Abstract
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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Acknowledgements
The authors extend heartfelt thanks to the anonymous reviewers for their invaluable feedback in shaping this manuscript. Gratitude is also expressed to those directly or indirectly contributing to the information and design of the manuscript. Their collaborative efforts have significantly enhanced the quality and clarity of the research presented.
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This project was supported by internal funding of Kashiv Biosciences. No external funding was received for this project. NP, HP, and GS are employees of Kashiv Biosciences and MCC provided part-time consultancy service for this project.
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NP performed information and data extraction, conducted data analysis, and drafted the manuscript. HP analysed the results and contributed to manuscript writing. GS proposed the initial project idea, provided inputs, and helped in editing the manuscript. MCC, as corresponding author, designed the research methodology, oversaw the project, edited the manuscript, and provided inputs and feedback from the first to the final draft of the manuscript.
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Corresponding editor: Alok Bhattacharya
This article is part of the Topical Collection: The Rare Genetic Disease Research Landscape in India.
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Patel, N., Pandya, H., Sangle, G. et al. Enhancing access to treatment for Gaucher disease in India: The need for indigenous manufacturing. J Biosci 49, 38 (2024). https://doi.org/10.1007/s12038-024-00427-w
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DOI: https://doi.org/10.1007/s12038-024-00427-w