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...

Full description

Bibliographic Details
Main Author: Supian, Azuwana
Format: Thesis
Language:English
Published: 2022
Subjects:
Online Access:http://eprints.usm.my/59508/
http://eprints.usm.my/59508/1/24%20Pages%20from%20AZUWANA%20BINTI%20SUPIAN.pdf
_version_ 1848884190617010176
author Supian, Azuwana
author_facet Supian, Azuwana
author_sort Supian, Azuwana
building USM Institutional Repository
collection Online Access
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.
first_indexed 2025-11-15T19:02:46Z
format Thesis
id usm-59508
institution Universiti Sains Malaysia
institution_category Local University
language English
last_indexed 2025-11-15T19:02:46Z
publishDate 2022
recordtype eprints
repository_type Digital Repository
spelling usm-595082023-10-19T02:10:57Z http://eprints.usm.my/59508/ 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. 2022-01 Thesis NonPeerReviewed application/pdf en http://eprints.usm.my/59508/1/24%20Pages%20from%20AZUWANA%20BINTI%20SUPIAN.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.
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
title 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_short 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
topic RS1-441 Pharmacy and materia medica
url http://eprints.usm.my/59508/
http://eprints.usm.my/59508/1/24%20Pages%20from%20AZUWANA%20BINTI%20SUPIAN.pdf