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OBJECTIVES: The rising costs of drugs have necessitated the exploration of innovative payment methods in healthcare systems. Risk-sharing agreements (RSAs) have been implemented in many countries as a value-based payment mechanism to manage the uncertainty associated with expensive technologies. This study aimed to investigate stakeholder perspectives on value-based payment in the Singaporean context, providing insights for future directions in health technology assessment and financing. METHODS: This descriptive qualitative inquiry involved participant interviews conducted between October 2021 and April 2022. Thematic analysis was conducted in two phases to analyze the interview transcripts. RESULTS: Seventeen respondents participated in the study, and five key themes emerged from the analysis. Stakeholders viewed RSAs as moderately positive, despite limited experience with them. They emphasized the importance of clearly defining objectives and establishing transparent criteria for implementing these schemes. The current data infrastructure was identified as both a barrier and facilitator, as RSAs impose administrative burdens. To successfully implement these payment mechanisms, capacity building, and effective stakeholder engagement that fosters mutual trust and cocreation are crucial. CONCLUSION: This study confirms previously identified barriers and facilitators to successful RSA implementation while contextualizing them within the Singaporean setting. The findings suggest that value-based payment has the potential to address uncertainty and improve access to healthcare technologies, but these barriers must be addressed for the schemes to be effective.
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Pesquisa Qualitativa , Participação dos Interessados , Avaliação da Tecnologia Biomédica , Singapura , Humanos , Avaliação da Tecnologia Biomédica/organização & administração , Participação no Risco Financeiro/organização & administração , Entrevistas como AssuntoRESUMO
AIM: Kidney failure patients in the Philippines have free choice on their kidney replacement therapy (KRT), with a majority choosing haemodialysis (HD) over peritoneal dialysis (PD) and transplantation despite the inadequate coverage of HD. Although national health insurance coverage is limited, KRT remains to be one of the top benefits pay-outs in the country. The study aims to identify the most cost-effective policy strategy for financing KRT in the Philippines, in the context of a universal healthcare policy. METHODS: A Markov model was developed to estimate and compare the costs and benefits of different policy options with the comparator being partial HD coverage. Direct medical, non-medical and indirect costs were measured, while outcomes were reported through quality-adjusted life years (QALYs). Parameters were derived from the kidney disease registry, hospital statistics from a tertiary hospital and a patient survey. RESULTS: The results of the cost-effectiveness analysis showed that shifting to a PD-First policy provides better value-for-money with an incremental cost-effectiveness ratio (ICER) of 570 029 Philippine Pesos (PHP) per QALY gained, compared with the ICER of the PD-First combined with pre-emptive transplant option of 577 989 PHP per QALY gained. Expanding existing HD coverage to 156 sessions was the least cost-effective policy (1 522 437 PHP per QALY gained). CONCLUSION: Government should consider shifting to a PD-First strategy and support policies that promote kidney transplants among existing PD and HD patients. This study also highlights the need for proper evaluation of partial coverage policies to ensure that government investments represent good value-for-money and patients receive optimal care.
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Custos de Cuidados de Saúde , Falência Renal Crônica/economia , Falência Renal Crônica/terapia , Terapia de Substituição Renal/economia , Assistência de Saúde Universal , Cobertura Universal do Seguro de Saúde/economia , Redução de Custos , Análise Custo-Benefício , Humanos , Falência Renal Crônica/diagnóstico , Transplante de Rim/economia , Diálise Peritoneal/economia , Filipinas , Qualidade de Vida , Diálise Renal/economia , Resultado do TratamentoRESUMO
There is growing interest globally in using real-world data (RWD) and real-world evidence (RWE) for health technology assessment (HTA). Optimal collection, analysis, and use of RWD/RWE to inform HTA requires a conceptual framework to standardize processes and ensure consistency. However, such framework is currently lacking in Asia, a region that is likely to benefit from RWD/RWE for at least two reasons. First, there is often limited Asian representation in clinical trials unless specifically conducted in Asian populations, and RWD may help to fill the evidence gap. Second, in a few Asian health systems, reimbursement decisions are not made at market entry; thus, allowing RWD/RWE to be collected to give more certainty about the effectiveness of technologies in the local setting and inform their appropriate use. Furthermore, an alignment of RWD/RWE policies across Asia would equip decision makers with context-relevant evidence, and improve timely patient access to new technologies. Using data collected from eleven health systems in Asia, this paper provides a review of the current landscape of RWD/RWE in Asia to inform HTA and explores a way forward to align policies within the region. This paper concludes with a proposal to establish an international collaboration among academics and HTA agencies in the region: the REAL World Data In ASia for HEalth Technology Assessment in Reimbursement (REALISE) working group, which seeks to develop a non-binding guidance document on the use of RWD/RWE to inform HTA for decision making in Asia.
