Your browser doesn't support javascript.
loading
Show: 20 | 50 | 100
Results 1 - 2 de 2
Filter
Add more filters










Database
Language
Publication year range
1.
Lancet Oncol ; 22(10): e430-e434, 2021 10.
Article in English | MEDLINE | ID: mdl-34592192

ABSTRACT

During the past decade, health technology assessment bodies have faced new challenges in establishing the benefits of new drugs for individuals and health-care systems. A topic of increasing importance to the field of oncology is the so-called agnostic regulatory approval of targeted therapies for cancer (independent of tumour location and histology) granted on the basis of basket trials. Basket trials in oncology offer the advantage of simultaneously evaluating treatments for multiple tumours, even rare cancers, in a single clinical trial. To address the novel challenges introduced by these trials, an interdisciplinary panel was convened on behalf of the Transparency Committee of the French National Authority for Health to clarify an approach designed to guarantee a transparent, reproducible, and fair assessment of histology-agnostic treatments for reimbursement by the French National Health Insurance Fund. The requirements of this approach include the need for randomisation, clinically relevant endpoints, appropriate correction for multiple significance testing, characterisation of subgroup heterogeneity, and validation of underlying biomarker assays. A prospectively designated external control is encouraged when the implementation of a direct comparison is deemed infeasible. We also underline the importance of recording outcomes from basket trials in a registry for use as future external controls.


Subject(s)
Antineoplastic Agents/therapeutic use , Clinical Trials as Topic , Neoplasms/drug therapy , Research Design , Technology Assessment, Biomedical , Antineoplastic Agents/adverse effects , France , Government Agencies , Humans , Molecular Targeted Therapy , Neoplasms/genetics , Neoplasms/pathology , Treatment Outcome
2.
Eur Respir Rev ; 23(132): 193-214, 2014 Jun.
Article in English | MEDLINE | ID: mdl-24881074

ABSTRACT

Idiopathic pulmonary fibrosis (IPF) is the most frequent chronic idiopathic interstitial pneumonia in adults. The management of rare diseases in France has been organised by a national plan for rare diseases, which endorsed a network of expert centres for rare diseases throughout France. This article is an overview of the executive summary of the French guidelines for the management of IPF, an initiative that emanated from the French National Reference Centre and the Network of Regional Competence Centres for Rare Lung Diseases. This review aims at providing pulmonologists with a document that: 1) combines the current available evidence; 2) reviews practical modalities of diagnosis and management of IPF; and 3) is adapted to everyday medical practice. The French practical guidelines result from the combined efforts of a coordination committee, a writing committee and a multidisciplinary review panel, following recommendations from the Haute Autorité de Santé. All recommendations included in this article received at least 90% agreement by the reviewing panel. Herein, we summarise the main conclusions and practical recommendations of the French guidelines.


Subject(s)
Idiopathic Pulmonary Fibrosis/diagnosis , Idiopathic Pulmonary Fibrosis/therapy , Pulmonary Medicine/standards , Consensus , Diagnostic Imaging/standards , Evidence-Based Medicine/standards , France/epidemiology , Humans , Idiopathic Pulmonary Fibrosis/epidemiology , Predictive Value of Tests , Risk Factors , Treatment Outcome
SELECTION OF CITATIONS
SEARCH DETAIL
...