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Health Technology Assessment
Given that there are finite resources available for improving health and providing health care, decision-makers often face difficult trade-offs in allocating resources to the most effective interventions in terms of prevention as well as diagnosis and treatment. Economic evaluations as part of Health technology assessment (HTA) can support the decision-making process by producing transparent and robust evaluations of the costs and consequences of new health technologies. Using rigorous methods, economic evaluation and HTA provides information on the likelihood that a health technology can benefit patients in relation to the costs.
Working with a range of stakeholders including patients, clinicians, service providers, government agencies and communities, we aim to provide high-quality evidence to inform policymakers on the costs, consequences and wider impact of health interventions. We work on a range of health priority areas, including mental health, sexual health, and non-communicable diseases. We are also exploring how to improve the current methodology and the way in which health economics evidence is synthesised in order to develop ever more robust tools to best inform policymakers.
- Case Study: Health Technology Assessment of Paediatric Medicines: European Landscape, Challenges and Opportunities inside the Conect4children Project
Drug development in the paediatric field has encountered more barriers than that in adults due to insufficient clinical evaluation. To tackle this problem, connect4children (c4c), a large collaborative European network, has been built to facilitate the development of drugs and therapies for children. The c4c project aims to provide a more efficient process in paediatric clinical trials and to create a network of stakeholders to encourage collaboration and reduce information asymmetries. Many researchers were engaged in this project, including Dr Elena Pizzo from University College London.
Dr Pizzo is a Principal Research Fellow in the Department of Applied Health Research at UCL. Funded by the National Institute for Health Research (NIHR), she works as a methodological expert in the Health Technology Assessment (HTA) group of the c4c programme. Using rigorous methods, HTA provides advice on whether a health technology should be used, which patients should benefit, and what the health benefits of the intervention are in relation to its costs. HTA is used in the drug development and evaluation process and informs decision-making processes relating to the pricing and reimbursement of medicines. At the start of the c4c programme, Dr Elena Pizzo and her colleagues reviewed the challenges and opportunities of paediatric drug development using HTA. In this review, particular attention was paid to market access for new paediatric medicines, patient access to treatment and methodology.
Market access for new paediatric medicines and patient access to treatment
HTA tools help assess the challenges and opportunities of paediatric drug development. Conducting clinical trials for paediatric medicines raises ethical, logistical, political, economic and regulatory issues. Dr Pizzo and colleagues summarised the efforts of European policymakers to address these problems. Thus, the paediatric regulation introduced a number of measures including promotion of paediatric drug labelling, deadlines for placing paediatric medicines on the market or rewards and incentives for paediatric medicines.
For instance, pharmaceutical companies that are working on the Paediatric Investigation Plan or have the Paediatric Use Marketing Authorization issued by the Paediatric Regulation can benefit from longer patent periods for their products. Moreover, paediatric investigation exemptions granted by European Medicines Agency (EMA) also facilitate the emergence of new medicines. Although these
initiatives have made great progress, some barriers to the development of paediatric drug remain. For example, the benefits provided by regulation do not outweigh the efforts needed to invest more and provide new medicines for children.
Methodological challenges of performing HTA analyses
Current HTA approaches in the paediatric field have methodological and systemic problems. In terms of methodological issues, Dr Pizo and colleagues have highlighted the difficulties of measuring health outcomes and health states in children. The difference in health gains to society between children and adults, the biases caused by legal guardians responding on behalf of children, and the delayed costs and benefits of treatment all present possible challenges to accurate results. In addition, systemic problems were also found to exist. For example, language barriers may hamper product comparisons and European countries have different reimbursement systems for paediatric medicines that receive the paediatric use marketing authorization. Therefore, innovative and sustainable solutions are needed to modify current HTA approaches and adapt them to the paediatric field.
References: Moretti, F., Ruiz, F., Bonifazi, F., Pizzo, E., Kindblom, J. M., & c4c HTA expert group. (2021). Health technology assessment of paediatric medicines: European landscape, challenges and opportunities inside the conect4children project. British journal of clinical pharmacology.
- Case Study: Assessing the Cost-Effectiveness of Mental Health Interventions Targeting Vulnerable Populations
How can interventions be designed to promote access to mental healthcare among vulnerable populations?
According to the World Health Organisation (WHO), one in eight individuals lives with a mental disorder. However, individuals with mental health conditions are generally poorly cared for, and treatment coverage is low. Thus, public expenditure on mental healthcare represents on average less than 2% of national health budgets (WHO, 2022).
Discrimination and social stigma are some of the main barriers for individuals seeking mental healthcare. Additionally, individuals with poor physical health often suffer from poor mental health simultaneously. In fact, studies show that when people with physical health issues receive mental health care, positive outcomes are achieved in terms of overall quality of life (Chiles et al., 2006; Simas et al., 2018; Cubillos et al., 2021).
