Objectif: Décrire la mise en œuvre, la certification et le retour d’expérience sur cinq ans d’un système de management de la qualité (SMQ) conforme à la norme ISO 9001:2015 au sein d’un service hospitalo-universitaire de pharmacotoxicologie regroupant des activités de pharmacovigilance, d’addictovigilance, de toxicovigilance et de réponse téléphonique à l’urgence, et analyser les bénéfices et les limites de cette démarche.Méthodes: Le SMQ a été déployé à partir de 2018 selon une approche structurée intégrant l’analyse du contexte et des parties intéressées, la cartographie des processus, la formalisation du système documentaire, la gestion des non-conformités, les audits internes et externes, les revues de processus et de direction, ainsi que le suivi d’indicateurs qualité.Résultats: La certification initiale ISO 9001:2015 a été obtenue en janvier 2021, suivie de deux audits de surveillance et d’un renouvellement de certification en février 2024. Sur cinq ans, 111 non-conformités ont été déclarées par 23 membres du personnel, générant 214 actions d’amélioration, dont 156 (73  %) ont été clôturées. La mise en œuvre du SMQ s’est accompagnée du déploiement d’outils structurants, notamment un logiciel métier dédié, et a permis le maintien de l’activité en situation de crise sanitaire. Les enquêtes de satisfaction ont montré un haut niveau de satisfaction des demandeurs (clarté de l’information : 99,2 % ; qualité des avis médicaux : 97,6  % ; délais de réponse : 95,1  %) ainsi que des autorités de tutelle. Aucun risque majeur n’a été identifié lors des audits.Conclusion: La mise en place d’un SMQ ISO 9001:2015 dans un service de Pharmacotoxicologie est faisable et bénéfique. Elle favorise une organisation robuste et agile, renforce la conformité réglementaire, la traçabilité et constitue un levier structurant pour le dialogue institutionnel. Cette expérience offre un modèle transposable à d’autres structures de vigilance.

OBJECTIVE:To describe the implementation, certification and five-year experience of an ISO9001:2015-compliant quality management system (QMS) within a university hospital Department of Pharmacotoxicology encompassing pharmacovigilance, addictovigilance, toxicovigilance and telephone-based emergency response activities, and to analyse the benefits and limitations of this approach. METHODS:The QMS was deployed from 2018 using a structured approach integrating context and stakeholder analysis, process mapping, documentation system formalisation, non-conformity management, internal and external audits, process and management reviews, and monitoring of quality indicators. RESULTS:Initial ISO9001:2015 certification was obtained in January 2021, followed by two surveillance audits and recertification in February 2024. Over five years, 111 non-conformities were reported by 23 staff members, generating 214 improvement actions, of which 156 (73%) were completed. QMS implementation was accompanied by the deployment of structuring tools, including a dedicated operational software solution, and enabled continuity of activity during a major health crisis. Satisfaction surveys showed high levels of satisfaction among service users (clarity of information: 99.2%; quality of medical advice: 97.6%; response times: 95.1%) and regulatory authorities. No major risks were identified during audits. CONCLUSION:Implementing an ISO9001:2015-based QMS in a Pharmacotoxicology ward is both feasible and beneficial. It supports a robust and agile organisational framework, strengthens regulatory compliance, traceability and constitutes a key driver for institutional dialogue. This experience provides a transferable model for other vigilance structures.

