What are Orphan drugs?

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.