Stakeholders' Views on Patient Access Schemes in Malaysia.

OBJECTIVES: The healthcare system aspires to ensure improved access to treatment while working within the existing financial constraints and increasing demands. Patient access schemes (PASs) are initiated to improve access. This study seeks the stakeholders' views on the PAS in the public healthcare sector in Malaysia. METHODS: This is an exploratory qualitative study using a web-based online survey and semistructured interviews. The participants were recruited by purposive sampling, where the Ministry of Health of Malaysia staff, pharmaceutical organization personnel, and patient advocacy organization personnel with experience in PAS were invited to participate. A total of 42 consenting participants answered the survey, whereas the face-to-face interview had 8 participants. Interviews were thematically analyzed using the qualitative data analysis software NVivo V.12. The Medical Research and Ethics Committee, Ministry of Health of Malaysia, approved the study. RESULTS: Only finance-based PAS was reportedly implemented, and it covered drugs for antineoplastic or immunomodulating therapy, alimentary tract and metabolism, sensory organs, and systemic hormonal preparations. A total of 3 major themes were identified. High upfront cost and high budget impact are a major concern leading to the need for PAS. Identifying the treatment needs was a major concern as well. The readiness of the health system to implement PAS will determine whether the PAS can be successfully implemented. Challenges similar to other jurisdictions were observed in Malaysia, concerns on data availability, the responsibility of the stakeholders, and the need for a legal framework. CONCLUSION: Most stakeholders responded positively that the PAS would grow. Trust among stakeholders and a structured access plan would enhance the implementation and ensure its success.

Stakeholder views of managed entry agreements: A literature review of national studies.

Access to highly expensive and innovative medicine is a challenge. Managed entry agreements/ arrangements (MEAs) consist of various forms of confidential agreements between pharmaceutical manufacturers and payers (hospitals, social insurance), which are mainly negotiated when there is uncertainty of the actual clinical benefit of the medicines, but high public expenditures are required. Continued and expanding use of these schemes requires a greater understanding of payer perceptions, experience, and orientation towards their current and future use. This literature review aimed to identify the stakeholders' concerns with regards to MEAs. A total of 20 articles were identified between 2007 and 2018. MEAs are mostly studied in the first world countries, whereas limited documentation is available for other nations. The reasons for rejection of proposed MEAs could possibly be related to existing low-cost effective treatments, lack of trust between stakeholders, high administrative burden and if the authorities believe they are funding the substantial proportion of the drug's development cost. Lack of well-designed and easy-to-use data collection methods for MEAs has an impact on the evidence generation. Most articles highlighted the lack of transparency of the agreements which may not benefit other health systems, especially for systems that are dependent on international referencing price. Stakeholders also identified the lack of expertise in assessing the pharmacoeconomic and health outcomes data and challenges in assessing risks up-front due to the complexity of the real-world data. Lack of transparency in most health systems are also a hindrance to further understanding and confidence in the MEAs. However, the views of the stakeholders may also evolve with experience. Though there are challenges in the designing and implementation of MEAs, close networking, improved human capital development especially in the area of health technology assessment and health economics would be able to close the gap in the implementation.

Efficacy of Oral Mixed Tocotrienols in Diabetic Peripheral Neuropathy: A Randomized Clinical Trial.

Importance: Management of painful diabetic peripheral neuropathy remains challenging. Most therapies provide symptomatic relief with varying degrees of efficacy. Tocotrienols have modulatory effects on the neuropathy pathway and may reduce neuropathic symptoms with their antioxidative and anti-inflammatory activities. Objective: To evaluate the efficacy of oral mixed tocotrienols for patients with diabetic peripheral neuropathy. Design, Setting, and Participants: The Vitamin E in Neuroprotection Study (VENUS) was a parallel, double-blind, placebo-controlled trial that recruited participants from January 30, 2011, to December 7, 2014, with 12 months of follow-up. This trial screened 14 289 patients with diabetes from 6 health clinics and ambulatory care units from 5 public hospitals in Malaysia. A total of 391 patients who reported neuropathic symptoms were further assessed with Total Symptom Score (TSS) and Neuropathy Impairment Score (NIS). Patients 20 years or older with a TSS of 3 or higher and an NIS of 2 or higher were recruited. Interventions: Patients were randomized to receive 200 mg of mixed tocotrienols twice daily or matching placebo for 12 months. Patients with hyperhomocysteinemia (homocysteine level ≥2.03 mg/L) received oral folic acid, 5 mg once daily, and methylcobalamin, 500 µg thrice daily, in both groups. Main Outcomes and Measures: The primary outcome was patient-reported neuropathy TSS (lancinating pain, burning pain, paresthesia, and asleep numbness) changes at 12 months. The secondary outcomes were NIS and sensory nerve conduction test result. Results: Of 391 eligible patients, 300 were recruited (130 [43.3%] male; mean [SD] age, 57.6 [8.9] years; mean [SD] duration of diabetes, 11.4 [7.8] years) and 229 (76.3%) completed the trial. The TSS changes between the tocotrienols and placebo groups at 12 months (-0.30; 95% CI, -1.16 to 0.56; P = .49) were similar. No significant differences in NIS (0.60; 95% CI, -1.37 to 2.65; P = .53) and sensory nerve conduction test assessments were found between both groups. In post hoc subgroup analyses, tocotrienols reduced lancinating pain among patients with hemoglobin A1C levels greater than 8% (P = .03) and normohomocysteinemia (homocysteine level <2.03 mg/L; P = .008) at 1 year. Serious adverse events in both groups were similar, except more infections were observed in the tocotrienols group (6.7% vs 0.7%, P = .04). Results reported were of modified intention-to-treat analyses. Conclusions and Relevance: Supplementation of oral mixed tocotrienols, 400 mg/d for 1 year, did not improve overall neuropathic symptoms. The preliminary observations on lancinating pain among subsets of patients require further exploration. Trial Registration: National Medical Research Registry Identifier: NMRR-10-948-7327 and clinicaltrials.gov Identifier: NCT01973400.