Objective The efficacy of balneotherapy in rheumatology remains unclear. We aimed to estimate its benefits and risks in rheumatology.Methods We conducted a systematic review of randomised trials assessing any European balneotherapy for a rheumatological indication in adults versus any control, on clinical outcomes. We searched PubMed, Cochrane Library, Embase and https://clinicaltrials.gov/ (up to 28 November 2023). We used the Cochrane risk of bias tool version 2, funnel plot and asymmetry tests. We used a random effects model with an inverse-variance weighting method for standardised mean difference (SMD) and risk ratio (RR). We used the Grading of Recommendations Assessment, Development and Evaluation approach for two primary outcomes, pain and quality of life (QoL) at 3 months, and two safety outcomes, withdrawal and any adverse event (AE).Results We included 29 trials in mechanical disorders, 9 in inflammatory diseases and 4 in fibromyalgia. The synthesis suggested a decrease in pain of a very low level of certainty (SMD: -0.72 (95% CI (-1.00; -0.44)), very serious risk of bias and of inconsistency, publication bias strongly suspected); an increase in QoL of a very low level of certainty (SMD: 0.56 (95% CI (0.37; 0.75)), very serious risk of bias and serious risk of inconsistency); inconclusive results regarding the risk of withdrawal (RR: 0.75 (95% CI (0.46; 1.20)), very serious risk of bias and serious risk of imprecision) and of AE (RR: 0.80 (95% CI (0.43; 1.50)), serious risk of bias and of inconsistency and very serious risk of imprecision).Conclusion The certainty of the effect of balneotherapy in rheumatology was very low.PROSPERO registration number CRD42023448206.

Single-arm control trials are increasingly proposed as a potential approach for treatment evaluation. However, the limitations of this design restrict its methodological acceptability. Regulatory agencies have raised concerns about this approach, although it is sometimes required in applications based solely on such studies. Consequently, the need for accurate indirect treatment comparisons has become critical, especially when constructing external control arms using routinely collected data as outcome measurements may differ from those recorded in the single-arm trial leading to potential misclassification of outcomes. This study aimed to quantify the bias from ignoring misclassification of a binary outcome within unanchored indirect comparisons, through simulations, and to propose a likelihood-based method to correct this bias (i.e., the outcome-corrected model). Simulations demonstrated that ignoring misclassification results in significant bias and poor coverage probabilities. In contrast, the outcome-corrected model reduced bias, improved 95% confidence interval coverage probability and root mean square error in various scenarios. The methodology was applied to two hepatocellular carcinoma trials illustrating a practical application. The findings underscore the importance of addressing outcome misclassification in indirect comparisons. The proposed correction method may improve reliability in unanchored indirect treatment comparisons.

Early screening of children with Autism Spectrum Disorder (ASD) needs to be improved to propose early interventions. Parental questionnaires are useful to help primary care professionals and nurseries to detect children’s autism signs. Our primary objective is to estimate the positive predictive value of an early detection kit composed of 2 parental questionnaires M-CHAT-R/F™ (Modified-CHecklist for Autism in Toddlers-Revised/Follow-up) and CSBS DP™-ITC (Communication and Symbolic Behavior Scales Developmental Profile-Infant Toddler Checklist) followed by a psychologist’s confirmation. Methods and Analysis: Boys and girls aged 16 to 30 months, seen at their general or paediatric practice or attending nurseries or early childcare centers, never diagnosed ASD, are eligible. We plan to have a cohort of 1700 children filling the screening kit and expect to detect about 81 children confirmed positive . We also expect to detect other neurodevelopmental disorders than ASD. Discussion: The use of 2 questionnaires and the intervention of trained psychologists should optimize the access to early detection of ASD near home and links between childhood professionals. Our study is coherent with the French organization and existing tools.

