Congenital Heart Disease in Adults: a cost- effectiveness analysis of Public Policies and Interventions

Abstract

Budget constraints pose significant challenges for countries with free and universal healthcare systems. To allocate resources effectively, cost-effectiveness studies play a crucial role. These studies compare healthcare policies, drugs, procedures, and technologies, taking into consideration costs, patient quality of life, and survival. Adults with Congenital Heart Disease (CHD) face a wide range of complications throughout their lives, including infective endocarditis (IE) and pulmonary hypertension (PH), among others. Preventive measures for IE, such as dental check-ups, often encounter economic barriers. Additionally, treatment options for pulmonary arterial hypertension (PAH) in CHD patients are limited due to a lack of specific clinical trials. This thesis has the following objectives: 1. Evaluate the cost-effectiveness of providing free dental care to high-risk IE patients within the United Kingdom (UK) National Health Service (NHS). The hypothesis is that free dental check-ups improve dental hygiene, reduce chronic "low-grade" bacteraemia, and ultimately decrease the risk of IE compared to the current NHS co-pay model. 2. Investigate the long-term cost-effectiveness of combination PAH therapy in adults with congenital heart disease and pulmonary arterial hypertension (PAH-CHD), using retrospective data. The hypothesis is that combination PAH therapy improves quality of life and reduces disease progression compared to PAH monotherapy. For both objectives, Markov Models were designed, and costs were considered from an NHS perspective. Quality-adjusted life-years (QALYs) and life-years (LYs) were measured as benefits. In the dental care model, a Markov model was based on aggregated data, while individual data from a UK healthcare centre was used to populate the PAH therapy model. Sensitivity analysis included deterministic and probabilistic approaches. This work showed that offering free dental care to high-risk IE patients resulted in gains of 0.47 QALYs, 0.48 LYs, and cost savings of £275.58 per patient. The Incremental Cost-Effectiveness Ratio (ICER) was £-582.61/QALY, indicating that free dental care not only improved health outcomes but was also cost saving. Moreover, offering combination PAH therapy vs. monotherapy resulted in an ICER of £7,446.00/QALY for the entire PAH-CHD population and £5,987.00/QALY for unrepaired PAH-CHD patients. However, for PAH patients after CHD defect correction, the ICER exceeded the UK's willingness-to-pay threshold (WTP), reaching £82,306.00/QALY. In summary, offering free dental care to high-risk IE patients appears to be cost-effective and cost-saving within UK WTP thresholds. Similarly, combination PAH therapy for PAH-CHD patients, especially those with unrepaired defects, is cost-effective. To improve healthcare management and budget allocation, clinicians should actively participate in policy decisions related to new treatments. Equipping them with cost-effectiveness study skills and data analysis tools empowers clinicians to shape the future of the national health systems and ensure optimal patient care.

Date of Award	31 Oct 2023
Original language	English
Supervisor	Konstantinos Dimopoulos (Director) & Antonio Martinez Rubio (Director)