**Executive Summary**
This document is the answer provided by the Minister of State in the Ministry of Health and Family Welfare to Unstarred Question No. 2381 regarding the treatment of Spinal Muscular Atrophy (SMA), which was to be answered on February 13th, 2026. The response provides information on government initiatives related to rare diseases, particularly SMA, under the National Policy for Rare Diseases (NPRD), 2021. It addresses financial support, treatment accessibility, and preventive measures.
**Key Points / Main Content**
* **National Policy for Rare Diseases (NPRD):**
* The government has framed the NPRD, 2021, to mitigate challenges posed by rare disease patients.
* More than 1125 Spinal Muscular Atrophy (SMA) patients have been registered since 2021 under NPRD, including in Andhra Pradesh.
* As of the date of writing, 63 rare diseases are identified under NPRD. SMA is categorized as Group-III rare disease with prohibitive treatment costs.
* **Financial Assistance:**
* NPRD facilitates financial assistance of up to Rs. 50 lakhs for treatment of identified rare diseases through designated Centers of Excellence (CoEs).
* Treatment and fund allocation is decided by the Rare Disease Committee in each CoE on a case-to-case basis, irrespective of income criteria.
* Exemption from Basic Customs Duty (BCD) and Integrated Goods & Services Tax (IGST) is extended to rare diseases’ drugs, medicines and Food for Special Medical Purposes (FSMP) subject to extant rules.
* A "Digital Portal for Crowd Funding & Voluntary Donations for Patients of Rare Diseases" has been launched.
* **Preventive Measures:**
* The Government encourages and supports State Governments in the implementation of a targeted preventive strategy through the CoEs.
* Under NPRD, CoEs are mandated to perform antenatal screening, neonatal screening (specified disorders), high-risk screening (antenatal, newborns, and children), etc., for prevention and control.
* CoEs are given one-time financial support of up to Rs. 5 crore for equipment procurement for strengthening patient care services for screening, diagnosis, and prevention (prenatal diagnosis) of rare diseases.
* **Legal Matters:**
* The High Court of Delhi issued certain directions regarding the adequacy of the Rs. 50 lakh cap, which was stayed by the Supreme Court. The Court directed the Government to comply with the extant terms and conditions of NPRD. The matter is sub judice in the Apex Court.
**Impact Analysis**
**Patients with Rare Diseases, Particularly Spinal Muscular Atrophy (SMA)**
* **Impact:** Affected by policies determining access to treatment, financial assistance, screening, and preventive measures.
* **Action Required:** To avail of the benefits and treatments, patients should consult with CoEs.
**State Governments**
* **Impact:** State Governments are primarily responsible for providing healthcare services.
* **Action Required:** To implement targeted preventive strategies through the CoEs and to support national policies related to rare diseases.
**Centers of Excellence (CoEs)**
* **Impact:** Designated for treatment, diagnostics, and preventive care for rare diseases.
* **Action Required:** To implement screening programs, provide treatment based on NPRD guidelines, and allocate funds as per the Rare Disease Committee decisions.
**Supreme Court**
* **Impact:** The Supreme Court is in the process of considering this topic, which may alter the current legal standards surrounding rare diseases.
* **Action Required:** N/A
Key Entities Referenced
National Policy for Rare Diseases (NPRD): A policy framed by the Government of India to mitigate challenges posed by rare disease patients, facilitating financial assistance and establishing Centers of Excellence (CoEs).
Spinal Muscular Atrophy (SMA): A rare genetic disease requiring expensive gene therapy treatment, which is a focal point of the policy discussion.
Centers of Excellence (CoEs): Designated centers facilitating treatment and offering support for rare diseases under NPRD guidelines.
Ministry of Health and Family Welfare: The primary ministry responsible for the policy and the answer provided to the parliamentary question.
Andhra Pradesh: Specifically mentioned state relevant to the discussion on SMA cases.
