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Within the life-cycle assessment of health technologies, real-world data (RWD) have until now been of secondary importance to clinical trial data. The availability of massive, better quality RWD, particularly with the emergence of connected devices, the improvement of methods for characterizing populations, make it possible to have a better insight into the effects of treatment, sometimes on a national scale the importance of RWD is likely to progress in the eyes of health technology assessors, going from being traditionally complementary to possibly replacing clinical trial data. This is the fundamental question that the round table, involving experts from the academic and/or hospital, institutional, and industrial worlds, set out to answer. This work served first to establish the current role of RWD in health technology assessment, by distinguishing the main purposes of RWD, the timing of the evaluation in relation to the life cycle of the technology, and then according to the party commissioning or receiving the outcomes of RWD-based studies. Secondly, the round table proposed six general recommendations for more intensive and decisive use of RWD in the assessment and decision-making process.

Resume L’article 51 de la loi de financement de la Securite sociale (LFSS) de 2018 ouvre la voie a des experimentations de nouvelles organisations des parcours de soins, assises sur des modalites de financement derogatoires aux regles actuelles. Le cadre tres ouvert du dispositif suscite une reelle adhesion des acteurs locaux, en temoigne les multiples candidatures recues par les pouvoirs publics en charge du dispositif. Pour maintenir cette dynamique et faire aboutir les projets d’experimentations, nos travaux ont porte sur l’enjeu a clarifier les roles et les responsabilites et a doter les parties prenantes d’un systeme d’evaluation specifique, tenant compte de la nature evolutive de ces projets et de l’agilite necessaire a leur conduite. Nous proposons en particulier une gradation de l’evaluation en 3 niveaux (faisabilite, resultats intermediaires, etude d’impact), qui permettrait de decider de la generalisation d’une experimentation sur la base de resultats intermediaires, ou a contrario d’arreter un projet si les indicateurs de faisabilite n’atteignent pas l’objectif fixe. Nos travaux enfin insistent sur l’importance du travail collectif et developpent les conditions de collaboration entre les acteurs interesses a l’amelioration des parcours de soins, professionnels de sante, offreurs de soins, acteurs economiques et societes savantes.

Resume Le partage et l’echange d’informations sur des sujets de sante, les paroles des patients et de leurs aidants sur differentes plateformes Internet, ainsi que leurs savoirs experientiels representent potentiellement de nouveaux environnements de recherche, en particulier sur les produits de sante. Les informations fournies dans le cadre d’une demarche individuelle et volontaire par les patients inscrits sur ces communautes « virtuelles » ou reseaux en ligne, sont autant de sources pour apprehender et comprendre finement l’usage des produits de sante, evaluer leur efficience et quantifier les possibles effets indesirables en conditions reelles, detecter des signaux faibles de pharmacovigilance ou « d’addictovigilance », mais aussi developper de nouveaux outils d’evaluation afin de generer de nouvelles hypotheses de travail. L’expression libre du vecu, du ressenti des patients et de la realite des experiences partagees ouvrent egalement le champ aux recherches societales autour des produits de sante, champ encore peu explore a ce jour. La recherche sur les produits de sante est soumise a une reglementation etablie et fait appel a des moyens et des methodologies qui jusqu’ici n’ont que peu evolue. Dans ce domaine, le developpement des communautes de patients en ligne offre de nouvelles perspectives, l’enjeu aujourd’hui etant de definir leur place au sein des moyens de recherche conventionnels. Cette place ne pourra etre acceptee par toutes les parties prenantes qu’a condition d’une bonne comprehension de leurs atouts, limites et contraintes. La table ronde reunie sur ce sujet s’est attachee a explorer et mieux comprendre l’ampleur du phenomene, les differentes typologies existantes des communautes virtuelles et des reseaux en ligne de patients, d’identifier les avantages possibles, les specificites et les limites methodologiques, reglementaires et deontologiques auxquelles les chercheurs sont confrontes actuellement et enfin, d’emettre les premieres recommandations dans ce domaine de recherche en croissance.

