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


Journal

Clinical pharmacology and therapeutics
ISSN: 1532-6535
Titre abrégé: Clin Pharmacol Ther
Pays: United States
ID NLM: 0372741

Informations de publication

Date de publication:
04 2020
Historique:
received: 16 07 2019
accepted: 02 09 2019
pubmed: 2 10 2019
medline: 28 10 2020
entrez: 2 10 2019
Statut: ppublish

Résumé

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.

Identifiants

pubmed: 31574163
doi: 10.1002/cpt.1638
pmc: PMC7158212
doi:

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

773-779

Commentaires et corrections

Type : CommentIn
Type : CommentIn

Informations de copyright

© 2019 The Authors. Clinical Pharmacology & Therapeutics published by Wiley Periodicals, Inc. on behalf of American Society for Clinical Pharmacology and Therapeutics.

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Auteurs

Hans-Georg Eichler (HG)

European Medicines Agency (EMA), Amsterdam, The Netherlands.
Medical University of Vienna, Vienna, Austria.

Franz Koenig (F)

Medical University of Vienna, Vienna, Austria.

Peter Arlett (P)

European Medicines Agency (EMA), Amsterdam, The Netherlands.

Harald Enzmann (H)

Federal Institute for Drugs and Medical Devices (BfArM), Bonn, Germany.
EMA's Committee for Medicinal Products for Human Use (CHMP), Amsterdam, The Netherlands.

Anthony Humphreys (A)

European Medicines Agency (EMA), Amsterdam, The Netherlands.

Frank Pétavy (F)

European Medicines Agency (EMA), Amsterdam, The Netherlands.

Brigitte Schwarzer-Daum (B)

Medical University of Vienna, Vienna, Austria.
EMA's Committee for Orphan Medicinal Products (COMP), Amsterdam, The Netherlands.

Bruno Sepodes (B)

EMA's Committee for Medicinal Products for Human Use (CHMP), Amsterdam, The Netherlands.
EMA's Committee for Orphan Medicinal Products (COMP), Amsterdam, The Netherlands.
Universidade de Lisboa, Lisbon, Portugal.

Spiros Vamvakas (S)

European Medicines Agency (EMA), Amsterdam, The Netherlands.

Guido Rasi (G)

European Medicines Agency (EMA), Amsterdam, The Netherlands.
University Tor Vergata, Rome, Italy.

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Classifications MeSH