Enhancing access to treatment for Gaucher disease in India: The need for indigenous manufacturing

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作者
Nidhi Patel
Heta Pandya
Ganesh Sangle
Mohua Chakraborty Choudhury
机构
[1] Kashiv Biosciences Pvt Ltd,DST Center for Policy Research
[2] Indian Institute of Science,undefined
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关键词
Eliglustat; enzyme replacement therapy (ERT); Gaucher disease; rare disease; substrate reduction therapy (SRT);
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摘要
Gaucher disease (GD) is a prevalent lysosomal storage disorder (LSD) that significantly impacts individuals’ lives. However, the exorbitant prices of GD medications pose a major hurdle in ensuring widespread availability and affordability of treatment in India. The country heavily relies on imported medications, leading to high costs and limited access for many patients. This article aims to address this issue by advocating for the establishment of indigenous manufacturing capabilities for GD medicines in India. Through an examination of the current landscape of GD treatment, including the availability, affordability, and challenges associated with imported medications, this article highlights the urgent need for localized production. By focusing on the potential benefits of indigenous manufacturing, such as reduced costs, increased accessibility, and enhanced availability, this research aims to provide insights and recommendations to policymakers, healthcare professionals, and relevant stakeholders. The findings underscore the importance of developing domestic manufacturing capabilities to address the affordability and accessibility challenges faced by GD patients in India. The research also emphasizes the potential positive impact on the healthcare system, the pharmaceutical industry, and the overall well-being of individuals with GD. Ultimately, this article seeks to stimulate discussions and actions towards creating a sustainable framework for indigenous manufacturing of GD medicines, thereby improving the lives of those affected by this rare and debilitating condition.
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