Article 51 of the French social security financing act for 2018 creates a derogatory funding system to support experimentation with new ways of organizing care pathways. The growing interest local leaders have expressed for this very open framework has led to a large number of candidate projects presented to public authorities in charge of the system. In order to sustain this dynamic movement and favor the success of these experimental projects, our work focused on clarifying the roles and responsibilities of project leaders and the need for a specific evaluation system that takes into account the evolving nature of these projects and the need for operational adaptability. We propose three levels of evaluation (feasibility, intermediary results and impact assessment) needed to decide on generalizing a given experiment based on its intermediary results, or on the contrary, terminating projects not meeting feasibility expectations. Our work emphasizes the importance of networking and of developing favorable conditions for fruitful collaboration between participating stakeholders-healthcare professionals, health providers, economic partners, learned societies-interested in improving the performance of care pathways.

The healthcare sector is currently facing a new paradigm, the explosion of "big data". Coupled with advances in computer technology, the field of "big data" appears promising, allowing us to better understand the natural history of diseases, to follow-up new technologies (devices, drugs) implementation and to participate in precision medicine, etc. Data sources are multiple (medical and administrative data, electronic medical records, data from rapidly developing technologies such as DNA sequencing, connected devices, etc.) and heterogeneous while their use requires complex methods for accurate analysis. Moreover, faced with this new paradigm, we must determine who could (or should) have access to which data, how to combine collective interest and protection of personal data and how to finance in the long-term both operating costs and databases interrogation. This article analyses the opportunities and challenges related to the use of open and/or "big data", from the viewpoint of pharmacologists and representatives of the pharmaceutical and medical device industry.

Early market access of health products is associated with a larger number of requests for information by the health authorities. Compared with these expectations, the growing expansion of health databases represents an opportunity for responding to questions raised by the authorities. The computerised nature of the health system provides numerous sources of data, and first and foremost medical/administrative databases such as the French National Inter-Scheme Health Insurance Information System (SNIIRAM) database. These databases, although developed for other purposes, have already been used for many years with regard to post-registration studies (PRS). The use thereof will continue to increase with the recent creation of the French National Health Data System (SNDS [2016 health system reform law]). At the same time, other databases are available in France, offering an illustration of "product use under actual practice conditions" by patients and health professionals (cohorts, specific registries, data warehouses, etc.). Based on a preliminary analysis of requests for PRS, approximately two-thirds appeared to have found at least a partial response in existing databases. Using these databases has a number of disadvantages, but also numerous advantages, which are listed. In order to facilitate access and optimise their use, it seemed important to draw up recommendations aiming to facilitate these developments and guarantee the conditions for their technical validity. The recommendations drawn up notably include the need for measures aiming to promote the visibility of research conducted on databases in the field of PRS. Moreover, it seemed worthwhile to promote the interoperability of health data warehouses, to make it possible to match information originating from field studies with information originating from databases, and to develop and share algorithms aiming to identify criteria of interest (proxies). Methodological documents, such as the French National Authority for Health (HAS) recommendations on "Les etudes post-inscription sur les technologies de sante (medicaments, dispositifs medicaux et actes). Principes et methodes" [Post-registration studies on health technologies (medicinal products, medical devices and procedures). Principles and methods] should be updated to incorporate these developments.

