PHARMACEUTICAL STRATEGIES IN ORPHAN DRUG DEVELOPMENT

Bhargavi Pandaga

Pandaga Bhargavi Priyadarshini Institute of Pharmaceutical Education and Research, 5th Mile, Pulladigunta, Guntur-522017, Andhra Pradesh, India.

Abstract

Pharmaceutical medicines known as orphan medications are created to treat uncommon illnesses that only a small percentage of people have and frequently have no proven treatments. Only a small number of licensed treatments are accessible despite the rising prevalence of rare diseases worldwide, mostly because of financial, regulatory, and scientific obstacles. With an emphasis on developing therapeutic modalities, technological breakthroughs, and regulatory frameworks, this paper offers a thorough analysis of orphan drug development. Treatment options for formerly untreatable uncommon diseases have increased due to recent trends showing a diversity of pharmacological modalities, including small molecules, gene and cell therapies, nucleic acid-based medicines, monoclonal antibodies, and synthetic proteins. Orphan drug approvals in the US have been greatly sped by regulatory incentives like the Orphan Drug Act, and development has been further aided by changing trial designs like n-of-one and expedited review procedures

Keywords: Orphan drug, regulatory pathways, treatment, drug development, rare diseases, patient advocacy rare diseases, regulatory

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