Management and clinical practice of multiple face and scalp actinic keratosis in France
2019 J Mark Access Health Policyhttps://www.doi.org/10.1080/20016689.2019.1605787 Actinic keratosis, cancerization zone, cryotherapy, face and scalp, photodynamic therapy, topical treatments,
Background: Actinic keratosis (AK) is characterized by the occurrence of thick and scaly skin areas caused by damage from ultraviolet radiation. The management of AK aims to reduce lesions and prevent their recurrence by regular monitoring. French guidelines, last updated in 2009, reflect European guidelines for the management of face and scalp AK. However, they do not address all current, available options. Objective: To assess the management of face and scalp AK in French clinical practice. Methods: A two-part online questionnaire comprising a survey among French dermatologists and an analysis of patient medical records was performed to describe AK patients treated with topical therapy, patients’ profiles, and characteristics of the affected lesion areas. Results: Decisions for topical treatments for face and scalp AK made by dermatologists were mainly driven by the lesion size. According to the last 10 patients they have seen, dermatologists were prescribing physical therapy in 53% of the cases, a combination of topical and physical therapy in 27% and topical only in 20%. Patient records revealed the average surface area targeted for treatment was 139 +/- 113cm(2). Conclusions: Discrepancies between the guidelines on the treatment of face and scalp AK and clinical practice exist. Further research may help to standardize the treatment.
Treatment patterns, persistence and adherence rates in patients with type 2 diabetes mellitus in Japan: a claims-based cohort study
2019 BMJ Openhttps://www.doi.org/10.1136/bmjopen-2018-025806 adherence, administrative claims-based study, antidiabetic drug therapy, dipeptidyl peptidase-4 inhibitors, persistence, type 2 diabetes, Nippon Boehringer Ingelheim, Eli Lilly Japan K.K., Kissei Pharmaceutical,, Medtronic Japan, MSD, Novartis Pharma KK, Novo Nordisk Pharma, Sanofi KK and, Takeda Pharmaceutical and contract research fees for collaborative research with, the Japan Diabetes Foundation. HK, KK, AO and YS are employees of Takeda, Pharmaceutical. SH was an employee of Takeda Pharmaceutical at the time the study, was conducted. FG and YO are employees of Creativ-Ceutical K.K.,
OBJECTIVE: To determine real-world trends in antidiabetic drug use, and persistence and adherence, in Japanese patients with type 2 diabetes mellitus (T2DM). DESIGN: Retrospective evaluation of administrative claims data (2011-2015) using the Japan Medical Data Center (JMDC) and Medical Data Vision (MDV) databases. SETTING: Analysis of two administrative claims databases for Japanese patients with T2DM. PARTICIPANTS: Adults (aged >/=18 years) with an International Classification of Diseases, 10th Revision code of T2DM and at least one antidiabetic drug prescription. MAIN OUTCOME MEASURES: Treatment patterns in untreated (UT) or previously treated (PT) patients receiving antidiabetic therapy; persistence with treatment at 12 months; adherence to treatment at 12 months. RESULTS: 40 908 and 90 421 patients were included from the JMDC and MDV databases, respectively. The most frequently prescribed therapy at the index (first prescription) date was dipeptidyl peptidase-4 inhibitor (DPP-4i) in UT patients (JMDC: 44.0%, MDV: 54.8%) and combination therapy in PT patients (74.6%, 81.1%). Most common combinations were DPP-4i plus: biguanide (BG; 11.4%, 10.9%), sulfonylurea (SU; 8.4%, 11.0%) or BG+SU (7.8%, 9.1%). In UT or PT patients from either database whose index prescription was for any antidiabetic drug class(es) other than DPP-4i, the most frequent add-on or switch was to DPP-4i. 12-month persistence with index monotherapy was highest with DPP-4i and BG. Adherence was high (>/=80%) for all monotherapy schedules, except insulin and glucagon-like peptide-1 agonist, and for the five most frequent two-drug and three-drug combinations. Persistence was greater in elderly UT patients and in those receiving =5 medications, but comparatively worse in UT patients with >/=3 index antidiabetic drug classes. CONCLUSIONS: The findings indicate that DPP-4i is the most commonly used antidiabetic drug class in Japanese patients with T2DM, and persistence and adherence to this antidiabetic drug class are high.
