Personalized Reimbursement Model (PRM) program: A real-world data platform of cancer drugs use to improve and personalize drug pricing and reimbursement in France.

OBJECTIVE: This article describes the Personalized Reimbursement Model (PRM) program methodology, limitations, achievement and perspectives in using real-world data of cancer drugs use to improve and personalize drug pricing and reimbursement in France. MATERIALS AND METHODS: PRM platform aggregates Electronic Pharmacy Records (EPR) data from French medical centers (PRM centers) to build retrospective cohorts of patients treated with injectable cancer drugs in a hospital setting. Data extracted on January 1st, 2020, from breast cancer (BC) patients who received trastuzumab, trastuzumab emtansin or pertuzumab since January 1st, 2011, and from lung cancer (LC) patients who received bevacizumab or atezolizumab since January 1st, 2015, enabled recovering their injectable cancer drugs history from diagnosis date until December 30th, 2019, and served as dataset for assessment. RESULTS: 123 PRM centers provided data from 30,730 patients (25,660 BC and 5,070 LC patients respectively). Overall, 20,942 (82%) of BC and 4,716 (93%) of LC patients were analyzed. Completion rate was above 98% for patients characteristics, diagnostic and treatment related data. PRM centers cover 48% and 33% of BC and LC patients in-hospital therapeutic management in France, respectively. Distribution of BC and LC patients therapeutic management, by medical center category and geographic location, was similar in PRM centers to all French medical centers, ensuring the representativeness of the PRM platform. CONCLUSION: PRM Platform enabled building a national database generating on demand Real-World Evidence based on EPR. This enabled the first performance-based risk-sharing arrangements based on PRM data, between the CEPS and Roche, for atezolizumab cancer immunotherapy in metastatic non-small cell lung cancer indication.

Pilot non dialysis chronic renal insufficiency study (P-ND-CRIS): a pilot study of an open prospective hospital-based French cohort.

BACKGROUND: Before establishing a prospective cohort, an initial pilot study is recommended. However, there are no precise guidelines on this subject. This paper reports the findings of a French regional pilot study carried out in three nephrology departments, before realizing a major prospective Non Dialysis Chronic Renal Insufficiency study (ND-CRIS). METHODS: We carried out an internal pilot study. The objectives of this pilot study were to validate the feasibility (regulatory approval, providing patients with information, availability of variables, refusal rate of eligible patients) and quality criteria (missing data, rate of patients lost to follow-up, characteristics of the patients included and non-included eligible patients, quality control of the data gathered) and estimate the human resources necessary (number of clinical research associates required). RESULTS: The authorizations obtained (CCTIRS - CNIL) and the contracts signed with hospitals have fulfilled the regulatory requirements. After validating the information on the study provided to patients, 1849 of them were included in three centres (university hospital, intercommunal hospital, town hospital) between April 2012 and September 2015. The low refusal rate (51 patients) and the characteristics of non-included patients have confirmed the benefit for patients of participating in the study and provide evidence of the feasibility and representativeness of the population studied. The lack of missing data on the variables studied, the quality of the data analyzed and the low number of patients lost to follow-up are evidence of the quality of the study. By taking into account the time spent by CRAs to enter data and to travel, as well as the annual patient numbers in each hospital, we estimate that five CRAs will be required in total. CONCLUSION: With no specific guidelines on how to realize a pilot study before implementing a major prospective cohort, we considered it pertinent to report our experience of P-ND-CRIS. This experience confirms that i) feasibility, ii) quality of data and iii) evaluating the resources required must be validated before carrying out a large prospective cohort study such as ND-CRIS.

The Non-Dialysis Chronic Renal Insufficiency study (ND-CRIS): an open prospective hospital-based cohort study in France.

BACKGROUND: Chronic kidney disease (CKD) amounts to a heavy burden for health services. There is no long-running epidemiological tool for CKD before dialysis. We here present the protocol for a cohort of patients with "non-dialysis" CKD receiving care in the Bourgogne-Franche-Comté region of France. The aim of this cohort was to periodically describe the characteristics of patients included and their care provision, to analyse evolution in care and patients' kidney function outcomes. METHODS: The ND-CRIS cohort is prevalent and incident. Patients are included in the cohort if over 18, with a glomerula filtration rate (GFR) <60 ml/min/1.73 m2, non-dialysed, informed on the research and not having opposed it, and followed by a nephrologist in one of the 9 centres in the region, (3 pilot centres joined by 6 others in 2015). All the patients are followed up, with varying time lapses according to the degree of GFR deterioration. Data is collected by clinical research assistants (CRAs) using a dedicated computerised case-report form (CRF). Professional practices are assessed using indicators defined by the French Health Authority. The follow-up of patients included should enable assessment of the evolution of their GFR and co-morbidities. The periodic descriptions should give insight into evolution in epidemiological terms. DISCUSSION: The ND-CRIS meets a need in epidemiological tools in France for CKD. The cohort does claim to be representative, of ND-CKD patients receiving care from nephrologists. The open and incident nature of the cohort and the number of patients included in the ND-CRIS should provide answers to questions that cannot be answered by smaller solely prevalent cohorts. The numbers of patients included over the study period (2391 patients in 3 centres in 3 years) suggests that the figure of 5000 patients should be reached by 2017. The participation of nephrologists and the rate of inclusions point to the feasibility of the implementation of this cohort. Beyond the information to be found in the CRFs, this cohort should also enable ad hoc studies, in particular in the area of pharmaco-epidemiology, and it could later serve as a research platform and as a public health surveillance tool.