Economic evaluations of precision medicine: case studies in diabetes

This thesis addressed the growing challenge of diabetes by exploring the cost-effectiveness of precision medicine interventions. Diabetes varies greatly among individuals, making precision treatments crucial for improving outcomes and reducing healthcare system burdens.The research encompassed a scoping review of precision medicine studies, an evaluation of a personalized diabetic retinopathy screening and a predictive model for diabetic foot ulcers, an assessment of a diabetes health economic model's transferability and a cost-effectiveness analysis of a glucose monitoring device for specific type 1 diabetes subgroups.Key findings highlighted a shortage of cost-effectiveness data in precision medicine for diabetes. Challenges included insufficient long-term efficacy and safety data, as well as missing essential biomarkers in practical use. Nonetheless, personalized diabetic retinopathy screening emerged as a cost-effective alternative while the prediction model for diabetic foot ulcers yielded mixed results compared to Dutch guidelines.Transferability of the health economic model showed variability among cohorts and outcomes, emphasizing the importance of model validation when applied to new populations. For glucose monitoring devices, cost-effectiveness was contingent on factors, with the highest probability observed in select subgroups.In summary, while precision medicine is increasingly adopted in diabetes management, its cost-effectiveness remains uncertain. This thesis contributes insights, indicating that personalized approaches for diabetic retinopathy and specific glucose monitoring devices can offer cost-effective solutions. However, improvements may be needed to enhance the cost-effectiveness of personalized diabetic foot ulcer referrals. This research underscores the significance of tailored diabetes care, identifying areas where precision medicine can deliver health benefits and cost savings

A personalised screening strategy for diabetic retinopathy:a cost-effectiveness perspective

Aims/hypothesis: In this study we examined the cost-effectiveness of three different screening strategies for diabetic retinopathy: using a personalised adaptive model, annual screening (fixed intervals), and the current Dutch guideline (stratified based on previous retinopathy grade). Methods: For each individual, optimal diabetic retinopathy screening intervals were determined, using a validated risk prediction model. Observational data (1998–2017) from the Hoorn Diabetes Care System cohort of people with type 2 diabetes were used (n = 5514). The missing values of retinopathy grades were imputed using two scenarios of slow and fast sight-threatening retinopathy (STR) progression. By comparing the model-based screening intervals to observed time to develop STR, the number of delayed STR diagnoses was determined. Costs were calculated using the healthcare perspective and the societal perspective. Finally, outcomes and costs were compared for the different screening strategies. Results: For the fast STR progression scenario, personalised screening resulted in 11.6% more delayed STR diagnoses and €11.4 less costs per patient compared to annual screening from a healthcare perspective. The personalised screening model performed better in terms of timely diagnosis of STR (8.8% less delayed STR diagnosis) but it was slightly more expensive (€1.8 per patient from a healthcare perspective) than the Dutch guideline strategy. Conclusions/interpretation: The personalised diabetic retinopathy screening model is more cost-effective than the Dutch guideline screening strategy. Although the personalised screening strategy was less effective, in terms of timely diagnosis of STR patients, than annual screening, the number of delayed STR diagnoses is low and the cost saving is considerable. With around one million people with type 2 diabetes in the Netherlands, implementing this personalised model could save €11.4 million per year compared with annual screening, at the cost of 658 delayed STR diagnoses with a maximum delayed time to diagnosis of 48 months. Graphical abstract: [Figure not available: see fulltext.]

Personalizing the Use of a Intermittently Scanned Continuous Glucose Monitoring (isCGM) Device in Individuals With Type 1 Diabetes:A Cost-Effectiveness Perspective in the Netherlands (FLARE-NL 9)

AIMS: Intermittently scanned continuous glucose monitoring (isCGM) is a method to monitor glucose concentrations without using a finger prick. Among persons with type 1 diabetes (T1D), isCGM results in improved glycemic control, less disease burden and improved health-related quality of life (HRQoL). However, it is not clear for which subgroups of patients isCGM is cost-effective. We aimed to provide a real-world cost-effectiveness perspective.METHODS: We used clinical data from a 1-year nationwide Dutch prospective observational study (N = 381) and linked these to insurance records. Health-related quality of life was assessed with the EQ-5D-3L questionnaire. Individuals were categorized into 4 subgroups: (1) frequent hypoglycemic events (58%), (2) HbA1c &gt; 70 mmol/mol (8.5%) (19%), (3) occupation that requires avoiding finger pricks and/or hypoglycemia (5%), and (4) multiple indications (18%). Comparing costs and outcomes 12 months before and after isCGM initiation, incremental cost-effectiveness ratios (ICERs) were calculated for the total cohort and each subgroup from a societal perspective (including healthcare and productivity loss costs) at the willingness to pay of €50,000 per quality-adjusted life year (QALY) gained.RESULTS: From a societal perspective, isCGM was dominant in all subgroups (ie higher HRQoL gain with lower costs) except for subgroup 1. From a healthcare payer perspective, the probabilities of isCGM being cost-effective were 16%, 9%, 30%, 98%, and 65% for the total cohort and subgroup 1, 2, 3, and 4, respectively. Most sensitivity analyses confirmed these findings.CONCLUSIONS: Comparing subgroups of isCGM users allows to prioritize them based on cost-effectiveness. The most cost-effective subgroup was occupation-related indications, followed by multiple indications, high HbA1c and the frequent hypoglycemic events subgroups. However, controlled studies with larger sample size are needed to draw definitive conclusions.</p

Personalizing the Use of a Intermittently Scanned Continuous Glucose Monitoring (isCGM) Device in Individuals With Type 1 Diabetes:A Cost-Effectiveness Perspective in the Netherlands (FLARE-NL 9)

AIMS: Intermittently scanned continuous glucose monitoring (isCGM) is a method to monitor glucose concentrations without using a finger prick. Among persons with type 1 diabetes (T1D), isCGM results in improved glycemic control, less disease burden and improved health-related quality of life (HRQoL). However, it is not clear for which subgroups of patients isCGM is cost-effective. We aimed to provide a real-world cost-effectiveness perspective.METHODS: We used clinical data from a 1-year nationwide Dutch prospective observational study (N = 381) and linked these to insurance records. Health-related quality of life was assessed with the EQ-5D-3L questionnaire. Individuals were categorized into 4 subgroups: (1) frequent hypoglycemic events (58%), (2) HbA1c &gt; 70 mmol/mol (8.5%) (19%), (3) occupation that requires avoiding finger pricks and/or hypoglycemia (5%), and (4) multiple indications (18%). Comparing costs and outcomes 12 months before and after isCGM initiation, incremental cost-effectiveness ratios (ICERs) were calculated for the total cohort and each subgroup from a societal perspective (including healthcare and productivity loss costs) at the willingness to pay of €50,000 per quality-adjusted life year (QALY) gained.RESULTS: From a societal perspective, isCGM was dominant in all subgroups (ie higher HRQoL gain with lower costs) except for subgroup 1. From a healthcare payer perspective, the probabilities of isCGM being cost-effective were 16%, 9%, 30%, 98%, and 65% for the total cohort and subgroup 1, 2, 3, and 4, respectively. Most sensitivity analyses confirmed these findings.CONCLUSIONS: Comparing subgroups of isCGM users allows to prioritize them based on cost-effectiveness. The most cost-effective subgroup was occupation-related indications, followed by multiple indications, high HbA1c and the frequent hypoglycemic events subgroups. However, controlled studies with larger sample size are needed to draw definitive conclusions.</p