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Medicina Baseada em Evidências , Mecanismo de Reembolso , Avaliação da Tecnologia Biomédica , Ásia , Análise Custo-Benefício , Confiabilidade dos Dados , Tomada de Decisões , Inquéritos e Questionários , TelecomunicaçõesRESUMO
This paper explores the characteristics of health technology assessment (HTA) systems and practices in Asia. Representatives from nine countries were surveyed to understand each step of the HTA pathway. The analysis finds that although there are similarities in the processes of HTA and its application to inform decision making, there is variation in the number of topics assessed and the stakeholders involved in each step of the process. There is limited availability of resources and technical capacity and countries adopt different means to overcome these challenges by accepting industry submissions or adapting findings from other regions. Inclusion of stakeholders in the process of selecting topics, generating evidence, and making funding recommendations is critical to ensure relevance of HTA to country priorities. Lessons from this analysis may be instructive to other countries implementing HTA processes and inform future research on the feasibility of implementing a harmonized HTA system in the region.
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Avaliação da Tecnologia Biomédica/organização & administração , Ásia , Tomada de Decisões , Humanos , Inquéritos e QuestionáriosRESUMO
BACKGROUND AND OBJECTIVE: This study aimed to assess the cost-effectiveness of two regimens regarded as the standard of care for the treatment of newly diagnosed, transplant-ineligible multiple myeloma in Singapore: (1) daratumumab, lenalidomide, and dexamethasone and (2) bortezomib, lenalidomide, and dexamethasone. Additionally, it aimed to explore potential strategies to manage decision uncertainty and mitigate financial risk. METHODS: A cost-effectiveness analysis from the healthcare system perspective was conducted using a partitioned survival model to estimate lifetime costs and quality-adjusted life years (QALYs) associated with daratumumab-based treatment and the bortezomib-based regimen. The analysis used data from the MAIA and SWOG S0777 trials and incorporated local real-world data where available. Sensitivity analyses were performed to evaluate the robustness of the findings, and a risk analysis was conducted to analyze various payer strategies in terms of their payer strategy and uncertainty burden (P-SUB), which account for the decision uncertainty and the additional cost of choosing a suboptimal intervention. RESULTS: The incremental cost-effectiveness ratio (ICER) for daratumumab, lenalidomide, and dexamethasone (DRd) compared with bortezomib, lenalidomide, and dexamethasone (VRd) was US $90,364 per QALY gained. The results were sensitive to variations in survival for DRd, postprogression treatment costs, cost of hospice care, and hazard ratio for progression-free survival. The scenarios explored indicated that structural assumptions, such as the time horizon of the analysis, significantly influenced the results due to uncertainties arising from immature trial data and treatment efficacy over time. Among the various payer strategies compared, an upfront price discount for daratumumab emerged as the best approach with the lowest P-SUB at US $14,708. CONCLUSION: In conclusion, this study finds that daratumumab as a first-line treatment for myeloma exceeds the cost-effectiveness threshold considered in this evaluation. An upfront price reduction is the recommended strategy to manage uncertainties and mitigate financial risks. These findings highlight the importance of targeted payer strategies to address specific types and sources of uncertainty.
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INTRODUCTION: Outcomes-based risk-sharing agreements (OBRSA) have been increasingly used worldwide to manage uncertainty in value assessments. This review aimed to summarize motivations, barriers, and facilitators to implementing OBRSAs with a specific focus on therapies for hematological cancer. AREAS COVERED: An integrative review was conducted based on a scoping of existing reviews on the topic and reports published by UK NICE. Findings from 16 articles and 10 reports were summarized and categorized into three themes: applications in blood cancer drugs, motivations for adoption, and barriers and facilitators to implementation. EXPERT OPINION: There was a dissociation between the theoretical basis for opting for OBRSAs, and reasons stated or inferred from practice. The administrative burden was considered a notable barrier to implementation, which affects not only payers and manufacturers but also healthcare providers. Effective stakeholder engagement and building mutual trust among key groups were identified as factors enabling successful implementation. The review raises essential considerations in implementing OBRSAs and implications for their future role, particularly for blood cancer drugs where uncertainty is rife. Carefully designed and managed schemes may remain an option for health systems to manage risks involved when funding high-cost treatments.
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Neoplasias Hematológicas , Humanos , Reino UnidoRESUMO
Multiple myeloma, a hematological malignancy, imposes a significant financial burden on healthcare systems. Health technology assessments (HTA) and economic evaluations play vital roles in reimbursement decisions and cost containment. This study aimed to explore healthcare utilization patterns and costs among myeloma patients in Singapore through a retrospective analysis of 605 patients treated at two cancer centers. Data encompassing demographics, treatment utilization, and billing were extracted from electronic records, and a cost analysis was performed from the perspective of the Singapore healthcare system. The results revealed common usage of immunomodulatory agents (52%) and proteasome inhibitors (37%), with bortezomib being the most frequently used targeted treatment. Treatment costs increased with disease progression, displaying variations depending on the therapeutic agent used. Notably, hospitalization costs due to adverse events were substantial, with pneumonia as the leading cause. This study highlights the high cost of myeloma therapy in Singapore, posing a financial burden for households. Findings may inform economic evaluations, evidence generation, reimbursement, and subsidy decisions. Leveraging real-world data from electronic records provides valuable insights into local healthcare utilization patterns. Future studies may explore integrating billing databases with clinical repositories for a more comprehensive analysis, and consider limitations such as incomplete clinical information and potential selection bias.
