Lise Rochaix

In a context of increasingly limited resources, a number of strategies, such as the adoption of decrementally cost-effective interventions (d-CEIs), which are both less clinically effective and less costly, could offer potential levers at enhancing both efficiency and equity in healthcare systems. These interventions are located in the SouthWest (S-W) quadrant of the cost-effectiveness plane, and have yet received little attention from researchers or HTA agencies as they are often perceived as per se “unethical” or “unacceptable”, hence the reference sometimes made to the S-W quadrant as the ‘Far West’. The purpose of our paper is to investigate policy-makers’ willingness to adopt d-CEIs using a choice experiment. We use a two-stage pairwise DCE survey to elicit (i) preferences for d-CEIs’ attributes in forced choices and (ii) adoption preferences, i.e. the determinants of d-CEIs’ adoption (unforced choices). We investigate the effect (and trade-offs) between three attributes: health loss (very small to significant), reversibility defined as the possibility to switch back to usual care (from possible to hardly possible) and cost-savings (from 5% to 15% of a fixed budget). Such trade-offs are contextualized by using two sensitivity attributes: disease severity (low and moderate) and savings uncertainty (low and high). Our final sample consists of 180 respondents with 46.7% originating from France and the remaining respondents from other EU countries. All attributes’ levels have a significant effect in the two decision stages. The “health loss” attribute dominates in the first stage followed by “reversibility”: we calculate that decision-makers would require 28.3% increase of budget savings to be indifferent between a scenario of small versus significant health losses and 14.5% budget savings to be indifferent between a scenario of possible and hardly possible reversibility. In contrast, the “reversibility” attribute dominates in the second stage suggesting that anticipated regret may play a role in adoption decisions.

Studying quasi-experimental data from French hospitals from 2010 to 2013, we test the effects of a substantial diagnosis-related group (DRG) tariff refinement that occurred in 2012, designed to reduce financial risks of French maternity wards. To estimate the resulting DRG incentives with regard to the choice between scheduled C-sections and other modes of child delivery, we predict, based on pre-admission patient characteristics, the probability of each possible child delivery outcome and calculate expected differences in associated tariffs. Using patient-level administrative data, we find that introducing additional severity levels and clinical factors into the reimbursement algorithm had no significant effect on the probability of a scheduled C-section being performed. The results are robust to multiple formulations of DRG financial incentives. Our paper is the first study that focuses on the consequences of a DRG refinement in obstetrics and develops a probabilistic approach suitable for measuring the expected effects of DRG fee incentives in the presence of multiple tariff groups.

Introduction End-stage renal disease (ESRD) affects 84 000 persons in France and costs an estimated €4.2 billion. Education about their disease empowers patients and allows improved management of their disease and better health outcomes. This study aims to explore whether the addition of an interactive web-based platform to patient education is effective and cost-effective and additionally whether complementing the platform with social functions and features improves its performance. Methods and analysis Patients with severe, ESRD or post-transplant will be randomised 1:1:1 to either standard therapeutic education; or education using a specific application; or the enhanced interactive app with social features. The total follow-up duration is 18 months. Primary endpoint is the cost utility of using app-based therapeutic intervention; secondary endpoints are: compliance with treatment guidelines, app use (professionals and patients), patients’ satisfaction, budget impact analysis. Ethics and dissemination The findings will inform the deployment and reimbursement of the application. The study has ethical approval by the Ile de France ethics committee. Dissemination of the results will be presented at conferences and in peer-reviewed publications.

To cite: Hassine A, Antoni G, Fender M, et al. Combined incentive actions, focusing on primary care professionals, to improve cervical cancer screening in women living in socioeconomically disadvantaged geographical areas: a study protocol of a hybrid cluster randomised effectiveness and implementation trial-RESISTE. BMJ Open 2022;12:e065952. Introduction Cervical cancer (CC) causes thousands of deaths each year. Nearly 100% of cases are caused by oncogenic strains of human papillomavirus (HPV). In most industrialised countries, CC screening (CCS) is based on the detection of HPV infections. For many reasons including lower adherence to CCS, underserved women are more likely to develop CC, and die from it. We aim to demonstrate that the use of incentives could improve screening rates among this population. Methods and analysis Our cluster randomised, controlled trial will include 10 000 women aged 30–65 years eligible for CCS, living in deprived areas in four French departments, two mainlands and two overseas, and who did not perform physician-based HPV testing within the framework of the nationally organised screening programme. HPV self-sampling kit (HPVss) will be mailed to them. Two interventions are combined in a factorial analysis design ending in four arms: the possibility to receive or not a financial incentive of €20 and to send back the self-sampling by mail or to give it to a health professional, family doctor, gynaecologist, midwife or pharmacist. The main outcome is the proportion of women returning the HPVss, or doing a physician-based HPV or pap-smear test the year after receiving the HPVss. 12-month follow-up data will be collected through the French National Health Insurance database. We expect to increase the return rate of HPV self-samples by at least 10% (from 20% to 30%) compared with the postal return without economic incentive. Ethics and dissemination Ethics approval was first obtained on 2 April 2020, then on July 29 2022. The ethics committee classified the study as interventional with low risk, thus no formal consent is required for inclusion. The use of health insurance data was approved by the Commission Nationale Informatique et Libertés on 14 September 2021 (ref No 920276). An independent data security and monitoring committee was established. The main trial results will be submitted for publication in a peer-reviewed journal. Trial registration number NCT04312178 .

