Context:
India faces significant challenges in ensuring the development, affordability, and accessibility of orphan drugs, especially compared to other countries like the United States and the European Union.
Relevance:
GS II: Health
Dimensions of the Article:
- Orphan Drugs
- Classification of Orphan Drugs
- India’s NPRD Classification for Rare Diseases
- Criteria for Orphan Drug Designation
- Incentives for Development
Orphan Drugs
- Orphan drugs 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. In the U.S., a disease is considered rare if it affects fewer than 2,00,000 people, while in the European Union, a disease must affect fewer than 1 in 10,000 people to be considered rare.
- Although 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.
- The lack of a clear definition complicates the identification of orphan drugs and the addressing of needs of patients affected by these conditions.
Classification of Orphan Drugs
Orphan drugs are categorized based on the diseases they target, their stage in the drug development process, and their regulatory status. This classification is crucial for understanding the availability and potential efficacy of treatments for rare diseases.
Disease Categories
- Genetic Disorders: These include conditions such as cystic fibrosis and Duchenne muscular dystrophy, which are inherited and typically require ongoing treatment throughout an individual’s life.
- Rare Cancers: Diseases like neuroblastoma and gliomas are categorized here. They are less common types of cancers, which often lack targeted treatments and carry a poorer prognosis.
- Metabolic Disorders: This category includes diseases like Gaucher’s disease and Fabry disease, which result from enzyme deficiencies affecting metabolism.
- Autoimmune Diseases: Conditions such as systemic sclerosis fall under this category, involving the immune system mistakenly attacking the body’s own tissues.
Regulatory Status
- Approved Orphan Drugs: These have received approval from regulatory bodies such as the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA), indicating they have passed rigorous testing for safety and efficacy.
- Orphan Drug Candidates: These are still undergoing clinical trials to gather necessary data on their effectiveness and safety before they can gain regulatory approval.
India’s NPRD Classification for Rare Diseases
India’s National Policy for Rare Diseases 2021 outlines a strategic classification to guide treatment approaches across different conditions:
- Group 1: Includes curable disorders through one-time interventions, such as Lysosomal Storage Disorders treatable with Hematopoietic Stem Cell Transplantation.
- Group 2: Encompasses diseases requiring long-term or lifelong management but are less costly to treat, such as Phenylketonuria and Maple Syrup Urine Disease.
- Group 3: Covers conditions where treatment is available but is often expensive and requires lifelong care, including diseases like Gaucher Disease and Pompe Disease.
Criteria for Orphan Drug Designation
To be designated as an orphan drug, a medication must meet specific criteria:
- Low Prevalence: The disease must affect a small percentage of the population.
- Unmet Medical Need: There must be a lack of approved treatments, or the orphan drug must offer significant therapeutic benefits over existing options.
- Evidence of Potential Benefit: Developers must provide scientific evidence demonstrating the drug’s potential to treat or alleviate the disease. This evidence can be from any stage of development, from preclinical studies to late-stage clinical trials.
Incentives for Development
Orphan drugs receive several incentives to stimulate their development, crucial for encouraging pharmaceutical companies to invest in treatments for rare diseases:
- Market Exclusivity: This prevents competitors from entering the market with similar products for a specific period.
- Tax Credits: Offered for research and development costs to reduce financial risk.
- Fee Waivers: For regulatory applications to lower the barrier to entry in the drug approval process.
-Source: The Hindu
