Using Patient Registries to Accelerate Rare Disease Research

In a recent interview with Clinical Leader, , PhD, of the Indo US Organization for Rare Diseases (IndoUSrare) talks about the challenges facing rare disease research and how the use of patient registries can accelerate research and clinical development. Patient registries allow the collection of real-world data (RWD) that can be used in research, but doing so requires collaboration between all stakeholders.

According to Rajasimha, “Government acts, policies, and guidelines have played a vital role in the progress of rare disease research. As an example, the Orphan Drug Act of 1983 spurred innovation leading to the approval of about 1,100 orphan drugs by 2023. Since the epidemiology or the prevalence and incidence of most rare diseases remain unknown, particularly in different populations, any policy updates are handicapped due to a lack of epidemiology data. The number of new rare diseases discovered in recent years due to the healthcare community’s ability to diagnose new rare and genetic diseases has taken the count close to 11,000 known rare diseases in 2023 (according to a recent report by RARE-X).”

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(Source: Clinical Leader, June 16^th, 2023)