Resume La mise sur le marche precoce de produits de sante s’accompagne d’une demande accrue d’informations par les autorites de sante. En regard de cette attente, l’enrichissement croissant des bases de donnees de sante represente une opportunite de reponse aux interrogations des autorites. L’informatisation du systeme de sante offre de nombreuses sources de donnees, en premier lieu avec les bases medico-administratives telles que le systeme national d’information inter-regimes de l’Assurance maladie (SNIIRAM). Ces bases de donnees, bien que developpees pour des objectifs autres, sont deja utilisees depuis de nombreuses annees a des fins d’etudes post-inscription (EPI). Leur utilisation n’ira que croissante avec la creation du systeme national des donnees de sante (SNDS) recemment etabli (loi de modernisation de notre systeme de sante de 2016). En parallele, il existe en France d’autres bases de donnees permettant d’approcher « l’utilisation en conditions reelles » des produits par les patients et les professionnels de sante (cohortes, registres specifiques, entrepots de donnees…). Sur la base d’une analyse preliminaire des demandes d’EPI, il apparait qu’environ deux tiers d’entre elles auraient trouve au moins en partie reponse dans les bases de donnees existantes. Le recours a ces bases comporte certains inconvenients, mais aussi de nombreux avantages qui sont listes. Afin d’en faciliter l’acces et d’en optimiser l’utilisation, il est apparu important d’exprimer des recommandations visant a faciliter cette evolution et a en garantir les conditions de validite technique. Parmi les recommandations formulees figure en particulier la necessite d’actions visant a favoriser la visibilite des travaux effectues dans le champ des EPI sur bases de donnees. En outre, il apparait utile de favoriser l’interoperabilite des entrepots de donnees de sante, de permettre l’appariement d’informations issues d’etudes de terrain avec celles provenant de bases de donnees, ou encore de developper et partager des algorithmes destines a identifier des criteres d’interet (proxies). Les documents methodologiques comme celui des recommandations de la Haute Autorite de sante (HAS) sur « les etudes post-inscription sur les technologies de sante (medicaments, dispositifs medicaux et actes). Principes et methodes » devraient, par leur actualisation, integrer ces evolutions.

Medical devices (MDs) cover a wide variety of products. They accompany changes in medical practice in step with technology innovations. Innovations in the field of MDs can improve the conditions of use of health technology and/or modify the organisation of care beyond the strict diagnostic or therapeutic benefit for the patients. However, these non purely clinical criteria seem to be only rarely documented or taken into account in the assessment of MDs during reimbursement decisions at national level or for formulary listing by hospitals even though multidimensional models for the assessment of health technologies have been developed that take into account the views of all stakeholders in the healthcare system In this article, after summarising the background concerning the assessment of health technologies in France, a definition of non-clinical criteria for the assessment of MDs is proposed and a decision tree for the assessment of MDs is described. Future lines of approach are proposed as a conclusion.

Resume Les dispositifs medicaux (DM) couvrent une large variete de produits. Ils accompagnent, au gre des innovations technologiques, l’evolution des pratiques medicales. Les innovations dans le champ du DM peuvent ameliorer les conditions d’utilisation de la technologie de sante et/ou modifier l’organisation des soins au-dela du strict benefice therapeutique ou diagnostique pour le patient. Cependant, ces criteres non purement cliniques semblent n’etre que rarement documentes ou pris en compte dans l’evaluation des DM lors des decisions de remboursement au niveau national ou de referencement au niveau des etablissements de sante alors meme que des modeles d’evaluation multidimensionnelle des technologies de sante, prenant en compte le point de vue de l’ensemble des acteurs du systeme de sante, ont ete elabores. Dans cet article, apres avoir rappele le contexte de l’evaluation des technologies de sante en France, une definition des criteres d’evaluation non clinique des DM est proposee et un arbre de decision pour l’evaluation des DM est decrit. Des pistes de reflexion sont proposees en conclusion.

