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Rare diseases are conditions that affect a small percentage of the population, yet they collectively impact over 70 million Indians. The National Policy for Rare Diseases (NPRD) 2021 aims to address their treatment challenges, but financial constraints persist. With treatment costs ranging from ₹10 lakh to ₹16 crore per patient annually, inadequate funding mechanisms hinder equitable access, exacerbating patient hardships.

Access to Treatment Challenges Despite NPRD 2021

Funding Inefficiencies

• Inadequate Financial Assistance: The one-time funding of ₹50 lakh per patient is insufficient for lifelong treatment of chronic rare diseases, leading to discontinuation of therapies.
• Non-Utilization of Allocated Funds: Despite ₹143.19 crore sanctioned to 12 Centres of Excellence (CoEs), delays in fund disbursal hinder access to treatment.
  For example: Several CoEs failed to utilize the funds effectively, delaying enzyme replacement therapy (ERT) for Gaucher disease patients, despite its proven benefits.
• Exclusion of Ultra-Rare Diseases: NPRD 2021 does not cover ultra-rare conditions like Acid Sphingomyelinase Deficiency (ASMD), depriving eligible patients of funding.
  For example: 32 ASMD patients in India lack government assistance, despite the availability of clinically approved therapies.

Administrative Inefficiencies

• Absence of Monitoring Mechanism: No structured oversight exists to track fund utilization and ensure timely treatment.
  For example: Parliamentary-backed interventions face execution delays, leaving 577 LSD patients ready for treatment but without support.
• Bureaucratic Delays in Disbursement: Lengthy approval procedures and lack of inter-agency coordination cause treatment interruptions.
  For example: Patients waiting for MoHFW’s crowdfunding portal funds often face delays due to unclear eligibility criteria and procedural bottlenecks.

Policy Gaps in NPRD 2021

• Lack of a Sustainable Funding Model: NPRD 2021 relies on one-time grants instead of a long-term funding structure, making continuous treatment impossible.
  For example: LSD patients require lifelong enzyme replacement therapy (ERT), but policy provisions end funding after ₹50 lakh.
• Insufficient Coverage of Rare Diseases: Not even 50% of over 450 rare diseases are included under NPRD 2021, leaving many patients without aid.
  For example: Pompe and Fabry diseases, despite having effective treatments, are not comprehensively covered under the policy.
• Weak Accountability in Implementation: Lack of performance audits results in poor execution and underutilization of funds at CoEs.
  For example: CoEs have failed to ensure transparent fund allocation, causing life-threatening delays for LSD patients.
• Limited Awareness and Accessibility: Doctors and patients lack awareness about rare disease treatments and funding avenues, limiting patient outreach.
  For example: Many eligible families are unaware of NPRD 2021 benefits, leading to under-enrollment in CoEs.
• No National Registry for Rare Diseases: Absence of a central database results in inaccurate patient estimations and delayed policy interventions.
  For example: A national registry could help track real-time treatment needs, ensuring better fund allocation and policy updates.

Measures for a Sustainable Funding Mechanism

• Creation of a Dedicated Rare Disease Fund: Establish a permanent government fund exceeding ₹50 lakh per patient to cover lifelong treatment costs.
  For example: Countries like Germany and the UK have specialized rare disease funds, ensuring continuous patient care.
• Public-Private Partnerships (PPP): Leverage CSR funding, pharma collaborations, and crowdfunding initiatives to supplement government resources.
  For example: Novartis’ CSR program in India has supported Spinal Muscular Atrophy (SMA) patients with costly gene therapies.
• Insurance Coverage for Rare Diseases: Expand Ayushman Bharat and state insurance schemes to include lifelong treatment coverage for rare diseases.
• Real-Time Monitoring System: Establish a digital platform to track fund utilization, patient treatment progress, and CoE accountability.
  For example: A blockchain-based tracking system can ensure transparent disbursal of funds without bureaucratic hurdles.
• Fast-Tracking National Programme on Rare Diseases: Accelerate the ₹974-crore programme to ensure faster diagnosis, affordable treatments, and better infrastructure.
  For example: Speeding up this initiative can benefit thousands of untreated LSD patients, reducing child mortality rates.

A comprehensive rare disease policy must go beyond intent and ensure effective implementation. Sustainable funding through a mix of government support, CSR initiatives, and public-private partnerships is crucial. Streamlining administrative processes, strengthening early diagnosis, and expanding insurance coverage can make treatment more accessible. A patient-centric approach, backed by strong institutional mechanisms, is essential to address the challenges faced by rare disease patients in India.

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