Rapid access to innovative medicinal products while ensuring relevant health technology assessment. Position of the French National Authority for Health

被引：33

作者：

Vanier, Antoine ^{[1
,2
]}

Fernandez, Judith ^{[1
]}

Kelley, Sophie ^{[1
]}

Alter, Lise ^{[1
]}

Semenzato, Patrick ^{[1
]}

Alberti, Corinne ^{[3
,4
]}

Chevret, Sylvie ^{[5
]}

Costagliola, Dominique ^{[6
]}

Cucherat, Michel ^{[7
]}

Falissard, Bruno ^{[8
]}

Gueyffier, Francois ^{[9
]}

Lambert, Jerome ^{[5
]}

Lengline, Etienne ^{[10
]}

Locher, Clara ^{[11
]}

Naudet, Florian ^{[12
,13
]}

Porcher, Raphael ^{[14
]}

Thiebaut, Rodolphe ^{[15
]}

Vray, Muriel ^{[16
]}

Zohar, Sarah ^{[17
,18
]}

Cochat, Pierre ^{[19
]}

Le Guludec, Dominique ^{[19
]}

机构：

[1] Haute Autor Sante, Hlth Technol Assessment Dept, La Plaine St Denis, France

[2] Univ Tours, Nantes Univ, Inserm, UMR U1246 Sphere, Nantes, France

[3] Univ Paris Cite, ECEVE, Inserm, Paris, France

[4] Robert Debre Mother Child Univ Hosp, AP HP, Inserm, CIC 1426,UEC, Paris, France

[5] Univ Paris Cite, ECSTRRA Team, UMR U1153, Inserm, Paris, France

[6] Sorbonne Univ, Inst Pierre Louis Epidemiol & Sante Publ IPLESP, Inserm, Paris, France

[7] Univ Claude Bernard Lyon 1, Pharmacol Dept, Villeurbanne, France

[8] Univ Paris Saclay, UMR CESP U1018, Inserm, UVSQ,AP HP, Paris, France

[9] Hosp Civils Lyon, Pole Sante Publ, Unidad Bases Donnees Clin & Epidemiol, Lyon, France

[10] Haute Autor Sante, Hematol Dept, AP HP, F-93210 Paris, France

[11] Rennes 1 Univ, EHESP, CHU Rennes, CIC 1414,Serv Pharmacol Clin,Irset,UMR S1085,Inser, Rennes, France

[12] Rennes 1 Univ, Inserm, CHU Rennes, EHESP,CIC 1414,Irset UMR S1085, Rennes, France

[13] Inst Univ France, Paris, France

[14] Univ Paris Cite, Ctr Rech Epidemiol & Stat CRESS UMR1153, Inserm, Paris, France

[15] Bordeaux 1 Univ, Bordeaux Populat Hlth SISTM Serv Informat Med, Inserm, CHU Bordeaux,Inria, Bordeaux, France

[16] Inst Pasteur Inserm, Unidad Epidemiol Malad Emergentes, Paris, France

[17] Inserm Univ Paris Cite, Sorbonne Univ, Ctr Rech Cordeliers, Paris, France

[18] Inria, HeKA, Paris, France

[19] Haute Autor Sante, Sci Board, La Plaine St Denis, France

来源：

BMJ EVIDENCE-BASED MEDICINE | 2024年 / 29卷 / 01期

关键词：

TRIAL; POPULATION; DESIGN;

D O I：

10.1136/bmjebm-2022-112091

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

[No abstract available]

引用

页码：1 / 5

页数：5

共 22 条

[1] An in silico approach helped to identify the best experimental design, population, and outcome for future randomized clinical trials [J].

Bajard, Agathe ;

Chabaud, Sylvie ;

Cornu, Catherine ;

Castellan, Anne-Charlotte ;

Malik, Salma ;

Kurbatova, Polina ;

Volpert, Vitaly ;

Eymard, Nathalie ;

Kassai, Behrouz ;

Nony, Patrice .

JOURNAL OF CLINICAL EPIDEMIOLOGY, 2016, 69 :125-136

[2] Real-world data: towards achieving the achievable in cancer care [J].

Booth, Christopher M. ;

Karim, Safiya ;

Mackillop, William J. .

NATURE REVIEWS CLINICAL ONCOLOGY, 2019, 16 (05) :312-325

[3] RANDOMIZATION OF 1ST PATIENT [J].

CHALMERS, TC .

MEDICAL CLINICS OF NORTH AMERICA, 1975, 59 (04) :1035-1038

[4] Informing single-arm clinical trials with external controls [J].

Davi, Ruthie ;

Mahendraratnam, Nirosha ;

Chatterjee, Arnaub ;

Dawson, C. Jill ;

Sherman, Rachel .

NATURE REVIEWS DRUG DISCOVERY, 2020, 19 (12) :821-822

[5] Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13 [J].

Davis, Courtney ;

Naci, Huseyin ;

Gurpinar, Evrim ;

Poplavska, Elita ;

Pinto, Ashlyn ;

Aggarwal, Ajay .

BMJ-BRITISH MEDICAL JOURNAL, 2017, 359

[6] Paying for Cancer Drugs That Prove Their Benefit [J].

Frank, Richard G. ;

Emanuel, Ezekiel J. .

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2021, 326 (16) :1579-1580

[7] Rare Cancer Trial Design: Lessons from FDA Approvals [J].

Gaddipati, Himabindu ;

Liu, Ke ;

Pariser, Anne ;

Pazdur, Richard .

CLINICAL CANCER RESEARCH, 2012, 18 (19) :5172-5178

[8] Regulatory approval of pharmaceuticals without a randomised controlled study: analysis of EMA and FDA approvals 1999-2014 [J].

Hatswell, Anthony J. ;

Baio, Gianluca ;

Berlin, Jesse A. ;

Irs, Alar ;

Freemantle, Nick .

BMJ OPEN, 2016, 6 (06)

[9] Using Big Data to Emulate a Target Trial When a Randomized Trial Is Not Available [J].

Hernan, Miguel A. ;

Robins, James M. .

AMERICAN JOURNAL OF EPIDEMIOLOGY, 2016, 183 (08) :758-764

[10] Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study [J].

Hwang, Thomas J. ;

Ross, Joseph S. ;

Vokinger, Kerstin N. ;

Kesselheim, Aaron S. .

BMJ-BRITISH MEDICAL JOURNAL, 2020, 371

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