Peter Lindgren

Background and purpose: Molecularly targeted cancer therapies challenge conventional health economic evaluation frameworks that are structured around tumour-specific indications, comparators, and trial designs. Existing models often rely on pooled estimates from heterogeneous early-phase evidence or single-indication analyses, creating uncertainty for reimbursement decision-makers. We propose a conceptual modelling framework that aligns cost-effectiveness analyses with the biological rationale of precision oncology, evaluating therapies according to shared molecular alterations across tumour types. Patient/material and methods: We examined the methodological limitations of conventional partitioned survival models (PSMs) commonly applied in oncology and evaluated their suitability for tumour-agnostic indications. Based on the collected literature, we developed a dynamic, modular PSM framework that integrates multiple tumour sites expressing a common biomarker. The framework supports pooled and tumour-specific analysis of cost-effectiveness and enables progressive disaggregation of subgroups as additional evidence becomes available. Results: The proposed modelling approach facilitates transparent synthesis of heterogeneous evidence across tumour types using epidemiologically informed weighting, while preserving the ability to estimate tumour-specific cost-effectiveness where data permit. It addresses key challenges in tumour-agnostic evaluation, including variation in standard of care, treatment effects, and resource use across cancer sites. The modular design promotes internal consistency, reduces duplication of analytical effort, and enables iterative re-assessment of both overall and subgroup-specific cost-effectiveness. Interpretation: A dynamic, weighted multi-site modelling framework represents a coherent and adaptable extension of current health-technology assessment-practice for tumour-agnostic therapies. By structuring evidence around molecular targets, the framework can improve transparency and robustness of cost-effectiveness estimates, thereby supporting more equitable and efficient reimbursement decisions in the context of precision oncology.

Radiofrequency renal denervation (RF RDN) is a catheter-based therapy for uncontrolled hypertension. This model-based analysis examined the cost-effectiveness of RF RDN in Sweden. Clinical events, costs, quality-adjusted life-years (QALYs) were projected over 10-year and lifetime horizons using a decision-analytic Markov model. Primary health states, included hypertension alone, myocardial infarction (MI), stroke, other symptomatic coronary heart disease (CHD), heart failure (HF), end-stage renal disease (ESRD), and death. Health state transitions were informed by multivariate risk equations. Clinical evidence from the SPYRAL HTN-ON MED trial informed the treatment effect modelled (-4.9 mmHg reduction in office systolic blood pressure (SBP) vs. sham). The base case was conducted from the Swedish healthcare payer perspective. The primary outcome was the incremental cost-effectiveness ratio (ICER),RF RDN vs. standard of care (SoC), evaluated against an assumed willingness-to-pay threshold of SEK 500,000 per QALY gained. Extensive sensitivity analyses were performed. At 10-years, the relative risks with RF RDN were 0.80 for stroke, 0.88 for MI, 0.89 for CHD, 0.72 for HF, 0.96 for ESRD, 0.86 for cardiovascular death and 0.93 for all-cause death. Over lifetime, RF RDN led to incremental costs of SEK 63,136 (total costs SEK 497,498 vs. SEK 434,362) and incremental QALY gain of 0.45 (14.79 vs. 14.34), yielding an ICER of SEK 139,280 per QALY gained. RF RDN was cost-effective across all scenarios and sensitivity analyses. Model projections suggest RF RDN to be a cost-effective therapy for uncontrolled including resistant hypertension in Sweden based on contemporary clinical evidence.

