RESUMO
BACKGROUND: Although health economic evaluations (HEEs) are increasingly common for therapeutic interventions, they appear to be rare for the use of risk prediction models (PMs). OBJECTIVES: To evaluate the current state of HEEs of PMs by performing a comprehensive systematic review. METHODS: Four databases were searched for HEEs of PM-based strategies. Two reviewers independently selected eligible articles. A checklist was compiled to score items focusing on general characteristics of HEEs of PMs, model characteristics and quality of HEEs, evidence on PMs typically used in the HEEs, and the specific challenges in performing HEEs of PMs. RESULTS: After screening 791 abstracts, 171 full texts, and reference checking, 40 eligible HEEs evaluating 60 PMs were identified. In these HEEs, PM strategies were compared with current practice (n = 32; 80%), to other stratification methods for patient management (n = 19; 48%), to an extended PM (n = 9; 23%), or to alternative PMs (n = 5; 13%). The PMs guided decisions on treatment (n = 42; 70%), further testing (n = 18; 30%), or treatment prioritization (n = 4; 7%). For 36 (60%) PMs, only a single decision threshold was evaluated. Costs of risk prediction were ignored for 28 (46%) PMs. Uncertainty in outcomes was assessed using probabilistic sensitivity analyses in 22 (55%) HEEs. CONCLUSIONS: Despite the huge number of PMs in the medical literature, HEE of PMs remains rare. In addition, we observed great variety in their quality and methodology, which may complicate interpretation of HEE results and implementation of PMs in practice. Guidance on HEE of PMs could encourage and standardize their application and enhance methodological quality, thereby improving adequate use of PM strategies.
Assuntos
Estudos de Avaliação como Assunto , Custos de Cuidados de Saúde , Pesquisa sobre Serviços de Saúde/métodos , Modelos Econômicos , Modelos Estatísticos , Análise Custo-Benefício , Técnicas de Apoio para a Decisão , Humanos , Reprodutibilidade dos Testes , Medição de Risco , Fatores de Risco , Resultado do TratamentoRESUMO
Introduction: Personalized medicine-based treatments in advanced cancer hold the promise to offer substantial health benefits to genetic subgroups, but require efficient biomarker-based patient stratification to match the right treatment and may be expensive. Standard molecular diagnostics are currently very heterogeneous, and tests are often performed sequentially. The alternative to whole genome sequencing (WGS) i.e. simultaneously testing for all relevant DNA-based biomarkers thereby allowing immediate selection of the most optimal therapy, is more costly than current techniques. In the current implementation stage, it is important to explore the added value and cost-effectiveness of using WGS on a patient level and to assess optimal introduction of WGS on the level of the healthcare system.Areas covered: First, an overview of current worldwide initiatives concerning the use of WGS in clinical practice for cancer diagnostics is given. Second, a comprehensive, early health technology assessment (HTA) approach of evaluating WGS in the Netherlands is described, relating to the following aspects: diagnostic value, WGS-based treatment decisions, assessment of long-term health benefits and harms, early cost-effectiveness modeling, nation-wide organization, and Ethical, Legal and Societal Implications.Expert opinion: This study provides evidence to guide further development and implementation of WGS in clinical practice and the healthcare system.