Resume Le concept de telemedecine a ete officialise en France avec la loi hopital patients sante territoires (HPST) de 2009 et le decret d’application de 2010. De nombreuses experimentations ont ete mises en œuvre et les institutions de regulation (Ministere, Agence regionale de sante [ARS], Haute autorite de sante [HAS]…) ont emis diverses orientations et recommandations tant sur le volet organisationnel que sur les attentes d’evaluation. C’est dans ce contexte que la table ronde a souhaite emettre des recommandations sur differents axes de la telesurveillance medicale (la place de la telesurveillance medicale, le parcours reglementaire, les principes d’evaluation, les modalites de prises en charge et les conditions pour un deploiement perenne et fluide). Si les nouvelles approches utilisant la telesurveillance medicale, font l’objet de nombreuses etudes conduisant au postulat qu’il existe un benefice a la fois sur un plan clinique et pour la qualite de vie du patient, la demonstration de l’impact sur l’organisation des soins et du benefice medico-economique restent a mieux preciser (criteres, methodes, moyens). De meme, les cadres contractuels du deploiement de la telesurveillance existent mais sont complexes ; ils font appels a de nombreux interlocuteurs (Direction generale de l’offre de soins [DGOS], ARS, HAS, Agence des systemes d’information partages de sante [ASIP], Commission nationale informatique et libertes [CNIL], Conseil national de l’ordre des medecins [CNOM]…) qui beneficieraient du partage d’une meme approche et de fluidifier les partenariats. A ce stade, l’enjeu est aussi de definir les conditions permettant la validation d’un modele economique stable pour engager des changements organisationnels. La problematique est ici de mener de front son evaluation et sa mise en œuvre. L’acces aux donnees patients, en particulier a celles des caisses d’Assurance maladie et leur exploitation, pourrait permettre une plus grande efficacite de la demarche. De plus, la non-fongibilite budgetaire des differentes enveloppes budgetaires des differents secteurs d’activites limite la consolidation des impacts economiques. Les modalites de financement devront donc etre adaptees a cette nouvelle repartition des roles, a la fois au cœur du systeme de sante et de l’ecosysteme industriel. Toutes ces evolutions contribueront a ce que les responsables de notre systeme de sante puissent porter cette nouvelle ambition en etroite relation avec tous les acteurs de cette economie.

Resume Le dispositif medical se caracterise par une grande heterogeneite (de l’abaisse langue a l’equipement d’imagerie, en passant par le dispositif implantable ou invasif), un cycle de vie court des produits avec des innovations incrementales recurrentes (de 18 mois a 5 ans) et un caractere operateur dependant : le plus souvent l’utilisateur clinicien doit realiser un acte de pose (prothese de hanche, stimulateur cardiaque), un acte therapeutique qui utilise un dispositif invasif non implantable (sonde d’ablation d’un foyer arythmique, ballonnet d’angioplastie, systeme de spondyloplastie par extension) voire un acte de suivi des dispositifs actifs implantes (suivi au long court des patients beneficiant d’un defibrillateur cardiaque, ou des malades parkinsoniens implantes d’un systeme de stimulation cerebrale profonde). La table ronde a souhaite se concentrer sur les procedures d’evaluation scientifique des dispositifs medicaux et des actes associes en vue de leur tarification et financement par l’Assurance maladie. Elle a ainsi pose pour postulat que le corpus de donnees disponibles etait suffisant et compatible avec une evaluation scientifique du benefice clinique. Les etudes post-inscription, qui participent a la continuite de l’evaluation des dispositifs medicaux, n’ont pas ete abordees. En outre, elle a choisi de centrer ses reflexions sur les dispositifs utilises dans les etablissements de sante (ES), les plus representatifs de l’association entre dispositifs et actes medicaux techniques. Le contexte des multiples procedures reglementaires, permettant la prise en charge des dispositifs medicaux et des actes, est rappele. Les problematiques liees plus specifiquement aux actes eux-memes, aux dispositifs medicaux non implantes et aux dispositifs medicaux innovants sont ensuite developpees. Dans chacune des trois situations, les propositions et les points de discussion de la table ronde sont presentes.

The way patients and their caregivers share information on various online platforms about health topics and their own experiential knowledge presents new potential environments for research, particularly as concerns health products. The information provided individually and voluntarily by patients who are members of these online communities is a new resource for identifying and understanding precisely how health products are used, assessing their effectiveness, quantifying potential adverse effects in real-life situations, detecting subtle signs that are significant for experts in pharmacovigilance and addiction studies, and developing new assessment tools to help form new working hypotheses. How patients freely express their experiences and feelings and the reality of what they share also opens the way for societal research into health products, a field that is still under-explored. Well-established regulations govern research into health products, which uses resources and methodologies that have changed little over the years. However, the development of online communities of patients presents new possibilities in this field. The challenge we face today is defining their place among traditional research techniques. This place cannot be accepted by all stakeholders unless we first establish a firm understanding of the advantages, limitations, and constraints of these communities. The round table on this topic endeavoured to: explore these issues and develop a better understanding of the phenomenon and the different varieties of online communities and networks for patients; identify possible advantages, special features, and methodological, regulatory, and ethical limitations that researchers currently face; and finally, to put forward the first recommendations in this growing field of research.

