Drug Repurposing: Unlocking access to rare solutions

Drug repurposing, a quick, cost-effective path to deliver existing treatments to patients, can serve to expedite and expand healthcare access for patients with rare diseases

In the marathon of drug development, where billions of dollars and over a decade of research are the norm, drug repurposing is quietly reshaping the landscape. This approach is particularly beneficial for rare diseases, where the treatment options are woefully limited. For conditions affecting fewer than 200,000 individuals, traditional development is economically daunting and repurposing emerges as a pivotal strategy, offering faster, affordable solutions for conditions that have long been left behind in traditional drug pipelines.

According to the National Institutes of Health (NIH), of the estimated 6,000 to 8,000 known rare diseases, only about 5 per cent have FDA-approved treatments. According to a report from the Ministry of Health and Family Affairs, approximately 96 million people in India are affected by various rare diseases, many of which are genetic or acquired through viral or bacterial infections. Over 70 per cent of these conditions lack a cure and often go unnoticed, presenting a significant opportunity for drug repurposing in India. In this context, the ability to bring effective therapies to market more quickly and at lower costs, positions drug repurposing as a crucial focus for the pharmaceutical industry.

Streamlined regulatory pathways

One of the biggest enablers of drug repurposing is the regulatory framework designed to expedite approvals for already-approved compounds. The FDA's 505(b)(2) pathway and the European Medicines Agency's (EMA) Hybrid Pathway offer routes for drug developers to rely on previously submitted clinical data, cutting down approval times significantly. According to research from Alacrita, a pharma and biotech consulting firm, between 2013 and 2018, nearly 50 per cent of all New Drug Applications (NDAs) approved by the FDA were based on the 505(b)(2) pathway.