Universität zu Köln

Michels, Sebastian, Wolf, Juergen and Hallek, Michael (2019). New concepts for approval of drugs in oncology beyond randomized clinical studies. Onkologe, 25. S. 83 - 90. HEIDELBERG: SPRINGER HEIDELBERG. ISSN 1433-0415

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Abstract

Background In recent years a large number of biomarker-stratified targeted and immunological treatment procedures were introduced in oncology. Higher efficacy has been shown in subgroups of patients with these drugs compared to the standard of care; however, due to the low numbers of patients within some subgroups confirmatory phase III trials are sometimes not feasible and the proof-of-principle is increasingly shifted towards early study phases. The approval authorities in the USA and the European Union have recognized these difficulties and established new instruments to accelerate and simplify drug approval. This development is accompanied by innovative trial designs that can provide relevant data for drug approval in early study phases. Objective Summary and discussion of innovative trial designs and approval procedures in oncology. Material and methods A systematic search was carried out in the medical literature database PubMed () for articles on approval procedures and trial designs in oncology as well as the websites of the European Medicines Agency (EMA), the US Food and Drug Administration (FDA) and the Federal Institute for Drugs and Medical Devices (Bundesamt fur Arzneimittel und Medizinprodukte, BfArM). Results and discussion A growing number of drugs are being approved within accelerated approval procedures in oncology. The FDA programs, such as the breakthrough designation and the accelerated approval procedures have been especially successful. Systematic analyses revealed that the time to the first use in patients was significantly shortened. Based on new designs, such as basket trials, approvals were granted in the USA for histology-independent personalized treatment for patient subgroups. Despite these achievements, criticism has arisen concerning the sometimes preliminary nature of data on safety and efficacy on which accelerated approval is based.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Michels, Sebastian UNSPECIFIED UNSPECIFIED UNSPECIFIED Wolf, Juergen UNSPECIFIED UNSPECIFIED UNSPECIFIED Hallek, Michael UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-142863
DOI:	10.1007/s00761-019-0598-x
Journal or Publication Title:	Onkologe
Volume:	25
Page Range:	S. 83 - 90
Date:	2019
Publisher:	SPRINGER HEIDELBERG
Place of Publication:	HEIDELBERG
ISSN:	1433-0415
Language:	German
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language SURROGATE END-POINTS; ACCELERATED APPROVAL; SURVIVAL; TRIAL; POSTAPPROVAL; CHEMOTHERAPY; THERAPEUTICS; ASSOCIATION; CRIZOTINIB; STRENGTH Multiple languages Oncology Multiple languages
Refereed:	Yes
URI:	http://kups.ub.uni-koeln.de/id/eprint/14286