Background Spread of resistant bacteria causes severe morbidity and mortality. Stringent control measures can be expensive and disrupt hospital organization. In the present study, we assessed the effectiveness and cost-effectiveness of control strategies to prevent the spread of Carbapenemase-producing Enterobacterales (CPE) in a general hospital ward (GW). Methods A dynamic, stochastic model simulated the transmission of CPE by the hands of healthcare workers (HCWs) and the environment in a hypothetical 25-bed GW. Input parameters were based on published data; we assumed the prevalence at admission of 0.1%. 12 strategies were compared to the baseline (no control) and combined different prevention and control interventions: targeted or universal screening at admission (TS or US), contact precautions (CP), isolation in a single room, dedicated nursing staff (DNS) for carriers and weekly screening of contact patients (WSC). Time horizon was one year. Outcomes were the number of CPE acquisitions, costs, and incremental cost-effectiveness ratios (ICER). A hospital perspective was adopted to estimate costs, which included laboratory costs, single room, contact precautions, staff time, i.e. infection control nurse and/or dedicated nursing staff, and lost bed-days due to prolonged hospital stay of identified carriers. The model was calibrated on actual datasets. Sensitivity analyses were performed. Results The baseline scenario resulted in 0.93 CPE acquisitions/1000 admissions and costs 32,050 €/1000 admissions. All control strategies increased costs and improved the outcome. The efficiency frontier was represented by: (1) TS with DNS at a 17,407 €/avoided CPE case, (2) TS + DNS + WSC at a 30,700 €/avoided CPE case and (3) US + DNS + WSC at 181,472 €/avoided CPE case. Other strategies were dominated. Sensitivity analyses showed that TS + CP might be cost-effective if CPE carriers are identified upon admission or if the cases have a short hospital stay. However, CP were effective only when high level of compliance with hand hygiene was obtained. Conclusions Targeted screening at admission combined with DNS for identified CPE carriers with or without weekly screening were the most cost-effective options to limit the spread of CPE. These results support current recommendations from several high-income countries.

This study measures inequality and inequity in the distribution of clinical trials on cancer drug development between 1996 and 2016, comparing the number of clinical trials with cancer need, proxied by prevalence, incidence, or survival rates for both rare and non-rare cancers. We leverage a unique global database of clinical trials activity and costs between 1996 and 2016, constructed for 227 different cancer types to measure for rare and non-rare cancers: i) inequalities and inequity of clinical trial activity, considering all trials as well as split by R&D stage; ii) inequalities and inequity in R&D investment proxied by trial enrollment and duration; iii) evolution of inequity over time. Inequalities are measured with concentration curves and indices and inequities measured with the health inequity index. We find four important results. First, we show pro-low need inequity across cancer types for both rare and non-rare cancers, for all need proxies. Second, we show inequity differs across R&D stages and between rare and non-rare cancers. The distribution of clinical trials for non-rare cancers disproportionately favors low-need non-rare cancers from earlier to later stages of R&D, whilst for rare cancers this only occurs in Phase 2 trials. Third, inequity analyses in R&D investment show that only trial enrollment for rare cancers and trial duration for non-rare cancers are disproportionately concentrated among low-need cancers. Finally, while pro-low need inequity has persisted between 1996 and 2016 for non-rare cancers, it has faded for rare cancers post-EU orphan drugs’ legislation.