Background and ObjectivesCreatine transporter deficiency (CTD) is a rare X-linked genetic disorder characterized by intellectual disability (ID). We evaluated the clinical characteristics and trajectory of patients with CTD and the impact of the disease on caregivers to identify relevant endpoints for future therapeutic trials. MethodsAs part of a French National Research Program, patients with CTD were included based on (1) a pathogenic SLC6A8 variant and (2) ID and/or autism spectrum disorder. Families and patients were referred by the physician who ordered the genetic analysis through Reference Centers of ID from rare causes and inherited metabolic diseases. After we informed the patients and their parents/guardians about the study, all of them gave written consent and were included. A control group of age-matched and sex-matched patients with Fragile X syndrome was also included. Physical examination, neuropsychological assessments, and caregiver impact were assessed. All data were analyzed using R software. ResultsThirty-one patients (27 male, 4 female) were included (25/31 aged 18 years or younger). Most of the patients (71%) had symptoms at <24 months of age. The mean age at diagnosis was 6.5 years. Epilepsy occurred in 45% (mean age at onset: 8 years). Early-onset behavioral disorder occurred in 82%. Developmental trajectory was consistently delayed (fine and gross motor skills, language, and communication/sociability). Half of the patients with CTD had axial hypotonia during the first year of life. All patients were able to walk without help, but 7/31 had ataxia and only 14/31 could walk tandem gait. Most of them had abnormal fine motor skills (27/31), and most of them had language impairment (30/31), but 12/23 male patients (52.2%) completed the Peabody Picture Vocabulary Test. Approximately half (14/31) had slender build. Most of them needed nursing care (20/31), generally 1-4 h/d. Adaptive assessment (Vineland) confirmed that male patients with CTD had moderate-to-severe ID. Most caregivers (79%) were at risk of burnout, as shown by Caregiver Burden Inventory (CBI) > 36 (significantly higher than for patients with Fragile X syndrome) with a high burden of time dependence. DiscussionIn addition to clinical endpoints, such as the assessment of epilepsy and the developmental trajectory of the patient, the Vineland scale, PPVT5, and CBI are of particular interest as outcome measures for future trials. Trial Registration InformationANSM Registration Number 2010-A00327-32.

Purpose MRI is the main imaging modality for pediatric brain tumors, but amino acid PET can provide additional information. Simultaneous PET-MRI acquisition allows to fully assess the tumor and lower the radiation exposure. Although symptomatic posterior fossa tumors are typically resected, the patient management is evolving and will benefit from an improved preoperative tumor characterization. We aimed to explore, in children with newly diagnosed posterior fossa tumor, the complementarity of the information provided by amino acid PET and MRI parameters and the correlation to histopathological results. Patients and Methods Children with a newly diagnosed posterior fossa tumor prospectively underwent a preoperative 11C-methionine (MET) PET-MRI. Images were assessed visually and semiquantitatively. Using correlation, minimum apparent diffusion coefficient (ADCmin) and contrast enhancement were compared with MET SUVmax. The diameter of the enhancing lesions was compared with metabolic tumoral volume. Lesions were classified according to the 2021 World Health Organization (WHO) classification. Results Ten children were included 4 pilocytic astrocytomas, 2 medulloblastomas, 1 ganglioglioma, 1 central nervous system embryonal tumor, and 1 schwannoma. All lesions showed visually increased MET uptake. A negative moderate correlation was found between ADCmin and SUVmax values (r = −0.39). Mean SUVmax was 3.8 (range, 3.3–4.2) in WHO grade 4 versus 2.5 (range, 1.7–3.0) in WHO grade 1 lesions. A positive moderate correlation was found between metabolic tumoral volume and diameter values (r = 0.34). There was no correlation between SUVmax and contrast enhancement intensity (r = −0.15). Conclusions Preoperative 11C-MET PET and MRI could provide complementary information to characterize pediatric infratentorial tumors.

Background: Unlicensed and off-label (UL/OL) prescriptions have been associated with an increased risk of drug-related problems. Data of their prevalence at hospital discharge remain insufficient. We aimed to describe the prevalence of UL/OL drugs in outpatient prescriptions at discharge in children. Methods: We conducted a retrospective study using the routinely collected health data of children at discharge from 2014 to 2016. The primary reference source for determining licensed labelling was the summaries of product characteristics (SPCs) in a French industry-independent formulary named Thériaque. We described the characteristics of UL/OL prescriptions at discharge and looked for predictors of UL/OL prescriptions. Results: We included 2536 prescriptions of 479 children. Licensed, OL, and UL prescriptions accounted for 58.6% (95% CI: 56.7–60.5), 39.2% (95% CI: 37.3–41.1), and 2.3% (95% CI: 1.7–2.9), respectively. A total of 323 (74%) children received at least one UL/OL drug. Among the licensed drugs, bronchodilators (8.8%) and analgesics (8.6%), and among the OL drugs, antibiotics (2.8%), were the most prescribed. The younger age of the children and higher number of drugs they received increased the probability of UL/OL prescriptions (unadjusted p-value of ≤0.05). Conclusion: The prevalence of UL/OL prescriptions is about 40% at discharge from a pediatric university hospital in France.

