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Introduction:
Union health and family welfare minister Harsh Vardhan has approved the National Policy for Rare Diseases 2021, according to a statement issued by the health ministry. The policy aims to bring down the high cost of treatment for rare diseases and was brought about after “multiple consultations with different stakeholders and experts in the area.
Policy:
- To address all these challenges, a very comprehensive National Policy for Rare Diseases 2021 has been finalized by the Ministry of H&FW after multiple consultations with different stakeholders and experts in the area.
- The Rare Diseases Policy 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 Department of Health Research, Ministry of Health & Family Welfare as convenor.
- Increased focus of research and development and local production of medicines will lower the cost of treatment for rare diseases. The policy also envisage creation of a national hospital based registry of rare diseases so that adequate data is available for definition of rare diseases and for research and development related to rare diseases within the country.
- The Policy 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 the high-risk parents.
- Screening will also be supported by Nidan Kendras set up by Department of Biotechnology. Policy also aims to strengthen tertiary health care facilities for prevention and treatment of rare diseases through designating 8 health facilities as Centre of Excellence and these CoEs will also be provided one-time financial support of up to Rs 5 crores for upgradation of diagnostics facilities.
Rare Diseases: Issues & Challenges:
- The field of rare diseases is complex and heterogeneous. The landscape of rare diseases is constantly changing, as there are new rare diseases and conditions being identified and reported regularly in medical literature.
- Apart from a few rare diseases, where significant progress has been made, the field is still at a nascent stage.
- For a long time, doctors, researchers and policy makers were unaware of rare diseases and until very recently there was no real research or public health policy concerning issues related to the field. This poses formidable challenges in development of a comprehensive policy on rare diseases.
The varying definitions of rare diseases
- 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 to suit their specific requirements and in context of their own population, health care system and resources.
- In the US, rare diseases are defined as a disease or condition that affects fewer than 200,000 patients in the country (6.4 in 10,000 people).
- EU defines rare diseases as a life-threatening or chronically debilitating condition affecting no more than 5 in 10,000 people.
- Japan identifies rare diseases as diseases with fewer than 50,000 prevalent cases (0.04%) in the country.
Challenges in treatment:
- Unavailability of treatment
Availability and access to medicines are important to reduce morbidity and mortality associated with rare diseases. Despite progress in recent years, effective or safe treatment is not available for most of the rare diseases. Hence, even when a correct diagnosis is made, there may not be an available therapy to treat the rare disease.
- Prohibitive cost of treatment
As the number of persons suffering from individual rare diseases is small, they do Snot constitute a significant market for drug manufacturers to develop and bring to market drugs for them. For this reason, rare diseases are also called ‘orphan diseases’ and drugs to treat them are called “orphan drugs”.
Data on how many people suffer from different diseases that are considered rare globally, is lacking in India. The cases identified so far have been diagnosed at tertiary hospitals. The lack of epidemiological data on incidence and prevalence of rare diseases impedes understanding of the extent of the burden of rare diseases and development of a definition.
Prevention & Control of Rare Diseases:
- The Central Government will work with the State governments to build capacity of health professionals at various levels.
- Though in the last two decades, due to advancement in technologies, understanding of the pathophysiological mechanisms of rare genetic disorders has somewhat improved, yet the treatment modalities are few and the available therapies may not lead to “cure’.
- Primary Prevention: This aims at preventing the occurrence of the disease, i.e., preventing birth of an affected child.
- Secondary prevention: This strategy focuses on avoiding the birth of affected fetus (prenatal screening and prenatal diagnosis), early detection of the disorders, appropriate medical intervention to ameliorate or minimize the manifestations (newborn screening).
- Tertiary prevention refers to provision of better care and medical rehabilitation to those rare disease patients who present at an advanced stage of the disease.
Government support:
- A provision for financial support up to Rs. 20 lakhs under the Umbrella Scheme of Rastriya Arogya Nidhi is proposed for treatment, of those rare diseases that require a one-time treatment (diseases listed under Group 1 in the rare disease policy).
- Beneficiaries for such financial assistance would not be limited to BPL families, but the benefit will be extended to about 40% of the population, who are eligible under Pradhan Mantri Jan Arogya Yojana.
- Besides, the Policy also envisages a crowd funding mechanism in which corporates and individuals will be encouraged to extend financial support through a robust IT platform for treatment of rare diseases.
- Funds so collected will be utilized by Centres of Excellence for treatment of all three categories of rare diseases as first charge and then the balance financial resources could also be used for research.
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