TY - JOUR AU - Reddy, D. Saikiran AU - Pramodkumar, T. M. AU - Reddy, Yugendar AU - Sirisha, K. PY - 2014/10/02 Y2 - 2024/03/28 TI - Orphan regulations for orphan drug development in India JF - Asian Journal of Pharmaceutics (AJP) JA - AJP VL - 8 IS - 2 SE - Articles DO - 10.22377/ajp.v8i2.350 UR - https://asiapharmaceutics.info/index.php/ajp/article/view/350 SP - AB - Through this review article an attempt has been made to put forward the challenges faced by rare disease drug development<br />and the current scenario of orphan drug legislations in India. An orphan drug is a pharmaceutical agent that is used to<br />treat a rare medical condition (viz., glioblastoma multiforme, nocardiosis, Tourette syndrome, etc). Developed countries<br />such as United States (US), Europe, Japan, and Australia have laid down legal framework for combating rare diseases. A path<br />breaking legislation was formulated by the US government way back in 1983, known as “Orphan Drugs Act (ODA).” The<br />key purpose of ODA was to incentivize R and D initiatives for such drugs to treat millions of population suffering from<br />“orphan diseases.” Though the percentage of patients suffering from “rare diseases” in India is reportedly higher than the<br />world average, unfortunately even today such cases get little help from our government. Indian government should also<br />encourage its domestic pharmaceutical industry to get engaged in research for orphan drugs by putting an “ODA” in place<br />and extending financial support, and regulatory concessions like smaller and shorter clinical trials, without further delay.<br />Thus, India could well‑demonstrate that the concept of orphan drugs for orphan diseases is really not orphan in India.<br />Key words: Orphan diseases, orphan drugs act, orphan drug ER -