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Context

Orphan drugs, critical in treating rare diseases, have increasingly gained attention in India following the implementation of the National Policy for Rare Diseases (NPRD) in 2021.

Read about rare diseases and challenges in India: https://www.iasgyan.in/daily-current-affairs/issues-in-treatment-of-rare-diseases

Despite this, 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.

What Are Orphan Drugs?

Orphan drugs are special medicines used to treat rare diseases, which can sometimes be life-threatening or cause long-term health issues. While these drugs are critical for those with rare diseases, developing them is very costly, and India faces unique challenges in making them available.

Orphan drugs are made specifically for rare diseases. These diseases affect a small number of people but can be very serious.

Global Standards:

Different countries have set criteria for orphan diseases:

In the U.S., a disease is rare if it affects fewer than 200,000 people.
In the European Union, a disease is rare if it affects fewer than 1 in 10,000 people.
In India, there is no exact definition for orphan diseases, but the National Policy for Rare Diseases (NPRD) provides some guidelines.

Classification of Orphan Drugs

Based on Disease Type:

Genetic Disorders: Diseases passed through genes, like cystic fibrosis.
Rare Cancers: Types of cancer like neuroblastoma.
Metabolic Disorders: For example, Gaucher’s disease, where the body struggles to process certain substances.
Autoimmune Diseases: Like systemic sclerosis, where the immune system attacks the body.

Based on Approval Status:

Approved Drugs: Recognized by agencies like the U.S. FDA or European Medicines Agency.
Candidates in Clinical Trials: Medicines still being tested for effectiveness and safety.

India’s Classification for Rare Diseases

Under the National Policy of Rare Diseases, India groups rare diseases into three categories to help with treatment plans:

Group 1: Diseases curable with one-time treatments, such as some Lysosomal Storage Disorders.

Group 2: Diseases requiring lifelong management but with lower treatment costs, like Maple Syrup Urine Disease (MSUD).

Group 3: Diseases needing high-cost, lifelong care, like Gaucher Disease.

Why Developing Orphan Drugs is Difficult in India?

Even though India produces all 450 of the world’s orphan medications (APIs), the majority of these medications are not readily available and are not reasonably priced here.

Developing orphan drugs is expensive, and the small number of patients makes it hard for companies to profit.

Since rare diseases affect few people, finding enough participants for clinical trials is challenging.

Many treatments are very expensive, such as enzyme replacement therapies (ERTs), costing crores of rupees per year, making them unaffordable for most Indian families.

India’s Current Challenges in Orphan Drug Development

While global frameworks such as the Orphan Drug Act (ODA) of 1983 in the U.S. have successfully incentivised pharmaceutical companies to invest in these drugs, India is still in the early stages of establishing a robust system that can address the needs of its rare disease patients.

India doesn’t have a clear definition for rare diseases or enough data, which makes drug development harder. Unlike the U.S. or EU, India doesn’t offer big tax breaks or other financial incentives for companies working on orphan drugs.

Currently, 14,615 cases are recorded in India’s rare disease registry, but a central national registry is needed for accurate data and better planning.

How Can India Improve Orphan Drug Availability?

A central registry would help track the prevalence of rare diseases. Introducing tax breaks, grants, and subsidies can motivate companies to develop orphan drugs. Policies to control prices and offer subsidies could make these drugs more affordable.

Sources:

HINDU