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171 results on '"Hatswell, Anthony J"'

20. Matching‐adjusted indirect comparison of bleeding outcomes in severe haemophilia A: Comparing valoctocogene roxaparvovec gene therapy, emicizumab prophylaxis, and FVIII replacement prophylaxis

Author

Astermark, Jan, primary, Buckner, Tyler W, additional, Frenzel, Laurent, additional, Hatswell, Anthony J, additional, You, Xiaojun, additional, Liu, Hai, additional, Goodman, Erin, additional, Santos, Sandra, additional, Hawes, Charles, additional, Hinds, David, additional, and Klamroth, Robert, additional

Published
2023
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24. A 24-month updated analysis of the comparative effectiveness of ZUMA-5 (axi-cel) vs. SCHOLAR-5 external control in relapsed/refractory follicular lymphoma

Author

Palomba, M Lia, primary, Ghione, Paola, additional, Patel, Anik R, additional, Nahas, Myrna, additional, Beygi, Sara, additional, Hatswell, Anthony J, additional, Kanters, Steve, additional, Limbrick-Oldfield, Eve H., additional, Wade, Sally W, additional, Ray, Markqayne D, additional, Owen, Jessica, additional, Neelapu, Sattva S, additional, Gribben, John, additional, Radford, John, additional, and Bobillo, Sabela, additional

Published
2023
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32. A Comparison of Clinical Outcomes from Updated Zuma-5 (Axicabtagene Ciloleucel) and the International Scholar-5 External Control Cohort in Relapsed/Refractory Follicular Lymphoma (R/R FL)

Author

Palomba, M. Lia, primary, Ghione, Paola, additional, Patel, Anik R., additional, Deighton, Kevin, additional, Jacobson, Caron, additional, Nahas, Myrna, additional, Jung, A Scott, additional, Hatswell, Anthony J, additional, Kanters, Steve, additional, Limbrick-Oldfield, Eve, additional, Wade, Sally W, additional, Thornton Snider, Julia, additional, Neelapu, Sattva S., additional, Riberio, Maria Teresa, additional, Gribben, John, additional, Radford, John, additional, Bobillo, Sabela, additional, and Ghesquieres, Herve, additional

Published
2021
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39. Creating historical controls using data from a previous line of treatment - Two non-standard approaches.

Author

Hatswell, Anthony J and Sullivan, William G

Subjects
*PROGRESSION-free survival, *CHRONIC lymphocytic leukemia, *INNOVATION adoption, *MEDICAL technology, *DISEASE progression
Abstract

Where medical interventions are licensed based on only uncontrolled study data (for example a single-arm trial), a common approach for estimating the incremental benefit is to compare the treatment to a 'historical control'; data collected from patients who did not receive the intervention. We illustrate with motivating examples two methods for the creation of historical controls where disease progression and overall survival are typically the key clinically meaningful endpoints. The first method utilises information routinely collected in a clinical trial programme: patients' time to disease progression on their previous line of treatment against which outcomes can be compared. The second uses published clinical outcomes for the prior line of treatment which can be extrapolated to estimate outcomes at the next line. As examples we use two pharmaceuticals licensed on the basis of uncontrolled clinical studies - idelalisib for double-refractory follicular lymphoma and ofatumumab for double-refractory chronic lymphocytic leukemia. Although subject to limitations that should be considered on a case-by-case basis, the methods may be appropriate when trying to quantify the clinical benefit of treatment based on limited and uncontrolled trial data. As a result, the methods can be used to inform health technology adoption decisions. [ABSTRACT FROM AUTHOR]

Published
2020
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41. Estimating the clinical effectiveness and value-based price range of erenumab for the prevention of migraine in patients with prior treatment failures: a US societal perspective

Author

Lipton, Richard B., primary, Brennan, Alan, additional, Palmer, Stephen, additional, Hatswell, Anthony J., additional, Porter, Joshua K., additional, Sapra, Sandhya, additional, Villa, Guillermo, additional, Shah, Neel, additional, Tepper, Stewart, additional, and Dodick, David, additional

Published
2018
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42. Regulatory approval of pharmaceuticals without a randomised controlled study: analysis of EMA and FDA approvals 1999–2014

Author

Hatswell, Anthony J, Baio, Gianluca, Berlin, Jesse A, Irs, Alar, and Freemantle, Nick

Subjects
United States Food and Drug Administration, Research, Antineoplastic Agents, Pharmacology and Therapeutics, Regulatory, United States, Europe, Government Agencies, CLINICAL PHARMACOLOGY, Product Surveillance, Postmarketing, Humans, Drug Approval, health care economics and organizations, Randomized Controlled Trials as Topic
Abstract

