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An Insight Of The Policy And Access To Orphan Drugs For Treating Rare Disease In Malaysia: Quantitative And Qualitative Analysis

An orphan drug is a medicine used to treat rare diseases. The disease has a very low population, with most of the diseases will impair the patients’ body functions and be life threatening. This study aimed to evaluate the rare disease policy and access to orphan drugs in Malaysia. Three phases have...

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Main Author: Supian, Azuwana
Format: Thesis
Language:English
Published: 2022
Subjects:
RS1-441 Pharmacy and materia medica
Online Access:http://eprints.usm.my/58455/1/AZUWANA%20BINTI%20SUPIAN%20-%20TESIS.pdf
http://eprints.usm.my/58455/
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spelling my.usm.eprints.58455 http://eprints.usm.my/58455/ An Insight Of The Policy And Access To Orphan Drugs For Treating Rare Disease In Malaysia: Quantitative And Qualitative Analysis Supian, Azuwana RS1-441 Pharmacy and materia medica An orphan drug is a medicine used to treat rare diseases. The disease has a very low population, with most of the diseases will impair the patients’ body functions and be life threatening. This study aimed to evaluate the rare disease policy and access to orphan drugs in Malaysia. Three phases have been completed. Phase one consisted of literature reviews to examine the current status of rare disease management in Malaysia and five other focus countries (Philippines, Singapore, Indonesia, Vietnam, and Thailand). They were examined based on the World Health Organisation’s (WHO) framework for action in strengthening health systems. A cross-sectional survey from ten public hospitals was also included in this phase to estimate the number of cases and treatment coverage for rare diseases in Malaysia. The second phase was designed by conducting 43 qualitative interviews and a focus group discussion on describing the perceptions among health stakeholders towards rare disease management. In the final stage, five Mucopolysaccharidosis type II (MPS II) patients’ record was retrieved and compared with the Guidelines for Treatment of Lysosomal Storage Diseases by Enzyme Replacement Therapy in Malaysia to estimate the costs involved in MPS II, including the treatment and monitoring patient from the point of healthcare provider perspective. The results suggest rare disease management remains challenging across Southeast Asia, as many of the focus countries face fundamental issues from basic healthcare systems to funding. Nonetheless, there are substantial improvement opportunities, including leveraging best practices worldwide and organising a multi-stakeholder and regional approach and strategy. From the survey, 1,249 patients were diagnosed with rare diseases in public hospitals. However, only 60% received their medications or supplements, and the rest continued with symptomatic treatment. Generally, health stakeholders are quite satisfied with the Malaysia health system and rare disease management compared to previous years. However, there is still a need for more improvement in the future. The focus group discussion presented many points and planning, and their ultimate goal is to have Malaysian rare disease and orphan drug act. It is found that the total cost of treatment per patient per year with orphan drug idursulfase and management of MPS II is MYR 710,289.35. In conclusion, Malaysia has made tremendous progress in managing rare diseases, but there are still opportunities for development in critical areas. To reduce the gap access to orphan drugs, all parties must clearly understand the rare disease management, structure, and national plan. Malaysia also should explore and develop a new financial model to ensure rare disease patients receive the care they need. 2022-02 Thesis NonPeerReviewed application/pdf en http://eprints.usm.my/58455/1/AZUWANA%20BINTI%20SUPIAN%20-%20TESIS.pdf Supian, Azuwana (2022) An Insight Of The Policy And Access To Orphan Drugs For Treating Rare Disease In Malaysia: Quantitative And Qualitative Analysis. PhD thesis, Perpustakaan Hamzah Sendut.
institution Universiti Sains Malaysia
building Hamzah Sendut Library
collection Institutional Repository
continent Asia
country Malaysia
content_provider Universiti Sains Malaysia
content_source USM Institutional Repository
url_provider http://eprints.usm.my/
language English
topic RS1-441 Pharmacy and materia medica
spellingShingle RS1-441 Pharmacy and materia medica
Supian, Azuwana
An Insight Of The Policy And Access To Orphan Drugs For Treating Rare Disease In Malaysia: Quantitative And Qualitative Analysis
description An orphan drug is a medicine used to treat rare diseases. The disease has a very low population, with most of the diseases will impair the patients’ body functions and be life threatening. This study aimed to evaluate the rare disease policy and access to orphan drugs in Malaysia. Three phases have been completed. Phase one consisted of literature reviews to examine the current status of rare disease management in Malaysia and five other focus countries (Philippines, Singapore, Indonesia, Vietnam, and Thailand). They were examined based on the World Health Organisation’s (WHO) framework for action in strengthening health systems. A cross-sectional survey from ten public hospitals was also included in this phase to estimate the number of cases and treatment coverage for rare diseases in Malaysia. The second phase was designed by conducting 43 qualitative interviews and a focus group discussion on describing the perceptions among health stakeholders towards rare disease management. In the final stage, five Mucopolysaccharidosis type II (MPS II) patients’ record was retrieved and compared with the Guidelines for Treatment of Lysosomal Storage Diseases by Enzyme Replacement Therapy in Malaysia to estimate the costs involved in MPS II, including the treatment and monitoring patient from the point of healthcare provider perspective. The results suggest rare disease management remains challenging across Southeast Asia, as many of the focus countries face fundamental issues from basic healthcare systems to funding. Nonetheless, there are substantial improvement opportunities, including leveraging best practices worldwide and organising a multi-stakeholder and regional approach and strategy. From the survey, 1,249 patients were diagnosed with rare diseases in public hospitals. However, only 60% received their medications or supplements, and the rest continued with symptomatic treatment. Generally, health stakeholders are quite satisfied with the Malaysia health system and rare disease management compared to previous years. However, there is still a need for more improvement in the future. The focus group discussion presented many points and planning, and their ultimate goal is to have Malaysian rare disease and orphan drug act. It is found that the total cost of treatment per patient per year with orphan drug idursulfase and management of MPS II is MYR 710,289.35. In conclusion, Malaysia has made tremendous progress in managing rare diseases, but there are still opportunities for development in critical areas. To reduce the gap access to orphan drugs, all parties must clearly understand the rare disease management, structure, and national plan. Malaysia also should explore and develop a new financial model to ensure rare disease patients receive the care they need.
format Thesis
author Supian, Azuwana
author_facet Supian, Azuwana
author_sort Supian, Azuwana
title An Insight Of The Policy And Access To Orphan Drugs For Treating Rare Disease In Malaysia: Quantitative And Qualitative Analysis
title_short An Insight Of The Policy And Access To Orphan Drugs For Treating Rare Disease In Malaysia: Quantitative And Qualitative Analysis
title_full An Insight Of The Policy And Access To Orphan Drugs For Treating Rare Disease In Malaysia: Quantitative And Qualitative Analysis
title_fullStr An Insight Of The Policy And Access To Orphan Drugs For Treating Rare Disease In Malaysia: Quantitative And Qualitative Analysis
title_full_unstemmed An Insight Of The Policy And Access To Orphan Drugs For Treating Rare Disease In Malaysia: Quantitative And Qualitative Analysis
title_sort insight of the policy and access to orphan drugs for treating rare disease in malaysia: quantitative and qualitative analysis
publishDate 2022
url http://eprints.usm.my/58455/1/AZUWANA%20BINTI%20SUPIAN%20-%20TESIS.pdf
http://eprints.usm.my/58455/
_version_ 1768007885485244416
score 13.211869

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