Advancing Rare Disease Drugs Through Real-World Evidence

Patients with rare diseases often suffer with few effective treatment options. Therapeutic development for these drugs targeting rare diseases increasingly depends on real-world evidence (RWE). In a new IQVIA blog, learn more about efforts in the UK to advance the development of drugs for rare diseases through the careful use of RWE.

According to professionals at IQVIA, “Nonetheless, the generation of RWE for rare disease comes with great challenges. One inherent challenge with all rare diseases is the low number of patients per disease. This not only means low availability of medical evidence, but also restricted access to data. Moreover, patients are usually treated across primary, secondary and tertiary care settings leading to information silos and the need to combine data from multiple sources that may not be compatible.  As most rare diseases have a genetic link, data sources will need to combine both clinical and genetic information. Finally, these diseases are often complex with expertise in pathophysiology, natural history, clinical manifestations, and treatment pathway being limited to very few specialists. To successfully, conduct an RWE study for rare diseases, not only will specialised data-sources be required but also a multidisciplinary team of epidemiologists, medics, geneticists, and statisticians are needed.”

To read more, click here.

(Source: IQVIA, February 27^th, 2023)