In a groundbreaking stride forward, Indian pharmaceutical companies, with the backing of government agencies, have achieved a remarkable feat within a year by developing treatments for four rare diseases, significantly slashing their cost of treatment by up to a staggering 100 times. These rare diseases, many of which afflict children, are primarily genetic in nature, posing severe health challenges to patients.
Among the four diseases targeted, Gaucher's Disease, characterised by liver or spleen enlargement, bone pain, and fatigue; Wilson's Disease, leading to copper accumulation in the liver alongside psychiatric symptoms; and Dravet/Lennox Gastaut Syndrome, causing complex seizure syndromes, are the focal points of this groundbreaking achievement.
The cost reduction has been nothing short of extraordinary. Treatment expenses for Gaucher's disease, employing Eliglustat capsules, plummeted from an exorbitant ₹ 1.8-3.6 crore per year to an astonishingly affordable ₹ 3.6 lakh. Similarly, the cost for Wilson's Disease treatment, utilising Trientine capsules, nosedived from ₹ 2.2 crore to a mere ₹ 2.2 lakh per year. Meanwhile, the treatment for Dravet Syndrome, involving Cannabidiol oral solution, witnessed a significant drop from ₹ 7-34 lakh per year to a range of ₹ 1-5 lakh.
Additionally, Tyrosinemia type 1 treatment, previously costing an astounding ₹ 2.2 crore to ₹ 6.5 crore annually, has now been drastically reduced to a manageable ₹ 2.5 lakh for the same duration. This transformation in affordability is life-changing, especially considering that without treatment, a child diagnosed with Tyrosinemia type 1 wouldn't survive beyond the age of 10. The medication Nitisinone has been pivotal in this remarkable cost reduction.
India grapples with an overwhelming population of 8.4 crore to 10 crore individuals affected by rare diseases, a significant 80% of which are genetic, necessitating early intervention and treatment due to the manifestation of symptoms at a young age.
Meanwhile, it's imperative to note the contrasting dynamics prevailing in the global pharmaceutical landscape, particularly in American pharmaceutical companies, often driven primarily by profit motives. This approach, while yielding advancements, can also significantly inflate treatment costs, rendering life-saving medications unaffordable for many individuals, especially those suffering from rare diseases. The focus on maximising profits may deter initiatives similar to India's recent strides in cost reduction, potentially limiting access to crucial treatments and impacting the overall well-being of the global population.
The pioneering efforts of Indian pharmaceutical companies not only signify a significant leap in accessibility to treatments for rare diseases but also underscore the importance of prioritising affordability and accessibility in healthcare, a value that should ideally guide pharmaceutical endeavours worldwide.
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