OBJECTIVES:To describe the PHAGEinLYON Clinic programme, set up in 2022 to improve access to phage therapy in France using pharmaceutical-grade phages. METHODS:All phage therapy requests received during 2022 were collected prospectively, and reviewed retrospectively to analyse the decision and the patient care pathway (NCT05883995). RESULTS:Of 143 requests for phage therapy, the indication was confirmed at a multidisciplinary team meeting for 57 (40%) patients. Forty-four patients were infected with bacteria that could be targeted easily by phages in France. Finally, 33 patients were treated, including 26 at the study institution, through a compassionate access programme or in a clinical trial. The main indication were complex bone and joint infections, endovascular infection and lung infection. In order to manage these patients, 172 pharmaceutical phage cocktails targeting Staphylococcus aureus and/or Pseudomonas aeruginosa were prepared: 57 for local injection and 99 for intravenous injection. During follow-up, 18 (69%) patients showed a favourable clinical evolution, and six (23%) patients required subsequent phage therapy, either with the same phage with greater exposure, or with a different phage from elsewhere. CONCLUSIONS:Implementation of the PHAGEinLYON Clinic programme in 2022 was associated with groundbreaking access to phage therapy in France.

BACKGROUND AND PURPOSE:Disabling dystonia despite optimal medical treatment is common in Wilson disease (WD). No controlled study has evaluated the effect of deep brain stimulation (DBS) on dystonia related to WD. This study was undertaken to evaluate the efficacy of DBS on dystonia related to WD. METHODS:A meta-analysis of an N-of-1 prospective, randomized, double-blind, multicenter DBS study was conducted at two French WD reference centers. Main inclusion criteria were patients with WD, stabilized for at least 6 months with significant disability due to dystonia despite optimized medical treatment. The subthalamic nucleus (STN) was targeted for bradykinetic patients with tonic dystonia, and the internal globus pallidus (GPi) was chosen for patients with hyperkinetic dystonia. Each patient underwent two periods of DBS "on" and two periods of DBS "off," each lasting 4 months. The order of stimulation conditions was randomized. The primary outcome was the change in the Canadian Occupational Performance Measure Performance (COPM-P) and Satisfaction scores after each 4-month period. Secondary outcomes were changes in the Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) severity and disability scores and Unified Wilson's Disease Rating Scale (UWDRS) scores. RESULTS:Between 12 May 2016 and 7 October 2022, three patients were included. Two patients received bilateral GPi DBS, and one received bilateral STN DBS. There was no change of COPM-P (p = 0.956), BFMDRS, and UWDRS scores. No serious adverse events were reported. CONCLUSIONS:STN or GPi DBS are ineffective on dystonia related to WD.

Purpose Toxic shock syndrome (TSS) is a rare disease responsible for significant morbidity and mortality. Intravenous immunoglobulin (IG) therapy in paediatric TSS could improve shock and organ failure, but more consistent efficacy and safety data are needed. Our objective was to determine whether a randomised clinical trial (RCT) assessing intravenous IG in TSS in children is feasible.Methods We performed a multicentre, feasibility, double-blind RCT assessing efficacy of high-dose intravenous IG versus albumin 4% (control group) within the first 12 hours of shock onset. Included patients were aged above 1 month and below 18 years with suspected TSS and septic shock. Feasibility was assessed by measuring inclusion rate, protocol compliance and missing data regarding death and the Pediatric Logistic Organ Dysfunction-2 (PELOD-2) Score. Other secondary clinical outcomes were evaluated during hospital stay, at 60 day and 1 year.Results 28 patients, admitted in 6 paediatric intensive care units during 36 consecutive months and followed for 1 year, received the allocated treatment: 13 in intravenous IG group, 15 in control group. The median age was 10.6 years and the sex ratio was 1. Inclusion rate was above 50%, protocol deviations were below 30% and missing data regarding death and PELOD-2 Score below 10%. No difference concerning secondary clinical outcomes between groups was observed, and more adverse events were reported in the control group.Conclusion It seems to be feasible to conduct an RCT assessing intravenous IG efficacy and safety in paediatric TSS but must be realised internationally, with choice of a clinically relevant endpoint and a specific design in order to be realistic.Trial registration number NCT02219165.

