression, treatment outcomes, and patient quality of life in a real-world context, thus enabling more informed decision-making and innovative trial designs. It can also give you more insight into how an IMP would react with other co-medications and treatments because you will get more situations where the trial drug is being taken alongside other medications.

Key Benefits of RWD in RWE Rare Disease Research

Access to a Global Pool of Patients: One of the most significant advantages of RWE studies is the ability to access a much larger and more diverse patient population than what is typically available in centralised clinical trials. For rare diseases, this is particularly crucial as patients are often scattered across the globe. RWD allows researchers to tap into international databases, patient registries, and health records, thus overcoming the geographical and logistical barriers that often hamper rare disease research. This globalisation can lead to more robust data sets, offering deeper insights and more generalisable findings.

Understanding Natural Disease Progression: Understanding natural disease progression through Real-World Data (RWD) provides researchers with critical insights into rare diseases. By analysing a variety of patient data over extended periods, researchers can discern patterns, variations, and outcomes of diseases, offering a comprehensive view of their progression and impact. This deep understanding, drawn from real-life contexts, is instrumental in designing effective treatments and care strategies, thereby enhancing both treatment effectiveness and patient outcomes.