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.
Goodsaid
Federico M
FM
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
Amur
Shashi
S
Aubrecht
Jiri
J
Burczynski
Michael E
ME
Carl
Kevin
K
Catalano
Jennifer
J
Charlab
Rosane
R
Close
Sandra
S
Cornu-Artis
Catherine
C
Essioux
Laurent
L
Fornace
Albert J
AJ
Hinman
Lois
L
Hong
Huixiao
H
Hunt
Ian
I
Jacobson-Kram
David
D
Jawaid
Ansar
A
Laurie
David
D
Lesko
Lawrence
L
Li
Heng-Hong
HH
Lindpaintner
Klaus
K
Mayne
James
J
Morrow
Peter
P
Papaluca-Amati
Marisa
M
Robison
Timothy W
TW
Roth
John
J
Schuppe-Koistinen
Ina
I
Shi
Leming
L
Spleiss
Olivia
O
Tong
Weida
W
Truter
Sharada L
SL
Vonderscher
Jacky
J
Westelinck
Agnes
A
Zhang
Li
L
Zineh
Issam
I
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