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Draft Policy On Rare Diseases Notified GS: 2 "EMPOWER IAS"

In news:

  • After being directed by the Madras High Court, the government has notified a draft policy on rare diseases.

 

More from the news:

  • The Centre first prepared such a policy in 2017 and appointed a committee in 2018 to review it.

 

What is a rare disease?

  • According to the World Health Organisation rare disease as an often debilitating lifelong disease or disorder with a prevalence of 1 or less per 1,000 population. 
  • According to the Indian Council of Medical Research (ICMR), it is one in 2,500 people or less.

 

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.

 

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.

 

Prevalence of Rare Diseases in India

  • Globally 6% to 8% of population being affected by rare diseases, to India, we have between 72 to 96 million people affected by rare diseases in the country, which is a significant number.
  • However, this is at best a general estimate and India will need to arrive at its own estimate and definition of rare diseases, derived chiefly from prevalence data, which is currently lacking.
  • So far only about 450 rare diseases have been recorded in India from tertiary care hospitals.
  • The most common rare diseases include Haemophilia, Thalassemia, Sicklecell 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 o f muscular dystrophies.

 

Overview of the 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.

 

The issue with the National Policy for Treatment of Rare Diseases 2017

  • No differentiation between one-time curative treatment disorders such as Hematopoietic Stem Cell Transplantation (HSCT) and disorders that require organ transplantation.
  • In 2017 the Public Interest Litigation (PIL) petition was filed by the Lysosomal Storage Disorders Support Society of India for placing both types of diseases into one group.
  • Madras High Court, on January 6, to consider the issue of providing medical care to those suffering from the rare Lysosomal Storage Disorders (LSD) as a “national emergency” and impressed upon the Centre as well as the State government to deliberate further.
  • The judges also recommended taking note of Delhi HC judgments before formulating and finalizing the policy.

 

Draft National Policy for Rare Diseases 2020

  • The draft policy provides for lowering the incidence of rare diseases based on an integrated preventive strategy.
  • It aims to provide access to affordable health care to patients of rare diseases that are amenable to a one-time treatment.
  • The focus of the draft policy is on the prevention of rare diseases as a priority for all the three groups of rare diseases identified by experts. 
  • For the purpose of the policy the term rare diseases are categorized into 3 groups:
    • Disorders amenable to one time curative treatment.
      1. Disorders amenable to organ transplantation
      2. Disorders amenable to treatment with Hematopoietic Stem Cell Transplantation (HSCT)
    • Diseases requiring long term / lifelong treatment having a relatively lower cost of treatment and benefit has been documented in the literature and annual or more frequent surveillance is required.
      1. Disorders managed with special dietary formulae or Food for special medical purposes (FSMP)
      2. Disorders that are amenable to other forms of therapy (hormone/ specific drugs)
    • Diseases for which definitive treatment is available but challenges are to make an optimal patient selection for the benefit, very high cost, and lifelong therapy.
  • Financial supportA maximum of ₹15 lakh could be provided to each patient under the umbrella scheme of Rashtriya Arogya Nidhi and the beneficiaries would not be limited to families below the poverty line. 
    • Also, it would be extended to 40% of the population in accordance with the norms of the Pradhan Mantri Jan Arogya Yojana.
  • The draft policy provides for lowering the incidence of rare diseases based on an integrated preventive strategy encompassing awareness generation and screening programs and, within the constraints on resources and competing for health care priorities.

Criticism of the Policy

  • Rare disease advocacy and support groups have called out the policy for several discrepancies including a lack of funding to support families and individuals with a rare disease.
  • It identifies eight hospitals across India as ‘centres of excellence’ and leaves it up to them to set up crowdfunding initiatives for patients that require it, or for families to themselves look at crowdfunding options for treatment.
  • The problem is that the policy caps resources for different diseases. While treatment for one particular disease might amount to Rs 10 lakh, another disease may require Rs 1 crore to cover the cost of medications, which are only bound to increase over time.
  • The policy has been criticised for acting more like a set of guidelines to understand the disease, rather than specifying actions which must be taken by the government to offer support to those affected.
  • Public health and hospitals being a State subject, the Central Government shall encourage and support the States in their endeavour towards screening and prevention of rare diseases. However, while screening has been emphasised as a preventive measure, there is no mention of how and when screening will take place, nor has it been stated how screening will be implemented.

 

Umbrella Scheme of Rashtriya Arogya Nidhi (RAN)

  • To provide financial assistance to patients, living below the poverty line and who are suffering from major life-threatening diseases, to receive medical treatment at any of the super-specialty Hospitals/Institutes or other Government hospitals. 
  • It was set up in 1997. It is registered under the Societies Registration Act, 1860, as a Society.
  • Under the Umbrella Scheme of RAN, funds are not released to States/UTs, but to the hospitals where eligible patients receive treatment.
  • Financial assistance up to Rs.15 lakh is provided for eligible patients in the form of ‘one-time grant’ to the Medical Superintendent of the Government hospital where the treatment is being received. The scheme has three components, namely:
    • Rashtriya Arogya Nidhi (RAN) – to provide financial assistance to patients suffering from life-threatening diseases other than Cancer.
    • Health Minister’s Cancer Patients Fund (HMCPF) - to provide financial assistance to patients suffering from Cancer.
    • Scheme for financial assistance for patients suffering from specified rare diseases.

 

Need of nationwide policy:

  • 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.
  • 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 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.

 

Source)

https://www.livemint.com/news/india/govt-may-treat-rare-disease-patients-at-one-time-treatment-cost-up-to-rs-15-lakh-11578941088572.html