Why in the News?

The Delhi High Court recently issued directives to enhance the availability of “orphan drugs” to combat rare diseases.

About the Delhi High Court Verdict:

The Delhi High Court issued directions aimed at improving the availability of “orphan drugs,” which are used to treat rare diseases.
This intervention seeks to address challenges related to the high cost of these treatments and the barriers to access for patients with rare diseases in India.

Definition: According to the World Health Organization (WHO), rare diseases are debilitating, lifelong conditions that affect 1 or fewer individuals in 1,000.
Conditions Recognized as Rare Diseases in India: Approximately 55 conditions, including Gaucher’s disease, Lysosomal Storage Disorders (LSDs), and certain muscular dystrophies, are classified as rare diseases.
National Registry: The Indian Council of Medical Research (ICMR) manages the National Registry for Rare and Other Inherited Disorders (NRROID), which has documented 14,472 patients with rare diseases.

Group 1: Diseases that can be treated with a one-time curative procedure (e.g., certain enzyme replacement therapies).
Group 2: Conditions requiring long-term or lifelong treatment, which are relatively less expensive and have documented benefits. Regular medical check-ups are necessary for patients.
Group 3: Diseases for which effective treatments are available, but they are highly costly and require ongoing, lifelong therapy. Selecting beneficiaries for these treatments presents a challenge due to the high costs.

National Policy for Rare Diseases (NPRD) 2021: Launched to provide financial support for the treatment of rare diseases. Patients receiving treatment at designated Centres of Excellence (CoE) can get financial assistance up to Rs 50 lakh.
Centres of Excellence: The CoEs include institutions such as AIIMS in Delhi, PGIMER in Chandigarh, and the Institute of Postgraduate Medical Education and Research at Kolkata’s SSKM Hospital.
Crowdfunding and Voluntary Donations Portal (2022): The Health Ministry launched a digital platform that allows donors to contribute toward the treatment of rare disease patients at CoEs. The portal provides details about patients, their conditions, estimated treatment costs, and bank account information of the CoEs.

Limited Treatment Options: Therapies are available for fewer than 5% of rare diseases, resulting in less than 10% of patients receiving disease-specific treatment.
High Treatment Costs: Many existing therapies for rare diseases are prohibitively expensive, putting a significant financial burden on patients and their families.
Regulatory Delays: Approval processes, such as those from the Drug Controller General of India (DCGI), can be slow. For instance, delays in approving US-based Sarepta Therapeutics’ medicines in India have affected the timely availability of treatments.
Bureaucratic Hurdles: Decision-making delays and administrative red tape further complicate access to necessary drugs, impacting patient care.
Challenges in Beneficiary Selection: Due to high treatment costs, identifying and prioritizing beneficiaries for financial assistance is difficult, potentially leaving some patients without support.

Streamline Regulatory Approvals: Expedite the approval process for orphan drugs by reducing bureaucratic hurdles and establishing a fast-track mechanism for essential treatments, ensuring timely access to life-saving medications.
Increase Financial Support and Expand Coverage: Enhance the funding cap under the National Policy for Rare Diseases and extend financial assistance to more patients, while encouraging public-private partnerships and innovative funding mechanisms like insurance coverage for rare disease treatments.