Rare Diseases in India and National Policy on them

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Context: Union Ministry of Health and Family Welfare published a national policy for treatment of rare diseases, listing 450 diseases as rare. The government has announced a ‘one-time financial assistance’ for Rare diseases. The standing finance committee has approved a proposal for adding a sub-component under the umbrella scheme of Rashtriya Arogya Nidhi (RAN) for provision of one-time financial assistance to those below threshold poverty line for specified rare diseases which require one-time treatment.

Relevance:
Prelims: Current events of national and international importance.
Mains: GS II-

  • Issues relating to development and management of Social Sector/Services relating to Health, Education, Human Resources.

 

Rare Diseases:
  • A rare disease is a health condition of low prevalence that affects a small number of people compared with other prevalent diseases in the general population.
  • There is no universally accepted definition of rare diseases and the definitions usually vary across different countries.
  • Though rare diseases are of low prevalence and individually rare, collectively they affect a considerable proportion of the population.
  • 80% of rare diseases are genetic in origin and hence disproportionately impact children.
  • In India, there are 56-72 million people affected by rare diseases.

Definition:

  • India does not have a definition of rare diseases because there is a lack of epidemiological data on its incidence and prevalence.
  • 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) in India.

 

Types:

  • Most rare diseases are genetic, and are present throughout a person’s entire life, even if symptoms do not immediately appear
  • In Europe, a disease or disorder is defined as rare when it affects less than 1 in 2000 citizens.
  • Rare diseases are characterized 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 anemia and primary immunodeficiency 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.
National Policy for Treatment of Rare Diseases:

Why need such a policy?

  • As per the policy, out of all rare diseases in the world, less than five percent have therapies available to treat them.
  • In India, roughly 450 rare diseases have been recorded from tertiary hospitals, of which the most common are Haemophilia, Thalassemia, Sickle-cell anemia, auto-immune diseases, Gaucher’s disease, and cystic fibrosis.
  • Three Categories of Rare Diseases:
    • diseases requiring one-time curative treatment,
    • diseases which need long-term treatment but the cost is low, and
    • diseases that require life-long treatment and the cost is high

Features of the policy:

  • While the policy has not yet put down a detailed roadmap of how rare diseases will be treated.
  • It has mentioned some measures, which include creating a patient registry for rare diseases, arriving at a definition for rare diseases that are suited to India, taking legal and other measures to control the prices of their drugs, etc.
  • It intends to kickstart a registry of rare diseases, which will be maintained by the Indian Council of Medical Research (ICMR).
  • 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.

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.

 

Need of nationwide policy:
  • The state has responsibility for providing affordable, accessible and reliable health-care services to every citizen.
  • In fact, the constitution also mentions the importance of health-care services under articles like 21, 38 and 47 and thus the state cannot evade this responsibility under the pretext of non-justifiability of articles.
  • 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.
  • 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.
  • Although the proportion of rare diseases is much less than the other diseases, it does not reduce the importance of the life of the person affected by rare diseases.
    • Thus national policy would remove this adverse distinction and would make the government committed equally to all people.
Criticism of the move:
  • Need for Continuous Treatment:
    • Most of the rare diseases for which treatment is available are progressive. They require continuous support and not just one-time assistance as an interim arrangement can never be a substitute for a policy.
    • The withdrawn Policy highlighted the measures and steps, both in the short as well as in the long term, that need to be taken to deal comprehensively with rare diseases. The policy sought to strike a balance between access to treatment with health system sustainability.
  • Pushes Families in Poverty:
    • Its impact on families is often catastrophic in terms of emotional as well as financial burden. The exorbitant cost of treatment per patient, which ranges anywhere from ₹25 lakh and ₹4 crore per year, is out of reach even for middle-class families.
  • Significant Population Impacted:
    • While there is no registry of rare disease patients in India (the policy provided for one), according to the government’s own estimates there are between 70-90 million patients.
  • Difficulty in R&D:
    • Rare diseases are difficult to research upon as the patient pool is very small and it often results in inadequate clinical experience. The policy envisaged an R&D framework that cannot be attained through one-time financial support.
  • Not covered under Health Insurance:
    • Private insurance companies treat genetic disorders as pre-existing conditions and, on that ground, exclude them from coverage. Since most rare diseases are genetic, patients are routinely denied insurance cover.

Way Forward:

  • As health is a state subject, state governments have a critical role to play in providing and organizing whatever support these patients require.
  • Karnataka is emerging as a model state in this regard with the establishment of a rare disease care center of excellence at the Indira Gandhi Institute for Child Health (IGICH) in Bengaluru. Other states should follow this initiative to provide the much-needed succor to these patients without wasting further time.

 



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