Why is it in the news?

• The Delhi High Court has recently issued directives to improve access to “orphan drugs,” which are used to treat rare diseases.
• These diseases, as defined by the World Health Organization (WHO), are lifelong and debilitating conditions that affect 1 or fewer people per 1,000, presenting significant treatment challenges.

More about the news

• In India, 55 conditions, such as Gaucher’s disease and certain forms of muscular dystrophy, are classified as rare diseases. The National Registry for Rare and Other Inherited Disorders (NRROID) has recorded 14,472 patients with these diseases.
• However, therapies exist for less than 5% of these conditions, meaning fewer than 1 in 10 patients receive the necessary care. Even when treatments are available, they are often prohibitively expensive, despite government financial aid, leading to legal battles over access to funds.
• Rare diseases in India are categorized into three groups: Group 1 includes diseases treatable with a one-time procedure; Group 2 involves conditions requiring long-term treatment; and Group 3 covers diseases needing costly, lifelong treatment. Identifying beneficiaries for these treatments remains a challenge.
• The 2021 National Policy for Rare Diseases (NPRD) offers financial assistance of up to Rs 50 lakh for patients treated at select Centres of Excellence (CoE). Additionally, a crowdfunding portal launched in 2022 enables donors to support treatment.
• By August 2024, Rs 24 crore had been allocated for rare disease treatments, with Rs 3.15 crore, Rs 34.99 crore, and Rs 74 crore allocated in prior years. Further, Rs 35 crore has also been released to improve patient care services.
• Orphan drugs, which are often patented and costly due to high development expenses and a limited market, remain largely inaccessible. Manufacturing these drugs in India could reduce prices, but government incentives like tax breaks are necessary.
• Although patients can import these medicines duty-free, companies are still subject to customs duties and GST, increasing the cost. The Delhi High Court has set a 30-day deadline for processing exemptions for these drugs.
• In 2019, orphan drugs were exempted from price controls, but the High Court expressed concerns, calling for more balanced pricing.
• Under the Patents Act of 1970, the government can authorize third-party manufacturing of treatments if the patent holder fails to provide them. It can also acquire patents to ensure access.
• The court further highlighted the lack of research and development in rare diseases and recommended negotiating with pharmaceutical companies to lower costs.
• Regulatory delays, such as those involving Sarepta Therapeutics, have also hindered access to treatment, impacting patients at AIIMS and other CoEs.