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REGISTRY OF RARE DISEASES

Why in news?

The Union Ministry of Health and Family Welfare published a national policy for the treatment of 450 ‘rare diseases’.

Overview of the Registry of rare Diseases policy:

  • Among other measures, the policy intends to kickstart a registry of rare diseases, which will be maintained by the Indian Council of Medical Research (ICMR).
  • According to the policy, rare diseases include genetic diseases, rare cancers, infectious tropical diseases, and degenerative diseases.
  • Under the policy, there are three categories of rare diseases — requiring one-time curative treatment, diseases that require long-term treatment but where the cost is low, and those needing long-term treatments with high cost. Some of the diseases in the first category include osteopetrosis and immune deficiency disorders, among others.
  • As per the policy, the assistance of Rs 15 lakh will be provided to patients suffering from rare diseases that require a one-time curative treatment under the Rashtriya Arogya Nidhi scheme. The treatment will be limited to the beneficiaries of Pradhan Mantri Jan Arogya Yojana.

Background:

The policy was created on the direction of the Delhi High Court to the Ministry of Health and Family Welfare. This was in response to writ petitions for free treatment of such diseases, due to their “prohibitively” high cost of treatment. Hence, a policy was deemed necessary to devise a “multipronged” and “multisectoral” approach to build India’s capacity for tackling such ailments, including by gathering epidemiological data, arriving at a definition and estimating the cost of such diseases.

What is a rare disease?

A rare disease, also referred to as an orphan disease, is any disease that affects a small percentage of the population.

  • Most rare diseases are genetic, and are present throughout a person’s entire life, even if symptoms do not immediately appear.

Characteristics:

  • Rare diseases are characterised by a wide diversity of symptoms and signs that vary not only from disease to disease but also from patient to patient suffering from the same disease. Relatively common symptoms can hide underlying rare diseases, leading to misdiagnosis.
  • The most common rare diseases recorded in India are Haemophilia, Thalassemia, sickle-cell anaemia and primary immuno deficiency in children, auto-immune diseases, Lysosomal storage disorders such as Pompe disease, Hirschsprung disease, Gaucher’s disease, Cystic Fibrosis, Hemangiomas and certain forms of muscular dystrophies.

Definition:

While there is no universally accepted definition of rare diseases, countries typically arrive at their own descriptions, taking into consideration disease prevalence, its severity and the existence of alternative therapeutic options.

In the US, for instance, a rare disease is defined as a condition that affects fewer than 200,000 people.

The same definition is used by the National Organisation for Rare Disorders (NORD).

Concerns and challenges:

  • Rare diseases pose a significant challenge to health care systems because of the difficulty in collecting epidemiological data, which in turn impedes the process of arriving at a disease burden, calculating cost estimations and making correct and timely diagnoses, among other problems.
  • Many cases of rare diseases may be serious, chronic and life-threatening. In some cases, the affected individuals, mostly children, may also suffer from some form of a handicap.
  • As per the 2017 report, over 50 per cent of new cases are reported in children and these diseases are responsible for 35 per cent of deaths in those below the age of one, 10 per cent of deaths between the ages of one and five, and 12 per cent between five and 15.

Need of nationwide policy:

  1. State has responsibility for providing affordable, accessible and reliable health-care services to every citizen. In fact constitution also mentions importance of health-care services under articles like 21, 38 and 47 and thus state cannot evade this responsibility under the pretext of non-justifiability of articles.
  1. Given the low volumes at which the drugs needed to treat such diseases would be consumed, pharmaceutical companies have little commercial incentive to produce them. Thus, a nationwide policy on orphan drugs could incentivize these players.
  2. Even if pharmaceutical companies are incentivized to develop drugs to treat rare diseases, pharmaceutical companies remain beholden to the laws of economics and, given the low demand for orphan drugs, price these drugs as high as they choose to. Hence there has to be regulation of the government in restricting the exorbitant prices of the drugs.
  3. Although proportion of rare diseases is much less than the other diseases, it does not reduce the importance of the life of person affected by rare diseases. Thus national policy would remove this adverse distinction and would make government committed equally to all people.
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September 2022
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