Pour une bonne allocation des ressources de la puissance publique, l’objectif de l’évaluation socioéconomique est d’apprécier, avec des critères transparents, l’utilité et l’intérêt collectif de projets d’investissement public ou de politiques publiques, y compris vis-à-vis de la protection de la santé des personnes dont l’enjeu est d’une importance particulière. Pourtant, la prise en compte des effets sur la santé dans les évaluations est souvent réduite à des tentatives isolées et exploratoires. Le manque d’outils ou de valeurs monétaires permettant d’évaluer et de valoriser les effets relatifs à la santé des personnes – on parlera d’« effets de santé » dans ce rapport pour couvrir à la fois les maladies et la qualité de vie, terme utilisé en économie de la santé1 – apparaît comme l’une des raisons de leur absence des évaluations socioéconomiques ex ante, ce qui revient finalement à considérer qu’ils sont nuls lors de la prise de décision. Dans ce contexte, mais également pour encourager le transfert des avancées méthodologiques du secteur de la santé (tel le recours à un indicateur synthétique de santé) vers d’autres secteurs et rendre explicite un coût de l’inaction, le Comité d’experts des méthodes d’évaluation socioéconomique, installé par France Stratégie et le Secrétariat général pour l’investissement, a lancé un groupe de travail sur l’évaluation socioéconomique des effets de santé des projets d’investissement public. Il a confié la présidence de ce groupe à Benoît Dervaux, économiste de la santé, maître de conférence-praticien hospitalier de santé publique à l’université de Lille et au Centre hospitalier universitaire de Lille (UMR 1167 RID-AGE), et Lise Rochaix, professeure agrégée des universités en sciences économiques à l’université de Paris 1 Panthéon-Sorbonne, affiliée à l’École d’économie de Paris et responsable scientifique de la chaire Hospinnomics, EEP et AP-HP. Ce groupe a cherché à établir une méthodologie d’estimation des coûts tangibles et intangibles des effets de santé qui puisse être appliquée à différents secteurs, dont quatre en particulier : dommages psychologiques des inondations, bénéfices de bénéfices de santé de l’activité physique dans l’espace public.

Introduction L’objectif principal de cet essai randomisé multicentrique (11 centres hospitaliers) est de démontrer l’efficience (analyse coût–utilité) d’une plateforme digitale (DOCMADI©) d’éducation thérapeutique possédant une composante communautaire et destinée aux patients avec IRC stade 5, dialysés ou transplantés, dans le cadre d’une promotion AP–HP, PRME 2015 (ministère de la Santé). Description Randomisation en 3 bras (pas d’accès à la plateforme [G1], accès sans [G2] et avec [G3] fonctionnalités communautaires). Les patients complètent 3 questionnaires en ligne (M0 conditionnant l’inclusion, M9, M18). Méthodes Critère de jugement principal : différence des coûts rapportés à la différence de survie ajustée à la qualité de vie. Critères secondaires : bénéfices cliniques (survie à M18, observance), effet sur l’organisation des soins (appels au service, passages aux urgences, événements indésirables évités), analyse des coûts et conséquences de l’intervention. Résultats Entre 05/2018 et 12/2020, 815 patients ont été randomisés et 539 inclus (G1 = 180, G2 = 189, G3 = 170), dont 78 au stade 5, 67 dialysés et 394 transplantés. Au 01/06/2021, 433 patients ont complété M9 et 323 M18. Les premiers résultats indiquent des freins à l’inclusion liés aux médecins (difficulté de toute étude clinique), aux patients (non-remplissage à M0) et à des problèmes techniques (logiciel ePro). L’utilisation de la plateforme a fortement augmenté en 2020, en particulier lors de la première vague épidémique de COVID-19, validant son intérêt comme outil d’information pour les centres participants. De nombreux « likes », commentaires et questions sur les forums ont permis de mieux comprendre les préoccupations des patients durant la crise sanitaire et validé l’utilité des fonctions sociales. Chiffres clefs d’utilisation de la plateforme pour l’année 2020 : 20 787 pages lues, 4657 téléchargements et jeux-tests joués plus de 1000 fois. Conclusion Les patients ont manifesté leur intérêt pour notre outil. L’analyse coût/utilité prévue en fin d’étude permettra d’évaluer son efficacité médico-économique.

Purpose This study investigates the impact of an intensive case management program on sick leave days, permanent work incapacity levels and treatment costs for severe vocational injuries set up by the French National Insurance Fund in five health insurance districts. Methods The method employed relies on a four-step matching procedure combining Coarsened Exact Matching and Propensity Score Matching, based on an original administrative dataset. Average Treatment effects on the Treated were estimated using a parametric model with a large set of covariates. Results After one-year follow-up, workers in the treatment group had higher sickness absence rates, with 22 extra days, and the program led to 2.7 (95% CI 2.3–3.1) times more diagnoses of permanent work incapacity in the treatment group. With an estimated yearly operational cost of 2,722 € per treated worker, the average total extra treatment cost was 4,569 € for treated workers, which corresponds to a cost increase of 29.2% for the insurance fund. Conclusions The higher costs found for the treatment group are mainly due to longer sick leave duration for the moderate severity group, implying higher cash transfers in the form of one-off indemnities. Even though workers in the treated group have more diagnoses of permanent work incapacity, the difference of severity between groups is small. Our results on longer sick leave duration are partly to be explained by interactions between the case managers and the occupational physicians that encouraged patients to stay longer off-work for better recovery, despite the higher costs that this represented for the insurance fund and the well-documented adverse side effects of longer periods off-work.