ABSTRACT

A medication intended to treat an uncommon medical ailment (such as Huntington's disease, myoclonus disease, Tourette syndrome, etc.) is known as an orphan drug. Pharmaceutical corporations pay little attention to them since the limited patient population makes the significant investment in drug research unjustifiable. Although the definition of rare diseases differs depending on the jurisdiction, it usually refers to the condition's occurrence, severity, and life expectancy of alternative treatments. The FDA initially established guidelines for orphan pharmaceuticals, which include funds for phase I/II clinical trials, tax credits for clinical research, marketing exclusivity for a predetermined amount of time, and exemption from application filing fees. Pharmaceuticals and natural items used in the diagnosis, treatment, or prevention of rare disorders are referred to as orphan medications. The European Orphan Pharmaceutical Regulation was established to promote the growth of medications to treat uncommon medical conditions and ensure that patients would have adequate access to specialized, high-quality care. A group of pharmacists enquired of the Indian authorities to enact the Orphan Drug Act in 2001 during the Indian Drugs Manufacturers Association (IDMA) convention. The population of India afflicted with uncommon diseases is reliant on orphan drugs authorised in developed countries. Developing nations also feel compelled to takeaction to advance orphan medication development and manufacture. The economic development of India's pharmaceutical industries is negatively impacted by the lack of regulation surrounding orphan pharmaceuticals. A detailed study of the orphan drug approval process in the US, EU and Indiais discussed in this review article. Keywords: Orphan Drug, FDA, IDMA, European Regulatory, Orphan Drug Act,orphan medication
Publication date: 01/12/2024

https://ijbpas.com/pdf/2024/December/MS_IJBPAS_2024_8563.pdf

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https://doi.org/10.31032/IJBPAS/2024/13.12.8563