Researchers from UCL aim to better understand how mental health issues can be effectively addressed and to conduct economic evaluations of interventions that integrate mental health care alongside physical healthcare.
Is it cost-effective to provide vulnerable populations with simultaneous access to mental and physical health care?
As part of the Lucy Project, group of students from the UCL MSc in Health Economics and Decision Science, together with Prof Neha Batura from the UCL Institute for Global Health and researchers from the Great Ormond Street Hospital for Children conducted a cost-effectiveness analysis of an intervention providing vulnerable populations with simultaneous access to mental and physical health care. By providing easily accessible mental health support to paediatric patients treated for long-term physical conditions, the Lucy Project aimed to address the lack of integrated mental health care in the UK. The outcomes were captured through the parent-reported Strengths and Difficulties Questionnaires (SDQ) and the Paediatric Quality of Life Inventory (PedsQL) measured at baseline and at six months (Clarke et al. 2022). The findings of the study showed that, at a cost-effectiveness threshold defined by the National Institute for Health and Care Excellence (£20,000 per quality-adjusted life year gained), the intervention was cost-effective under different scenarios. In the baseline scenario, the intervention was cost-effective in 75% of cases. In the practical case scenario, which took into account the cost-effectiveness of the intervention in a more streamlined care setting, the intervention was cost-effective in 94% of cases.
Is simultaneous access to mental and physical health care for vulnerable populations in non-medical settings cost-effective?
Prof Rachael Hunter from the UCL Institute of Epidemiology and Health Care, together with other researchers from across the UK conducted an economic evaluation of a prison-based intervention (“Engager”) to address mental health problems. In a two-group randomised controlled trial, 280 prisoners who reported suffering from anxiety, depression or phobias were randomly allocated to either standard care or "Engager", an innovative complex care. Participants were followed up for 12 months following release from prison. The results of the study showed that the “Engager” intervention is not cost-effective under NICE cost-effectiveness threshold (£20,000-£30,000 per quality-adjusted life year gained). However, the authors of the study highlighted the challenges of providing health care to patients with multiple needs. Prisoners with health issues often also have to deal with other problems such as substance abuse, and monetary or housing problems. The authors also show the importance of including a wide range of costs and outcomes in prison-based economic and suggest that follow-up beyond 12 months after release could provide additional information on the cost-effectiveness of these types of interventions.
Conclusion
The two studies conducted by UCL researchers highlight the complexity of assessing the cost-effectiveness of interventions targeting vulnerable populations, whether children or prisoners. The results of any economic evaluation depend on the type of employed methodological approach, particularly the perspective of the analysis. Moreover, the economic implications of negative mental health may represent a greater economic loss than the health care costs of the interventions. This is particularly true when considering the indirect costs of mental health problems (e.g. late return to the labour market).
References: Chiles, J. A., Lambert, M. J., & Hatch, A. L. (1999). The impact of psychological interventions on medical cost offset: A meta-analytic review. Clinical Psychology: Science and Practice, 6(2), 204. Clarke, H., Morris, W., Catanzano, M., Bennett, S., Coughtrey, A. E., Heyman, I., ... & Batura, N. (2022). Cost-effectiveness of a mental health drop-in centre for young people with long-term physical conditions. BMC Health Services Research, 22(1), 1-12. Cubillos, L., Bartels, S. M., Torrey, W. C., Naslund, J., Uribe-Restrepo, J. M., Gaviola, C., ... & Marsch, L. A. (2021). The effectiveness and cost-effectiveness of integrating mental health services in primary care in low-and middle-income countries: systematic review. BJPsych bulletin, 45(1), 40-52. Hunter, R. M., Anderson, R., Kirkpatrick, T., Lennox, C., Warren, F., Taylor, R. S., ... & Byng, R. (2022). Economic evaluation of a complex intervention (Engager) for prisoners with common mental health problems, near to and after release: a cost-utility and cost-consequences analysis. The European Journal of Health Economics, 23(2), 193-210. Simas, T. A. M., Flynn, M. P., Kroll-Desrosiers, A. R., Carvalho, S. M., Levin, L. L., Biebel, K., & Byatt, N. (2018). A systematic review of integrated care interventions addressing perinatal depression care in ambulatory obstetric care settings. Clinical obstetrics and gynecology, 61(3), 573. Waisel, D. B. (2013). Vulnerable populations in healthcare. Current Opinion in Anesthesiology, 26(2), 186-192. World Health Organization. (2022). World mental health report: transforming mental health for all: executive summary. In World mental health report: transforming mental health for all: executive summary.
- Case Study (Video): Optimal Treatment for mHSPC from a Health Economics Standpoint
At the 2022 Advanced Prostate Cancer Consensus Conference (APCCC) Hybrid Meeting, Caroline Clarke presents on health economics in metastatic hormone sensitive prostate cancer (mHSPC).