Govt Treatment Centers In India
Centres of Excellence (COE)
The following are Centers of Excellence for Rare Diseases. Patients and Families are requested to visit the nearest center and register to avail treatment:
- All India Institute of Medical Sciences, New Delhi
- Maulana Azad Medical College, New Delhi
- Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow
- Post Graduate Institute of Medical Education and Research, Chandigarh
- Centre for DNA Fingerprinting & Diagnostics, Hyderabad
- King Edward Medical Hospital, Mumbai
- Institute of Post-Graduate Medical Education and Research, Kolkata
- Center for Human Genetics (CHG) with Indira Gandhi Hospital, Bengalur
- All India Institute of Medical Sciences, Jodhpur
- Institute Of Child Health and Hospital for Children Egmore, Chennai
- Government Medical College, Thiruvananthapuram, Kerala
Activities of the COEs
- Treatment of rare diseases
- Screening – Antenatal, neonatal (specified disorders), and high-risk
screening (antenatal & in newborns and children) - Diagnostics – Cytogenetic, molecular, metabolic
- Prevention via prenatal screening & diagnosis, education & training at
all levels - Research on low-cost diagnostics & therapeutics
Nidan Kendras
Nidan Kendras have been set up by the Department of Biotechnology (DBT) under
the Unique Methods of Management and Treatment of Inherited Disorders (UMMID)
project for genetic testing and counseling services. These centers offer
screening, genetic testing, and counseling for rare diseases. If a Nidan Kendra
has facilities for treatment, it may do so under the guidance of a COE.
List of Nidan Kendras
- Lady Hardinge Medical College (LHMC), Delhi
- Nizam’s Institute of Medical Sciences (NIMS), Hyderabad, Telangana
- All India Institute of Medical Sciences (AIIMS), Jodhpur
- Army Hospital Research & Referral, Delhi
- Nil Ratan Sircar (NRS) Medical College and Hospital, Kolkata
Government of India Support in Treatment
The following initiatives shall be taken for patients of rare diseases:
- Financial support up to Rs. 20 lakh under the
Umbrella Scheme of Rashtriya Arogaya Nidhi (RAN)
shall be provided by the Central Government for treatment of those
rare diseases that require a one-time treatment (Group 1). Beneficiaries
are not limited to BPL families but extend to ~40% of the population
(as per PM Jan Arogya Yojana norms), for treatment in Government
tertiary hospitals only.(Refer to the NPRD2021 Document or list of diseases below.)
- State Governments can consider supporting patients of rare diseases
that can be managed with special diets, hormonal supplements, or other
relatively low-cost interventions (Group 2).(Refer to the NPRD2021 Document or list of diseases below.)
- The Government aims to create an alternate funding mechanism through a
digital platform for voluntary individual and corporate donors to
contribute toward the treatment costs of patients with rare diseases.The notified hospitals will share patient information, disease details,
estimated treatment costs, and bank account info for donations or
contributions through an online system.
Groups of Disorders as per Policy
Group 1
Disorders amenable to one-time curative treatment:
- Disorders amenable to treatment with Hematopoietic Stem Cell
Transplantation (HSCT):- Certain Lysosomal Storage Disorders (LSDs) for which Enzyme Replacement Therapy (ERT) is unavailable
- Severe Mucopolysaccharoidosis (MPS) Type I in the first 2 years of life
- Adrenoleukodystrophy (early stages, before severe neurological signs)
- Immune deficiency disorders like SCID, CGD, Wiskott-Aldrich Syndrome, etc.
- Osteopetrosis
- Fanconi Anemia
- Disorders amenable to organ transplantation:
- Liver Transplantation – Metabolic Liver Diseases:
- Tyrosinemia
- Glycogen Storage Disorders (GSD) I, III, IV (due to poor metabolic control,
multiple liver adenomas, high risk for Hepatocellular carcinoma,
or significant liver dysfunction) - Maple Syrup Urine Disease (MSUD)
- Urea cycle disorders
- Organic acidemias
- Renal Transplantation:
- Fabry disease
- Autosomal Recessive Polycystic Kidney Disease (ARPKD)
- Autosomal Dominant Polycystic Kidney Disease (ADPKD)
- Combined liver and kidney transplants for certain cases (e.g., Methyl Malonic Aciduria)
- Liver Transplantation – Metabolic Liver Diseases:
Group 2
Diseases requiring long-term or lifelong treatment with relatively
lower costs, where benefits are well-documented. Annual or frequent
surveillance is required:
- Disorders managed with special dietary formulae or Food for
Special Medical Purposes (FSMP):- Phenylketonuria (PKU)
- Non-PKU hyperphenylalaninemia
- Maple Syrup Urine Disease (MSUD)
- Tyrosinemia type 1 and 2
- Homocystinuria
- Urea Cycle Enzyme defects
- Glutaric Aciduria types 1 & 2
- Methyl Malonic Acidemia
- Propionic Acidemia
- Isovaleric Acidemia
- Leucine sensitive hypoglycemia
- Galactosemia
- Glucose-galactose malabsorption
- Severe food protein allergy
- Disorders amenable to other forms of therapy
(hormone/specific drugs):- NTBC for Tyrosinemia Type 1
- Osteogenesis Imperfecta – Bisphosphonates therapy
- Growth Hormone therapy for GH deficiency, Prader-Willi Syndrome,
Turner Syndrome, Noonan Syndrome - Cystic Fibrosis – Pancreatic enzyme supplementation
- Primary Immunodeficiency disorders – IVIG and subcutaneous therapy
- Sodium Benzoate, Arginine, Citrulline, Phenylacetate (for Urea Cycle
disorders), Carbaglu, Megavitamin therapy (Organic acidemias,
mitochondrial disorders) - Hemin for Acute Intermittent Porphyria, High dose Hydroxocobalamin, etc.
- Large neutral amino acids, mitochondrial cocktail therapy, Sapropterin, etc.
Group 3
Diseases where definitive treatment is available but poses challenges
in patient selection, cost, and lifelong therapy.
Group 3a
Sufficient evidence for good long-term outcomes:
- Gaucher Disease (Type I & III without severe neurological impairment)
- MPS Type I (Hurler Syndrome, attenuated forms)
- MPS II (Hunter Syndrome, attenuated form)
- Pompe Disease (infantile & late-onset if diagnosed early)
- Fabry Disease (before significant end-organ damage)
- MPS IVA (before complications)
- MPS VI (before complications)
- DNAase for Cystic Fibrosis
Group 3b
Very high treatment cost; long-term follow-up data is limited or
involves small patient numbers:
- Cystic Fibrosis (Potentiators)
- Duchenne Muscular Dystrophy (Antisense oligonucleotides, PTC)
- Spinal Muscular Atrophy (Antisense oligonucleotides: IV, oral & gene therapy)
- Wolman Disease
- Hypophosphatasia
- Neuronal ceroid lipofuscinosis
Disclaimer: This information is based on the NPRD 2021.
For full details, please refer to the complete NPRD 2021 document.