GOVERNMENTOFINDIA
MINISTRYOFHEALTH AND FAMILYWELFARE
DEPARTMENTOFHEALTH AND FAMILYWELFARE
LOK SABHA
UNSTARRED QUESTION No. 2381
TO BE ANSWERED ON13thFEBRUARY2026
TREATMENT OF SPINALMUSCULAR ATROPHY
2381. DR. BYREDDY SHABARI:
Will the Minister of HEALTH AND FAMILYWELFARE be pleased to state:
(a) the number of Spinal Muscular Atrophy (SMA) cases reported in the country during
the last five years, State-wise, including Andhra Pradesh;
(b) whether the Government acknowledges that infants belonging to economically weaker
families are unable to access SMA gene therapy due to its cost of about 16 crore per
injection and if so, the details thereof;
(c) whether the National Policy for Rare Diseases adequately covers ultra-expensive life-
saving treatments required for infants suffering from SMAand if so, the details thereof;
(d) whether the Government proposes to extend financial support, special approvals or
negotiated pricing mechanisms for SMAgene therapy and if so, the details thereof; and
(e) the steps taken or proposed to be taken by the Government to strengthen early
diagnosis, newborn screening and genetic counselling for SMA across the country,
including Andhra Pradesh?
ANSWER
THE MINISTER OFSTATE IN THE MINISTRYOFHEALTH AND FAMILY
WELFARE
(SHRIPRATAPRAO JADHAV)
(a) Health beinga State subject, the primary responsibility of providinghealthcare services is
that of the State Governments. However, to mitigate the challenges posed by rare disease
patients in India, the Government has framed the National Policy for Rare Diseases (NPRD),
2021. Under NPRD, more than 1125 Spinal Muscular Atrophy (SMA) patients have been
registered since 2021from across the country, includingAndhra Pradesh.
(b) As on date, 63 rare disease are identified under NPRD. Spinal Muscular Atrophy is
categorized as Group-III rare disease with a prohibitive treatment cost. Some of the steps
takenbythe Government tosupport rare disease patientsare asfollows:
NPRD facilitates financial assistance of up to Rs. 50 lakhs for treatment of identified
rare diseases through the designated Centers of Excellence (CoEs). As per NPRD
guidelines, treatment and fund allocation to each patient is decided by the dedicated
Rare DiseaseCommittee ineachCoEafterclinicalexaminationoncase-to-casebasis,
irrespective of anyincome criteria. Exemption from Basic Customs Duty (BCD) and Integrated Goods & Services Tax
(IGST) is extended to rare diseases’ drugs, medicines and Food for Special Medical
Purposes(FSMP),subject to the extantrules.
For financial assistance of high-cost rare diseases, a “Digital Portal for Crowd
Funding & Voluntary Donations for Patients of Rare Diseases” has also been
launched to fill the funding gap between the Government assistance and the actual
treatmentcost.
(c) and (d) Vide Order dt. 04.10.2024 in W.P.(C) 5315/2020 & CM APPL. 19189/2020,
4237/2023, Hon’ble High Court of Delhi issued certain directions to the Government
regarding adequacy of the Rs. 50 lakh cap for high-cost treatment of some rare diseases.
However, that Order was stayed by the Hon’ble Supreme Court vide Order dt. 09.12.2024 in
Petition for Special Leave to Appeal (C) No. 28777/2024, wherein the Court directed the
Government to comply with the extant terms and conditions of NPRD. Therefore, the matter
issub judice inthe Apex Court.
(e) The Government encourages and support the State Governments in implementation of a
targeted preventive strategy through the CoEs. Some of the steps taken by the Government
are asfollows:
Under NPRD, the CoEs are mandated to perform Antenatal Screening, Neonatal
Screening (specified disorders), High-Risk Screening (Antenatal, newborns and children)
etc.asmethodsof preventionand control of rare diseases.
The CoEs are also given one-time financial support of up to Rs. 5 crore for
procurementof equipmentfor strengthening patient care services for screening, diagnosis
andprevention(prenatal diagnosis) of rare diseases.
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