Abstract Aim To characterize patients with neurogenic bladder (NGB), their treatment patterns, healthcare resource utilization, and associated costs based on records from a primary care database in the United Kingdom. Methods This was a retrospective, descriptive, observational study of anonymized data from the Clinical Practice Research Datalink and Hospital Episode Statistics databases (selection period, 1 January 2004 to 31 December 2016). Adults with a definitive or probable diagnosis of NGB and ≥1 referral to a urologist were included. Results The study cohort included 3913 patients with definitive (n?=?363) or probable (n?=?3550) NGB. Patients had a mean of 8.6 (standard deviation [SD], 7.6) comorbidities, and mean Anticholinergic Cognitive Burden Scale score of 6.6 (SD, 5.9). During 12 months? follow-up, urinary tract infection (UTI) and urinary incontinence were the most common complications. Most patients (92.2%) received ≥1 prescription for an antimuscarinic agent or mirabegron, and 53.9% of patients received prescriptions for UTI-specific antibiotics. The mean number of visits to a general practitioner for any cause was 67.7 (SD, 42.6) per individual. Almost half (46.7%) of the study cohort visited a specialist during the 12-month follow-up period, and 11.0% had ≥1 hospital admission. Total mean per patient costs for healthcare resource utilization was £2395. Conclusions The burden of illness, healthcare resource needs, and associated costs among patients with NGB are considerable. Drug prescribing patterns are consistent with the symptoms and complications of NGB, although increased awareness of drugs with anticholinergic activity among prescribers may help to reduce the cumulative anticholinergic burden in this vulnerable population.

The goal of the treatment of a disease has moved from treating organs and diseases through symptoms, biological parameters and imaging towards treating a human being as a whole. The treatments should deliver benefits that patients can personally perceive. However, the patient’s perspective does not always match the one of those surrounding them. Illustratively, patients’ symptom assessments are more predictable for daily health status, whereas clinicians’ symptom measurements are more related to clinical outcomes. The term, patient-reported outcomes (PROs), includes any data that are reported directly by the patient without an intermediary, such as a family member or a healthcare professional. The use of PROs in oncology trials is increasing and the U.S. Food and Drug Administration has published guidelines on the review and evaluation of PROs. However, while PROs are increasingly used in clinical trials, they are rarely used in daily clinical practice. Further, healthcare payers are concerned with issues related to relevance, quality, and interpretability of these outcomes.

