Are Novel, Nonrandomized Analytic Methods Fit for Decision Making? The Need for Prospective, Controlled, and Transparent Validation

被引：52

作者：

Eichler, Hans-Georg ^{[1
,2
]}

Koenig, Franz ^{[2
]}

Arlett, Peter ^{[1
]}

Enzmann, Harald ^{[3
,4
]}

Humphreys, Anthony ^{[1
]}

Petavy, Frank ^{[1
]}

Schwarzer-Daum, Brigitte ^{[2
,5
]}

Sepodes, Bruno ^{[4
,5
,6
]}

Vamvakas, Spiros ^{[1
]}

Rasi, Guido ^{[1
,7
]}

机构：

[1] EMA, Amsterdam, Netherlands

[2] Med Univ Vienna, Vienna, Austria

[3] Fed Inst Drugs & Med Devices BfArM, Bonn, Germany

[4] EMAs Comm Med Prod Human Use CHMP, Amsterdam, Netherlands

[5] EMAs Comm Orphan Med Prod COMP, Amsterdam, Netherlands

[6] Univ Lisbon, Lisbon, Portugal

[7] Univ Tor Vergata, Rome, Italy

来源：

CLINICAL PHARMACOLOGY & THERAPEUTICS | 2020年 / 107卷 / 04期

关键词：

D O I：

10.1002/cpt.1638

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

Real-world data and patient-level data from completed randomized controlled trials are becoming available for secondary analysis on an unprecedented scale. A range of novel methodologies and study designs have been proposed for their analysis or combination. However, to make novel analytical methods acceptable for regulators and other decision makers will require their testing and validation in broadly the same way one would evaluate a new drug: prospectively, well-controlled, and according to a pre-agreed plan. From a European regulators' perspective, the established methods qualification advice procedure with active participation of patient groups and other decision makers is an efficient and transparent platform for the development and validation of novel study designs.

引用

页码：773 / 779

页数：7

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