Oncology from an HTA and Health Economic Perspective
2019 Recent Results Cancer Reshttps://www.doi.org/10.1007/978-3-030-01207-6_3 Cost-Benefit Analysis, Decision Making, Medical Oncology/*economics, *Models, Economic, Neoplasms/*economics, Randomized Controlled Trials as Topic, Research Design, *Technology Assessment, Biomedical, Cost-effectiveness, Decision-making, Health technology assessment, Oncology drugs,
In this chapter, we will present and discuss the challenges of assessing oncology products from a health economic perspective. We will provide a brief introduction on the need for economic evaluation in health care and focus on cost-effectiveness and comparative aspects of the evaluation of oncology products, which are of paramount interest to HTA decision-making bodies using economic evaluation in their decision-making framework. As the burden of oncology is well-documented, we do not discuss it in detail here. Before we address the specific issue of oncology, we will briefly define the critical aspects of HTA assessment and also define what a cost-effectiveness analysis is and why economic modelling is the most appropriate tool to assess the cost-effectiveness of oncology products. We will touch upon the prices of oncology drugs and the questions that high prices raise regarding funding and availability. We then present an overview of the general structure of an oncology cost-effectiveness model. Usually, this is quite simple, representing response, progression, advanced-stage disease and death. Despite the relative simplicity of these models, some issues may render the evaluation more complex; we will touch upon these in this chapter: Issue with clinical inputs due to the design of randomised clinical trials (e.g. cross-over designs involving a treatment switch) Need for survival extrapolation and limitations of current parametric models Rare conditions with limited economic and comparative evidence available High pace of clinical development Finally, we will conclude with a discussion of the uncertainty around the evaluation of oncology products and the major evolution expected in health economics in oncology.
Cost-effectiveness of fluocinolone acetonide implant (ILUVIEN(R)) in UK patients with chronic diabetic macular oedema considered insufficiently responsive to available therapies
2019 BMC Health Serv Reshttps://www.doi.org/10.1186/s12913-018-3804-4 Cost-effectiveness, Diabetic macular oedema, Fluocinolone acetonide implant, Incremental cost-effectiveness ratio, Treatment cost,
BACKGROUND: Diabetic macular oedema (DMO) may lead to visual loss and blindness. Several pharmacological treatments are available on the National Health Service (NHS) to United Kingdom patients affected by this condition, including intravitreal vascular endothelial growth factor inhibitors (anti-VEGFs) and two types of intravitreal steroid implants, releasing dexamethasone or fluocinolone acetonide (FAc). This study aimed to assess the value for money (cost-effectiveness) of the FAc 0.2 mug/day implant (ILUVIEN(R)) in patients with chronic DMO considered insufficiently responsive to other therapies. METHODS: We developed a Markov model with a 15-year time horizon to estimate the impact of changes in best-corrected visual acuity in DMO patients on costs and quality-adjusted life years. The model considered both eyes, designated as the “study eye”, defined at model entry as phakic with an ongoing cataract formation or pseudophakic, and the “fellow eye”. The model compared the FAc 0.2 mug/day implant with a 700 mug dexamethasone implant (pseudophakic patients only) or with usual care, defined as a mixture of laser photocoagulation and anti-VEGFs (phakic and pseudophakic patients). Costs were estimated from the perspective of the NHS and Personal Social Services; full NHS prices were used for drugs. RESULTS: In patients who were pseudophakic at baseline, at 36 months, the FAc implant provided an additional gain of 4.01 and 3.64 Early Treatment Diabetic Retinopathy Study (ETDRS) letters compared with usual care and the dexamethasone implant, respectively. Over the 15-year time horizon, this translated into 0.185 additional quality-adjusted life years (QALYs) at an extra cost of pound3066 compared with usual care, and 0.126 additional QALYs at an extra cost of pound1777 compared with dexamethasone. Thus, incremental cost-effectiveness ratios (ICERs) were pound16,609 and pound14,070 per QALY gained vs. usual care and dexamethasone, respectively. In patients who were phakic at baseline, the FAc 0.2 mug/day implant provided an additional gain of 2.96 ETDRS letters at 36 months compared with usual care, which, over 15 years, corresponded to 0.11 additional QALYs at an extra cost of pound3170, resulting in an ICER of pound28,751 per QALY gained. CONCLUSION: The FAc 0.2 mug/day implant provided good value for money compared with other established treatments, especially in pseudophakic patients.
Measuring the health-related quality of life in young children: how far have we come?
