Context: In a significant move, the Delhi High Court recently directed measures to enhance the availability of orphan drugs—medications specifically designed to treat rare diseases.
Rare diseases, as defined by the WHO, are conditions that affect fewer than 1 in 1,000 people. In India, 55 medical conditions, including Gaucher’s disease and certain muscular dystrophies, are classified as rare diseases.

• However, less than 5% of these diseases have available therapies, leading to significant challenges for patients seeking treatment.

Government Initiatives and Funding Policies

• National Policy for Rare Diseases (NPRD): In response to the challenges faced by patients with rare diseases, the Indian government launched the National Policy for Rare Diseases (NPRD) in 2021. This policy provides financial assistance of up to Rs 50 lakh for treatments at designated Centres of Excellence (CoEs), including prestigious institutions such as AIIMS and PGIMER.

• Digital Portal for Crowdfunding & Voluntary Donations: To facilitate crowdfunding, the Health Ministry launched a digital portal where patients can present their treatment needs, costs, and banking details to potential donors.

• As of August 2024, Rs 24 crore had been allocated to CoEs for treating rare disease patients, with significant disbursements in previous years (Rs 3.15 crore in 2021-22, Rs 34.99 crore in 2022-23, and Rs 74 crore in 2023-24).

• Legal Provisions: Under the Patents Act of 1970, the government can allow third parties to manufacture orphan drugs if they are not made available by the patent holder. This approach includes negotiating with pharmaceutical companies to ensure drug availability and potentially acquiring patents to facilitate local production.

Challenges in the Availability of Orphan Drugs

• High Cost of Orphan Drugs: Many orphan drugs are patented, making them prohibitively expensive. The small market size and high development costs deter pharmaceutical companies from producing these medications profitably.

• Regulatory and Customs Hurdles: While patients importing orphan drugs are exempt from customs duty, pharmaceutical companies still face an 11% customs duty and a 12% GST when bringing these medications to India. The Delhi High Court has mandated that necessary exemptions for these drugs be processed within 30 days.

• Lack of Price Control: In January 2019, the Department of Pharmaceuticals exempted orphan drugs from price controls. The Delhi High Court criticized this exemption, emphasizing that it creates barriers to access for patients.

• Delayed Approvals: The approval process for orphan drugs by the Drug Controller General of India (DCGI) often faces delays, affecting timely access to treatments.

About Rare Disease

• Rare diseases are defined by the World Health Organization (WHO) as debilitating conditions affecting fewer than 1 in 1,000 individuals.

• In India, around 55 medical conditions, including Gaucher’s disease and various forms of muscular dystrophy, fall under this classification.

• The National Registry for Rare and Other Inherited Disorders (NRROID) has documented 14,472 rare disease patients in the country, revealing the urgent need for effective treatments.

• Classification of Rare Diseases: Rare diseases in India are categorized into three groups based on treatment options:

• Group 1: Diseases that can be treated with a one-time curative procedure.

• Group 2: Conditions requiring long-term or lifelong treatment, which are less costly but necessitate regular check-ups.

• Group 3: Diseases for which effective but expensive treatments are available and often require lifelong administration.

Conclusion

• While efforts are being made to address rare diseases in India, significant challenges remain in terms of affordability, availability, and regulatory delays.

• A comprehensive strategy involving government policy reforms, tax incentives, and increased research is essential to improve access to orphan drugs.