Clinical trials often last several months or even several years. As the trial progresses, it can be tempting to find out whether the data obtained already answers the question posed at the start of the trial in order to stop inclusions or monitoring earlier. However, knowing and taking into account interim results can sometimes compromise the integrity of the results, which is counterproductive. To minimise this risk and ensure that the treatments are assessed reliably, safety and/or efficacy criteria are monitored during the study by a Data Monitoring Committee. After receiving the results confidentially, the Data Monitoring Committee assesses the benefit/risk ratio of the study treatment and recommends that the trial be continued, modified or terminated. Data Monitoring Committee members issuing these recommendations have an important responsibility: a hasty decision to end the trial may lead to inconclusive results unable to answer the initial question and, inversely, delaying the decision to end the trial may expose the subjects to potentially ineffective or even harmful interventions. The Data Monitoring Committee's task is therefore particularly complex. With this in mind, the round table discussion at the Giens workshops was a chance to review the scientific justification for creating Data Monitoring Committees and to recall the need for their members to receive comprehensive training on the complexities of multiple analyses, confidentiality requirements applying to the results and the need for them to be aware that recommendations to end a trial must be based on data that is robust enough to assess the benefit/risk ratio of the treatment studied.

Endocrine disruptors (ED) are ubiquitous pollutants, possibly implicated in chronic disease. Exposure of vulnerable populations; including neonates, infants and children; must therefore be limited. Informing parents is now a public health challenge. We conducted a quantitative cross-sectional study at the Lyon Mother and child Hospital. We used questionnaires to assess the beliefs and knowledge about ED of parents and pediatric healthcare professionals in the pediatric ward in Lyon, France. A total of 746 questionnaires were completed: 444 for professionals and 302 for parents. The majority of both populations had already heard of ED but only 10% of parents and 5% of professionals felt sufficiently informed. Professionals answered better than parents (73% vs. 60%). The main source of information was similar: media. Only 20% of professionals had read a scientific article about ED and 4% have followed a training. Environmental exposure and EDs is an increasing concern for parents but specific knowledge remains scare for parents and professionals. Specific training is needed.