2019 Journal of Market Access & Health Policyhttps://www.doi.org/10.1080/20016689.2019.1618661
Real-world clinical evidence on rivaroxaban, dabigatran, and apixaban compared with vitamin K antagonists in patients with nonvalvular atrial fibrillation: a systematic literature review
2019 Expert Rev Pharmacoecon Outcomes Reshttps://www.doi.org/10.1080/14737167.2018.1518134 Administration, Oral, Anticoagulants/adverse effects/pharmacology/*therapeutic use, Atrial Fibrillation/complications/*drug therapy, Dabigatran/adverse effects/pharmacology/therapeutic use, Hemorrhage/chemically induced, Humans, Pyrazoles/adverse effects/pharmacology/therapeutic use, Pyridones/adverse effects/pharmacology/therapeutic use, Rivaroxaban/adverse effects/pharmacology/therapeutic use, Stroke/etiology/*prevention & control, Vitamin K/antagonists & inhibitors, Warfarin/adverse effects/pharmacology, Anticoagulation, direct oral anticoagulants, nonvalvular atrial fibrillation, real-world evidence, stroke prevention,
INTRODUCTION: Several comparative real-world effectiveness studies on direct oral anticoagulants (DOACs) have been conducted, but an overview of the available evidence remains to be developed, which could provide a better understanding of the value of DOACs relative to vitamin K antagonists (VKAs). Areas covered: A systematic literature review was conducted on the available real-world evidence (RWE) of three DOACs (rivaroxaban, dabigatran, and apixaban) compared with VKAs (e.g. warfarin), in patients with nonvalvular atrial fibrillation (NVAF).This systematic literature review included RWE published up to December 2016. Studies with > 50 patients reporting on incident and prevalent NVAF cases were included. The following databases were searched: Medline, Embase, and the Cochrane Library. Outcomes of interest included thromboembolic events, all-cause mortality, bleeding events, and nonpersistence. Of the 562 RWE DOACs articles retrieved, 49 presented results for rivaroxaban versus VKAs, 79 for dabigatran versus VKAs, and 18 for apixaban versus VKAs. Substantial heterogeneity was found across patient population, outcome definition, and follow-up period. Major bleeding, ischemic stroke, and intracranial hemorrhage were the most frequent outcomes analyzed. Expert commentary: Overall, the RWE studies were aligned with the Phase 3 trials. However, conflicting results were reported for several outcomes of interest.
Adherence and persistence to hyperlipidemia medications in patients with atherosclerotic cardiovascular disease and those with diabetes mellitus based on administrative claims data in Japan
2019 Atherosclerosishttps://www.doi.org/10.1016/j.atherosclerosis.2018.12.026 Adherence, Cardiovascular disease, Diabetes mellitus, Hyperlipidemia, Persistence,
BACKGROUND AND AIMS: Real-world data on treatment patterns in Japanese hyperlipidemia patients with diabetes mellitus (DM) or prior atherosclerotic cardiovascular diseases (ASCVD) are lacking. METHODS: This is a retrospective, longitudinal cohort analysis of administrative claims data (Japan Medical Data Center [JMDC] and Medical Data Vision [MDV] databases) for patients prescribed a new hyperlipidemia medication between 2014 and 2015. Patients were followed for >/=12 months. Outcomes included prescribing patterns, persistence (discontinuations), and adherence (proportion of days covered). RESULTS: Data were analyzed for 11,718 and 27,746 DM, and 4101 and 14,356 ASCVD patients from the JMDC and MDV databases, respectively. Among previously-untreated patients, index prescriptions were primarily for moderate statins in the DM (JMDC: 74.7%, MDV: 77.5%) and ASCVD (JMDC: 75.4%, MDV: 78.5%) sub-cohorts. Combinations were rarely prescribed (=2.5%). Previously-treated patients were most frequently prescribed combinations in the DM (JMDC: 46.7%, MDV: 53.6%) and ASCVD (JMDC: 49.3%, MDV: 53.3%) sub-cohorts. Intensive statins were rarely used by previously-untreated (=1%) or previously-treated (=8%) patients in either sub-cohort. Approximately half of previously-untreated patients discontinued hyperlipidemia therapy within 12 months. Adherence was >/=80% across most drug classes. CONCLUSIONS: Many Japanese hyperlipidemia patients with DM or ASCVD are prescribed single-agent lipid-lowering therapy. Use of intensive therapy is lower than expected, and is suggestive of under-treatment. The low persistence rates are concerning, and warrant further study.
