Showing papers by "Louis P. Garrison published in 2013"

Споразумения за споделяне на риска, основани на представянето - добра практи- ка на дизайна, приложението и оценката: Доклад на работната група на ISPOR Performance-Based Risk-Sharing Arrangements - Good Practices for Design, Implementation, and Evaluation: Report of the ISPOR Good Practices for Performance- Based Risk-Sharing Arrangements Task Force

Abstract: 1Pharmaceutical Outcomes Research & Policy Program, Department of Pharmacy, University of Washington, Seattle, WA, USA; 2Office of Health Economics, London, UK; 3Institute of Health and Wellbeing, University of Glasgow, Glasgow, UK; 4ESSEC Business School, Cergy Pontoise, France; 5F. Hoffmann-La Roche AG, Basel, Switzerland; 6Center for Medical Technology Policy, Baltimore, Maryland, USA; 7Institute of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, The Netherlands; 8Agenzia Italiana del Farmaco, Rome, Italy; 9Access to Medicines GlaxoSmithKline plc, Brentford, UK

The Cost-Effectiveness of Initial Treatment of Multiple Myeloma in the U.S. With Bortezomib Plus Melphalan and Prednisone Versus Thalidomide Plus Melphalan and Prednisone or Lenalidomide Plus Melphalan and Prednisone With Continuous Lenalidomide Maintenance Treatment

Abstract: The outlook for transplant-ineligible multiple myeloma patients has improved enormously over recent years with the incorporation of new agents into standard regimens. Novel regimens combine melphalan and prednisone (MP) with bortezomib (VMP), with thalidomide (MPT), and with lenalidomide with (MPR-R) and without (MPR) lenalidomide maintenance. The efficacy, safety, and cost-effectiveness of these regimens have not yet been compared; therefore, we conducted a pharmacoeconomic analysis using data from randomized controlled trials versus MP. Using a Markov model developed from a U.S. payer's perspective, we compared VMP with MPT and MPR-R over a lifetime horizon. MPT and MPR-R were chosen because, like VMP, they are superior to MP in response and outcomes. Data from the Velcade as Initial Standard Therapy in Multiple Myeloma (VISTA; VMP), Intergroupe Francophone du Myelome (IFM) 99–06 (MPT), and MM-015 (MPR-R) trials were used. The IFM 99–06 study was selected because of the superior activity in this study compared with other MPT studies. Using patient-level (VMP) and published (MPT, MPR-R) data, we estimated the health-state transition and adverse event probabilities for each regimen, related costs, and state-specific utility estimates. Costs (in 2010 U.S. dollars) and health outcomes were discounted at 3%. Discounted lifetime direct medical costs were lowest with VMP at $119,102. MPT cost $142,452 whereas MPR-R cost $248,358. Incremental cost-effectiveness ratio calculations projected that VMP would confer cost savings and better health outcomes relative to MPT and MPR-R. We conclude that VMP is highly likely to be cost-effective compared with MP, MPT, and MPR-R.

Ipilimumab in 2nd line treatment of patients with advanced melanoma: a cost-effectiveness analysis

Abstract: Objective:To estimate the cost-effectiveness of ipilimumab (3 mg/kg) compared with best supportive care (BSC) in pre-treated advanced melanoma patients.Methods:The analysis was based on a US payer perspective and lifetime time horizon. A three-state Markov model was developed representing clinical outcomes, quality-of-life, and healthcare resource use of patients treated with ipilimumab and BSC. Transitions between states were modeled using overall and progression-free survival data from the MDX010-20 trial. Utility data were from a melanoma-specific study of the health state preferences of the general population. Disease management costs expressed in 2011 US Dollars were based on healthcare resource use observed in a US retrospective medical chart study. Uncertainty was analyzed using one-way and probabilistic sensitivity analyses.Results:The gain in life years and QALYs from introducing ipilimumab over BSC were 1.88 years (95% CI = 1.62–2.20) and 1.14 (95% CI = 1.01–1.34) QALYs, respectively, ov...

Differential pricing of new pharmaceuticals in lower income European countries

Abstract: Pharmaceutical companies adjust the pricing strategy of innovative medicines to the imperatives of their major markets. The ability of payers to influence the ex-factory price of new drugs depends on country population size and income per capita, among other factors. Differential pricing based on Ramsey principles is a second-best' solution to correct the imperfections of the global market for innovative pharmaceuticals, and it is also consistent with standard norms of equity. This analysis summarizes the boundaries of differential pharmaceutical pricing for policymakers, payers and other stakeholders in lower-income countries, with special focus on Central-Eastern Europe, and describes the feasibility and implications of potential solutions to ensure lower pharmaceutical prices as compared to higher-income countries. European stakeholders, especially in Central-Eastern Europe and at the EU level, should understand the implications of increased transparency of pricing and should develop solutions to prevent the limited accessibility of new medicines in lower-income countries.

