RUBIH2 - Use of NGS in haematological malignancies: From real world data to national recommendations, an innovative program to evaluate the impact of healthcare technology on patient care

Abstract

Background: Knowledge of molecular abnormalities in haematological malignancies led to better understanding of their role in disease development, progression and resistance to treatments. However, effective impact of NGS analyses need to be evaluated. RuBIH2 programme aims to determine appropriate prescription, clinical utility and validity, cost and territorial organisation of NGS under a governmental funded “Programme de Recherche Médico-Economique” (Health Economics Research Program). Methods: We considered 5 haematological malignancies: myelodysplasia, acute lymphoid leukaemia, acute myeloid leukaemia, lymphoma and lymphoproliferative disorders and myeloproliferative disorders. 26 French biomolecular platforms are involved. The program is split into 4 work packages (WP). WP1: micro-costing, WP2: appropriate prescribing guidelines, WP3: evaluation of clinical validity, WP4: recommendations on national organization. Results: WP1: is a micro-costing of NGS on 5 biomolecular platforms to assess production cost depending on the volume of analyses performed, type of pathology and panel size. Based on a bottom-up approach and onsite observations, we calculated an average mean cost. WP2: is to define recommended diagnostic tests according to pathologies and patients’ characteristics. National key opinion leaders (clinicians and biologists) defined a NGS minimal panel depending on therapeutic possibilities. Biological analyses performed at diagnosis, relapse or during follow-up will be classified as mandatory, recommended, under evaluation or obsolete. WP3: is a real-world data study of NGS analyses carried out by 26 biomolecular platforms. Biological and clinical information are collected in an eCRF. Analysis will describe the impact of NGS on patients’ pathways. WP4: will integrate data collected during WP 1-3 to define an optimal national organization and bundled payment scheme. Conclusions: Final objective is to improve patient care and access to NGS based on better stratification and definition of analyses to perform at each stage of patient's care pathways: diagnostic, relapse and follow-up. Clinical trial identification: NCT03750994. Legal entity responsible for the study: Assistance Publique Hôpitaux de Paris. Funding: Direction Générale de l'Offre de Soins (DGOS) - French Ministry of Health. Disclosure: All authors have declared no conflicts of interest.