Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
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In 2012, the National Institute for Health and Care Excellence assessed dasatinib, nilotinib, and standard-dose imatinib as first-line treatment of chronic phase chronic myelogenous leukemia (CML). Licensing of these alternative treatments was based on randomized controlled trials assessing complete cytogenetic response (CCyR) and major molecular response (MMR) at 12 months as primary end points. We use this case study to illustrate the validation of CCyR and MMR as surrogate outcomes for overall survival in CML and how this evidence was used to inform National Institute for Health and Care Excellence's recommendation on the public funding of these first-line treatments for CML. ⋯ This case study illustrates the consideration of surrogate outcome evidence in health technology assessment. Although it is often recommended that the acceptance of surrogate outcomes be based on randomized controlled trial data demonstrating an association between the treatment effect on both the surrogate outcome and the final outcome, this case study shows that policymakers may be willing to accept a lower level of evidence (i.e., observational association).
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Randomized Controlled Trial Multicenter Study
Item-level psychometric properties for a new patient-reported psoriasis symptom diary.
This research evaluated the psychometric properties of a new Psoriasis Symptom Diary, identified diary responder definitions for use in determining whether a patient has experienced clinically meaningful change, and refined diary item content for use in future clinical trials. ⋯ The 16-item Psoriasis Symptom Diary demonstrated favorable psychometric properties and is a brief, useful tool for measuring patient-based symptoms and the impact of chronic plaque psoriasis.
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Comparative Study
Cost-utility of metal-on-metal hip resurfacing compared to conventional total hip replacement in young active patients with osteoarthritis.
Metal-on-metal hip resurfacing arthroplasty (MoM HRA) has emerged as an alternative to total hip arthroplasty (THA) for younger active patients with osteoarthritis (OA). Birmingham hip resurfacing is the most common MoM HRA in Alberta, and is therefore compared with conventional THA. ⋯ The results show that, on average, MoM HRA was preferred to THA for younger and male patients, but THA is still a reasonable option if the patient or clinician prefers given the small absolute differences between the options and the confidence ellipses around the cost-effectiveness estimates.
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This article aims to provide an overview of the current literature focusing on the reimbursement of personalized medicine across the European Union. The article starts by describing types of perspectives that are possible (general public, patient, payer, provider, service commissioner, and policymaker). ⋯ The article touches on the strategic challenges and the use of economic evidence to introduce personalized medicine from a health policy perspective. The article also draws on empirical studies that have explored patients' and clinicians' views of examples of personalized medicine to illustrate the challenges for developing patient-centered and timely health care services.
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Reduced mortality with low-dose computed tomography (LDCT) lung cancer screening was demonstrated in a large randomized controlled study of high-risk individuals. Cost-effectiveness must be assessed before routine LDCT screening is considered. We aimed to evaluate the cost-effectiveness of LDCT lung cancer screening in Israel. ⋯ Our analysis suggests that baseline LDCT lung cancer screening in Israel presents a good value for the money and should be considered for inclusion in the National List of Health Services financed publicly.