A5033526386- Health Systems, Economic Evaluations, Quality of Life
- Economic and Financial Impacts of Cancer
- Statistical Methods in Clinical Trials
- Lung Cancer Treatments and Mutations
- Pharmaceutical Economics and Policy
- Lymphoma Diagnosis and Treatment
- Cancer Immunotherapy and Biomarkers
- CAR-T cell therapy research
- Biosimilars and Bioanalytical Methods
- Healthcare cost, quality, practices
- Hemophilia Treatment and Research
- Melanoma and MAPK Pathways
- Polyomavirus and related diseases
- Gastrointestinal motility and disorders
- Advanced Causal Inference Techniques
- Global Health Care Issues
- Gastroesophageal reflux and treatments
- Chronic Lymphocytic Leukemia Research
- Lung Cancer Diagnosis and Treatment
- Healthcare Policy and Management
- Bacteriophages and microbial interactions
- Migraine and Headache Studies
- Gastrointestinal Tumor Research and Treatment
- Cutaneous Melanoma Detection and Management
- Meta-analysis and systematic reviews
University College London
2016-2025
Red Hat (United Kingdom)
2018-2024
Royal Statistical Society
2022-2024
Delta Air Lines (United States)
2022
University of Exeter
2020
BresMed
2014-2019
University of Sheffield
2019
Ibero American University
2016
The efficacy of pharmaceuticals is most often demonstrated by randomised controlled trials (RCTs); however, in some cases, regulatory applications lack RCT evidence.To investigate the number and type these approvals over past 15 years European Medicines Agency (EMA) US Food Drug Administration (FDA).Drug approval data were downloaded from EMA website 'Drugs@FDA' database for all decisions on published 1 January 1999 to 8 May 2014. details eligible extracted, including therapeutic area,...
In the pivotal ZUMA-5 trial, axicabtagene ciloleucel (axi-cel; an autologous anti-CD19 chimeric antigen receptor T-cell therapy) demonstrated high rates of durable response in relapsed/refractory (r/r) follicular lymphoma (FL) patients. Here, outcomes from are compared with international SCHOLAR-5 cohort, which applied key trial eligibility criteria simulating randomized controlled conditions. data were extracted institutions 5 countries, and 1 historical clinical for r/r FL patients who...
Background: Frequent migraine with four or more headache days per month is a common, disabling neurovascular disease. From US societal perspective, this analysis models the clinical efficacy and estimates value-based price (VBP) for erenumab, fully human monoclonal antibody that inhibits calcitonin gene-related peptide receptor.Methods: A Markov health state transition model was developed to estimate incremental costs, quality-adjusted life-years (QALYs), range erenumab in prevention. The...
Plain Language SummaryWhat is the study about?Hemophilia A a genetic bleeding disorder that causes for long periods because of lower than normal levels factor VIII (FVIII), protein important blood clotting. FVIII replacement therapy typical approach to manage bleeds, but new and improved therapies are becoming widely available. Prophylaxis with medication emicizumab gene valoctocogene roxaparvovec two such novel treatments approved use in adults severe hemophilia without inhibitors.Two...
Health-related quality of life is often collected in clinical studies, and forms a cornerstone economic evaluation. This study had two objectives, firstly to report compare pre- post-progression health state utilities advanced melanoma when valued by different methods secondly explore the validity progression-based utility modelling compared based upon time death. Utilities were generated from ipilimumab MDX010-20 trial (Clinicaltrials.gov Identifier: NCT00094653) using condition-specific...
Objective To establish how real-world evidence (RWE) has been used to inform single technology appraisals (STAs) of cancer drugs conducted by the National Institute for Health and Care Excellence (NICE). Methods STAs published NICE from April 2011 October 2018 that evaluated treatments were reviewed. Information regarding use RWE directly company-submitted cost-effectiveness analysis was extracted categorized topic. Summary statistics describe emergent themes, a narrative summary provided...
The SCHOLAR-5 study examines treatment patterns and outcomes of real-world follicular lymphoma (FL) patients on 3rd line (LoT) or higher, for whom existing data are limited. is a retrospective cohort using from adults (≥ 18 years) with grade 1-3a FL, initiating ≥3rd LoT after June 2014 at major centers in the US Europe. Objective response rate (ORR), complete (CR), progression-free survival (PFS) overall (OS) were analyzed by LoT. Time-to-event assessed Kaplan-Meier methods. Of 128 patients,...