Assessing the potential cost-effectiveness of retesting IHC0, IHC1+, or FISH-negative early stage breast cancer patients for HER2 status

Abstract: BACKGROUND Fluorescence in situ hybridization (FISH) and immunohistochemistry (IHC) tests are commonly used to assess human epidermal growth factor 2 (HER2) status of tumors in patients with breast cancer. This analysis evaluates the likely cost-effectiveness of expanded retesting to assess HER2 tumor status in women with early stage breast cancer. METHODS We developed a decision-analytic model to estimate the incremental cost-effectiveness ratio (ICER) of expanded reflex testing from a US payer perspective. Expanded reflex testing is defined as retesting tumor specimens from patients whose tumors are IHC0, IHC1+, or FISH-negative on their first test. In the base case, we assumed that 80% of patient tumors are initially IHC-tested and 20% are FISH-tested. Testing outcomes for IHC and FISH with and without retesting were based on published meta-analyses. The cost of tests and treatment and the long-term health outcomes were obtained from the literature. RESULTS In the base case, we estimated that 2.27% of women who received expanded reflex testing would be HER2-positive and receive trastuzumab treatment: the projected ICER was $36,721 per life year or $39,745 per quality-adjusted life year (QALY). This varied between $47,100 per QALY and $35,500 per QALY if we assumed that 1%-8% of patients retested were then HER2+, respectively. The results of deterministic and probabilistic sensitivity analysis were robust. This strategy would result in 4700 (2000-17,000) patients being eligible to receive trastuzumab treatment annually. CONCLUSIONS Retesting patients who are IHC0, IHC1+, or FISH-negative is projected to be a cost-effective clinical strategy. Cancer 2013;119:3113–3122. © 2013 American Cancer Society.

Can and should value-based pricing be applied to molecular diagnostics?

Abstract: Current pricing and reimbursement systems for diagnostics are not efficient. Prices for diagnostics are often driven by administrative practices and expected production cost. The purpose of the paper is to discuss how a value-based pricing framework being used to ensure efficient use and price of medicines could also be applied to diagnostics. Diagnostics not only facilitates health gain and cost savings, but also information to guide patients' decisions on interventions and their future 'behaviors'. For value assessment processes we recommend a two-part approach. Companion diagnostics introduced at the launch of the drug should be assessed through new drug assessment processes considering a broad range of value elements and a balanced analysis of diagnostic impacts. A separate diagnostic-dedicated committee using value-based pricing principles should review other diagnostics lying outside the companion diagnostics-and-drug 'at-launch' situation.

Understanding the Economic Value of Molecular Diagnostic Tests: Case Studies and Lessons Learned

Abstract: Ten years after completion of the Human Genome Project, progress towards making “personalized medicine” a reality has been slower than expected. The reason is twofold. Firstly, the science is more difficult than expected. Secondly, limited progress has been made in aligning economic incentives to invest in diagnostics. This paper develops nine case studies of “success” where diagnostic tests are bringing personalized medicine into clinical practice with health and economic impact for patients, healthcare systems, and manufacturers. We focus on the availability of evidence for clinical utility, which is important not only for clinicians but also for payers and budget holders. We find that demonstrating diagnostic clinical utility and the development of economic evidence is currently feasible (i) through drug-diagnostic co-development, and (ii) when the research is sponsored by payers and public bodies. It is less clear whether the diagnostic industry can routinely undertake the work necessary to provide evidence as to the clinical utility and economic value of its products. It would be good public policy to increase the economic incentives to produce evidence of clinical utility: otherwise, opportunities to generate value from personalized medicine—in terms of both cost savings and health gains—may be lost.

Original article Ipilimumab in 2nd line treatment of patients with advanced melanoma: a cost-effectiveness analysis

Abstract: Objective: To estimate the cost-effectiveness of ipilimumab (3 mg/kg) compared with best supportive care (BSC) in pretreated advanced melanoma patients. Methods: The analysis was based on a US payer perspective and lifetime time horizon. A three-state Markov model was developed representing clinical outcomes, quality-of-life, and healthcare resource use of patients treated with ipilimumab and BSC. Transitions between states were modeled using overall and progression-free survival data from the MDX010-20 trial. Utility data were from a melanoma-specific study of the health state preferences of the general population. Disease management costs expressed in 2011 US Dollars were based on healthcare resource use observed in a US retrospective medical chart study. Uncertainty was analyzed using one-way and probabilistic sensitivity analyses. Results: The gain in life years and QALYs from introducing ipilimumab over BSC were 1.88 years (95% CI ¼ 1.62– 2.20) and 1.14 (95% CI ¼ 1.01–1.34) QALYs, respectively, over the lifetime time horizon. The estimated incremental cost of treating with ipilimumab vs BSC was $146,716 (95% CI ¼ $130,992–$164,025). The estimated incremental cost-effectiveness ratios were $78,218 per life year gained and $128,656 per QALY gained. Ipilimumab was 95% likely to be cost-effective at a willingness-to-pay of $146,000/QALY. Limitations: Ipilimumab’s method of action causes a tumor response pattern that differs from the Response Evaluation Criteria in Solid Tumors upon which the model is based, leading to a potential under-estimate of quality-oflife of ipilimumab patients. Survival and QALY gains were related to the time horizon of the analysis. Sensitivity analyses indicated that qualitative conclusions regarding the cost-effectiveness of ipilimumab were unchanged when the method of quality adjustment and the time horizon were varied. Conclusion: The analysis shows that the estimated cost-effectiveness of ipilimumab is within what has been shown to be acceptable to payers for oncology products in the US.