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1. The missing links in government’s efforts to tackle rare diseases
With India having commemorated Rare Disease Day 2025 on 28 February, it is time to review the progress made in formulating rare disease policies while promoting patient care. The National Policy for Rare Diseases (NPRD) 2021 was finally announced following decades of pleas and petitions from various advocacy groups. Further, a structured network of clinicians was created across 12 Centres of Excellence (CoEs) spread across 11 states to treat eligible patients with rare diseases.
These patients may be few in number, but doesn’t every life count? Treatment can save lives that otherwise would have had no hope.
The Gaps in Implementation
The creation of clinical expertise and healthcare capacity to treat rare diseases in India is laudable. However, while the intent is exemplary, the execution is somewhat lacking due to a vital missing link—the lack of a sustainable funding mechanism. Access to treatment is another glaring gap.
These are critical issues because the treatment of any rare disease entails exorbitant expenses that are out of reach for most patients and their families. Therefore, a sustainable funding mechanism for rare diseases is indispensable if these patients are to be given a fresh lease on life.
Rare diseases pose a substantial healthcare challenge, especially lysosomal storage disorders (LSDs). As they are chronic genetic disorders, they require lifelong management. Although India has over 450 rare diseases, treatment options are available for less than 50 per cent. NPRD 2021 was meant to address these issues, but its implementation has been inadequate.
Challenges in Treatment Access
Many patients across the country remain without access to lifesaving medication and treatments. Consider LSDs, which are classified under Group 3 (a) disorders. The Drugs Controller General of India has approved enzyme replacement therapy (ERT), which is the country’s most effective therapy.
The 12 CoEs in 11 states are empanelled by the Ministry of Health and Family Welfare (MoHFW) to onboard and treat eligible patients. The MoHFW registers eligible patients with notified rare disorders on its national crowdfunding portal.
Out of all rare disease patients, 577 LSD patients are ready for immediate treatment support. However, barely a fraction has received life-saving treatment, despite the availability of therapy and funds. This is happening because no structured monitoring mechanism exists to supervise the implementation of NPRD 2021. As a result, institutional interventions are delayed despite parliamentary backing.
Funding and Financial Gaps
Currently, one-time funding of ₹50 lakh is provided for eligible LSD patients. However, nearly 40 LSD patients across India have exhausted this financial assistance. With their treatment discontinued, they are only waiting for the inevitable.
This is not surprising since approximately 30 per cent of children with rare disorders succumb to their condition before reaching their fifth birthday. Likewise, without a sustainable funding mechanism, the lives of many other eligible rare disease patients are hanging in the balance.
Non-utilisation of Funds and Administrative Delays
A lack of funds is not the only issue. Non-utilisation of funds is also causing treatment delays. The MoHFW has sanctioned ₹143.19 crore to the 12 CoEs, but many have failed to utilise the funds effectively, delaying both treatment and access to therapies.
This needless state of affairs is primarily due to minimal accountability and little transparency in fund utilisation by the CoEs.
Moreover, ultra-rare conditions such as Acid Sphingomyelinase Deficiency (ASMD) are excluded from NPRD 2021, even though clinically proven, approved therapies are available. There are 32 ASMD patients in India, but since their condition is not notified under NPRD 2021, government funding is not provided.
Recommendations to Strengthen Rare Disease Management
Keeping these policy gaps and administrative delays in mind, the MoHFW could create a robust monitoring mechanism that:
A. Provides sustainable funds for people with chronic and ultra-rare conditions:
(i) In at least three CoEs, nearly 40 patients have run out of their one-time funding support of ₹50 lakh and presently await more assistance.
(ii) Breaking the continuum of care leaves patients in a dangerous situation.
(iii) The MoHFW could create a dedicated rare disease fund providing much higher assistance than the current ₹50 lakh limit.
B. Accelerates the introduction of the National Programme on Rare Diseases:
(i) Under NPRD 2021, the ₹974-crore programme aims to address crucial gaps in treating rare disorders.
(ii) Since most rare disease patients are children, including LSD patients who need prompt intervention, this programme’s implementation must be expedited.
C. Ensures timely and equitable treatment access for eligible rare diseases:
(i) The National Rare Disease Committee (NRDC) Impact Report found that some conditions, like Gaucher disease, were ranked 10/10 in terms of progress.
(ii) Despite this, only a fraction of diagnosed, eligible patients has received ERT, although it is a notified condition.
(iii) Streamlining the operations of CoEs and expediting funds disbursal are necessary for timely treatment.
D. Promotes stronger implementation of the NPRD 2021 framework:
(i) A rigorous monitoring mechanism could help immediately address several administrative delays.
(ii) An independent monitoring body backed by real-time tracking for funds utilisation and patient treatment status will ensure better oversight of NPRD 2021’s implementation.
Conclusion
These measures are imperative to provide rare disease patients with a fighting chance at a near-normal life. Any further delay in implementing these measures would be akin to a death sentence for many rare disease patients, including innocent children who deserve a chance to live without constant fear of an uncertain future.
Disclaimer:
This analysis is based on the editorial content published in Indian Express and is intended solely for informational and educational purposes. The views, opinions, and interpretations expressed herein are those of the author of original article. Readers are encouraged to refer to the original article for complete context and to exercise their own judgment while interpreting the analysis. The analysis does not constitute professional advice or endorsement of any political, economic, or social perspective.
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