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BACKGROUND: Globally, there is increasing interest in the use of real-world data (RWD) and real-world evidence (RWE) to inform health technology assessment (HTA) and reimbursement decision-making. Using current practices and case studies shared by eleven health systems in Asia, a non-binding guidance that seeks to align practices for generating and using RWD/RWE for decision-making in Asia was developed by the REAL World Data In ASia for HEalth Technology Assessment in Reimbursement (REALISE) Working Group, addressing a current gap and needs among HTA users and generators. METHODS: The guidance document was developed over two face-to-face workshops, in addition to an online survey, a face-to-face interview and pragmatic search of literature. The specific focus was on what, where and how to collect RWD/ RWE. RESULTS: All 11 REALISE member jurisdictions participated in the online survey and the first in-person workshop, 10 participated in the second in-person workshop, and 8 participated in the in-depth face-to-face interviews. The guidance document was iteratively reviewed by all working group members and the International Advisory Panel. There was substantial variation in: (a) sources and types of RWD being used in HTA, and (b) the relative importance and prioritization of RWE being used for policy-making. A list of national-level databases and other sources of RWD available in each country was compiled. A list of useful guidance on data collection, quality assurance and study design were also compiled. CONCLUSION: The REALISE guidance document serves to align the collection of better quality RWD and generation of reliable RWE to ultimately inform HTA in Asia.
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Formulação de Políticas , Avaliação da Tecnologia Biomédica , Humanos , Projetos de Pesquisa , Inquéritos e Questionários , ÁsiaRESUMO
BACKGROUND: The Philippines has the highest cumulative COVID-19 cases and deaths in the Western-Pacific. To explore the broader health impacts of the pandemic, we assessed the magnitude and duration of changes in hospital admissions for 12 high-burden diseases and the utilization of five common procedures by lockdown stringency, hospital level, and equity in patient access. METHODS: Our analysis used Philippine social health insurance data filed by 1,295 hospitals in 2019 and 2020. We calculated three descriptive statistics of percent change comparing 2020 to the same periods in 2019: (1) year-on-year, (2) same-month-prior-year, and (3) lockdown periods. FINDINGS: Disease admissions declined (-54%) while procedures increased (13%) in 2020 versus 2019. The increase in procedures was caused by hemodialysis surpassing its 2019 utilization levels in 2020 by 25%, overshadowing declines for C-section (-5%) and vaginal delivery (-18%). Comparing months in 2020 to the same months in 2019, the declines in admissions and procedures occurred at pandemic onset (March-April 2020), with some recovery starting May, but were generally not reversed by the end of 2020. Non-urgent procedures and respiratory diseases faced the largest declines in April 2020 versus April 2019 (range: -60% to -70%), followed by diseases requiring regular follow-up (-50% to -56%), then urgent conditions (-4% to -40%). During the strictest (April-May 2020) and relaxed (May-December 2020) lockdown periods compared to the same periods in 2019, the declines among the poorest (-21%, -39%) were three-times greater than in direct contributors (-7%, -12%) and two-times more in the south (-16%, -32%) than the richer north (-8%, -10%). Year-on-year admission declines across the 12 diseases and procedures (except for hemodialysis) was highest for level three hospitals. Compared to public hospitals, private hospitals had smaller year-on-year declines for procedures, because of increases in utilization in lower level private hospitals. INTERPRETATION: COVID-19's prolonged impact on the utilization of hospital services in the Philippines suggests a looming public health crisis in countries with frail health systems. Through the periodic waves of COVID-19 and lockdowns, policymakers must employ a whole-of-health strategy considering all conditions, service delivery networks, and access for the most vulnerable. FUNDING: Open Philanthropy.
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The World Health Organization (WHO) launched the OneHealth Tool (OHT) to help low and middle income countries to develop their capacities for sector-wide priority setting. In 2016, we sought to use the OHT to aid the Philippine Health Insurance Corporation (PHIC), the national health insurer of the Philippines, in decisions to expand benefit packages using cost-effectiveness analyses. With technical support from the WHO, we convened health planning officers from the Philippine Department of Health (DOH) and the Philippine Health Insurance Corporation (PHIC) conduct generalized cost-effective analyses (GCEA) of selected un-financed noncommunicable disease interventions using OHT. We collected epidemiological and cost data through health facility surveys, review of literature such as cost libraries and clinical practice guidelines, and expert consultations. Although we were unable to use GCEA results directly to set policy, we learnt important policy lessons which we outline here that might help inform other countries looking to inform service coverage decisions. Additionally, the entire process and GCEA visualizations helped high-level policymakers in the health sector, who have traditionally relied on ad hoc decision making, to realize the need for a systematic and transparent priority-setting process that can continuously provide the evidence needed to inform service coverage decisions.