India’s Emerging Pharmaceutical Opportunity in Rare-Disease Drugs

Syllabus: GS2/ Health, GS3/ Economy

Context

• The growing burden of rare diseases has highlighted the need to strengthen India’s rare-disease pharmaceutical ecosystem and leverage its capabilities in affordable drug manufacturing.

What are Rare Diseases?

• Rare diseases are conditions that occur infrequently within a population.
  • They are characterized by three key markers: Total number of people with the disease, Prevalence and Availability /Non-availability of treatment options.
• The World Health Organization (WHO) defines a rare disease as a condition that affects a small percentage of the population, typically fewer than 1 in 1,000 to 2,000 people. 
• There are around 8,000 rare diseases globally, while 1,004 rare genetic disorders have been reported in India.
• Drugs developed to treat rare diseases are called orphan drugs.

The Global Orphan Drug Market

• The US Orphan Drug Act, 1983, introduced financial and regulatory incentives for developing drugs for rare diseases.
• The United States had approved only 38 orphan drugs before the Act, compared with 1,122 approvals by 2022.
• Despite these incentives, the high cost of research, development and treatment continues to restrict patient access.

Challenges in Developing Rare-Disease Drugs

• Commercial incentives: The small number of patients suffering from individual rare diseases restricts the potential market for their medicines, reducing the commercial incentives for pharmaceutical companies to invest in research and development.
• Clinical trials: Identifying sufficiently large patient cohorts is difficult because individual rare diseases affect relatively few people.
• Regulatory constraints: Complex regulatory procedures and the need for additional clinical studies increase the time and cost involved in introducing orphan drugs in India.

Potential for India’s Rare-Disease Pharmaceutical Sector

• Diverse patient population: India’s large population and higher prevalence of certain genetic disorders due to endogamy provide opportunities for patient identification and clinical trials.
• Manufacturing capabilities: India’s expertise in generic medicines, biosimilars and advanced therapies, including CAR-T Cell Therapy, provides a strong foundation for orphan drug manufacturing.
  • Skilled professionals, established infrastructure and relatively low production costs can support affordable, small-scale production.
• International collaboration: Partnerships between foreign pharmaceutical companies, Indian research institutions and patient organisations can accelerate drug development and clinical trials.

Measures to Promote Rare-Disease Drug Development in India

• Under Rule 101 of the New Drugs and Clinical Trials Rules 2019, the Central Drugs Standard Control Organisation (CDSCO) has specified six countries — including the U.S. and the U.K.—and if one of these countries approves a drug, the drug need not have a new clinical trial conducted in India before its maker seeking the Indian regulator’s approval.
• The Department of Pharmaceuticals has launched the Production Linked Incentive (PLI) Scheme for Pharmaceuticals, offering financial incentives to selected manufacturers for domestic production of orphan drugs.

Way Ahead

• India’s established pharmaceutical manufacturing capabilities, diverse patient population and growing biotechnology sector provide opportunities to expand into rare-disease drug development.
• A coordinated approach combining research support, streamlined regulation, sustainable financing and assured patient access can help India develop a globally competitive orphan drug industry while addressing the unmet healthcare needs of its population.

Source: TH