Introduction The efficacy of pharmaceuticals is most often demonstrated by randomised controlled trials (RCTs); however, in some cases, regulatory applications lack RCT evidence. Objective To investigate the number and type of these approvals over the past 15 years by the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). Methods Drug approval data were downloaded from the EMA website and the ‘Drugs@FDA’ database for all decisions on pharmaceuticals published from 1 January 1999 to 8 May 2014. The details of eligible applications were extracted, including the therapeutic area, type of approval and review period. Results Over the period of the study, 76 unique indications were granted without RCT results (44 by the EMA and 60 by the FDA), demonstrating that a substantial number of treatments reach the market without undergoing an RCT. The majority was for haematological malignancies (34), with the next most common areas being oncology (15) and metabolic conditions (15). Of the applications made to both agencies with a comparable data package, the FDA granted more approvals (43/44 vs 35/44) and took less time to review products (8.7 vs 15.5 months). Products reached the market first in the USA in 30 of 34 cases (mean 13.1 months) due to companies making FDA submission before EMA submissions and faster FDA review time. Discussion Despite the frequency with which approvals are granted without RCT results, there is no systematic monitoring of such treatments to confirm their effectiveness or consistency regarding when this form of evidence is appropriate. We recommend a more open debate on the role of marketing authorisations granted without RCT results, and the development of guidelines on what constitutes an acceptable data package for regulators.

Published
2016

44. Frequentist and Bayesian meta-regression of health state utilities for multiple myeloma incorporating systematic review and analysis of individual patient data.

Author

Hatswell, Anthony J., Burns, Darren, Baio, Gianluca, and Wadelin, Frances

Abstract

This analysis presents the results of a systematic review for health state utilities in multiple myeloma, as well as analysis of over 9,000 observations taken from registry and trial data. The 27 values identified from 13 papers are then synthesised in a frequentist nonparametric bootstrap model and a Bayesian meta-regression. Results were similar between the frequentist and Bayesian models with low utility on disease diagnosis (approximately 0.55), raising to approximately 0.65 on first line treatment and declining slightly with each subsequent line. Stem cell transplant was also found to be a significant predictor of health-related quality of life in both individual patient data and meta-regression, with an increased utility of approximately 0.06 across different models. The work presented demonstrates the feasibility of Bayesian methods for utility meta-regression, whilst also presenting an internally consistent set of data from the analysis of registry data. To facilitate easy updating of the data and model, data extraction tables and model code are provided as Data S1. The main limitations of the model relate to the low number of studies available, particularly in highly pretreated patients. [ABSTRACT FROM AUTHOR]

Published
2019
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48. Comparative effectiveness of ZUMA-5 (axi-cel) vs SCHOLAR-5 external control in relapsed/refractory follicular lymphoma

Author

Ghione, Paola, Palomba, M Lia, Patel, Anik R, Bobillo, Sabela, Deighton, Kevin, Jacobson, Caron A, Nahas, Myrna, Hatswell, Anthony J, Jung, A Scott, Kanters, Steve, Snider, Julia Thornton, Neelapu, Sattva S, Ribeiro, Maria Teresa, Brookhart, M Alan, Ghesquieres, Herve, Radford, John, and Gribben, John G

Abstract

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, clinical outcomes from ZUMA-5 are compared to the international SCHOLAR-5 external control cohort, which applied key ZUMA-5 trial eligibility criteria to attempt to simulate RCT conditions. SCHOLAR-5 data were extracted from institutions in five countries, and from one historical clinical trial, for r/r FL patients who initiated a ≥3rdline of therapy after July 2014. Patient characteristics were balanced through propensity scoring on pre-specified prognostic factors using standardized mortality ratio (SMR) weighting. Time-to-event outcomes were evaluated using weighted Kaplan-Meier analysis. Overall response rate (ORR) and complete response (CR) rate were compared using weighted odds ratios. The 143 patients identified in SCHOLAR-5 reduced to an effective sample of 85 patients after SMR weighting, versus 86 patients in ZUMA-5. Median follow-up time was 25.4 and 23.3 months for SCHOLAR-5 and ZUMA-5. The median overall survival (OS) and progression-free survival (PFS) in SCHOLAR-5 were 59.8 months and 12.7 months, and were not reached in ZUMA-5. The hazard ratios for OS and PFS were 0.42 (95% confidence interval [CI]: 0.21-0.83) and 0.30 (95%CI: 0.18-0.49). The ORR and CR rate were 49.9% and 29.9% in SCHOLAR-5 compared to 94.2% and 79.1% in ZUMA-5, for odds ratios of 16.2 (95%CI: 5.6-46.9) and 8.9 (95%CI: 4.3-18.3). Compared to available therapies, axi-cel demonstrated an improvement in meaningful clinical endpoints. This study suggests axi-cel may address an important unmet need for r/r FL patients. Study can be found at NCT03105336 https://www.clinicaltrials.gov/ct2/show/NCT03105336.

Published
2024
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