Voluntary exploratory data submissions to the US FDA and the EMA: experience and impact.
Heterogeneity in the underlying mechanisms of disease processes and inter-patient variability in drug responses are major challenges in drug development. To address these challenges, biomarker strategies based on a range of platforms, such as microarray gene-expression technologies, are increasingly being applied to elucidate these sources of variability and thereby potentially increase drug development success rates. With the aim of enhancing understanding of the regulatory significance of such biomarker data by regulators and sponsors, the US Food and Drug Administration initiated a programme in 2004 to allow sponsors to submit exploratory genomic data voluntarily, without immediate regulatory impact. In this article, a selection of case studies from the first 5 years of this programme - which is now known as the voluntary exploratory data submission programme, and also involves collaboration with the European Medicines Agency - are discussed, and general lessons are highlighted.
Genomics Group, Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, US FDA, Building 51, 10903 New Hampshire Avenue, Silver Spring, Maryland 20903-002, USA. Federico.Goodsaid@fda.hhs.gov
Federico M
FM
Goodsaid
Shashi
S
Amur
Jiri
J
Aubrecht
Michael E
ME
Burczynski
Kevin
K
Carl
Jennifer
J
Catalano
Rosane
R
Charlab
Sandra
S
Close
Catherine
C
Cornu-Artis
Laurent
L
Essioux
Albert J
AJ
Fornace
Lois
L
Hinman
Huixiao
H
Hong
Ian
I
Hunt
David
D
Jacobson-Kram
Ansar
A
Jawaid
David
D
Laurie
Lawrence
L
Lesko
Heng-Hong
HH
Li
Klaus
K
Lindpaintner
James
J
Mayne
Peter
P
Morrow
Marisa
M
Papaluca-Amati
Timothy W
TW
Robison
John
J
Roth
Ina
I
Schuppe-Koistinen
Leming
L
Shi
Olivia
O
Spleiss
Weida
W
Tong
Sharada L
SL
Truter
Jacky
J
Vonderscher
Agnes
A
Westelinck
Li
L
Zhang
Issam
I
Zineh
D000410:Alanine Transaminase / Q000097:blood; D001384:Azetidines / Q000009:adverse effects / Q000627:therapeutic use; D001596:Benzylamines / Q000009:adverse effects / Q000627:therapeutic use; D002292:Carcinoma, Renal Cell / Q000175:diagnosis; D017277:Drug Approval; D005060:Europe; D005472:Fluorouracil / Q000009:adverse effects; D020869:Gene Expression Profiling; D005819:Genetic Markers; D006801:Humans; D007391:International Cooperation; D007680:Kidney Neoplasms / Q000175:diagnosis; D016030:Kidney Transplantation; D010597:Pharmacogenetics; D010879:Piperazines / Q000493:pharmacokinetics / Q000627:therapeutic use; D000068799:Prasugrel Hydrochloride; D057285:Precision Medicine; D013876:Thiophenes / Q000493:pharmacokinetics / Q000627:therapeutic use; D014481:United States; D014486:United States Food and Drug Administration
D001384:Azetidines; D001596:Benzylamines; D005819:Genetic Markers; D010879:Piperazines; D013876:Thiophenes; C426686:ximelagatran; D000410:Alanine Transaminase; D000068799:Prasugrel Hydrochloride; D005472:Fluorouracil