Context: Recently, Union Health Minister approved the ‘National Policy for Rare Diseases 2021’ which aims to lower the high cost of treatment for rare diseases, with increased focus on indigenous research and local production of medicines.
About Rare Diseases

• It is a disease that affects a smaller percentage of the population compared to other diseases. Its prevalence is very low.
• The World Health Organisation defines a rare disease as an often debilitating lifelong disease or disorder condition with a prevalence of 1 or less, per 1000 population. 

Note: It is estimated that there are about 7000 rare diseases. Most of the rare diseases (about 80%) are genetic and hence affects a large number of children.

• Some rare diseases are not inherited such as some rare cancers, some autoimmune diseases, infectious tropical diseases, etc.
• Unfortunately, there are no treatments for many rare diseases.

Provisions of the National Rare Disease Policy 2021

• It aims to lower the high cost of treatment for rare diseases with increased focus on indigenous research with the help of a national consortium to be set up with the Department of Health Research, Ministry of Health and Family Welfare.
• It also talks about the creation of national hospital based registry of rare diseases so that adequate data is available for definition of such diseases and for research and development related to rare diseases within the country.
• It also focuses on early screening and prevention through primary and secondary health care infrastructure such as Health and Wellness Centres and District Early Intervention Centres (DEICs) and through counselling for high-risk patients.
• It aims to strengthen tertiary health care facilities for prevention and treatment of rare diseases through designating eight health facilities as ‘Centres of Excellence’ and these will also be provided one-time financial support of up to Rs 5 crore for upgradation of diagnostics facilities.
• Patients of rare diseases will be eligible for a one-time treatment under the Ayushman Bharat Pradhan Mantri Jan Arogya Yojana (AB-PMJAY).
• The government will provide Financial support of up to Rs. 20 lakh under the Umbrella Scheme of Rashtriya Arogya Nidhi for treatment of those rare diseases listed under Group 1.

Note: “Beneficiaries for such financial assistance will not be limited to BPL families, but the benefit will be extended to about 40 per cent of the population who are eligible under the Pradhan Mantri Jan Arogya Yojana".
The policy categorizes rare diseases into three groups

• Group 1: Disorders amenable to one-time curative treatment;
• Group 2: Diseases requiring long term or lifelong treatment; and
• Group 3: Diseases for which definitive treatment is available, but challenges are to make an optimal patient selection for benefit.

Further, for group 2, the State Governments can consider supporting specific patients. It includes a rare disease that can be managed with special diets or hormonal supplements or other relatively low-cost interventions (Diseases listed under Group 2).

• Voluntary Crowdfunding: The government has said that it will assist in voluntary crowd-funding for the treatment of Group 3. It is because it will be difficult to fully finance the treatment of high-cost rare diseases of Group 3.

Objections to the Policy

• The policy offers no support to patients awaiting treatment since the earlier National Policy for Treatment of Rare Diseases 2017 was kept on hold.
• The policy has left patients with Group 3 rare diseases to fend for themselves. It has absolutely no consideration for Group 3 patients who require lifelong treatment support.

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.

Additional Information
Rashtriya Arogya Nidhi scheme: It provides financial assistance to patients living below the poverty line and who are suffering from major life-threatening diseases, to receive medical treatment.