Why will there never be a randomized trial for NTRK-rearranged tumors?

Type de document :

Article dans une revue scientifique

DOI :

10.1016/j.annonc.2023.04.001

PMID :

URL permanente :

http://hdl.handle.net/20.500.12210/89812

Titre :

Why will there never be a randomized trial for NTRK-rearranged tumors?

Auteur(s) :

Penel, Nicolas [Auteur]

METRICS : Evaluation des technologies de santé et des pratiques médicales - ULR 2694
Lebellec, Loic [Auteur]
Centre Hospitalier Régional Universitaire [CHU Lille] [CHRU Lille]
Blay, Jean-Yves [Auteur]
Centre Léon Bérard [Lyon]

Titre de la revue :

Annals of Oncology

Nom court de la revue :

Ann Oncol

Date de publication :

2023-04-19

ISSN :

1569-8041

Discipline(s) HAL :

Sciences du Vivant [q-bio]

Résumé en anglais : [en]

The optimal assessment of the risk-to-benefit ratio of a new treatment, particularly in oncology, has always been based on the results of prospective randomized trials, ideally carried out in a double-blind manner with a ...
Lire la suite >The optimal assessment of the risk-to-benefit ratio of a new treatment, particularly in oncology, has always been based on the results of prospective randomized trials, ideally carried out in a double-blind manner with a large sample size and overall survival as the primary endpoint. This is still the best level of evidence available and has been an indisputable metric for discussing approval and reimbursement with health authorities for decades. This remains true in clinical oncology. However, it is no longer an appropriate standard in personalized medicine, particularly when focusing on rare subtypes. Indeed, molecular screening can categorize common diseases into several ultra-rare pathologies, each grouping a limited number of patients. For example, patients with NTRK-rearranged tumors are textbook cases. They could be identified and receive personalized treatment; but, in some countries, access to treatment is suspended pending the results of randomized trials. However, the demand is insurmountable.Lire moins >

Langue :

Anglais

Audience :

Internationale

Vulgarisation :

Non

Établissement(s) :

Université de Lille
CHU Lille

Collections :

METRICS : Evaluation des technologies de santé et des pratiques médicales - ULR 2694

Date de dépôt :

2023-11-15T02:08:45Z
2024-03-28T10:39:19Z

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