The Burden of Allergic Rhinitis and Impact of GRAZAX® Grass Allergy Immunotherapy Treatment on Quality of Life in Germany and The Netherlands: Results from A Qualitative Study
2019 Int J Aller Medicationshttps://www.doi.org/10.23937/2572-3308.1510038
Number needed to treat based on real-world evidence for non-vitamin K antagonist oral anticoagulants versus vitamin K antagonist oral anticoagulants in stroke prevention in patients with non-valvular atrial fibrillation
2019 J Med Econhttps://www.doi.org/10.1080/13696998.2019.1606001 C13, C31, Non-vitamin K antagonists, atrial fibrillation, meta-analysis, number needed to treat, real-world evidence, stroke prevention, vitamin K antagonists,
Aims: Non-vitamin K antagonist oral anticoagulants (NOACs) and vitamin K antagonists (VKAs) are used to prevent stroke in patients with atrial fibrillation (AF). This paper aimed to evaluate the clinical efficacy and safety of NOACs when compared to VKAs by calculating the number needed to treat (NNT) at 2 years using incidence rates and hazard ratios (HRs) derived from a meta-analysis of studies conducted in real-world settings. Materials and methods: HRs were sourced from a published systematic literature review and a meta-analysis of real-world evidence on the use of NOACs vs VKAs. Rivaroxaban, dabigatran, and apixaban vs VKAs were investigated. The efficacy outcomes included: a composite of ischaemic stroke and systemic embolism (IS/SE), ischaemic stroke (IS), and all-cause mortality. The safety analysis assessed major bleeding and intracranial haemorrhage (ICH). Results: Superiority of NOACs vs VKAs was observed in 10/15 comparisons. Treating patients with rivaroxaban and dabigatran was associated with a reduced risk of IS and all-cause mortality compared to VKAs, with one death prevented every 22 and 32 patients, respectively, and one IS prevented every 206 and 166 patients, respectively. Rivaroxaban was significantly associated with a reduced risk of IS/SE compared to VKA (NNT: 107). No significant differences were observed between apixaban and VKAs. Dabigatran and apixaban were associated with a reduced risk of major bleeding compared to VKA (NNT: 59 and 38, respectively). No significant difference was observed between rivaroxaban and VKAs regarding major bleeding. Rivaroxaban, dabigatran, and apixaban were significantly associated with a reduced risk of ICH (NNT: 205, 115, and 108, respectively). Limitations: Heterogeneity in definitions of major bleeding across studies. Conclusions: The NNT calculation, when approached and interpreted properly, is a practical measure of the effectiveness of a treatment. The calculation based on HRs showed that NOACs are safe and effective alternatives to VKAs in real life.
Cost-effectiveness analyses using real-world data: an overview of the literature
2019 J Med Econhttps://www.doi.org/10.1080/13696998.2019.1588737 I10, I19, economic evaluation, literature review, modeling, real-world,
OBJECTIVES: Real-world evidence (RWE) may provide good estimates of absolute event probabilities and costs in patients in actual clinical practice, but their use in decision-analytic models poses many challenges. A literature review based on a systematic search was conducted to summarize the limitations of using RWE in decision-analytic modeling reported in the literature but also to identify existing recommendations about real-world modeling. METHODS: A literature search was performed on Medline and Embase databases, as well as relevant websites. No restrictions in language or geographical scope were imposed. RESULTS: A total of 14 references were included. RWE is recognized as a valuable source of data for market access and reimbursement, and as a complement to clinical trial evidence for treatment pathways, resource use, long-term natural history, and effectiveness. The main limitations identified in the literature were: confounding bias, missing data, lack of accurate data related to drug exposure and outcomes, errors during the record-keeping process, protection of private data, and insufficient numbers of patients. Although most submission guidelines recognized the potential biases associated with RWE, guidance on the appropriate methods to deal with these biases, and approaches to review different relevant evidence to inform model development, were scarce. Several initiatives have attempted to provide guidance on the use of RWE in decision-modeling. CONCLUSIONS: RWE is likely to be particularly valuable for informing healthcare policy-makers when formulating appropriate treatment pathways, encouraging the optimal allocation of scarce resources, and improving aggregate patient outcomes. However, little guidance is available on the relative merits of using efficacy and/or effectiveness evidence in Health Technology Appraisal submissions. Further research is needed to better understand these methods and their potential applications in a broader range of scenarios and simulation studies, and their impact on economic modeling.