Arginase 1 deficiency (ARG1-D) is a ultrarare disease with manifestations that cause mobility and cognitive impairment that progress over time and may lead to early mortality. Diseases such as ARG1-D have a major impact also outside of the health care sector and the aim of this study was to estimate the current burden of disease associated with ARG1-D from a societal perspective. The study was performed as a web-based survey of patients with ARG1-D and their caregivers in four European countries (France, Portugal, Spain, United Kingdom). The survey was distributed at participating clinics and included questions on e.g., symptoms (including the Gross Motor Function Classification System, GMFCS, and cognitive impairment), health care use, medication, ability to work, caregiving, and impact on health-related quality-of-life (HRQoL) using the EQ-5D-5L. The estimated total mean societal cost per patient and year was £63,775 (SD: £49,944). The cost varied significantly with both mobility impairment (from £49,809 for GMFCS level 1 to £103,639 for GMFCS levels 3-5) and cognitive impairment (from £43,860 for mild level to £99,162 for severe level). The mean utility score on the EQ-5D-5L for patients was 0.498 (SD: 0.352). The utility score also varied significantly with both mobility impairment (from 0.783 for GMFCS level 1 to 0.153 for GMFCS level 3-5) and cognitive impairment (from 0.738 for mild level to 0.364 for severe level). Similar to other studies of rare diseases, the study is based on a limited number of observations. However, the sample appear to be reasonably representative when comparing to previous studies of ARG1-D. This study shows that ARG1-D is associated with a high societal cost and significant impact on HRQoL. Earlier diagnosis and better treatment options that can postpone or withhold progression may therefore have a potential for improved HRQoL and savings for the patient, caregiver, and society.

Background: In 2015, the Swedish government in an unprecedented move decided to allocate 150 million € to provide funding for new drugs for hepatitis C. This was triggered by the introduction of the first second generation of direct-acting antivirals (DAAs) promising higher cure rates and reduced side effects. The drugs were cost-effective but had a prohibitive budget impact. Subsequently, additional products have entered the market leading to reduction in prices and expansions of the eligible patient base. Methods: We estimated the social surplus generated by the new DAAs in Stockholm, Sweden, for the years 2014–2019. The actual use and cost of the drugs was based on registry data. Effects on future health care costs, indirect costs and QALY gains were estimated using a Markov model based primarily on Swedish data and using previous generations of interferon-based therapies as the counterfactual. Results: A considerable social surplus was generated, 15% of which was appropriated by the producers whose share fell rapidly over time as prices fell. Most of the consumer surplus was generated by QALY gains, although 10% was from reduced indirect costs. QALY gains increased less rapidly than the number of treated patients as the eligibility criteria was loosened. Conclusions: The transfer of funds from the government to the regions helped generate substantial surplus for both consumers and producers with indirect costs playing an important role. The funding model may serve as a model for the financing of innovative treatments in the future.

Aims/hypothesis: The risk of complications and medical consequences of type 2 diabetes are well known. Hospital costs have been identified as a key driver of total costs in studies of the economic burden of type 2 diabetes. Less evidence has been generated on the impact of individual diabetic complications on the overall societal burden. The objective of this study was to analyse costs of hospital-based healthcare (inpatient and outpatient care) and work absence related to individual macrovascular and microvascular complications of type 2 diabetes in Sweden in 2016. Methods: Data for 2016 were retrieved from a Swedish national retrospective observational database cross-linking individual-level data for 1997–2016. The database contained information from population-based health, social insurance and socioeconomic registers for 392,200 people with type 2 diabetes and matched control participants (5:1). Presence of type 2 diabetes and of diabetes complications were derived using all years, 1997–2016. Costs of hospital-based care and of absence from work due to diabetes complications were estimated for the year 2016. Regression analysis was used for comparison with control participants to attribute absence from work to individual complications, and to account for joint presence of complications. Results: Use of hospital care for complications was higher in type 2 diabetes compared with control participants in 2016: 26% vs 12% had ≥1 hospital contact; there were 86,104 vs 24,608 outpatient visits per 100,000 people; and there were 9894 vs 2546 inpatient admissions per 100,000 people (all p < 0.001). The corresponding total costs of hospital-based care for complications were €919 vs €232 per person (p < 0.001), and 74.7% of costs were then directly attributed to diabetes (€687 per person). Regression analyses distributed the costs of days absent from work across diabetes complications per se, basic type 2 diabetes effect and unattributed causes. Diabetes complications amounted to €1317 per person in 2016, accounting for possible complex interactions (25% of total costs of days absent). Key drivers of costs were the macrovascular complications angina pectoris, heart failure and stroke; and the microvascular complications eye diseases, including retinopathy, kidney disease and neuropathy. Early mortality in working ages cost an additional €579 per person and medications used in risk-factor treatment amounted to €418 per person. Conclusions/interpretation: The economic burden of complications in type 2 diabetes is substantial. Costs of absence from work in this study were found to be greater than of hospital-based care, highlighting the need for considering treatment consequences in a societal perspective in research and policy. Graphical abstract