Economic evaluation is a systematic framework for assessing and combining clinical and economic evidence to inform health policy decisions. Economic evaluations may be conducted alongside RCTs or through decision analytic modelling (DAM). For wider policy questions outside of specific RCT populations, DAM provides an explicit quantitative framework for the synthesis of evidence from multiple sources to estimate costs and benefits of competing interventions, together with associated measures of cost-effectiveness and uncertainty.
Health Technology Assessment of CAR T-Cell Therapies
Summary: CAR T-cell therapies are innovative treatments for cancer. They are associated with high, upfront costs and a limited evidence base. This creates challenges for key stakeholders, including reimbursement decision-makers. This research involved research involved conducting independent health technology assessments (HTAs) of the CD19 CAR T-cell therapies, tisagenlecleucel and axicabtagene ciloleucel. Evidence was attained through primary data collections, systematic literature reviews and clinical evidence synthesis, and the use of advanced decision analytic modelling approaches. This evidence informed the bespoke decision analytic models that were developed to evaluate cost effectiveness and budget impacts of these therapies. Value of information analyses were conducted to estimate the value of simultaneously eliminating all uncertainty of all uncertain parameters in the respective cost-utility models. The parameters, which contributed most to the overall decision uncertainty, were identified. The impact of performance-linked reimbursement agreements on the cost effectiveness and budget impact of these therapies was explored.
Tisagenlecleucel for relapsed/refractory acute lymphoblastic leukemia in the Irish healthcare setting: Cost-effectiveness and value of information analysis. Carey N, Leahy J, Trela-Larsen L, McCullagh L, Barry M. International Journal of Technology Assessment in Health Care. 2022. DOI: https://doi.org/10.1017/S0266462322000356
A text-mining tool generated title-abstract screening workload savings: performance evaluation versus single-human screening. Carey N, Harte M, Mc Cullagh L. Journal of Clinical Epidemiology. 2022. DOI: https://doi.org/10.1016/j.jclinepi.2022.05.017
Theoretical Cost Implications of Funding Drugs that are Not Cost Effective
Healthcare systems internationally are financially challenged by the increasing utilization of high-cost drugs. The reimbursement of drugs that are not cost effective is associated with opportunity cost and thus may negatively impact on the sustainability of healthcare provision. In our analysis, we estimate the theoretical excess expenditure (in the first year of reimbursement) that would be incurred by the Irish payer, should 18 drugs (that fit our pre-specified inclusion and exclusion criteria) be reimbursed at their list (submitted) price rather than at a cost-effective price. We estimate that this theoretical excess expenditure would be £108.2 million. This theoretical excess expenditure could instead fund homecare provision for 1850 patients with dementia for one year. This study demonstrates the importance of an HTA process to identify potential excess expenditure before a drug-reimbursement decision is made.
Estimating the Theoretical Cost Implications of Funding New Drugs Considered Not to Be Cost-Effective. Kennedy C, McCullagh L, Adams R, Trela-Larsen L, Tilson L, Barry M, Value in Health, 2021, ISSN 1098-3015, https://doi.org/10.1016/j.jval.2021.03.012.
Health Technology Assessment of PCSK9 inhibitors
National Centre for Pharmacoeonomics, Trinity College Dublin Department of Statistics
PCSK9 inhibitors are a new class of medicines for the treatment of hypercholesterolemia in patients who fail to reach LDL-C targets on maximal tolerated statin therapy. The therapies licensed to date (evolocumab and alirocumab) are monoclonal antibodies administered as a fortnightly or monthly injections. Given the high cost of therapy and the size of the licensed population, the potential budget impact of these agents is significant. The heterogeneous nature of the population means that the cost-effectiveness of the agents will vary across different patient characteristics and risk profiles. However the analysis of population subgroups adds to the complexity of a health technology assessment. The aim of this study is to identify the subgroups of the population who will benefit most from PCSK9 inhibitor treatment and to quantify the cost effectiveness of the intervention in these subgroups
Cost-Effectiveness of Rivaroxaban and Dabigatran Etexilate Thromboprophylaxis after Total Hip and Knee Replacement
The objective was to evaluate the cost effectiveness of rivaroxaban and dabigatran etexilate compared to enoxaparin for the prophylaxis of venous thromboembolism in patients undergoing elective total hip replacement (THR) and total knee replacement (TKR) in the Irish healthcare setting. The evaluation was conducted from the Irish health payer perspective. A static decision tree model was developed with a 180 day post surgery time horizon. Analysis indicated that rivaroxaban is less costly and more effective when compared to either enoxaparin or dabigatran etexilate post THR and TKR.
McCullagh L, Tilson L, Walsh C, Barry M. A Cost-Effectiveness Model Comparing Rivaroxaban and Dabigatran Etexilate with Enoxaparin Sodium as Thromboprophylaxis after Total Hip and Knee Replacement in the Irish Health Care Setting. Pharmacoeconomics 2009;27(10):829-846
The Pharmacoeconomic Evaluation Process in Ireland
National Centre for Pharmacoeconomics, Trinity College Dublin Department of Statistics
The movement of all drugs submitted to the NCPE (from January 2010 to December 2015 inclusive) was investigated. Over the study period, the NCPE evaluated 230 drugs for which reimbursement was sought. In total, a positive reimbursement recommendation has been made in 165 cases (72 % of all drugs submitted, or 79 % when drugs undergoing the process at the time of analysis were excluded). A price reduction was deemed necessary in 71 (43 %) of the 165 cases, and a recommendation to restrict/monitor usage was made in 35 cases (21 %).
