Delhi High Court Issues Directives to Improve Access to Orphan Drugs for Rare Disease Patients

(Source: Indian Express; Section: Explained; Page: 13)

Topic: GS2 – Social Justice – Health

Context:

The Delhi High Court recently issued directives to improve the availability of “orphan drugs,” which are used 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.

 Analysis of News:   

Classification of Rare Diseases in India

In India, rare diseases are categorized into three groups based on the treatment’s complexity:

• Group 1: Diseases treatable with one-time curative procedures.
• Group 2: Conditions requiring lifelong but relatively affordable treatment.
• Group 3: Diseases requiring lifelong, costly treatment with limited access to effective care.

Financial Assistance and Government Policy

• The National Policy for Rare Diseases (NPRD), launched in 2021, provides financial assistance of up to ₹50 lakh for treatment at designated Centers of Excellence (CoEs).
• These CoEs, such as AIIMS and PGIMER, play a pivotal role in providing care to patients. Additionally, a crowdfunding platform has been created to facilitate voluntary donations for treatment.

High Cost of Orphan Drugs

• Many orphan drugs are patented, making them prohibitively expensive. Pharmaceutical companies are reluctant to manufacture them due to high development costs and a limited market.
• Although patients importing these drugs are exempt from customs duties, companies still face significant taxes.
• The Delhi High Court has emphasized the need for tax exemptions and quicker processing of these medicines to lower costs.

Legal and Policy Interventions

• The government can take steps under the Patents Act to allow third parties to manufacture these medicines, ensuring their availability.
• The High Court has called for more research and negotiations with pharmaceutical companies to lower treatment costs and boost domestic production.

Challenges in Implementation

• The approval process for orphan drugs is often slow, delaying access to essential treatments. The National Rare Diseases Committee highlighted the need for faster regulatory decisions to prevent disruptions in patient care.

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.

What are the Challenges posed by Rare Diseases in India?

1. Unavailability of treatment- Less than 50% of the 450-odd rare diseases identified in India are treatable. Most patients typically receive only basic treatment that alleviates symptoms. 2. Unaffordable Treatment Costs- Some rare disease’s treatment, requires exorbitantly priced antidotes and supportive medication, which poses the challenge of affordability. 3. Low Focus on R&D for drug development- The rare disease is not considered as a significant market by the drug manufacturers, as the number of persons suffering from individual rare diseases is small. Hence, these diseases are treated as ‘orphan diseases‘ and the drugs are treated as ‘orphan drugs‘ by the pharma giants. 4. Late diagnosis- Rare disease takes on an average of seven years for their diagnosis in India. Delay in diagnosis or a wrong diagnosis increases the suffering of the patients exponentially. 5. Lack of trained healthcare professionals- Lack of trained healthcare professionals to interpret the signs and symptoms of rare diseases in the initial stages, has compounded the challenge posed by rare diseases in India.

Practice Question:  Discuss the challenges faced in the treatment of rare diseases in India, particularly in relation to the high cost of orphan drugs and the role of government policy. What steps can be taken to improve access and affordability for patients? (150 words/10 m)