Objective The objective of this study was to develop guidance contributing to improved consistency and quality in economic evaluations of personalised medicine (PM), given current ambiguity about how to measure the value of PM as well as considerable variation in the methodology and reporting in economic evaluations of PM. Methods A targeted literature review of methodological papers was performed for an overview of modelling challenges in PM. Expert interviews were held to discuss best modelling practice. A systematic literature review of economic evaluations of PM was conducted to gain insight into current modelling practice. The findings were synthesised and used to develop a set of draft recommendations. The draft recommendations were discussed at a stakeholder workshop and subsequently finalised. Results Twenty-two methodological papers were identified. Some argued that the challenges in modelling PM can be addressed within existing methodological frameworks, others disagreed. Eighteen experts were interviewed. They believed large uncertainty to be a key concern. Out of 195 economic evaluations of PM identified, 56% addressed none of the identified modelling challenges. A set of 23 recommendations was developed. Eight recommendations focus on the modelling of test-treatment pathways. The use of non-randomised controlled trial data is discouraged but several recommendations are provided in case randomised controlled trial data are unavailable. The parameterisation of structural uncertainty is recommended. Other recommendations consider perspective and discounting; premature survival data; additional value elements; patient and clinician compliance; and managed entry agreements. Conclusions This study provides a comprehensive list of recommendations to modellers of PM and to evaluators and reviewers of PM models.

Objective: Lung cancer is one of the most common cancers worldwide and its survival is still poor. The objective of our study was to estimate long-term survival of Hungarian lung cancer patients at first time based on a nationwide review of the National Health Insurance Fund database.Methods: Our retrospective, longitudinal study included patients aged ≥20 years who were diagnosed with lung cancer (ICD-10 C34) between January 1, 2011 and December 31, 2016. Survival rates were evaluated by year of diagnosis, patient gender and age, and morphology of lung cancer.Results: 41,854 newly diagnosed lung cancer patients were recorded. Mean age at diagnosis varied between 64.7 and 65.9 years during study period. One- and 5-year overall survival rates for the total population were 42.2 and 17.9%, respectively. Survival was statistically associated with gender, age and type of lung cancer. Female patients (n = 16,362) had 23% better survival (HR: 0.77, 95% confidence interval (CI): 0.75–0.79; p < 0.001) than males (n = 25,492). The highest survival rates were found in the 20–49 age cohort (5Y = 31.3%) and if the cancer type was adenocarcinoma (5Y = 20.5%). We measured 5.3% improvement (9.2% adjusted) in lung cancer survival comparing the period 2015–2016 to 2011–2012 (HR: 0.95 95% CI: 0.92–0.97; p = 0.003), the highest at females <60 year (0.86 (adjusted HR was 0.79), interaction analysis was significant for age and histology types.Conclusion: Our study provided long-term Lung cancer survival data in Hungary for the first time. We found a 5.3% improvement in 5-year survival in 4 years. Women and young patients had better survival. Survival rates were comparable to–and at the higher end of–rates registered in other East-Central European countries (7.7%–15.7%).

Clinical data and cost-effectiveness analyses from several countries support the use of low-dose computed tomography (LDCT) to screen patients with high risk of lung cancer (LC). This study aimed to explore the economic value of screening LC with LDCT in Hungary. Cohorts of screened and nonscreened subjects were simulated in a decision analytic model over their lifetime. Five steps in the patient trajectory were distinguished: no LC, nondiagnosed LC, screening, diagnosed LC, and post-treatment. Patient pathways were populated based on the Hungarian pilot study of screening, the Nederlands-Leuvens Longkanker Screenings Onderzoek (NELSON) LC screening trial, and local incidence and prevalence data. Healthcare costs were obtained from the National Health Insurance Fund. Utility data were obtained from international sources and adjusted to local tariffs. Scenarios according to screening frequency, age bands (50-74, 55-74 years), and smoking status were analyzed. Annual LDCT-based screening compared with no screening for 55- to 74-year-old current smokers showed 0.031 quality-adjusted life-year (QALY) gains for an additional €137, which yields €5707 per QALY. Biennial screening for the same target population showed that purchasing 1 QALY would cost €10 203. The least cost-effective case was biennial screening of the general population aged 50 to 74 years, which yielded €37 931 per QALY. Screening LC with LDCT for a high-risk population could be cost-effective in Hungary. For the introduction of screening with LDCT, targeting the most vulnerable groups while having a long-term approach on costs and benefits is essential.

The implementation of adequate financing and reimbursement of personalized medicine (PM) in Europe is still turbulent. The views and experience of stakeholders about barriers in financing and reimb...

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