Journal of market access & health policy最新文献

Introduction: Outcome-based agreements (OBAs) are occasionally deployed to relieve the burden of high drug prices on healthcare budgets. However, it is not clear when manufacturers are willing to collaborate in establishing such agreements. Therefore, we explored the feasibility of OBAs from the manufacturer's point of view.

Methods: Dutch market-access experts from eight major pharmaceutical companies, globally active in the field of oncology, were interviewed. Opinions were compiled, and interviewees and their colleagues were then given the chance to review the manuscript for additional comments.

Results: Most interviewees believe that OBAs can be useful in providing access to off-label use of authorised medicines, especially when no alternative treatment is available for seriously ill patients. For the licenced indications, manufacturers seem to be more inclined to collaborate when there is a potential incentive to improve market-access (e.g., if the product is not used because of concerns regarding its effectiveness). However, manufacturers are less likely to collaborate when there are greater financial risks for the company. Further concerns were definition of outcome or performance, the impact of compliance on the effectiveness of a drug, administrative burden, uncertainty regarding revenue recognition and the challenges of reimbursing combination therapies.

Discussion: Market-access interviewees were generally positive about OBAs, however they were more reluctant towards OBAs for registered indications with low response-rate. The definition of performance or outcome and its clinical relevance and validity, the feasibility of OBAs and their administrative burden are relevant aspects that need to be addressed in advance. Ideally, countries should collaborate to share the outline of OBAs and create shared databases to accumulate evidence.

It is widely acknowledged that using p-value thresholds as the basis for making decision on health care spending is not appropriate. In the context of medical decision making, we argue that patient preferences need to be a stronger factor. Depending on attitudes to risk, patients might prefer a medical treatment that performs on average worse than a comparator but offers a small probability of a large gain such as a cure. However, what has been labeled 'value of hope' is not yet fully reflected in the decision-making process of drug approval and health technology assessment (HTA). Therefore, patient risk preferences should be formally incorporated within the decision-making framework for regulatory and reimbursement decisions.

Background: In the United States (US), colorectal cancer (CRC) is the second leading cause of cancer-related deaths. With the majority of the US population covered by employer-based health plans, employers can play a critical role in increasing CRC screening adherence, which may help avert CRC-related deaths. Therefore, it is important for self-insured employers to consider the impact of appropriate utilization of CRC screening options. Objective: To evaluate the impact of increasing multitarget stool DNA [mt-sDNA (Cologuard®)] use among CRC screeners from the perspective of a US self-insured employer. Methods:A 5-year Markov model was developed to quantify the budget impact of increasing mt-sDNA from 6% to 15% among average-risk screeners using colonoscopy, fecal immunological test, and mt-sDNA. Data on direct medical costs were obtained from published literature, Medicare CPT codes, and the Healthcare cost and Utilization project. Indirect costs included productivity loss due to workplace absenteeism for CRC screening and treatment. Results: With a hypothetical population of 100,000 employees with screeners aged 50-64 years, compared to status quo, increased mt-sDNA utilization resulted in no differences in the numbers of cancers detected and the overall direct and indirect cost savings were ~$214,000 ($0.04 per-employee-per-month) over 5 years. Most of the savings were due to a reduction in the direct medical expenditure related to CRC screening, adverse events, and productivity loss due to colonoscopy screening. Similar results were observed in the model simulation among screeners aged 45-64 years. Conclusion: Increased utilization of mt-sDNA for CRC screening averts direct and indirect medical costs from a self-insured US employer perspective.

Background: The practice of non-medical switch (NMS) from a reference biological (originator) to a biosimilar is widely accepted in some countries. However, there is little documentation on the impact of NMS from one originator to another originator. Objectives: To assess the consequences for patients of NMS from one biological originator to another, based on existing literature. The focus was on efficacy and cost of treatment with TNF-α-inhibitors in three disease areas. Methods: A literature search was conducted in Ovid (PubMed, EMBASE) and abstracts from meetings in key therapeutic areas, to identify studies reporting efficacy, safety or costs by switching between originator biologics. Results: 167 references were identified and abstracts screened; 36 papers reviewed in full text, and 6 fulfilled the inclusion criteria. Three clinical studies of NMS had very small sample sizes, but suggested that NMS is beneficial. The remaining three studies used administrative data with little clinical information, indicating that NMS was disadvantageous and associated with increased health care utilization and costs. Conclusions: There is very limited documentation on NMS from one originator biological to another, and the literature suffers from methodological limitations. The results are mixed and preclude drawing an overriding conclusion. Future studies, are warranted.