Resume Les etudes realisees apres l’autorisation de mise sur le marche (AMM) ayant pour objectif d’identifier, caracteriser ou quantifier un ou des risques, appelees Post-authorization safety studies (PASS), ont ete renforcees ces dernieres annees, avec la mise en place des plans de gestion de risque (PGR), dont le cadre reglementaire europeen a ete defini en 2005 et qui vient d’etre modifie recemment dans le cadre de la revision communautaire. Ces etudes de securite, interventionnelles ou non, portent sur un medicament commercialise, qu’il soit ou non utilise dans les conditions de l’autorisation de mise sur le marche (AMM). En dehors de ces etudes de securite, d’autres etudes ayant un objectif principal qui n’est pas l’evaluation du risque, notamment l’evaluation de l’efficacite, la description des conditions de prescription et d’utilisation en situation reelle, la pharmacocinetique, l’impact de sante publique peuvent completer les donnees de securite disponibles. La Table Ronde a analyse les etudes de securite post-AMM des plans de gestion du risque des industriels presents et a degage des caracteristiques relatives aux actions proposees. Quant au cahier des charges et au choix de la methodologie, seul un canevas general a pu etre esquisse compte tenu de la complexite et de la diversite des situations de risque medicamenteux.

Les etudes post-inscription documentent, en situation reelle, les conditions de mise sous traitement, les populations reellement traitees, les durees de traitement, l'observance, les benefices des traitements, l'impact du traitement sur les strategies therapeutiques, l'organisation des soins et l'impact de sante publique, ... Leurs donnees sont utilisees notamment lors d'une reevaluation anticipee ou lors du renouvellement d'inscription au remboursement. Cent trente quatre demandes de la Commission de la Transparence (CT) et/ou du Comite Economique des Produits de Sante (CEPS) ont ete faites depuis 1997 et leurs resultats sont pris en compte et participent a la reevaluation du Service Medical Rendu (SMR) et de l'Amelioration du Service Medical Rendu (ASMR) anticipes. Au cours de cette table ronde des Rencontres Nationales de Pharmacologie Clinique de Giens, les difficultes de realisation des etudes post-inscription medicaments ont ete identifiees et des propositions ont ete formulees dans les domaines du dialogue autour de la demande, de la previsibilite et de l'anticipation des demandes et de la formation des medecins.

Non-interventional research is an essential approach to gathering data in different situations and it often complements other research methodologies, such as biomedical research and research aimed at evaluating usual care. Yet the legislative framework for this type of research is nonexistent, and this void poses a number of problems for non-interventional researchers, including an absence of any guarantee of quality and therefore of reliability, a limited recognition of the research beyond our borders, cumbersome administrative procedures, and a lack of visibility. In light of the growing demand for data, particularly in post marketing authorisation for drugs, which largely relies on non-interventional methods, the Round Table participants have issued a set of proposals for a future legislative framework for this type of research.

