National Health Policy for Rare Diseases

Rare diseases in India

• According to the government, so far only about 450 diseases have been recorded in India from tertiary care hospitals that are globally considered as rare diseases.
• The most commonly reported diseases include 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.
• There are 7,000 - 8,000 rare diseases, but less than 5% have therapies available.
• About 95% rare diseases have no approved treatment and less than 1 in 10 patients receive disease specific treatment.
• Where drugs are available, they are expensive.

National Health Policy for Rare Diseases, 2020

• The Delhi High Court directed the Centre to finalise the National Health Policy for Rare Diseases of 2020 by March 31 and make operational provision of crowdfunding envisaged under the law for treatment of high-cost rare diseases.
• Justice Prathiba M. Singh passed the direction while hearing two different pleas concerning children, who are suffering from duchenne muscular dystrophy, a condition that causes progressive muscle degeneration and weakness in the victim.

About National Policy for Rare Diseases, 2020

• It proposes to set up a registry under the Indian Council of Medical research (ICMR) to create a database.
• To provide financial assistance of up to Rs 15 lakh to Ayushman Bharat beneficiaries for rare diseases that require a one-time treatment in tertiary hospitals only.
• It also suggests voluntary crowdfunding as an alternate means of financial support and notifying government hospitals to facilitate treatment.
• Alternatively, the draft proposes to set up a digital platform for voluntary crowdfunding.
• The draft policy also categorises rare diseases under three categories based on clinical experiences and treatment availability.
• The policy also states that in the absence of data to clearly define rare diseases, such diseases in India will construe the three categories as identified in the policy.

It offers no clarity on long-term financial assistance, appears designed on donations

Criticism of the Draft Policy

• The government, in its policy paper, has proposed financial support of up to ?15 lakh under an umbrella scheme of the Rashtriya Arogya Nidhi for those rare diseases that require a one-time treatment.
• Beneficiaries for such financial assistance would not be limited to Below Poverty Line (BPL) families, but extended to 40% of the population eligible as per the norms of the Pradhan Mantri Jan Arogya Yojana, for their treatment in government tertiary hospitals only.
• “The 2017 policy on rare diseases had proposed a ?100 crore corpus fund allocation for the treatment of patients with rare diseases, which was a Centre-State partnership of 60:40, with each State forming a technical committee to evaluate patients’ applications. That has come down to ?15 lakh support for diseases needing one-time treatment. Clearly, now, after a major lag, the policy has come with no hope in sight,” noted a release from the Lysosomal Storage Disorders Support Society.
• It appears the entire policy is drafted to justify that government cannot provide treatment due to high cost as it is resource constrained.
• The policy has adopted a very narrow scope limited to only 3 categories, while ignoring those where treatment is yet to be developed and R&D is required.

What is the news?

• The Delhi High Court has set up a special committee to find a time-bound solution on ways to provide treatment and therapy options to patients suffering from rare diseases.
• Justice Prathiba M. Singh ordered the committee to also give “immediate concrete proposals for crowdfunding of the costs of treatment for children with rare diseases”.
• The High Court’s direction came while hearing a bunch of petitions filed by patients suffering from rare diseases such as Duchenne Muscular Dystrophy (DMD) and Hunter’s syndromes seeking direction to the government to provide them uninterrupted free treatment in view of the exorbitant cost of treatment.

What is DMD and Hunter’s syndromes?

• DMD is a condition that causes progressive muscle degeneration and weakness in the victim.
• Hunter’s syndromes is a rare disease that is passed on in families. It mostly affects boys and their bodies cannot break down a kind of sugar that builds bones, skin, tendons, and other tissues.