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Too nay deewana banaya to mein deewana bana
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تو نے کیا کیا نہ بنایا کوئی کیا کیا نہ بنا
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The most expensive injection in the world costs Rs 35 crore.
Zolgensma
What is it and why is the price so high?
The drug, first approved by the US Food and Drug Administration on May 24, 2019, was approved by the UK's National Health Service (NHS) on March 9, 2020.
The drug is manufactured by Novartis Gene Therapies, a Swiss bio-pharmaceutical company based in the United States. This drug is life-changing for young people with spinal muscular atrophy and their families. Spinal muscular atrophy is a major genetic disease of death in infants and young children, which is why NHS England has explored the mountains to make this treatment available, according to Novartis. Spinal Muscular Atrophy is a rare hereditary disease caused by a deficiency of a missing gene or functional survival motor neuron 1 (SMN1) gene. This results in rapid and irreversible damage to motor neurons, affecting vital muscle functions such as breathing, swallowing and basic movement.
Because these neurons control the movement of muscles throughout the body, their deficiency can lead to paralysis, severe muscle weakness, and impaired mobility. The prevalence of SMA is about 1 in 10,000 children. 1 in 54 people have a genetic defect in SMA and there is a 25% chance of having a child with SMA in two carriers.
Zolgensma is the second and most effective medicine for this disease. Its high cost is due to its small market size and life-saving ability in the pharmaceutical industry.
This disease is rare and that is why we need a very specific medicine. It takes a lot of time to research the skills needed to build it and its surroundings. It is also a one-time dose. The fewer the cases, the higher the cost.
The cost of Zolgensma is 2. 2.125 million - less than 50% of the current standard 10-year cost of chronic SMA therapy in the United States, and over time, it is expected that SMA patients will be treated and cared for. Expenses in the healthcare system will be saved, and payments will be made with payers to ensure access.
Zolgensma sets consistent pricing under a worldwide pricing framework, however final pricing and compensation decisions are made locally.
According to a 2016 study published in the Journal of Health Economics, it takes about 2.6 billion and more than 10 years to develop a new drug. A study by the Massachusetts Institute of Technology (MIT) found that only 14% of drugs in clinical trials eventually get FDI approval because a certain amount of the drug does not succeed in research.
Researchers from MIT and Dana Farber Cancer Institute have suggested financing expensive drugs so that patients have to pay in installments, provided the drug works.