Abstract Background Attention‐deficit hyperactivity disorder (ADHD) is a frequent comorbidity in children with epilepsy, which management mostly relies on the usual treatments of ADHD, especially methylphenidate. Supplementation with polyunsaturated n‐3 Fatty Acid (PUFA) has been proposed as an alternative therapeutic approach in ADHD without epilepsy but has never been evaluated in epilepsy‐associated ADHD. Methods A multicenter double blind randomized placebo‐controlled trial evaluating supplementation with PUFA, in eicosapentaenoic‐ and docosahexaenoic‐acid form, conjugated to a phospholipid vector (PS‐Omega3) in children aged >6 and <16‐years old, and suffering from any type of epilepsy and ADHD (inattentive or combined type) according to DSM‐V. After a 4‐week baseline period, patients were allocated (1:1) either to placebo group or to PS‐Omega 3 group and entered a 12 week‐double‐blind treatment period which was followed by a 12 week‐open‐label treatment period. The primary outcome was the reduction of the ADHD‐rating scale IV attention‐deficit subscore after 12 weeks of treatment. Results The study was stopped early because of lack of eligible participants and the expected sample size was not reached. Seventy‐four patients were randomized, 44 in PS‐Omega3, and 30 in the placebo group. The reduction after 12 weeks of treatment in the inattention subscore of the ADHD‐IV scale was −1.57 in the PS‐Omega3 group, and −2.90 in the placebo group (p = 0.33, α = 5%). Results were similar after 24 weeks of treatment and for all other ADHD‐related secondary outcomes, with no difference between placebo and PS‐Omega3. Conclusion Our study remaining underpowered, no formal conclusion about the effect of Ps‐Omega3 could be drawn. However, our data strongly suggested that the PS‐Omega 3 formulation used in the current study did not improve ADHD symptoms in children with epilepsy. Plain Language Summary Supplementation with polyunsaturated n‐3 Fatty Acid (PUFA) has been proposed in ADHD but has never been evaluated in patients with both epilepsy and ADHD. To address this issue, we conducted a multicenter double blind randomized placebo‐controlled trial evaluating supplementation with PUFA in children with epilepsy and ADHD. The study was stopped early because of lack of eligible participants, hampering formal conclusion. However, the evolution of the ADHD symptoms at 12 and 24 weeks did not differ between placebo and PUFA supplementation, strongly suggesting that PUFA did not improve ADHD symptoms in children with epilepsy.

The polypill strategy could become widely accepted in cardiovascular prevention due to reduced costs and its simplicity, which promote compliance. Aspirin is often included as a component of the polypill for primary prevention, but three powerful recent trials failed to show any favorable net benefit even in high-risk subgroups. Our objective is to estimate the net benefit associated with aspirin in primary cardiovascular prevention.We simulated the impact of different polypill compositions combining pravastatin, ramipril, hydrochlorothiazide, with or without aspirin, on a realistic French virtual population between 35 and 65 years old. We assessed how this impact on myocardial infarction and stroke varied according to gender, diabetes, and arterial hypertension. We identified the subgroup of individuals whose specific benefit from aspirin was greater than twice the risk of serious bleeding it induced.The absolute benefit associated with aspirin was reduced by co-prescriptions. No subgroup of women benefited from aspirin, and the subgroup of women with a clear net benefit represented 128 women out of 529,421. Men at high risk of cardiovascular death, or with diabetes and hypertension, had a benefit from aspirin exceeding the risk of bleeding induced, but this risk represented more than half of the benefit. No subgroup analyzed did show a benefit greater than twice the risk of bleeding. The proportion of men whose expected benefit from aspirin was greater than twice the risk of bleeding represented 3% of all men. An optimal polypill strategy in primary prevention between the ages of 35 and 65, combining three drugs but not aspirin, can hope to save two out of three strokes and more than one out of two myocardial infarctions. It would prevent a major cardiovascular accident every 16 to 193 individuals treated according to the subgroups considered.Until proven otherwise, aspirin has only a limited place in individuals between 35 and 65 years without a cardiovascular history. We showed how simulating therapeutic strategies on a realistic virtual population could be used for best applying available evidence.

Evidence-based medicine is the cornerstone of shared-decision making in healthcare today. The public deserves clear, transparent and trust-worthy information on drug efficacy. Yet today, many drugs are prescribed and used without solid evidence of efficacy. Clinical trials and randomised clinical trials (RCTs) are the best method to evaluate drug efficacy and side effects. In a shared medical decision-making approach, general practitioners need drug assessment based on patient-important outcomes. The aim of project rebuild the evidence base (REB) is to bridge the gap between the data needed in clinical practice and the data available from clinical research. The drugs will be assessed on clinical patient important outcomes and for a population. Using the Cochrane tools, we propose to analyse for each population and outcome: 1) a meta-analysis based on RCTs with a low risk of bias overall; 2) an evaluation of results of confirmatory RCTs; 3) a statistical analysis of heterrogeneity between RCTs and 4) an analysis of publication bias. Depending on the results of these analyses, the evidence will be categorized in 4 different levels: firm evidence, evidence (to be confirmed), signal or absence of evidence. Project REB proposes a method for reading and interpreting RCTs and their meta-analysis to produce quality data for general practitioners to focus on risk-benefit assessment in the interest of patients. If this data does not exist, it could enable clinical research to better its aim.

