Syllabus
- Mains – GS 2 (Governance)
Context: The Union Government has given full exemption from basic customs duty on all drugs and food for special medical purposes imported for personal use for treatment of all Rare Diseases listed under the National Policy for Rare Diseases 2021.
- The exemption has been granted by the Central Board of Indirect Taxes and Customs (CBIC) by substituting “Drugs, Medicines or Food for Special Medical Purposes (FSMP)” instead of “drugs or medicines”.

Source: The Hindu
About Rare Diseases:
- Rare diseases (also called “Orphan” diseases) are broadly defined as diseases that infrequently occur in a population and three markers are used (the total number of people with the disease, its prevalence, and the availability/non-availability of treatment options).
- The World Health Organisation (WHO) defines a rare disease as having a frequency of less than 6.5-10 per 10,000 people.
- Example: Lysosomal Storage Disorders (LSD), Pompe disease, cystic fibrosis, muscular dystrophy, spina bifida, haemophilia etc.
- About 95% rare diseases have no approved treatment and less than 1 in 10 patients receive disease-specific treatment.
National Policy for Rare Diseases, 2021
- It categories ‘rare disease’ into three groups:
- Group 1: Disorders amenable to one-time curative treatment.
- Group-2: Diseases requiring long term/lifelong treatment having relatively lower cost of treatment and benefit has been documented in literature and annual or more frequent surveillance is required.
- Group 3:- Diseases for which definitive treatment is available but challenges are to make optimal patient selection for benefit, very high cost and lifelong therapy.
- The government would notify selected Centres of Excellence at premier government hospitals for comprehensive management of rare diseases.
- Provision for financial support of up to Rs. 50 lakhs to the patients suffering from any category of the Rare Diseases and for treatment in any of the Centre of Excellence (CoE) mentioned in NPRD-2021, outside the Umbrella Scheme of Rashtriya Arogaya Nidhi.
- In order to receive financial assistance for treatment of rare disease, the patient of the nearby area may approach the nearest Centre of Excellence to get him assessed and avail the benefits.
- Eight (08) Centres of Excellence (CoEs) have been identified for diagnosis, prevention and treatment of rare diseases.
- Five Nidan Kendras have been set up for genetic testing and counselling services.
Preventive measures adopted by the policy
The preventive measures adopted by the National Policy for Rare Diseases are as follows:
- Preventing birth of an affected child.
- Prenatal Screening
- Prenatal Screening by invasive testing
- New born screening
- Early postnatal diagnosis and treatment
Constitutional Provisions in the context of the new policy for rare diseases
- Article 38 says that the state will secure a social order for the promotion of welfare of the people.
- Providing affordable healthcare is one of the ways to promote welfare.
- Article 41 imposes duty on state to provide public assistance in cases of unemployment, old age, sickness and disablement etc.
- Article 47 makes it the duty of the State to improve public health, securing of justice, human condition of works, extension of sickness, old age, disablement and maternity benefits and also contemplated.
Challenges to be addressed by the policy
The National Policy for Rare Diseases, 2021, will address the following challenges:
- The research and development for majority of rare diseases is less as very little is known about pathophysiology of these diseases.
- The patient pool of rare diseases is less.
- Lack of availability and accessibility of medicines to rare diseases.
- Cost of treatment of rare disease is highly expensive.
Way Forward:
There is an immediate need to create awareness amongst the general public, patients & their families and doctors, training of doctors for early and accurate diagnosis. As resources are limited and have multiple uses, the policy makers have to make a choice of prioritizing certain set of interventions over others. There should be policy measures for supporting R&D and drug development for rare diseases.
Source: The Hindu