National Policy for Rare Diseases 2021

Why in news?

The Union Health and Family Welfare ministry recently approved the National Policy for Rare Diseases 2021.

What are rare diseases?

Rare diseases are broadly defined as diseases that infrequently occur in a population; three markers are used:
1. the total number of people with the disease
2. its prevalence
3. the availability/non-availability of treatment options
WHO defines rare disease as often debilitating lifelong disease or disorder with a prevalence of 1 or less, per 1000 population.
However, different countries have their own definitions.
It is defined in a way to suit their specific requirements and in context of their own population, health care system and resources.
As per an estimate, there are 7,000 known rare diseases with an estimated 300 million patients in the world.
- Of this, 70 million are in India.
They include inherited cancers, autoimmune disorders, congenital malformations, Hirschsprung’s disease, Gaucher disease, cystic fibrosis, muscular dystrophies and Lysosomal Storage Disorders (LSDs).

What are the key provisions in the policy?

Objective - The policy intends at lowering the cost of treatment of rare diseases (diseases listed under Group 1 in the rare disease policy).
It also aims at increasing the focus on indigenous research and local production of medicines.
Support - Those who are suffering from rare diseases that require one-time treatment will have the financial support of up to Rs 20 lakh under the umbrella scheme of Rashtriya Arogya Nidhi.
Financial assistance will not be limited to just the BPL families.
Around 40% of the population covered under the Pradhan Mantri Jan Arogya Yojana will benefit from the policy.
Funding - The policy will make use of a crowdfunding mechanism to cover the cost of treatment of rare diseases.
As part of it, corporates and individuals will be encouraged to extend financial support through a robust IT platform.
Registry - A national hospital-based registry of rare diseases will be created.
This is to ensure that adequate data and comprehensive definitions of such diseases are available for those interested in research and development.
Detection - The policy aims to screen and detect rare diseases at early stages, which will in turn help in their prevention.
It aims to achieve this through the help of Health and Wellness Centres, District Early Intervention Centres and counselling.

What is the significance?

Rare diseases are difficult to research upon.
Also, availability and accessibility to medicines are important in the process of treatment.
Fund support from government is vital for continual treatment of those with rare diseases.
The Supreme Court and various high courts had expressed concern about the lack of a national policy for rare diseases.
The policy thus aims to help the nation overcome these fundamental challenges.

What are the shortcomings?

Much of the effort in the rare diseases sector, from the medical side, has been to evolve formal definitions.
This is because it would support the development of and commercialisation of drugs for treatment, and improve funding for research on rare diseases.
The Policy meets much of the demands, but falls short of delivering the complete mandate.
As per the Policy, diseases such as LSD for which definitive treatment is available, but costs are prohibitive, have been categorised as Group 3.
However, no funding has been allocated for the immediate and lifelong treatment needs, for therapies already approved by the Drugs Controller General of India.

What can be done?

Experts point out that the costs to help already-diagnosed patients might be in the range of Rs. 80-100 crore annually.
To note, the Centre has worked out cost-sharing agreements with Kerala, Tamil Nadu and Karnataka.
If it can extend this with other States too, its share of the annual costs will be halved.
The Centre can, however, still set aside a substantial corpus to fund life-saving treatments, even as it rolls out the policy.

Source: The Hindu, Hindustan Times