: Monday, 17 March 2025 2:21 pm
DinRlabh diseases are conditions that affect a small portion of the population, yet they are collectively affecting more than 70 million people in India. National Policy 2021 for rare diseases tries to deal with their treatment challenges, but financial limitations remain an important obstacle. Every year, with treatment expenses ranging from ₹ 10 lakh to ₹ 16 crore per patient, insufficient funding mechanisms make it difficult for patients to reach the necessary care, which further increases their problems. Most rare diseases have a genetic base and often cause severe, chronic health problems. Many have a lack of proper diagnostic methods or treatment protocols and existing treatment usually manage symptoms instead of providing treatment. In India, like many developing countries, there is no universally accepted definition of rare diseases. The burden of rare diseases in India is quite high, which is about one third of global patients. The national policy for rare diseases suggests that about 50–100 million people in India are affected by these conditions.
There are more than 450 rare diseases in the country, including famous conditions such as spinal muscular atrophy and gauchar disease. About 8 to 10 million Indians are suffering from rare diseases, of which more than 75% are children. The high rate of sickness and mortality associated with these serious conditions is one of the main reasons why many of these children do not survive even adulthood. A lump sum funding of ₹ 50 lakh per patient is insufficient for lifelong management of chronic rare diseases, resulting in stopping treatment.
Although ₹ 143.19 crore has been allocated to 12 excellence centers, the delay in fund distribution hinders access to essential remedies. Some excellence centers have delayed the use of funds effectively, causing delay in enzyme replacement therapy for patients with gaucher disease, despite its established benefits, in 2021, there are not high conditions such as acid sphingomilenaz deficiencies in 2021, so that eligible patients remain without financial support.
In India, patients with acid sphingomelinege deficiency are without government assistance, even though medically approved treatment is available. There is no inspection to monitor the use of funds and provide timely treatment. The implementation of the initiatives supported by the Parliament is delayed, causing patients with lysosomal storage disorder without the necessary help. Treatment is obstructed due to long acceptance process and poor inter-agency coordination.
Patients waiting for funds from the crowdfonding portal often face delays due to vague eligibility criteria and procedural obstacles. National Rare Disease Policy 2021 depends on a lump sum grant rather than a permanent funding model, making the ongoing treatment impractical. Patients with lyrical storage disorder require life enzyme replacement therapy, but policy provisions eliminate funding after ₹ 50 lakhs. The National Rare Disease Policy 2021 covers less than half of more than 450 rare diseases, which leads to many patients without any help.
Conditions such as pompes and fabry diseases, whose effective treatment is available, is not fully covered by policy. The absence of display audit leads to low use of funds in ineffective implementation and excellence centers. These centers have delayed providing transparent funds, resulting in severe delays for patients with lysosomal storage disorder. Both doctors and patients lack information about the treatment and financing options available, which hinders outreach efforts.
Many families who qualify for the National Rare Disease Policy 2021 benefits are not known about them, resulting in low enrollment rate in centers. Lack of centralized databases leads to incorrect estimate and slow policy reactions of the patient. A national registry can effectively monitor real -time treatment requirements, facilitating better fund distribution and timely policy adjustment. To cover lifetime treatment expenses, it is necessary to create a permanent government fund of more than ₹ 50 lakh per patient.
Countries such as Germany and the UK have set up special funds for rare diseases, which ensures persistent patient care. We should use corporate social responsibility funding, partnership with pharmaceutical companies and crowdfunding efforts to increase government resources. For example, the corporate social responsibility initiative of Novartis in India has provided assistance to patients with spinal muscular atrophy for expensive gene therapy.
It is important to expand Ayushman India and State Insurance programs to include lifelong treatment for rare diseases. Additionally, a digital platform should be developed to monitor the use of funds, to track the progression of the patient and ensure accountability of COE. Applying blockchain-based tracking systems can guarantee transparent fund distribution without bureaucracy obstacles. To ensure quick diagnosis, affordable treatment and better infrastructure, faster the initiative of ₹ 974 crore.
Rising this program rapidly, thousands of untreated patients can benefit greatly and can help reduce child mortality. A complete rare disease policy should only focus on effective execution by moving beyond intentions. Permanent financing is necessary by combining government assistance, corporate social responsibility and public-private participation. Simplifying administrative processes, increasing early diagnosis and making insurance coverage will make treatment more accessible. In India, a patient-focused approach supported by strong institutional structures is important to deal with the challenges faced by rare diseases. Healthcare professionals require training to increase their diagnosis accuracy.
Pregnant mothers with family history of rare diseases should undergo compulsory delivery prenat and postpartum care. The government should establish a clear definition of rare diseases, increase budget allocation, allocate funds for drug development and treatments and expand the number of excellence centers. These steps will strengthen the national policy of rare diseases 2021. Both public and private sectors should be involved in the financing of social support programs for rare diseases through CSR initiatives and financing cooperation. Additionally, removing GST from all life -saving drugs for rare diseases will help make these drugs more economical.
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