Orphan drugs

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About Orphan drugs:

These are pharmaceutical agents developed specifically to treat rare (orphan) diseases.
- These diseases, though affecting only a small portion of the population, often lead to life-threatening or chronically debilitating conditions. Definitions of orphan drugs vary depending on the regulatory framework.
- There is no formal prevalence-based definition in India, the NPRD of 2021 outlines a framework for diagnosing and treating rare diseases, with a low prevalence threshold expected.
Orphan drugs are categorised based on the types of diseases they target and their regulatory status.
Diseases such as genetic disorders, rare cancers, metabolic disorders, and autoimmune conditions frequently fall under the orphan disease category.
- Genetic disorders include conditions like cystic fibrosis and Duchenne muscular dystrophy,
- Rare cancers like neuroblastoma and gliomas also qualify for orphan drug development.
- Metabolic disorders, such as Gaucher’s disease and Fabry disease, and autoimmune diseases, like systemic sclerosis, also benefit from orphan drugs.
For a drug to receive orphan drug designation, it must meet certain criteria that vary across countries.
- Typically, the disease in question must have a low prevalence, the condition must lack approved treatments, or the orphan drug must provide significant benefits over current treatment options.
- Developers of orphan drugs must also provide scientific evidence that the drug has the potential to treat or alleviate the condition.
- This evidence can be presented at any stage of drug development, from preclinical research to late-phase clinical trials.
Once designated, orphan drugs receive several incentives to encourage their development, including market exclusivity, tax credits for research and development (R&D) expenses, and fee waivers for regulatory applications.

Q1: What is Duchenne muscular dystrophy?

It is a rare genetic disorder. It was first described by the French neurologist Guillaume Benjamin Amand Duchenne in 1860. It is characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact.

News: Understanding orphan drugs: what are they, how are they classified and what are their challenges in the Indian context