McCullagh L, Barry M. The Pharmacoeconomic Evaluation Process in Ireland. PharmacoEconomics 2016; 34(12), 1267-1276.
Health technology assessment of novel drug treatments for advanced melanoma
National Centre for Pharmacoeconomics
Recent years have seen significant therapeutic advances in the treatment of metastatic melanoma. New immunotherapies such as pembrolizumab and nivolumab have demonstrated reductions in tumour size and prolonged responses in selected patients. Combination targeted treatment with BRAF and MEK inhibitors have been associated with lengthy periods of disease stabilisation. There are many uncertainties relating to the optimal clinical use of these agents, and their provision is associated with significant costs and resource use implications for the health service. The purpose of this health technology assessment (HTA) is to estimate the cost effectiveness of the treatments used in Ireland for metastatic melanoma.
A re-evaluation of the cost-effectiveness of regorafenib 5 years after reimbursement
National Centre for Pharmacoeconomics, Materials and Surface Science Institute (MSSI) University of Limerick, Royal College of Surgeons Ireland, National Cancer Registry of Ireland
Awarding Body: Health Research Board – Applied Partnership Award 2016. (Part of the Utility of High-Tech Drug Analysis to the Decision Maker project)
This analysis will compare the estimates of cost-effectiveness predicted by the applicant prior to receiving reimbursement and the actual cost effectiveness based on a revised set of cost effectiveness model inputs (e.g. revised numbers of patients receiving the drug, revised expenditure for the drug, revised duration of treatment, revised dosing, revised health outcomes i.e. overall survival, updated costs for healthcare resource use associated with colorectal cancer) since time of reimbursement.
Cost-effectiveness of Vaccination in Ireland
National Immunisation Advisory Committee
The NCPE has, together with the National Immunisation Advisory Committee, conducted evaluations of the cost effectiveness of several vaccination programmes which have supported decision making in the Irish healthcare setting.
Cost effectiveness of hepatitis B vaccination strategies in Ireland: an economic evaluation Lesley Tilson, Lelia Thornton, Darina O’Flanagan, Howard Johnson, Michael Barry. European Journal of Public Health. Jun 2008;18(3):275-82.
Economic Evaluation of a Universal Childhood Pneumococcal Conjugate Vaccination Strategy in Ireland. Tilson L, Usher C, Butler K, Fitzsimons J, O’Hare F, Cotter S, O’Flanagan D, Johnson H, Barry M. Value in Health. 2008;11(5):898-903.
Cost effectiveness of HPV vaccine in reducing the risk of cervical cancer in Ireland due to HPV types 16 & 18 using a transmission dynamic model. Usher C, Tilson L, Olsen J, Jepsen M, Walsh C, Barry M. Vaccine 2008 Oct 16;26(44):5654-61.
Cost-effectiveness of universal rotavirus vaccination in reducing rotavirus gastroenteritis in Ireland. Tilson L, Jit M, Schmitz S, et al. Vaccine 2011; 29 (43): 7463-7473.
Evaluating the neonatal BCG vaccination programme in Ireland. Usher C, Adams R, Schmitz S, Kieran J, O’Flanagan D, O’Donnell J, Connolly K, Corcoran B, Butler K, Barry M, Walsh C. Archives of Public Health. 2016 Jul 13;74(1):28.
Population-level impact, herd immunity, and elimination after human papillomavirus vaccination: a systematic review and meta-analysis of predictions from transmission-dynamic models. Brisson M, Bénard É, Drolet M, Bogaards JA, Baussano I, Vänskä S, Jit M, Boily MC, Smith MA, Berkhof J, Canfell K, Usher C, Walsh C, et al. The Lancet Public Health. 2016 Nov 30;1(1):e8-17.
Other Related Publications
Cost-effectiveness of natriuretic peptide-based screening and collaborative care: a report from the STOP-HF (St. Vincent’s Screening To Prevent Heart Failure) Study.
Ledwidge M. T, O’Connell E, Gallagher J, Tilson L, James S, Voon V, Bermingham M, Tallon E, Watson C, O’Hanlon R, Barry M and McDonald K. European Journal of Heart Failure, 17:672-679. doi: 10.1002/ejhf.286
Cost-effectiveness of population-based screening for colorectal cancer: a comparison of guaiac-based faecal occult blood testing, faecal immunochemical testing and flexible sigmoidoscopy. L Sharp, L Tilson, S Whyte, A O’Ceilleachair, C Walsh, C Usher, P Tappenden, J Chilcott, A Staines, M Barry and H Comber. Br J Cancer. 2012:106; 805-816.
Using resource modelling to inform decision making and service planning: the case of colorectal cancer screening in Ireland. Sharpe L, Tilson L, Whyte S, O Ceilleachair A, Walsh C, Usher C, Tappenden P, Chilcott J, Staines A, Barry M, Comber H. BMC Health Services Research 2013, 13:105 doi:10.1186/1472-6963-13-105
Pharmacoeconomic Evaluation in Ireland: A Review of the Process Tilson L, O’Leary A, Usher C, Barry M. Pharmacoeconomics 2010;28(4): 307-322.