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The offer of anti-cancer drugs has recently been disrupted by the introduction of checkpoint inhibitors on the market. Currently, one anti-CTLA-4, two anti-PD-1 and two anti-PD-L1 are authorized in the European Union, in seven different types of cancer. The clinical development of these therapies is still in full swing: in July 2017, more than 1 500 clinical trials were evaluating anti-PD-1, anti-PD-L1 and anti-CTLA-4 drugs in about twenty different locations and this number continues to increase. In the short term in France, other immunotherapies, the CAR-T cells, will complete this therapeutic arsenal. These immunotherapies appear as a real revolution in the treatment of some cancers. Nevertheless, many issues are associated with these therapies, particularly regarding the identification of good responders, the proper use of these drugs including the management of therapeutic strategies and safety profile, as well as the organization of care. In addition, the expenses associated with ipilimumab, nivolumab and pembrolizumab are substantial and almost tripled in one year, going from 120 million euros in 2015 to more than 340 million euros in 2016. This raises the question of the ability of the current healthcare system to maintain equitable access to innovation and best treatments for all patients. For all these reasons, the French National Cancer Institute decided to dedicate its thematic annual report on these innovative immunotherapies, targeting in particular checkpoint inhibitors and CAR-T cells, in order to produce an inventory of current data and an analysis regarding the different issues associated with these therapies.

Resume Les progres technologiques dans l’aide au diagnostic et le traitement de l’information pourraient a terme modifier l’organisation des soins de premier recours. Les reformes francaises de 2009 et 2010 conduisent a etudier l’hypothese d’une utilisation par les pharmaciens du TDR pour l’angine streptococcique. Cette perspective apparait d’interet majeur notamment pour l’orientation de populations ne consultant pas ou peu. Mais, elle reclame notamment une cooperation locale entre medecins et pharmaciens, afin d’optimiser l’orientation et les soins. Elle met en evidence le risque d’une diminution de la qualite de la prise en charge si des protocoles rigoureux, la conscience du risque transfere et la tracabilite des actes devaient faire defaut. L’hypothese etudiee met en exergue des points clefs de methode extrapolables a d’autres tests, et a leur possible categorisation selon leurs regles d’usage. Sous ces conditions, les progres technologiques nous semblent, dans le nouveau cadre legislatif francais, ouvrir un horizon considerable a la cooperation medico-pharmaceutique, au profit de la qualite, de l’equite et de l’efficience des soins de premier recours. L’arrete du 24 novembre 2011 qui prevoit la possible delegation du test sous la responsabilite du medecin, ne modifie ni l’analyse ni les conclusions de cet article.

e15031Background: The launch of CAR-T cells raises major issues in terms of therapeutic strategies, safety, organization of care, financial sustainability. Regarding these challenges and the strong...

Resume Le bon usage des medicaments peut etre defini simplement comme l’utilisation du bon medicament, a la bonne dose, pendant la duree necessaire, pour un patient donne qui le tolere correctement. Il est impossible, etant donne le nombre de medicaments et le nombre infini des situations cliniques individuelles de ne pas baser la prescription sur une reference. La reference peut etre unique, lorsque la situation clinique est simple et le medicament, a choisir, unique. Dans ce cas une bonne formation initiale et continue sert de reference au choix. L’usage du RCP (Resume des Caracteristiques du Produit) choisi permet d’obtenir une prescription plus precise et plus sure. Dans beaucoup de cas l’usage de telle ou telle strategie therapeutique peut conduire au resultat souhaite. Dans ces cas, des referentiels scientifiques permettent d’aider le prescripteur a choisir la meilleure strategie. Enfin les strategies therapeutiques peuvent avoir une efficacite egale et des couts tres differents. Des referentiels peuvent etre emis pour diminuer le cout d’une prescription donnee. Les difficultes issues de references ou de referentiels sont : 1. Ils ne peuvent pas couvrir l’integralite des situations cliniques. 2. Ils devraient etre evolutifs a mesure que la science progresse et cela est difficile. 3. La multiplicite des emetteurs aboutit a des messages confus parfois contradictoires. 4. Leur pertinence et leur efficacite devraient etre evaluees. 5. Ils peuvent etre inutilisables pour le prescripteur en raison de leur complexite ou de l’eloignement des situations pratiques. Nous proposons un constat et un certain nombre de solutions pour ameliorer la qualite et la pertinence des references ou referentiels : des structures de coordination choisissant les themes et definissant les methodes de redaction, la creation d’un repertoire des referentiels, un cahier des charges de tout referentiel avec exigence de qualite, une formation initiale et continue des soignants destinataires des referentiels. Il est par ailleurs necessaire de mettre en place des mesures d’impact de ces referentiels et de s’imposer une actualisation adaptee periodique. Tout cela devrait permettre une amelioration mesurable de la qualite des soins.

Proper use of drugs can be defined as the use of the right product, in a correct dosage, during an adequate length of time, for a given patient and provided he has no serious side effects. It is virtually impossible, with such a number of drugs, such a number of clinical situations to prescribe adequately without using references or guidelines. References may lead to a unique choice, when the diagnosis is certain and the drug to be given is unique. With a good initial and continuous medical education, doctors can take easily this type of decision. The Summary of Products Characteristics (SPC) helps them; by sticking to this fundamental reference, prescription might be more precise and safe. In a lot of clinical situations the choice between a large numbers of therapeutic strategies necessitates use of a guideline based on scientific knowledge. Finally, a given therapeutic strategy can be as effective as and considerably less expensive than another. In such cases, payers can drive doctors to the prescription of the less expensive strategy. Some difficulties are common to all references and guidelines: 1. A lot of clinical situations are not covered by guidelines. 2. Guidelines should be updated each time there is a modification of knowledge: it is extremely difficult to do. 3. A great number of guidelines exist, issued by scientific community, health authorities or the payers. Sometime you can find a proposition in a guideline and the reverse in another guideline. It could be confusing. 4. Guidelines should be evaluated rigorously to know if they fulfil their goals. 5. Some of those guidelines simply cannot help doctors. They are too complex or do not take into account practical situations. We have made an inventory of those various guidelines and their weaknesses and we propose some solutions to increase their utility. We propose an analysis of the situation and some solutions to improve the quality and the relevance of the guidelines: to create groups of coordination to choose the aims and the elaboration methods, to initiate a register of guidelines, to list the rules for any new guideline with quality control, to propose a continue medical education for all the health workers. It is necessary to measure the impact of any new recommendation and to do periodical actualisation. The goal of these recommendations is to improve global quality of health.