A Review on Orphan Drug Dynamics: To Treat Rare Health Conditions

Abstract

Orphan drugs are pharmaceutical products designed to treat rare diseases, which affect a small percentage of the population. This article provides a detailed overview of orphan drugs, particularly focusing on the regulatory landscape, challenges, and ongoing initiatives in India. It examines the improvements in India's regulatory framework, including expedited review processes and exemptions from certain clinical trial requirements, aimed at facilitating the development and accessibility of orphan drugs. The article also highlights the contributions of Indian pharmaceutical companies in the orphan drug sector, showcasing a success story from Sun Pharmaceutical Industries Limited, which developed a significant orphan drug. Additionally, the article explores the challenges faced by India in orphan drug development due to its large population and limited resources, alongside the global advancements in gene therapy and drug repurposing. Despite the absence of a clear regulatory pathway for orphan drug development in India, recent policy changes indicate progress. The study advocates for a collaborative approach between the government, regulatory bodies, industries, and patient advocacy groups to accelerate the development of orphan drugs. Furthermore, it discusses the role of emerging technologies and business models in speeding up the process of creating novel treatments for rare diseases. The article also suggests that international collaboration and regulatory harmonization could improve access to these life-saving therapies, particularly in resource-constrained countries. With the continuous advancement of research and regulatory frameworks, there is hope for improving the treatment landscape for rare diseases globally, especially in countries like India.