ORDI media statement on National Policy for Rare Diseases, 2021

Ordi

The National Policy for Rare Diseases, 2021 abridges the ‘right to health’ by ignoring true needs of Rare Disease community in India

Hyderabad, 2021: This statement is with reference to the National Policy for Rare Diseases, 2021 (“NPRD, 2021”) released by the Health Ministry on March 30, 2021; a long awaited and long delayed document whose journey began in 2017. All the advocacy, angst, and anticipation has amounted to naught, for the rare disease community in India. This is because not much has been altered from the draft National Policy for Rare Diseases released in 2020. If anything, it is another iteration that fails to acknowledge and address the real challenges of rare disease patients and their families.

Rare Disease Policy fails to meet the dire treatment needs of Rare Disease Patients

No specific funding allocated for the treatment of Rare Diseases (Group 2 and Group 3 disorders); rather it suggests relying on Crowdfunding which is not a practical solution!

Rare Diseases does have a definition! This will hamper the research, local drug development activities, and the long-term plan for the Rare Disease Community.

Group 2 disorders are left at the discretion of State whereas the State isn’t well equipped with knowledge, infrastructure or funding support system to tackle such disorders. States need guidance and directions from the Central Government.

For a country as huge as India, 8 Centres of Excellence for management of Rare Diseases is insufficient!

Organization for Rare Diseases India (ORDI) strongly believes that there are many things that are woefully missing in the NPRD 2021. For one thing, the Government has taken a rather cavalier approach to the issue of affordability of the care needed to sustain a life of dignity for persons living with rare diseases. To tackle the issue of affordability, the NPRD 2021 suggests that an ailing/dying patient or their family should rely on crowdfunding for treatment. Picture the scene: a person desperately in need of medical intervention or continued medicines, must themselves or with help from their family, organise fundraisers, so that they may continue living. A stark picture indeed, one that amplifies either the fact that the struggles of the rare disease community don’t matter, or worse, that the Government doesn’t believe it has a role to play in ensuring holistic, affordable care for all rare diseases. Persons living with rare diseases, irrespective of age, gender, economic status, caste, religion or another societal classification, have a right to dignified treatment, supportive care, and funding options to afford treatment and supportive care.

A person living with a rare disease may have a first line treatment (e.g., a therapy/drug for their particular disease), a second line treatment (i.e., drugs actually developed for other diseases that are repurposed to manage their symptoms individually, but don’t treat the disease as a whole), and/or palliative and rehabilitative treatment (e.g., physiotherapy, nutrition supplements and foods, etc.) Many times, a patient needs one or more of these three forms of treatment, in addition to frequent diagnostics (e.g., lab work, medical scans, etc.). In some cases, patients also use assistive aids and medical devices (e.g., C-PAP machines). The NPRD, 2021 does not account for all these various elements that come together to form one coherent treatment package for patients, and therefore fails to understand the true extent of the financial, emotional, and mental costs.

The NPRD invokes the Rashtriya Arogya Nidhi (RAN) Scheme, stating that INR 20 Lakh Rupees would be given to diseases falling within Group 1 of the NPRD, 2021 (i.e., disorders amenable to one-time curative treatment. This includes diseases such as Mucopolysaccharoidosis (MPS) type I within first 2 years of age. Adrenoleukodystrophy (early stages), before the onset of hard neurological signs, Immune deficiency disorders like Severe Combined Immunodeficiency (SCID), Chronic Granulomatous disease, Wiskot Aldrich Syndrome, Osteopetrosis, Fanconi Anemia etc. This amount is inadequate to cover the costs of treatments for these diseases, which are actually needed more than once. State Governments have been given the discretion to provide for the treatment needed to manage diseases listed in Group 2 (i.e., diseases that require long term care). The NPRD, 2021, does not give details on funding, allocation, responsibilities, and Government’s role in providing supportive care (i.e., care that comes from the second line of treatment). It merely leaves it to the States to figure out how they want to handle it.

All this while, despite health being a state subject, the states have done nothing significant for Rare Disease patients; even in cases that have treatment as low as diet management. Expecting the clueless State to manage group 2 and 3 disorders on its own without any sort of guidance is a huge flaw in the policy! The woefully inadequate funding allocated by the Government, and the offhand suggestion of relying on crowdfunding and CSR initiatives, means that patients and their families are left largely in the lurch when it comes to affording any kind of care. Similarly, the NPRD 2021, mentions the need to encourage local innovation and manufacturing, as a way to reduce costs of drugs, devices and other therapies. While this is encouraging, the fact that no real commitment or allocation has been made as to how much money will go into this or should ideally go into this, is a real spirit dampener. It means that more time will be lost in trying to set up systems to promote this kind of research. For example, setting up a research fund, with little to no guidance on how projects are selected, what kind of oversight should be exercised on such projects, and how the fund itself will be administered and sustained.

There is yet another challenge to be addressed- and that is data. How has the list of rare diseases currently in the NPRD 2021 been collated without clearly defining or setting out the parameters of what would be considered a rare disease? Without data on the number of persons living with a certain rare disease, how will companies know the demand, or how to structure their clinical trials? How will any Government funding, allocation, and Schemes work, if we don’t have a clear metric to decide what diseases are considered rare diseases? Finally, there are 7000+ known rare diseases. A piece-meal approach, that counts only some rare diseases and not all, is a terrible one with terrible consequences. Not being “listed” amounts to not being counted, and being counted, could mean the difference between life or death.

“The NPRD, 2021, is a mockery of the right to health each person in India enjoys, because it ignores the true needs of the rare disease community in India” says Mr. Prasanna Shirol, Founder Director, ORDI. “The lack of foresight and grounding in the rare disease community’s reality shines through the cold and thoughtless drafting of the NPRD 2021. A truly shocking thing, given the hundreds of High Court cases, individual representations, and representations spanning almost two decades by patient advocacy groups. But we will continue to fight on – till every person living with a rare disease, irrespective of age, or any socio-economic factors, has the right to health.”

ORDI is currently conducting discussions with their stakeholders to collate necessary information and submit an extensive report to Ministry of Health and Family Welfare (MoHFW) to review the policy.

About Organization of Rare Diseases India (ORDI):
Founded in Feb 2014, ORDI is a patient cantered non-profit organization with the mission to improve health of patients with rare diseases across India through awareness, advocacy, collaborations, and information dissemination. ORDI represents the collective voice of all patients with rare diseases in India to effect government policy making. ORDI has identified and taken on the grand challenges facing the rare diseases community in India and is committed to systematically and holistically addressing those challenges.

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