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 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

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 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

Impact Of Pharmacist Homecare Service On Medication Adherence And Knowledge Among Hypertensive Patients In Seremban

Hypertension is one of the major public health problems in Malaysia due to its high prevalence (42.6% in 2006), lack of awareness amongst the general population, its poor control and its impact on cardiovascular morbidity and mortality. A common reason for inadequate control of hypertension is low adherence with antihypertensive medicine regimen. Therefore, this research was conducted to assess and evaluate patients’ adherence and to examine the impact of “Pharmacist Homecare Service (PHS)” provision on the patients’ adherence, blood pressure reduction and knowledge among hypertensive patients

Are new models needed to optimize the utilization of new medicines to sustain healthcare systems?

Medicines have made an appreciable contribution to improving health. However, even high-income countries are struggling to fund new premium-priced medicines. This will grow necessitating the development of new models to optimize their use. The objective is to review case histories among health authorities to improve the utilization and expenditure on new medicines. Subsequently, use these to develop exemplar models and outline their implications. A number of issues and challenges were identified from the case histories. These included the low number of new medicines seen as innovative alongside increasing requested prices for their reimbursement, especially for oncology, orphan diseases, diabetes and HCV. Proposed models center on the three pillars of pre-, peri- and post-launch including critical drug evaluation, as well as multi-criteria models for valuing medicines for orphan diseases alongside potentially capping pharmaceutical expenditure. In conclusion, the proposed models involving all key stakeholder groups are critical for the sustainability of healthcare systems or enhancing universal access. The models should help stimulate debate as well as restore trust between key stakeholder groups

Rare disease in Malaysia: Challenges and solutions.

ObjectiveRare diseases are often underdiagnosed, and their management is frequently complicated by a lack of access to treatment and information about the diseases. To allow for better policy planning, we sought to examine the current status of managing rare diseases in Malaysia.MethodsThis study was conducted in two phases. In the first phase, we triangulated information from reviews of journal publications, documents from the Malaysian government and in-depth interviews among selected key healthcare stakeholders in Malaysia. The second phase was designed as a cross-sectional survey to estimate the number of cases and treatment coverage for rare diseases in Malaysia.ResultsMalaysia has no official definition of rare disease yet but currently in the process of reviewing them for Malaysia. There are 13 rare disease specialists and a dozen medical doctors in genetic clinics around Malaysia, mainly in public health facilities. From the survey, 1,249 patients were diagnosed with rare diseases in public hospitals. Only 60% received their medications or supplements, and the rest continued with symptomatic treatment.ConclusionGenerally, Malaysia has made significant progress in the management of rare diseases, but there are still opportunities for development in critical areas. Ultimately, if all healthcare providers, government, society, and politicians work together to manage rare diseases, we will see an improvement in patient outcomes