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

被引:0
|
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
来源
关键词
Eliglustat; enzyme replacement therapy (ERT); Gaucher disease; rare disease; substrate reduction therapy (SRT);
D O I
暂无
中图分类号
学科分类号
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.
引用
收藏
相关论文
共 50 条
  • [31] Regression of smoldering myeloma with treatment of Gaucher disease
    Barley, Kevin
    Parekh, Anshuman
    Salam, Syed
    Mendu, Damodara Rao
    Shukla, Ravi Prakash
    Vatti, Deepa
    Verina, Daniel
    Stauffer, Chanan
    Salib, Christian
    El Jamal, Siraj
    Teruya-Feldstein, Julie
    Duffield, Amy S.
    Leshchenko, Violetta V.
    Jagannath, Sundar
    Balwani, Manisha
    Parekh, Samir
    [J]. BLOOD ADVANCES, 2024, 8 (07) : 1634 - 1638
  • [32] Skeletal manifestations in Gaucher disease: Presentation and treatment
    Lebel, E
    Itzchaki, M
    Elstein, D
    Hadas-Halpern, I
    Abrahamov, A
    Zimran, A
    [J]. ISRAEL MEDICAL ASSOCIATION JOURNAL, 1999, 1 (04): : 267 - 271
  • [33] THE NEED FOR STANDARDIZATION OF IMMUNOGENICITY ASSAYS FOR GAUCHER DISEASE ENZYME REPLACEMENT THERAPIES
    Keutzer, J.
    Richards, S.
    Graham, S.
    Yee, J.
    [J]. CLINICAL THERAPEUTICS, 2009, 31 : S207 - S207
  • [34] GAUCHER DISEASE - TREATMENT OF HYPERSPLENISM WITH SPLENIC EMBOLIZATION
    THANOPOULOS, BD
    FRIMAS, CA
    MANTAGOS, SP
    BERATIS, NG
    [J]. ACTA PAEDIATRICA SCANDINAVICA, 1987, 76 (06): : 1003 - 1007
  • [35] Recommendations on Diagnosis, Treatment, and Monitoring for Gaucher Disease
    Martins, Ana Maria
    Valadares, Eugenia Ribeiro
    Porta, Gilda
    Coelho, Janice
    Semionato Filho, Jose
    Dudeque Pianovski, Mara Albonei
    Kerstenetzky, Marcelo Soares
    Pombo Montoril, Maria de Fatima
    Aranda, Paulo Cesar
    Pires, Ricardo Flores
    Vale Mota, Ronald Moura
    Bortolheiro, Teresa Cristina
    [J]. JOURNAL OF PEDIATRICS, 2009, 155 (04): : S10 - S18
  • [36] Treatment of multiple myeloma in patients with Gaucher disease
    Machaczka, Maciej
    Lerner, Richard
    Klimkowska, Monika
    Hagglund, Hans
    [J]. AMERICAN JOURNAL OF HEMATOLOGY, 2009, 84 (10) : 694 - 696
  • [37] Patterns of renal disease in indigenous populations in India
    Mani, MK
    [J]. NEPHROLOGY, 1998, 4 : S4 - S7
  • [38] ANALYSIS OF SOCIAL WORK STUDY MATERIAL IN INDIA - NEED FOR INDIGENOUS THOUGHT
    NAGPAUL, H
    [J]. INDIAN JOURNAL OF SOCIAL RESEARCH, 1970, 11 (03): : 358 - 371
  • [39] Enhancing innovation between scientific and indigenous knowledge: pioneer NGOs in India
    Maria-Costanza Torri
    Julie Laplante
    [J]. Journal of Ethnobiology and Ethnomedicine, 5
  • [40] Enhancing innovation between scientific and indigenous knowledge: pioneer NGOs in India
    Torri, Maria-Costanza
    Laplante, Julie
    [J]. JOURNAL OF ETHNOBIOLOGY AND ETHNOMEDICINE, 2009, 5 : 29