Objective: To assess long-term efficacy of droxidopa for treatment of neurogenic orthostatic hypotension (nOH) in patients concomitantly on dopa decarboxylase (DDC) inhibitors (DDCIs)Background: nOH is a non-motor symptom of Parkinson disease (PD). Treatment of PD frequently includes DDCIs. Droxidopa, a treatment for symptomatic nOH, is converted to norepinephrine by DDC.Design/Methods: Patients were enrolled in a prospective cohort study comparing outcomes before initiation of droxidopa and after 1, 3, and 6 months of treatment. Patients recorded monthly number of falls using a questionnaire and dizziness/lightheadedness using Orthostatic Hypotension Symptom Assessment, Item 1 (OHSA-1). Post-hoc analysis compared outcomes in patients on DDCIs vs without DDCIs. “DDCI treatment” defined as receiving carbidopa alone or in combination with levodopa or entacapone. Influence of DDCIs was compared across time points using generalized linear mixed models adjusting for repeated measures within individuals.Results: 168 patients were included (PD: 32.1%, autonomic failure: 66.1%, other: 1.8%). 61.8% of patients on DDCIs (n=55) reported ≥1 fall at baseline vs 46.9% of patients not on DDCIs (n=113). Patients on DDCIs experienced significant reduction in fall rate (30.1%, 36.4 point reduction) and improvement in OHSA-1 scores (−1.8) from baseline to 6 months (both P<0.05). Patients not on DDCIs also experienced significant OHSA-1 scores improvement (−1.9, P<0.05), but fall rate reduction was not significant (39.8%, 6.2 point reduction, P=0.49). The differences in change from baseline to 6 months for fall risk and OHSA-1 score were not significant between groups (P=0.15, P=0.96, respectively). DDCI users (66.6%) had a greater prevalence of >1 falls vs nonusers (46.6%) at baseline.Conclusions: These post-hoc analyses suggest that DDCI users and nonusers experienced reductions in fall rate and dizziness/lightheadedness with droxidopa. Additional studies are needed to examine the impact of DDCIs on droxidopa because the current study was not powered for subgroup analyses and data were patient self-reported.Disclosure: Dr. Kymes has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Lundbeck and CVS Health. Dr. Kymes holds stock and/or stock options in Lundbeck that is greater than $10,000 in value which sponsored research in which Dr. Kymes was involved as an investigator. Dr. Francois has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Lundbeck and Creativ-Ceutical. Dr. Francois holds stock and/or stock options in Lundbeck. Dr. Sullivan has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Lundbeck. Dr. McLeod has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Xcenda, LLC. Dr. Duhig has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Xcenda, LLC. Dr. Ogbonnaya has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Xcenda LLC. Dr. Quillen has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Xcenda LLC. Dr. Cannon has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Lundbeck. Dr. Biaggioni has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Lundbeck, Theravance Biopharma. Dr. Biaggioni has received research support from Lundbeck (investigator-initiated grant) and Theravance Biopharma (Clinical Trial). Dr. Shibao has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Lundbeck. Dr. Shibao h s received research support from Lundbeck. Dr. Yue has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Xcenda LLC. Dr. Hauser has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with AbbVie Inc., Acorda Therapeutics, Academy for Continued Healthcare Learning, Acadia Pharmaceuticals, Inc., Adamas Pharmaceuticals, AstraZeneca, ApoPharma, Back Bay Life Science, Biotie Therapies, Bracket, Cerecor, Inc., ClearView Healthcare Partners, ClinicalMind Medical and Therapeutic Communications, CNS Ratings, LLC., Cowen and Company, Cynapsus Therapeutics, DDB Health LLC, Decision Resources Group (DRG), Eli Lilly & Company, eResearch Technology, Inc., Expert Connect, Extera Partners, GE Healthcare, Health Advances, HealthLogix, Health and Wellness Partners, Huron Consulting Group, Impax Laboratories, Impel Neuropharma, Intec Pharma Ltd., Jazz Pharmaceuticals, Kashiv Pharma LLC, Kyowa Kirin Pharmaceutical Development, Ltd., LCN Consulting, LifeMax, Life Sciences, Lundbeck LLC, The Lockwood Group, MEDACorp, Medscape, Medtronic, Michael J. Fox Foundation, Mitsubishi Tanabe Pharmaceuticals, Movement Disorder Society, National Institutes of Health (NIH), Neurocea LLC, Neurocrine Biosciences, Neuroderm, Neuropore Therapies, Orbes Medical Group, Outcomes Insights, Parkinson Study Group, Peerview Press, Pennside Partners, Pfizer, Inc., Pharma Two B, Ltd, Phase Five Communications, Prescott Medical Group, Prexton Therapeutics, Prilenia Development Ltd., Projects in Knowledge, Putnam Associates, Quintiles, RMEI Medical Education for Better Outcomes, SAI Med Partners LLC, Sarepta Therapeutics, Schlesinger Associates, Scion Neurostim, LLC, Seagrove Partners, LLC, Slingshot Insights, Sunovion Pharmaceuticals, Inc., Sun Pharma, Teva Pharmaceutical Industries, US WorldMeds, Vista Research, WebMD, Windrose Consulting Group. Dr. Hauser has received royalty, license fees, or contractual rights payments from the University of South Florida. Dr. Hauser has received research support from AbbVie Inc., Acorda Therapeutics, AstraZeneca, Axovant Sciences , Biogen Inc., Cavion, Enterin Inc., Impax Laboratories, LLC., Intec Pharma Ltd, Jazz Pharmaceuticals, NeuroDerm Ltd., Lundbeck, Michael J Fox Foundation for Parkinson’s Research, F. Hoffman-La Roche, Dart NeuroScience LLC, Prexton Therapeutics, Revance Therapeutics Inc., Sunovion Pharmaceuticals.

