Background: Little is known about the costs associated with vitamin K antagonist (VKA) treatment in patients with non-valvular atrial fibrillation (NVAF) in France.
Objectives: To evaluate monthly per-patient costs attributable to VKA treatment in NVAF patients from a French societal perspective.
Study design: Retrospective data were obtained from 7 international normalised ratio (INR) monitoring centres in France. Patients older than 18 years of age with NVAF treated with VKA were recruited. Additional patient-level data assessing resource use corresponding with VKA treatment were collected via self-completed questionnaires. Unit costs applicable to 2015 were multiplied by resource use and summed to generate VKA treatment costs.
Results: 363 patients were included; 53% were men. The majority of patients received fluindione (72%). The number of INR tests per patient per month was 1.69 (95% CI, 1.59–1.80). The monthly patient cost was €39.72 (€36.23–43.21) from the French societal perspective. Direct medical costs comprised 76% of overall costs, with drug costs representing 7.4% (€2.4); direct non-medical and indirect costs comprised 10% and 14% respectively.
Conclusions: Costs associated with VKA treatment in NVAF cannot be estimated only with drug costs. When direct and indirect attributable costs associated with VKA treatment are considered, the VKA treatment costs are more substantial.

Background: Drug prices in the United States are the highest in the world, restricting access to the domestic lower income population. President Trump campaigned heavily on promises to reduce drug prices.Methods: A literature review was conducted through PUBMED, EMBASE, Media and grey literature to consolidate and analyze publications addressing President Trump’s promises from the campaign trail as well as the shortcomings and achievements of the Trump administration.Results: Major promises ranged from repealing and replacing Obamacare, expanding coverage, allowing cross-state insurance purchasing, and reducing drug prices. Main accomplishments of the Trump administration have been two executive orders (13765 and 13813), the proposition of the American Health Care Act, and the passing of the Tax Cuts and Jobs Act of 2017. The American Patients First blueprint further revealed the administration’s strategies for lowering drug prices. The administration has also engaged in unconventional strategies, such as via bilateral leverage directly with trade partners.Conclusion: The Trump administration has not yet been able to fulfill the major campaign promises, primarily the ones requiring legislative and/or administrative action. If enough legislative action is accomplished to implement the Trump administration’s promises, the effects on the pharmaceutical industry could be direct, yet minimal, unless Medicare can directly negotiate with manufacturers.

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.

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 /=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.

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.

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.

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.

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 (/=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.