National Policy for Rare Diseases, 2021

Rare Diseases: WHO defines a rare disease as a lifelong disease or disorder that often highly weakens an individual. It has a prevalence of 1 or less per 1000 population.

• Example: Haemophilia, Thalassemia, Sickle cell anaemia, auto-immune diseases among others.
• However, every country has its own definition for rare diseases.
• The US defines rare diseases as a disease or condition that affects fewer than 200,000 patients in the country.
• Likewise, the EU defines rare diseases as life-threatening or chronically debilitating (weakening) conditions. It should affect no more than 5 in 10,000 people.

About National Policy For Rare Diseases,2021:

• Aim: The policy aims to lower the incidence and prevalence of rare diseases based on an integrated and comprehensive preventive strategy.
• The strategy includes awareness generation, counselling programmes, providing affordable Health Care among others.

Key Features of the National Policy For Rare Diseases, 2021:

• Categorisation: The policy categorizes rare diseases into three groups:
  • Group 1: Disorders amenable to one-time curative treatment;
  • Group 2: Diseases requiring long term or lifelong treatment; and
  • Group 3: Diseases for which definitive treatment is available, but challenges are to make an optimal patient selection for benefit.
• Government Support:
  • The government will provide Financial support of up to Rs. 20 lakh under the Umbrella Scheme of Rashtriya Arogya Nidhi for treatment of those rare diseases listed under Group 1.
  • Moreover, Beneficiaries for such financial assistance would not be limited to BPL families.
    • About 40% of the population, eligible under Pradhan Mantri Jan Arogya Yojana, will also be eligible for assistance.
  • For group 2, the State Governments can consider supporting specific patients.
    • It includes a rare disease that can be managed with special diets or hormonal supplements or other relatively low-cost interventions (Diseases listed under Group 2).
• Voluntary Crowdfunding: The government has said that it will assist in voluntary crowd-funding for the treatment of Group 3. It is because it will be difficult to fully finance the treatment of high-cost rare diseases of Group 3.

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.

National Policy for Rare Diseases, 2021

The Policy aims at lowering the incidence and prevalence of rare diseases based on an integrated and comprehensive preventive strategy encompassing awareness generation, premarital, post-marital, pre-conception and post-conception screening and counselling programmes to prevent births of children with rare diseases, and within the constraints on resources and competing health care priorities, enable access to affordable health care to patients of rare diseases.

Initiatives for treatment support for patients of rare diseases under the Policy are as follows: -

• Financial support up to Rs. 20 lakhs under the Umbrella Scheme of Rashtriya Arogaya Nidhi shall be provided by the Central Government for treatment, of those rare diseases that require a one-time treatment (diseases listed under Group 1). Beneficiaries for such financial assistance would not be limited to BPL families, but extended to about 40% of the population, who are eligible as per norms of Pradhan Mantri Jan Arogya Yojana, for their treatment in Government tertiary hospitals only.
• State Governments can consider supporting patients of such rare diseases that can be managed with special diets or hormonal supplements or other relatively low-cost interventions (Diseases listed under Group 2).
• Keeping in view the resource constraints, and a compelling need to prioritize the available resources to get maximum health gains for the community/population, the Government will endeavour to create alternate funding mechanism through setting up a digital platform for voluntary individual and corporate donors to contribute to the treatment cost of patients of rare diseases.
• Voluntary crowd-funding for treatment: Keeping in view the resource constraint and competing health priorities, it will be difficult for the Government to fully finance treatment of high-cost rare diseases. The gap can however be filled by creating a digital platform for bringing together notified hospitals where such patients are receiving treatment or come for treatment, on the one hand, and prospective individual or corporate donors willing to support treatment of such patients. The notified hospitals will share information relating to the patients, diseases from which they are suffering, estimated cost of treatment and details of bank accounts for donation/ contribution through online system. Donors will be able to view the details of patients and donate funds to a particular hospital. This will enable donors from various sections of the society to donate funds, which will be utilized for treatment of patients suffering from rare diseases, especially those under Group 3. Conferences will be organized with corporate sector companies to motivate them to donate generously through digital platform. Ministry of Corporate Affairs will be requested to encourage PSUs and corporate houses to contribute as per the Companies Act as well as the provisions of the Companies (Corporate Social Responsibility Policy) Rules, 2014 (CSR Rules). Promoting health care including preventive health care is included in the list in the Schedule for CSR activities.

Treatment cost of the patient will be first charge on this fund. Any leftover fund after meeting treatment cost can be utilized for research purpose also.

At present financial assistance to poor patients, living below threshold poverty line and also to the population, who are eligible as per norms of Pradhan Mantri Jan Arogya Yojana under Ayushman Bharat, suffering from specified rare diseases for their treatment at Government Hospitals or Institutes having super specialty facilities / Government tertiary hospitals is being provided under the Umbrella Scheme of Rashtriya Arogya Nidhi (RAN). The budget allocation for the current financial year 2021-2022 for rare diseases is Rupees 25 Crore.

National Policy for Rare Diseases, 2021 provides for National Consortium for Research and Development on therapeutics for Rare Diseases with an expanded mandate to include research & development, technology transfer and indigenization of therapeutics for rare diseases. It will be convened by Department of Health Research (DHR) with ICMR as a member.

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 rare diseases.
• The most commonly reported diseases include Haemophilia, Thalassemia, Sickle-cell Anaemia and Primary Immuno Deficiency in children, auto-immune diseases, and 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% of 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 the 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, and 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.

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