Patient-Reported Falls and Fear of Falling in a Prospective Study of Droxidopa for Treatment of Neurogenic Orthostatic Hypotension (S18.002)
Objective: To assess the long-term effectiveness of droxidopa in reducing the risk of falling and fear of falling (FoF) in patients with neurogenic orthostatic hypotension (nOH)Background: nOH, a sustained blood pressure drop upon standing, may increase fall incidence, which can trigger FoF and future fall risk. Droxidopa is approved to treat nOH symptoms; fall benefits were suggested in pivotal trials.Design/Methods: Patients newly initiating droxidopa enrolled in an open-label, prospective cohort study. The number of falls in the past month and FoF were recorded at baseline, 1, 3, and 6 months using a falls questionnaire and the Short Falls Efficacy Scale-International. P values were calculated using paired t test for continuous measures and generalized estimating equations with multinomial distributions for categorical measures.Results: 179 patients were enrolled (Parkinson disease, 33%; autonomic failure without identifiable cause, 65%; other, 2%); 135, 119, and 105 remained at 1, 3, and 6 months. Patients reported fewer falls in the previous month at 1, 3, and 6 months vs baseline (P≤0.01, P=0.06, P<0.05, respectively). Among patients who fell ≥1 time at baseline, fewer falls were reported during droxidopa treatment; 59% fell ≥3 times at baseline and only 36% fell ≥3 times at 6 months. At 1, 3, and 6 months, significantly less concern about falling vs baseline were indicated by reductions in FoF scores (all P<0.0001) and categorical changes in level of concern about falling (all P<0.01).Conclusions: In this “real-world” study patients taking droxidopa were at a lower risk of falling after 1, 3, and 6 months of treatment. Those who fell ≥1 time in the prior month reported fewer falls per month vs baseline. Patients reported reduced FoF during treatment. These results suggest persistence of beneficial effects at 6 months of treatment, but conclusions are limited by the lack of a control cohort.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. 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. Shibao has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Lundbeck. Dr. Shibao has 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, LL ., 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. 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).
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.
PSS5 COMPARING TWO TREAT-AND-EXTEND ANTI-VEGF REGIMENS FOR MANAGING WET AGE-RELATED MACULAR DEGENERATION – IMPLICATIONS FOR THE NATIONAL HEALTH SYSTEM IN FRANCE
2019 Value in Healthhttps://www.doi.org/10.1016/j.jval.2019.09.2567
Non-profit drug research and development: the case study of Genethon
2019 J Mark Access Health Policyhttps://www.doi.org/10.1080/20016689.2018.1545514 Non-profit, ethics, orphan drugs, pharmaceutical industry, pricing, research and development,
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.
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.
Heterogeneous Recommendations for Oncology Products Among Different HTA Systems: A Comparative Assessment
2019 Recent Results Cancer Reshttps://www.doi.org/10.1007/978-3-030-01207-6_4 Decision Making, Medical Oncology/*economics, Resource Allocation, *Technology Assessment, Biomedical, Cost-effectiveness, Has, Health technology assessment, IQWiG/G-BA, Nice,
Rising budget constraints and demands for healthcare services create additional complexity within the decision process for resource allocation. Innovations and scientific progress have been shown to be key drivers of the increase in healthcare expenditures (1). In the context of rising medical care costs and limited resources, Health Technology Assessment (HTA) was developed as a tool to inform decision-making and to provide the rationalization behind these decisions driving resource allocation and spending for health technology products. Furthermore, HTA agencies make the decision-making process more transparent. The HTA approach involves evaluating multiple aspects of a new product’s value in order to maximize health gain provided within the setting of limited resources.
A systematic literature review on the use of platelet transfusions in patients with thrombocytopenia
2019 Hematologyhttps://www.doi.org/10.1080/16078454.2019.1662200 Thrombocytopenia, chronic liver disease, health care costs, immune thrombocytopenia, platelet count, platelet transfusion, thrombopoietin (receptor agonist[s]), transfusion reaction,
Objective: Investigate globally, current treatment patterns, benefit-risk assessments, humanistic, societal and economic burden of platelet transfusion (PT). Methods: Publications from 1998 to June 27, 2018 were identified, based on databases searches including MEDLINE(R); Embase and Cochrane Database of Systematic Reviews. Data from studies meeting pre-specified criteria were extracted and validated by independent reviewers. Data were obtained for efficacy and safety from randomized controlled trials (RCTs); data for epidemiology, treatment patterns, effectiveness, safety, humanistic and societal burden from real-world evidence (RWE) studies; and economic data from both. Results: A total of 3425 abstracts, 194 publications (190 studies) were included. PT use varied widely, from 0%-100% of TCP patients; 1.7%-24.5% in large studies (>1000 patients). Most were used prophylactically rather than therapeutically. 5 of 43 RCTs compared prophylactic PT with no intervention, with mixed results. In RWE studies PT generally increased platelet count (PC). This increase varied by patient characteristics and hence did not always translate into a clinically significant reduction in bleeding risk. Safety concerns included infection risk, alloimmunization and refractoriness with associated cost burden. Discussion: In RCTs and RWE studies there was significant heterogeneity in study design and outcome measures. In RWE studies, patients receiving PT may have been at higher risk than those not receiving PT creating potential bias. There were limited data on humanistic and societal burden. Conclusion: Although PTs are used widely for increasing PC in TCP, it is important to understand the limitations of PTs, and to explore the use of alternative treatment options where available.