International audience; The concept of telemedicine was formalised in France in the 2009 "Hospital, patients, health territories" (loi hôpital, patients, santé, territoire) law and the 2010 decree through which it was applied. Many experiments have been carried out and the regulatory institutions (Ministry, Regional Health Agency [Agence régionale de santé, ARS], French National Health Authority [Haute autorité de santé, HAS], etc.) have issued various guidance statements and recommendations on its organisation and on the expectations of its evaluation. With this background, the round table wanted to produce recommendations on different areas of medical telemonitoring (the role of telemonitoring, the regulatory system, the principles for assessment, methods of use and conditions for sustained and seamless deployment). Whilst many studies carried out on new medical telemonitoring approaches have led to the postulate that it offers benefit, both clinically and in terms of patient quality of life, more information is needed to demonstrate its impact on the organisation of healthcare and the associated medico-economic benefit (criteria, methods, resources). Similarly, contractual frameworks for deployment of telemonitoring do exist, although they are complicated and involve many different stakeholders (Director General fo the Care Offering [Direction générale de l'offre de soins, DGOS], ARS, HAS, Agency for Shared Health Information Systems [Agence des systèmes d'information partagés de santé, ASIP], French National Data Protection Commission [Commission nationale informatique et libertés, CNIL], French National Medical Council [Conseil national de l'Ordre des médecins, CNOM], etc.) that would benefit from a shared approach and seamless exchange between the partners involved. The current challenge is also to define the conditions required to validate a stable economic model in order to promote organisational change. One topical issue is placing the emphasis on its evaluation and operation. Access to patient data, particularly data from the health insurance funds and the use of these data, may enable the process to be more effective. In addition, the budgetary non-fungibility of the various financial envelopes for the different areas of work, restricts the consolidation of financial impact. Funding methods will need to be adapted to this new distribution of roles, both at the centre of the healthcare system and in the industrial ecosystem. All of these changes will help the leaders of our healthcare system to bring this new ambition closer to all of the people working in the health economy.

Medical devices are many and various, ranging from tongue spatulas to implantable or invasive devices and imaging machines; their lifetimes are short, between 18 months and 5 years, due to incessant incremental innovation; and they are operator-dependent: in general, the clinical user performs a fitting procedure (hip implant or pacemaker), a therapeutic procedure using a non-implantable invasive device (arrhythmic site ablation probe, angioplasty balloon, extension spondyloplasty system, etc.) or follow-up of an active implanted device (long-term follow-up of an implanted cardiac defibrillator or of a deep brain stimulator in Parkinson's patients). A round-table held during the XXVIII(th) Giens Workshops meeting focused on the methodology of scientific evaluation of medical devices and the associated procedures with a view to their pricing and financing by the French National Health Insurance system. The working hypothesis was that the available data-set was sufficient for and compatible with scientific evaluation with clinical benefit. Post-registration studies, although contributing to the continuity of assessment, were not dealt with. Moreover, the focus was restricted to devices used in health establishments, where the association between devices and technical medical procedures is optimally representative. An update of the multiple regulatory protocols governing medical devices and procedures is provided. Issues more specifically related to procedures as such, to non-implantable devices and to innovative devices are then dealt with, and the proposals and discussion points raised at the round-table for each of these three areas are presented.

Studies conducted after the marketing authorisation with the objective of identification, characterization or quantification of one or more risks (called PASS "Post-Authorisation Safety Studies"), have been strengthened in the past years with the implementation of the concept of risk management plans (RMPs), established in 2005 in the European regulatory framework and recently amended as part of the community revision. These safety studies, interventional or not, are related to a marketed drug, whether or not the drug is used within the market authorisation conditions. Apart from these safety studies, other studies whose primary objective is not risk assessment, including assessment of efficacy, description of prescription data and use in real life, pharmacokinetics, public health impact ... can complete available safety data.The Giens Round Table examined PASS from the risk management plans of a sample of marketing authorisation holders (participants to the Round Table) and identified the main characteristics of proposed actions. Concerning the specifications and the choice of methodology, only a general outline has been sketched in view of the complexity and diversity of drug risks situations.

Post-registration studies describe parameters of real-life clinical practice such as the treated population, conditions of treatment initiation, treatment duration, adherence, associated benefits/risks as well as the impact on treatment strategies, healthcare procedures and on public health. The results of these studies are used in particular for re-evaluation, re-registration or in applications for reimbursement. Since 1997, 134 requests for post-registration studies have been made either by the French Transparency Committee (CT) and/or the Committee for Pricing of Healthcare Products (CEPS) and the results of these studies were taken into account in the re-evaluation of Actual Benefit (AB) or Improvement in Actual Benefit (IAB). During the roundtable discussion on this subject at the National Clinical Pharmacology Meeting held at Giens (France), the difficulties in performing such studies were identified and proposals were made to predict and anticipate requests for these studies but also for the training of physicians.

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