National Policy for Rare Diseases 2021
The National Policy for Rare Diseases 2021 which is declared on 30th March 2021 is a new comprehensive policy of India for prevention and management of rare diseases.
Ministry of Health & Family Welfare is the implementing authority of National Policy for Rare Diseases 2021
What is a Rare Disease?
- Rare diseases don’t have a universal or standard definition.
- A disease that occurs infrequently or rarely in population is generally considered as a rare disease, and it has been defined by different countries in terms of its prevalence – that is either in absolute terms or in terms of prevalence per 10,000 population.
- Any country defines a rare disease in the context of its own population, resources and health care system.
- According to WHO– rare disease or disorder have a prevalence of 1 or less, per 1000 population.
The Table below shows how different countries in world defines Rare diseases depending on their prevalence in population.
Challenges of Rare Diseases in India
- The field of rare diseases is very big, complex and heterogeneous. Prevention, treatment and management of rare diseases has multiple challenges associated with them.
- One of the biggest challenge is Early diagnosis of rare diseases owing to a variety of factors that include
- lack of awareness among primary care physicians,
- lack of adequate screening as well as diagnostic facilities .
- Another big challenge is in the research and development for the majority of rare diseases as less is known about the pathophysiology or the history of these diseases particularly in the Indian context.
- Rare diseases patients pool is very small so it is difficult to research upon and it often results in inadequate clinical experience.
- Prohibitively expensive cost of treatment of rare diseases is also a big challenge.
- Availability and accessibility to medicines is important to reduce morbidity and mortality caused by rare diseases.
Institutional demands for National Policy for Rare Diseases 2021
There has been some decent progress in recent years in context of rare diseases, but there is also a need to augment effective and safe treatment for rare diseases. Various High Courts and the Supreme Court of India have also expressed concern regarding lack of a national policy for rare diseases.
Key points of National Policy for Rare Diseases 2021
- Policy divides Rare diseases into 3 Groups and Specifies action needed for each group:
- Group 1: Disorders amenable to one-time curative treatment Eg: Osteopetrosis, Severe Combined Immunodeficiency (SCID), Fanconi Anemia
- 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. Eg: Galactosemia, Phenylketonuria (PKU), Glucose galactose malabsorbtion.
- 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 Eg: Gaucher Disease, Hurler Syndrome, Hunter syndrome.
- The Policy aims to lower the high cost of treatment for rare diseases by increasing focus on indigenous research with the help of a National Consortium which will be set under Department of Health Research, Ministry of Health & Family Welfare.
- By increasing focus of research and development and by local production of medicines the cost of treatment for rare diseases will be lowered down.
- The policy talks about creation of a national hospital based registry of rare diseases so that adequate data is available for definition of rare diseases and for R&D related to rare diseases within the India.
- Focus will be increased on early screening and prevention through primary and secondary health care infrastructure -such as
- Health and Wellness Centres
- District Early Intervention Centres (DEICs)
- through counselling for the high-risk parents.
- Nidan Kendras set up by Department of Biotechnology will also support in screening
- The Indian tertiary health care system will also be strengthened for prevention and treatment of rare diseases by designating 8 health facilities as Centre of Excellence across India and these CoEs will also be provided with a one-time financial support of upto Rs 5 crores for upgradation of diagnostics facilities for rare diseases.
- Also there will be a Financial support up to Rs. 20 lakhs under the Umbrella Scheme of Rastriya Arogya Nidhi for treatment, of those rare diseases that require a one-time treatment (diseases listed under Group 1 in the rare disease policy).
- Not only BPL families, but the benefit will be extended to approx. 40% of the population, who are eligible under Pradhan Mantri Jan Arogya Yojana.
- Policy also envisages a crowd funding mechanism in which individuals and corporates will be encouraged to provide financial support through a robust IT platform for treatment of rare diseases. Funds collected from crowd funding will be utilized by Centres of Excellence for treatment of all three categories of rare diseases
Official Policy Document: Here
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