The lack of adequate treatment for many patients with opioid use disorder (OUD) has led to high medical costs ($90B in 2020). An analysis of the cost-effectiveness (cost-utility) of reSET-O, the first and only FDA-approved prescription digital therapeutic (PDT) for the treatment of OUD, is needed to inform value assessments and healthcare decision making. To evaluate the cost-utility of reSET-O in conjunction with treatment-as usual (TAU) compared to TAU alone. A third-party payer-perspective decision analytic model evaluated the cost-effectiveness of reSET-O + TAU relative to TAU (i.e., oral buprenorphine, face-to-face counseling, and contingency management [immediate rewards for negative drug tests logged]) alone over 12 weeks. Clinical effectiveness data (retention in therapy and health state utilities) were obtained from the peer-reviewed literature, while resource utilization and cost data were obtained from a published claims data analyses. Over 12 weeks, the addition of reSET-O to TAU resulted in a gain of 0.003 quality-adjusted life years (QALYs), and $1,014 lower costs, resulting in economic dominance vs. TAU. reSET-O + TAU's was economically dominant (less costly, more effective) vs. TAU alone over 12 weeks, a result that was driven by a reduction in medical costs after initiation of reSET-O observed in a recent real-world claims analysis.

Background: In recent years, innovation in oncology has created new challenges for pricing and reimbursement systems. Oncology medicines with multiple indications face a number of access challenges: (1) the number of assessments and administrative burden; (2) aligning price to different values of the same product; (3) managing clinical uncertainty at time of launch; and (4) managing budget uncertainty. These challenges impact a range of stakeholders and can result in delayed patient access to life-saving treatments. Consequently, countries have taken steps to facilitate patient access. Methods: Drawing on the experience across Europe we have reviewed different mechanisms countries have adopted that address these challenges. These include approaches aimed directly at the issue, multi-year-multi-indication (MYMI) agreements (BE, NL), and other approaches to manage access: flexible access agreements for new indications with clinical uncertainty (UK); development of a new agreement for each new indication (IT); and immediate access for new indications and bundled assessments (DE). Results: MYMI agreements are valuable where existing rules mean that every indication faces the same upfront evaluation process that delays patient access. They are also useful in managing budget impact and uncertainty. Other approaches that adopt an indication-specific approach helps manage clinical uncertainty at the time of launch and realise different values for the same product. They can help align price to value, even though indication-based pricing does not exist. Bundled assessments reduce the administrative burden for stakeholders, and the benefits of immediate reimbursement is that patient access is not delayed. Conclusion: The challenges for medicines with multiple indications impact a range of stakeholders and can result in delayed patient access to life-saving treatments. MYMI agreements have created a more pragmatic approach to HTA for medicines with multiple indications to ensure both fast and broad patient access. Continued innovation in oncology will require further innovative approaches in pricing and reimbursement. It is important that policymakers, payers and manufacturers engage in early discussions and are willing to find new solutions to help accelerate patient access to innovative therapies.

Background:The optimisation of vaccine policies before their implementation is beholden upon public health decision makers, seeking to maximise population health. In this case study in Serbia, the childhood vaccines under consideration included pneumococcal conjugate vaccination (PCV), rotavirus (RV) vaccination and varicella zoster virus (VZV) vaccination. Objective: The objective of this study is to define the optimal order of introduction of vaccines to minimise deaths, quality adjusted life years (QALYs) lost, or hospitalisation days, under budget and vaccine coverage constraints. Methods: A constrained optimisation model was developed including a static multi-cohort decision-tree model for the three infectious diseases. Budget and vaccine coverage were constrained, and to rank the vaccines, the optimal solution to the linear programming problem was based upon the ratio of the outcome (deaths, QALYs or hospitalisation days) per unit of budget. A probabilistic decision analysis Monte Carlo simulation technique was used to test the robustness of the rankings. Results: PCV was the vaccine ranked first to minimise deaths, VZV vaccination for QALY loss minimisation and RV vaccination for hospitalisation day reduction. Sensitivity analysis demonstrated the most robust ranking was that for PCV minimizing deaths. Conclusion: Constrained optimisation modelling, whilst considering all potential interventions currently, provided a comprehensive and rational approach to decision making.

