Protocolisation, Use and Development of Anti-Cancer Drugs in the Context of T2A (Case-Mix Based Payment System) Set-Up

Drugs used in oncology represent more than half of the innovative and costly drugs which are not covered by a Group Homogène de Soins (DRG type classification) within the context of the case-mix based payment system (termed T2A). For these drugs, good practice reference guidelines have been drawn up by scientific societies and registration agencies. Recognised indications, relevant indications and situations where the treatment should not be prescribed are defined by the National Institute of Cancer. The reference guidelines should lead towards the good use of these drugs and allow the sick funds to control prescriptions. They should evolve with time, which means that bibliographic monitoring and independent expert opinion is necessary to update them as science provides new data. Manufacturers are involved in this process which in no case should undermine developmental efforts leading to registration. The objective of this protocolisation is to allow all patients early and legitimate access to drugs representing real therapeutic progress. These reference guidelines should be integrated into the life-cycle of a drug and should give rise to new developments allowing the good use of cancer products in situations which have been properly validated

Protocolisation, utilisation et développement des médicaments anticancéreux dans le cadre de la mise en place de la T2A

Les médicaments utilisés en cancérologie représentent plus de la moitié des médicaments innovants et coûteux pris en charge hors d'un groupe homogène de soins dans le cadre de la tarification à l'activité. Pour ces médicaments, des référentiels de bon usage élaborés par les sociétés savantes, les agences d'enregistrement et l'Institut National du Cancer (INCa) définissent les indications reconnues, les indications pertinentes et les situations où le traitement ne doit pas être prescrit. Ces référentiels devraient tendre à un bon usage de ces médicaments et permettre un contrôle des prescriptions par les organismes payeurs. Ces référentiels se doivent d'être évolutifs, d'où la nécessité d'une veille bibliographique et de l'appel à des experts indépendants pour une actualisation en fonction des données nouvelles de la science. Les industriels sont impliqués dans cette démarche qui ne doit en rien se substituer aux efforts des développements pouvant conduire à un enregistrement. L'objectif de cette protocolisation est de permettre un accès précoce et légitime de tous les patients aux médicaments représentant un réel progrès thérapeutique. Ces référentiels doivent s'intégrer dans la vie du médicament et doivent permettre de nouveaux développements permettant le bon usage des produits de cancérologie dans des situations correctement validées

The 2020 BMT CTN Myeloma Intergroup Workshop on Immune Profiling and Minimal Residual Disease Testing in Multiple Myeloma

The fifth annual Blood and Marrow Transplant Clinical Trials Network Myeloma Intergroup Workshop on Immune Profiling and Minimal Residual Disease Testing in Multiple Myeloma was conducted as one of the American Society of Hematology Annual Meeting Scientific Workshops on Thursday December 3, 2020. This workshop focused on four main topics: (1) integrating minimal residual disease into clinical trial design and practice; (2) the molecular and immunobiology of disease evolution and progression in myeloma; (3) adaptation of next-generation sequencing, next-generation flow cytometry, and cytometry by time of flight techniques; and (4) chimeric antigen receptor T-cell and other cellular therapies for myeloma. In this report, we provide a summary of the workshop presentations and discuss future directions in the field

ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children.

Paediatric Strategy Forums have been created by the multistakeholder organisation, ACCELERATE, and the European Medicines Agency to facilitate dialogue between all relevant stakeholders and suggest strategies in critical areas of paediatric oncology drug development. As there are many medicines being developed for B-cell malignancies in adults but comparatively few in children with these malignancies, a Paediatric Strategy Forum was held to discuss the best approach to develop these products for children. It was concluded that as current frontline therapy is highly successful, despite associated acute toxicity, de-escalation of this or substitution of presently used drugs with new medicines can only be undertaken when there is an effective salvage regimen, which is currently not available. Therefore priority should be given to developing treatment for patients with relapsed and refractory mature B-cell lymphomas. The consensus of the clinicians attending the meeting was that CAR T-cells, T-cell engagers and antibody drug conjugates (excluding those with a vinca alkaloid–like drug) presently have the greatest probability of providing benefit in relapse in view of their mechanism of action. However, as producing autologous CAR T-cells currently takes at least 4 weeks, they are not products which could be quickly employed initially at relapse in rapidly progressing mature B-cell malignancies but only for the consolidation phase of the treatment. Global, industry-supported, academic-sponsored studies testing compounds from different pharmaceutical companies simultaneously should be considered in rare populations, and it was proposed that an international working group be formed to develop an overarching clinical trials strategy for these disease groups. Future Forums are planned for other relevant paediatric oncologic diseases with a high unmet medical need and relevant molecular targets.</p