Home / NewTech / The Union Ministry of Health publishes the Rare Disease Directive. Implementation could be difficult

The Union Ministry of Health publishes the Rare Disease Directive. Implementation could be difficult

The government will provide up to Rupee 15 billion in one-time orphan treatment under its Rashtriya Arogaya Nidhi program in line with the much anticipated draft rare disease policy.

According to a draft national policy on rare diseases released on Monday, the beneficiaries would not only be limited to families below the poverty line, but would also cover 40 percent of the population living in accordance with the Ayushman Bharat-Pradhan Mantri norms and Jan Arogya Yojana are only eligible for treatment in state tertiary hospitals.

The Union Ministry of Health intends to report certain medical institutes as centers of excellence for rare diseases.

These include AIIMS, New Delhi, Maulana Azad Medical College, New Delhi, the Sanjay Gandhi Postgraduate Institute for Medical Sciences, Lucknow and Postgraduate Institute for Medical Education and Research, Chandigarh.

   The Union Ministry of Health publishes the Rare Disease Directive. Implementation could be difficult.

] In India only about 450 diseases of tertiary care hospitals were registered, which are considered rare diseases worldwide.

"The cost of treating patients in these centers of excellence is covered by donations received through the online digital platform." According to the draft directive.

The Ministry published the draft directive on its website and received comments and suggestions by February 10.

Rare diseases of group 1, e.g. B. Lysosomal Storage Disorders (LSDs)

The program finances immune deficiency diseases, chronic granulomatous diseases, osteopetrosis, Fabry disease and liver or kidney transplantation.

The stated policy is intended to provide financial support for rare diseases such as Gaucher disease and spinal muscular atrophy, Hurler syndrome and Wolman disease, which require lifelong treatment.

"The government will seek to provide an alternative funding mechanism through the creation of ads." A digital platform for individual and corporate voluntary donors to help cover the treatment costs of rare disease patients, "the draft said.

Rare diseases are serious and chronic diseases and often life-threatening. Patients who suffer from rare diseases, especially Lysosomal Storage Disorders (LSDs), often lead a very incompetent life. There are approximately 50 LSDs, of which only five in India Approved treatment options are available.

The Ministry of Health had formulated a national guideline for the treatment of rare diseases (NPTRD) in July 2017.

However, the implementation of the guideline was faced with certain challenges. A limiting factor in the implementation was the inclusion of States and the ambiguity t about how much the government could support in terms of tertiary care.

When politics was shared with state governments, issues such as the cost-effectiveness of rare disease interventions were raised.

  Rare diseases are serious and chronic diseases and are often life-threatening. Patients who suffer from rare diseases, especially Lysosomal Storage Disorders (LSDs), often lead very incapable lives.

Rare diseases are serious, chronic diseases and often life-threatening. They often live very inept lives. Photo credit: PTI

Policy had implementation problems and gaps, including the cost-effectiveness of supporting such health policies with limited resources, making implementation impossible.

Given policy challenges, a decision was made to rewrite national rare disease treatment policies, and in November 2018 the Ministry of Health set up a panel of experts to review this policy.

Prior to the reformulation of the policy, the previous policy was overridden In a non-statutory official gazette of December 18, 2018, until the revised directive was issued or until further orders were issued, whichever is earlier.

Finding that rare diseases are a major economic burden for every country and especially resources. The draft directive states that there are currently very few pharmaceutical companies producing rare disease drugs worldwide and there are no domestic manufacturers in India.

Due to the high costs, the government was unable to provide these drugs free of charge. It is estimated that for a child weighing 10 kg, the annual treatment costs for some rare diseases can vary from Rs 10 lakh to more than one crore per year, with the drug dose and cost increasing with age.

Up to now, only 450 illnesses have been registered in tertiary care hospitals in India, which according to the draft directive are considered rare worldwide.

The most frequently reported diseases include hemophilia, thalassemia, sickle cell anemia and primary immunodeficiency in children. Autoimmune diseases, lysosomal storage disorders such as Pompe disease, Hirschsprung disease, Gaucher disease, cystic fibrosis, hemangiomas and certain forms of muscular dystrophy.

Insufficient availability of epidemiological data on rare diseases, limited resources. In the competition for health priorities, the government is currently establishing a national register at the ICMR for rare diseases, with the aim of creating a database of various rare diseases.

Steps in this direction have already been taken by the ICMR. The registry is expected to provide sufficient information over time to arrive at a definition of rare diseases best suited to the country.


Updated date: January 15, 2020, 14:13:34 IST


Autoimmune diseases,

Certain forms of muscular dystrophy,

Chronic granulomatosis,

cystic fibrosis,

Draft national rare disease policy released on Monday

Draft directive,

Fabry disease,

Gaucher's disease,


Hirschsprung's disease,

Immunodeficiency disorders,

kidney transplant

Liver Transplantation,

Lysosomal storage disorders,

National Rare Disease Policy,


Pompe's disease,

Rare diseases,

Rare Diseases Policy.,

Ministry of Health of the Union,

Ysosomal Storage Disorders (LSDs)

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