Background. Drug-related problems (DRPs) are one of the leading causes of hospital readmissions. Children with chronic diseases are more likely to experience DRPs than adults. The burden and characteristics of drug-related readmissions at and after hospital discharge in children remain unclear. Objective. We aimed to summarize the impact of DRPs at and after hospital discharge on the risk of readmissions in children with chronic diseases. Methods. We conducted a systematic review searching PubMed from inception until January 2022. Study selection criteria were studies assessing the impact of different factors at discharge and after discharge on the risk of hospital readmissions in children with chronic diseases, reporting an assessment of DRPs. DRP could be the only risk factor assessed or one among others. Included studies were assessed with the Risk of Bias in Non -Randomized Studies - of Exposure (ROBINS -E) tool. We summarized the qualitative impact of the reported DRPs on hospital readmission as conclusive (significant association) or inconclusive. Results. Of the 4734 studies initially identified, 13 met inclusion criteria. Eleven studies were retrospective, using electronic health records. The studies assessed the impact of DRPs at or after discharge according to the type of medication (in 6 studies), number of medication (in 5 studies) and medication nonadherence (in 2 studies). From the 44 reported associations between DRPs and the risk of readmission 26 (59% [95% CI, 43%-73%]) were conclusive, of which 81% increased the risk and 19% decreased the risk, and 17 (39% [95% CI, 24%-55%]) were inconclusive. Conclusion. The impact of DRPs on hospital readmissions in children with chronic diseases displayed conflicting results, estimated associations having potentially a serious risk of bias. We need more evidence with a lower risk of bias. (c) 2022 Societe francaise de pharmacologie et de therapeutique. Published by Elsevier Masson SAS. All rights reserved.

Introduction. La médecine fondée sur les preuves (EBM) est la pierre angulaire de la décision médicale partagée. Le public mérite des informations claires, transparentes et dignes de confiance sur l’efficacité des médicaments. Pourtant, aujourd’hui, de nombreux médicaments sont prescrits et utilisés sans preuve solide de leur efficacité. Les essais cliniques randomisés (ECR) et leurs méta-analyses sont les meilleures études pour évaluer l’efficacité des médicaments et leurs effets indésirables, mais leurs résultats ne sont pas facilement interprétables en pratique et sont même parfois discutables par rapport aux données retenues. Dans une approche de décision médicale partagée, les médecins généralistes ont besoin que l’évaluation des médicaments soit fondée sur des résultats importants et pertinents pour le patient. L’objectif du projet Rebuild the Evidence Base (REB) est de combler le fossé entre les données nécessaires à la pratique clinique et les données disponibles de la recherche clinique. Méthodes et analyses. Les médicaments seront évalués selon des critères cliniques importants pour les patients et dans une population donnée. En utilisant les outils Cochrane, pour chaque population et critère d’évaluation choisis, seront réalisées : 1. une méta-analyse, fondée sur des essais contrôlés randomisés (ECR) avec un faible risque global de biais ; 2. l’évaluation des résultats issus des ECR de confirmation ; 3. l’évaluation de l’hétérogénéité statistique entre essais (I2), et 4. l’évaluation du risque de biais de publication. En fonction des résultats de ces analyses, les preuves seront évaluées selon quatre niveaux : preuve solide, résultat probant mais à confirmer, signal à confirmer, ou absence de preuve. Conclusion. Le projet REB propose une méthode de lecture et d’interprétation des essais cliniques randomisés et de leur méta-analyse afin de produire des données de qualité permettant aux médecins généralistes de se centrer sur l’évaluation du bénéfice-risque dans l’intérêt des patients. Si ces données n’existent pas, cela permettra à la recherche clinique de mieux définir ses objectifs.