Non-profit drug research and development (R&D) has the potential to deliver innovative treatments at affordable prices. Using the case study methodology, we discuss some ethical and economic issues, including the possible impact of non-profit companies on innovation efforts from for-profit firms. Like other non-profits, Genethon is willing to adopt an ethical attitude toward their donors by pricing their products affordably. It remains to be seen if the approach to internalize the marketing authorization, manufacturing and distribution activities prove to be efficient and sustainable. Also, the firm faces an ethical dilemma because lower prices of innovative drugs can dry the for-profit R&D in the area and prevent patient access to future innovations.

BACKGROUND: The interaction between positive, negative and depressive symptoms experienced by people with schizophrenia is complex. We used longitudinal data to test the hypothesis that depressive symptoms mediate the links between positive and negative symptoms. METHODS: We analyzed data from the European Schizophrenia Cohort, randomly sampled from outpatient services in France, Germany and the UK (N = 1208). Initial measures were repeated after 6 and 12 months. Depressive symptoms were identified using the Calgary Depression Scale for Schizophrenia (CDSS), while positive and negative symptoms were assessed with the Positive and Negative Syndrome Scale (PANSS). Latent variable structural equation modelling was used to investigate the mediating role of depression assessed at 6 months in relation to the longitudinal association between positive symptoms at baseline and negative symptoms at 12 months. RESULTS: We found longitudinal associations between positive symptoms at baseline and negative symptoms at 12 months, as well as between both of these and CDSS levels at 6 months. However depression did not mediate the longitudinal association between PANSS scores; all the effect was direct. CONCLUSIONS: Our findings are incompatible with a mediating function for depression on the pathway from positive to negative symptoms, at least on this timescale. The role of depression in schizophrenic disorders remains a challenge for categorical and hierarchical diagnostic systems alike. Future research should analyze specific domains of both depressive and negative symptoms (e.g. motivational and hedonic impairments). The clinical management of negative symptoms using antidepressant treatments may need to be reconsidered.

OBJECTIVE: To compare treatment patterns, persistence and adherence between fixed-dose combinations (FDCs) and two-pill combinations (TPCs) of oral antidiabetic drug (OAD) classes in Japanese patients with type 2 diabetes mellitus (T2DM) using administrative claims databases (Japan Medical Data Center [JMDC] and Medical Data Vision [MDV]). METHODS: This was a retrospective, longitudinal cohort analysis conducted between 2011 and 2015, in patients with T2DM receiving OADs as FDC or TPC. Outcomes included prescribing patterns, treatment persistence and adherence. RESULTS: Data from 3474 and 3066 patients receiving FDCs, and 4325 and 5192 patients receiving TPCs from the JMDC and MDV databases, respectively, was extracted. The most common OAD combination received by over half of all patients was dipeptidyl peptidase-4 inhibitor (DPP-4i) + thiazolidinediones (TZDs) (64.1% [JMDC] and 70.5% [MDV]). Overall, 12-month persistence rates were higher in patients receiving FDCs compared with TPCs (70.4 vs. 66.2% [JMDC], 75.6 vs. 55.7% [MDV]). In the JMDC population receiving FDCs or TPCs, persistence rates were highest with DPP-4i schedules (67.5-83.5%). Median time to discontinuation was significantly longer with biguanide + TZD, and DPP-4i + TZD FDC schedules (p < .05) than TPC; adherence rates were >/=80% across all antidiabetic drug classes in both database populations. CONCLUSIONS: Persistence with and adherence to OADs in Japanese patients with T2DM were greater with FDCs than with TPCs, which may suggest increased patient satisfaction due to reduced treatment burden. Further studies are warranted to investigate the impact of adherence and persistence of FDCs of OADs on glycemic control.

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.