Impact of Hypertension on Hospitalizations for Cardiovascular Diseases in a Worksite Population: An Epidemiologic Study Using Claims Data for Workers
2019 Am J Hypertenshttps://www.doi.org/10.1093/ajh/hpy177
BACKGROUND: Few of studies investigated the effect of antihypertensives among working population. We aimed to describe the impact of hypertension with and without antihypertensives on hospitalizations because of cardiovascular diseases (CVDs) in the Japanese working population. METHODS: This retrospective study included adults aged 40-64 years whose systolic and diastolic blood pressure (BP) readings taken between 2008 and 2015 were available in the Japanese claims database. Individuals were categorized as treated (T) or untreated (UT) depending on their hypertension treatment history. Time to first CVD-related hospitalization was compared among individuals with different BP levels using Cox models that included baseline characteristics with either baseline BP level or a time-dependent variable reflecting BP changes over time. RESULTS: Of 740,784 UT individuals (male: 61.9%, mean age: 47.9 years), 2,121 individuals were hospitalized due to a CVD over a 957.3-day average follow-up. Among 72,828 T individuals (male: 74.3%, mean age: 53.7 years), the corresponding figure was 470 individuals over an 813.4-day average follow-up. The risk of hospitalization increased with baseline hypertension severity among UT (hazard ratios [HRs] = 1.93, 2.82, and 6.32 for grade 1, grade 2, and grade 3, respectively; P < 0.0001, reference: optimal) but not among T individuals. UT individuals with hypertension at any given time had a significantly higher probability of hospitalization compared to nonhypertensive individuals (HR = 1.74, P < 0.0001). CONCLUSIONS: This study adds quantitative evidence on the impact of hypertension on the risk of CVD-related hospitalization in the Japanese working population. It suggests that antihypertensive treatment had beneficial effects in this relatively young, working population aged 40-64 years.
PCV67 BUDGET IMPACT OF RIVAROXABAN 2.5 MG IN COMBINATION WITH ASPIRIN IN TREATMENT OF PATIENTS WITH CORONARY ARTERY DISEASE (CAD) OR PERIPHERAL ARTERY DISEASE (PAD) IN GREECE
2019 Value in Health
Impact of methodological choices on a meta-analysis of real-world evidence comparing non-vitamin K antagonist oral anticoagulants with vitamin K antagonists for the treatment of patients with non-valvular atrial fibrillation
2019 Curr Med Res Opinhttps://www.doi.org/10.1080/03007995.2019.1647020 Non-valvular atrial fibrillation, anticoagulation, meta-analysis, real-world evidence, vitamin K antagonist,
Objective The aim of this study was to investigate the impact of methodological choices in a meta-analysis of real-world evidence (RWE) comparing three non-vitamin K antagonist oral anticoagulants with vitamin K antagonists (VKAs) for the treatment of patients with non-valvular atrial fibrillation (NVAF). Methods The meta-analysis was based on a systematic review of RWE studies enrolling incident and prevalent patients aged >/=18 years with NVAF and receiving either rivaroxaban, dabigatran, apixaban, or a VKA. Five different scenarios were considered to explore the impact of the initial meta-analysis assumptions: (1) using studies that involved only incident patients; (2) excluding studies that only reported crude values and did not consider any adjustment; (3) including all studies independently of possible database overlap; (4) using studies with data on different dosages for rivaroxaban and dabigatran; and (5) assigning quality weights to studies to assess quality of reporting. These scenarios were run on three outcomes: ischemic stroke (IS), myocardial infarction (MI), and intracranial hemorrhage (ICH). Results Across all scenarios, rivaroxaban was associated with significantly lower risks of IS and ICH than VKAs. In most scenarios, dabigatran was associated with significantly lower risks of IS and ICH. In all scenarios, apixaban was associated with a significantly lower risk of ICH. Conclusions Sensitivity analyses showed the impact of similar assumptions was different depending on the outcome, and the drug considered. The development of recommendations and guidelines for the inclusion of RWE in meta-analyses could prove useful in evaluating the effectiveness of health care interventions.