Which of the following statements is correct regarding the National Policy for Rare Diseases (NPRD) in India?

NPRD includes provisions for fast‑track approval and price negotiation of orphan medicines, aiming to improve access for rare disease patients. Relevant for GS3.

Explain the major challenges in ensuring affordable access to orphan drugs like Risdiplam for rare disease patients in India.

Focus on patent protection, high R&D costs, limited domestic manufacturing capacity, regulatory delays, and coordination gaps between centre and states under NPRD. Relevant for GS3.

Discuss the role of central and state governments in implementing the National Policy for Rare Diseases, with reference to the recent SMA patients' appeal for generic Risdiplam.

Structure the answer: (i) objectives of NPRD, (ii) central mechanisms – price negotiation, fast‑track approval, import facilitation, (iii) state responsibilities – procurement, distribution, monitoring, (iv) challenges – bureaucratic delays, funding, IP issues, (v) impact of advocacy like SMA petition in prompting executive action. Relevant for GS3.

SMA Patients & Parents Appeal to PM Modi f...

Patients with Spinal Muscular Atrophy and parents of minor sufferers have appealed to Prime Minister Narendra Modi for nationwide access to generic Risdiplam under the National Policy for Rare Diseases. The move highlights the urgency of early treatment for this progressive genetic disorder and underscores the policy’s role in ensuring equitable drug access for rare diseases.

Overview A group of individuals living with Spinal Muscular Atrophy (SMA) and the parents of minor patients have written to Prime Minister Narendra Modi seeking urgent intervention. Their demand is for the nationwide availability of the generic form of Risdiplam under the National Policy for Rare Diseases (NPRD) . The appeal underscores the time‑sensitive nature of SMA therapy, where any delay can lead to irreversible loss of motor function. Key Developments Patients and parents have formally petitioned the Prime Minister for fast‑track approval of generic Risdiplam across India. The request is anchored on the provisions of the National Policy for Rare Diseases (NPRD) , which mandates equitable access to approved treatments for rare conditions. Stakeholders highlight that SMA is a progressive, life‑limiting disease, making early intervention critical to prevent permanent deterioration. Important Facts SMA is a genetic disorder affecting the motor neurons that control voluntary muscles. It is classified as a rare disease because its prevalence is low, but the clinical impact is severe. The only disease‑modifying therapy currently available is Risdiplam , an oral drug that increases the production of functional SMN protein. While the branded version is expensive, a generic version could dramatically lower costs and expand reach. The National Policy for Rare Diseases (NPRD) was introduced to address gaps in diagnosis, treatment, and financial support for such conditions. It empowers the Ministry of Health and Family Welfare to negotiate drug pricing, facilitate import of orphan medicines, and ensure that patients across states receive uniform care. UPSC Relevance Understanding the interplay between health policy and rare disease management is essential for GS‑3 (Health) and GS‑2 (Polity). The case illustrates how advocacy groups can influence executive action, the role of central policies like the NPRD in shaping health outcomes, and the challenges of drug accessibility in a federal structure. It also touches upon ethical considerations (GS‑4) regarding equitable treatment for vulnerable populations. Way Forward To translate the petition into concrete action, the following steps are recommended: The Ministry should expedite the generic drug approval process under existing fast‑track mechanisms. State health departments need to align their procurement strategies with the central NPRD guidelines to avoid regional disparities. Awareness campaigns must be launched to educate patients and caregivers about the availability and proper use of Risdiplam . Periodic monitoring of treatment outcomes should be instituted to assess the impact of broader access on disease progression. Timely implementation will not only alleviate the suffering of SMA patients but also set a precedent for handling other rare diseases under the national policy framework.