Evidence about the Nigerian health indicators show that the quality of health care in Nigeria is low and inflation of health care prices also persists. Theoretically, by observing the market concentration, inferences can be drawn as to how hospitals conduct themselves, which allows the evaluation of the market performance. Therefore, the effects of market concentration on the health care price and quality were examined. Market concentration was measured by Herfindahl Hirschman Index (HHI) and four hospital concentration ratios (CR₄). The values of HHI were disaggregated into the less and more concentrated markets. Quality of health care was measured by the staff-nurse-patient ratio. Ordinary Least Square (OLS) was used to estimate the effects of market concentration on price and quality of health care. The price of health care was found to be 13.4% lower in the less concentrated markets than in the more concentrated market. Income significantly and positively influenced health care prices by 17.8%. Also, a low HHI lead to 33.4% increase in Staff-nurse Patient Ratio (SPR) indicating that the quality of health care was higher in less concentrated markets as hospitals increased the treatment intensity via staff-nurse patient ratio. A less concentrated market is linked with higher health care quality and lower health care prices. Therefore, a strategy that will reduce market concentration so as to enhance consumer welfare in terms of price and quality is recommended.

Background: Standard of care (SoC) for transfusion-dependent β-thalassemia (TDT) requires lifelong, regular blood transfusions as well as chelation to reduce iron accumulation. Objective: This study investigates the cost-effectiveness of betibeglogene autotemcel ('beti-cel'; LentiGlobin for β-thalassemia) one-time, gene addition therapy compared to lifelong SoC for TDT. Study design: Microsimulation model simulated the lifetime course of TDT based on a causal sequence in which transfusion requirements determine tissue iron levels, which in turn determine risk of iron overload complications that increase mortality. Clinical trial data informed beti-cel clinical parameters; effects of SoC on iron levels came from real-world studies; iron overload complication rates and mortality were based on published literature. Setting: USA; commercial payer perspective Participants: TDT patients age 2-50 Interventions: Beti-cel is compared to SoC. Main outcome measure: Incremental cost-effectiveness ratio (ICER) utilizing quality-adjusted life-years (QALYs) Results: The model predicts beti-cel adds 3.8 discounted life years (LYs) or 6.9 QALYs versus SoC. Discounted lifetime costs were $2.28 M for beti-cel ($572,107 if excluding beti-cel cost) and $2.04 M for SoC, with a resulting ICER of $34,833 per QALY gained. Conclusion: Beti-cel is cost-effective for TDT patients compared to SoC. This is due to longer survival and cost offset of lifelong SoC.

Background: Simulation modeling facilitates the estimation of long-term health and economic outcomes to inform healthcare decision-making. Objective: To develop a framework to simulate progression of Parkinson's disease (PD), capturing motor and non-motor symptoms, clinical outcomes, and associated costs over a lifetime. Methods: A patient-level simulation was implemented accounting for individual variability and interrelated changes in common disease progression scales. Predictive equations were developed to model progression for newly diagnosed patients and were combined with additional sources to inform long-term progression. Analyses compared a hypothetical disease-modifying therapy (DMT) with a standard of care to explore the drivers of cost-effectiveness. Results: The equations captured the dependence between the various measures, leveraging prior values and rates of change to obtain realistic predictions. The simulation was built upon several interrelated equations, validated by comparison with observed values for the Movement Disorder Society Unified PD Rating Scale (MDS-UPDRS) and UPDRS subscales over time. In a case study, disease progression rates, patient utilities, and direct non-medical costs were drivers of cost-effectiveness. Conclusions: The developed equations supported the simulation of early PD. This model can support conducting simulations to inform internal decision-making, trial design, and strategic planning early in the development of new DMTs entering clinical trials.