Ravulizumab and eculizumab are approved terminal complement inhibitor treatments for atypical hemolytic uremic syndrome (aHUS). was engineered from to have an increased half-life allowing reduced dosing frequency (8-weekly vs. 2-weekly). To account differences in respective clinical trials, a validated balancing technique used enable indirect comparison of ravulizumab treatment efficacy aHUS. Patient-level data four trials were available pooling with two trials. In the primary analysis,...
Background In the ZUMA-5 trial (Clinical trials identification: NCT03105336), axicabtagene ciloleucel (axi-cel; a chimeric antigen receptor T-cell therapy) demonstrated high rates of durable response in relapsed/refractory (r/r) follicular lymphoma (FL) patients and clear superiority relative to SCHOLAR-5 external control cohort. We update this comparison using 24-month data.Research design methods The cohort is comprised r/r FL who initiated ≥3rd line therapy after July 2014 meeting...
BackgroundTreatment options at third-line and beyond for patients with late-line metastatic colorectal cancer (mCRC) are limited, outcomes poor best supportive care (BSC). This study investigated the cost-effectiveness of trifluridine/tipiracil regorafenib relative to BSC alone in mCRC who have been previously treated with, or not considered candidates for, standard chemotherapies.Materials MethodsA partitioned survival model was constructed assess lifetime costs benefits accrued by...
The primary aim of this study was to perform a mapping the EORTC-QLQ-C30 scores EQ-5D-3L for SIRFLOX study; large dataset patients with previously untreated liver-only or liver-dominant metastatic colorectal cancer (mCRC). A secondary compare predictive validity existing mappings from conducted in other cancers.Questionnaires (completed within 529 patients) were used linear mixed regression model utility values (scored using UK tariff) as function five scores, nine symptom and global score...
Metastatic Merkel cell carcinoma (mMCC) is a rare and aggressive skin cancer. Until recently, there were no licensed treatment options for patients with mMCC, prognosis was poor. A cost-effectiveness analysis conducted avelumab, newly available option versus standard care (SC), from UK National Health Service perspective.A partitioned survival model developed to assess the lifetime costs effects of avelumab SC. Data JAVELIN 200 trial (NCT02155647) used inform estimates quality-adjusted...
Background While placebo-controlled randomised controlled trials remain the standard way to evaluate drugs for efficacy, historical data are used extensively across development cycle. This ranges from supplementing contemporary increase power of cross-trial comparisons in estimating comparative efficacy. In many cases, these approaches performed without in-depth review context data, which may lead bias and incorrect conclusions. Methods We discuss original ‘Pocock’ criteria use how has...
In health economic evaluations, quality of life should be measured with preference-based utilities, such as the EuroQol 5 Dimension 3-level (EQ-5D-3 L). Non-preference-based instruments (often disease-specific questionnaires) are commonly mapped to utilities. We investigated if relationship observed between Patient Assessment Constipation Quality Life (PAC-QOL) and EQ-5D-3 L in patients chronic idiopathic constipation (CIC) also applies opioid-induced (OIC). patient-level data from a...
In the majority of children and adolescents with epilepsy, optimal drug therapy adequately controls their condition. However, among remaining patients who are still uncontrolled despite mono-, bi- or tri-therapy chronic anti-epileptic treatment, a rescue medication is required. Western Europe, licensed medications available for first-line treatment prolonged acute convulsive seizures (PACS) vary widely, so comparators clinical economic evaluation not consistent. No European guidelines...
Background Evaluating long-term prognosis is important for physicians, patients and payers. This study reports the results of a model developed to predict survival UK receiving second-line ipilimumab. Methods MDX010-20 trial data were used ipilimumab versus best supportive care. Two aspects this analysis required novel approaches: 1) The overall Kaplan–Meier shape unusual: an initial steep decline observed before 'plateau'. 2) need extrapolate beyond end (4.6 years). Based upon clinician...
Patients with severe hemophilia A (SHA) in Italy are routinely treated standard half-life recombinant factor VIII (rFVIII) products. rFVIII Fc-fusion protein (rFVIIIFc) is an extended product that enables less frequent administration than rFVIII, which may support improved adherence. Available data indicate low breakthrough bleed rates and potentially long-term joint health for patients rFVIIIFc prophylaxis. This study assessed the cost effectiveness of versus from Italian healthcare...