<h3>Overview</h3> <p>A group of individuals living with <span class="key-term" data-definition="Spinal Muscular Atrophy — a hereditary neuromuscular disorder causing progressive muscle weakness; relevant to GS1: Health and GS3: Health policy.">Spinal Muscular Atrophy (SMA)</span> and the parents of minor patients have written to <span class="key-term" data-definition="Prime Minister Narendra Modi — head of the Government of India, responsible for executive decisions and policy direction (GS2: Polity).">Prime Minister Narendra Modi</span> seeking urgent intervention. Their demand is for the nationwide availability of the generic form of <span class="key-term" data-definition="Risdiplam — an oral medication that modifies SMN2 gene splicing to increase survival motor neuron protein, used for SMA treatment; illustrates drug approval and access issues (GS3: Health).">Risdiplam</span> under the <span class="key-term" data-definition="National Policy for Rare Diseases (NPRD) — a government framework aimed at providing diagnosis, treatment, and financial support for rare diseases; crucial for health governance (GS3: Health).">National Policy for Rare Diseases (NPRD)</span>. The appeal underscores the time‑sensitive nature of SMA therapy, where any delay can lead to irreversible loss of motor function.</p> <h3>Key Developments</h3> <ul> <li>Patients and parents have formally petitioned the Prime Minister for fast‑track approval of generic <strong>Risdiplam</strong> across India.</li> <li>The request is anchored on the provisions of the <strong>National Policy for Rare Diseases (NPRD)</strong>, which mandates equitable access to approved treatments for rare conditions.</li> <li>Stakeholders highlight that SMA is a progressive, life‑limiting disease, making early intervention critical to prevent permanent deterioration.</li> </ul> <h3>Important Facts</h3> <p>SMA is a <strong>genetic</strong> disorder affecting the motor neurons that control voluntary muscles. It is classified as a rare disease because its prevalence is low, but the clinical impact is severe. The only disease‑modifying therapy currently available is <strong>Risdiplam</strong>, an oral drug that increases the production of functional SMN protein. While the branded version is expensive, a generic version could dramatically lower costs and expand reach.</p> <p>The <strong>National Policy for Rare Diseases (NPRD)</strong> was introduced to address gaps in diagnosis, treatment, and financial support for such conditions. It empowers the Ministry of Health and Family Welfare to negotiate drug pricing, facilitate import of orphan medicines, and ensure that patients across states receive uniform care.</p> <h3>UPSC Relevance</h3> <p>Understanding the interplay between health policy and rare disease management is essential for GS‑3 (Health) and GS‑2 (Polity). The case illustrates how advocacy groups can influence executive action, the role of central policies like the <strong>NPRD</strong> in shaping health outcomes, and the challenges of drug accessibility in a federal structure. It also touches upon ethical considerations (GS‑4) regarding equitable treatment for vulnerable populations.</p> <h3>Way Forward</h3> <p>To translate the petition into concrete action, the following steps are recommended:</p> <ul> <li>The Ministry should expedite the generic drug approval process under existing fast‑track mechanisms.</li> <li>State health departments need to align their procurement strategies with the central <strong>NPRD</strong> guidelines to avoid regional disparities.</li> <li>Awareness campaigns must be launched to educate patients and caregivers about the availability and proper use of <strong>Risdiplam</strong>.</li> <li>Periodic monitoring of treatment outcomes should be instituted to assess the impact of broader access on disease progression.</li> </ul> <p>Timely implementation will not only alleviate the suffering of SMA patients but also set a precedent for handling other rare diseases under the national policy framework.</p>

Quick Reference

Key Insight

SMA patients push for generic Risdiplam under NPRD, spotlighting rare‑disease drug access in India.

Key Facts

SMA (Spinal Muscular Atrophy) is a rare genetic neuromuscular disorder requiring early treatment.
Risdiplam is the only disease‑modifying oral drug for SMA; the branded version costs > ₹2 lakhs per patient per year.
Patients and parents petitioned PM Narendra Modi in 2026 for nationwide generic Risdiplam under NPRD.
The National Policy for Rare Diseases (NPRD) (launched 2021) mandates fast‑track approval and equitable access to orphan medicines.
NPRD empowers the Ministry of Health to negotiate drug pricing, import orphan drugs, and coordinate state procurement.

Background

The appeal highlights the intersection of health policy, intellectual‑property law, and federal governance. It tests the effectiveness of NPRD in translating central directives into affordable treatment for rare disease patients across states.

Mains Angle

GS‑3 (Health) – Examine how central policies like NPRD can be operationalised to ensure affordable access to orphan drugs, and the role of advocacy in prompting executive action.

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Background & Context

Mains Answer Angle

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Practice Questions

Prelims MCQ

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Mains Essay

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Key Insight

Key Facts

Background

Mains Angle

SMA Patients & Parents Appeal to PM Modi for Nationwide Generic Risdiplam Access under NPRD

Overview

Key Facts

Background & Context

Mains Answer Angle

Full Article

Analysis

Practice Questions

Prelims MCQ

Mains Short Answer

Mains Essay

Quick Reference

Key Insight

Key Facts

Background

Mains Angle