Rare diseases, often chronic, progressive, debilitating, or life-threatening, affect a small percentage of the global population. In India, a rare disease typically refers to a condition impacting fewer than 1 in 10,000 individuals. With over 7,000 identified rare diseases, a significant majority have a genetic origin, presenting profound challenges for diagnosis, treatment, and ongoing care. Patients and their families face immense burdens, including diagnostic delays due to limited medical awareness and infrastructure, coupled with the prohibitive cost of specialized treatments, particularly for orphan drugs. This financial strain often pushes families into severe economic distress. Recognizing the urgent need to address the unmet medical requirements of this vulnerable group, India has been striving to develop a robust policy framework to ensure equitable access to essential healthcare and therapies.
National Policy for Rare Diseases (NPRD) 2021
The Government of India launched the National Policy for Rare Diseases (NPRD) 2021 to provide a structured approach for managing these conditions. The policy aims for early diagnosis, promotion of indigenous research, and improved access to affordable orphan drugs. Key objectives include:
• Establishing Centers of Excellence (CoEs) for comprehensive diagnosis and treatment.
• Promoting domestic research and development for therapies.
• Offering financial support for rare diseases requiring one-time curative treatment.
• Developing a national rare disease registry for epidemiological data.
NPRD 2021 categorizes diseases into three groups: Group 1 diseases, eligible for up to Rs. 50 lakh financial support from the Rashtriya Arogya Nidhi (RAN) for one-time curative treatment; and Group 2 and 3 diseases, which require long-term or lifelong high-cost treatment. For the latter, the policy promotes crowdfunding platforms and corporate social responsibility (CSR) initiatives to gather funds, while simultaneously emphasizing domestic drug production to reduce overall costs. The CoEs are pivotal in delivering comprehensive care.
Orphan Drugs: Definition and Challenges in India
Orphan drugs are pharmaceutical products developed for treating rare medical conditions. Their development is often economically unviable for companies due to the small patient pool, leading to limited research and high costs. To stimulate production, governments worldwide offer incentives like tax credits and market exclusivity. In India, several significant hurdles impede the accessibility of orphan drugs:
• Prohibitive Cost: The primary barrier is the exorbitant price, often millions of rupees annually per patient, reflecting high R&D expenses spread over a small user base.
• Limited Availability: Many globally approved orphan drugs are not readily accessible in India due to regulatory complexities, import restrictions, and the absence of a large commercial market for manufacturers.
• Domestic Manufacturing Gap: Despite India’s robust generic pharmaceutical industry, orphan drug manufacturing remains underdeveloped due to complex biotechnological processes and perceived low return on investment.
• Regulatory Hurdles: The drug approval process for rare diseases can be lengthy, delaying patient access, although efforts are underway to streamline these pathways.
Addressing these challenges requires a concerted approach, combining incentives for domestic manufacturing, fostering international collaborations, and implementing robust financial support to make these life-saving treatments attainable.
Government Initiatives and Accessibility Enhancements
Beyond the NPRD 2021, the Indian government has implemented several initiatives to improve rare disease management and orphan drug accessibility:
• Centers of Excellence (CoEs): Eight CoEs across India offer crucial diagnostic, clinical, and research facilities, enhancing diagnostic accuracy and treatment protocols for rare diseases.
• Financial Assistance: The NPRD offers up to Rs. 50 lakh for one-time curative treatment for eligible Group 1 rare disease patients, easing significant financial burdens.
• Crowdfunding Platform: A digital platform is encouraged by the policy to facilitate public and corporate contributions towards treatment costs for Group 2 and 3 rare disease patients.
• Customs Duty Exemption: In April 2023, a full customs duty exemption was granted for all imported drugs and special medical food used in rare disease treatments, substantially reducing costs.
• Research and Development: Government bodies like the Department of Biotechnology and ICMR actively fund and foster domestic R&D, promoting innovation in rare disease diagnostics and therapies.
These concerted efforts demonstrate a strong governmental commitment to alleviating challenges faced by rare disease patients and their families.
Frequently Asked Questions (FAQs)
1. What defines a rare disease in India?
In India, a rare disease is generally defined as a health condition affecting a very small percentage of the population, specifically less than 1 in 10,000 individuals. These conditions are often chronic, progressive, and life-threatening, requiring specialized medical attention and high-cost treatments.
2. What is the main objective of the National Policy for Rare Diseases (NPRD) 2021?
The NPRD 2021 aims to promote indigenous research and development, strengthen diagnostic and treatment facilities, create a national registry, and provide financial support for certain rare diseases. It seeks to ensure more equitable and affordable access to care.
3. Why are orphan drugs so expensive?
Orphan drugs are expensive primarily due to the high costs associated with their research and development, which are spread over a very small patient population. The complex manufacturing processes and limited market demand also contribute to their exorbitant pricing.
4. How does the government help reduce the cost of orphan drugs?
The government helps by providing financial assistance under schemes like RAN for Group 1 rare diseases and by granting full customs duty exemption on imported orphan drugs and food for special medical purposes. It also promotes domestic R&D to lower costs.
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