Skip to main content
Premium Trial:

Request an Annual Quote

Relabeling May Guide Retrospective Data Criteria


The conditions under which the US Food and Drug Administration accepted retrospective analyses from two pharmaceutical companies in updating labeling for a class of colorectal cancer drugs with pharmacogenetic information may serve as a model for other sponsors attempting to personalize treatments with genomic-biomarker assessments, according to an FDA official.

When FDA relabeled two colorectal cancer drugs — Amgen's Vectibix and Bristol-Myers Squibb/ImClone's Erbitux — informing doctors and patients that those with KRAS mutations in codon 12 or 13 do not respond to EGFR inhibitors, it marked the first time the agency used retrospective clinical trial data to update the label of a drug with PGx information.
However, just because the companies performed genomic analyses on samples collected during previously completed trials doesn't mean "they went on a fishing expedition," Lawrence Lesko, director of the Office of Clinical Pharmacology at FDA's Center for Drug Evaluation and Research, said at a conference hosted by Ohio State University Center for Personalized Healthcare.

Lesko lists "critical evidence" submitted by the sponsors, and notes that "if one wants to utilize retrospective data … then this would be a good way to do it."

BMS and Amgen's submissions hit a number of FDA's key criteria for accepting retrospective data, including presentation of a biologically plausible hypothesis, replication of findings, reduced bias, a pre-specified statistical plan, and the availability of an analytically valid test.

According to Lesko, the agency accepted the companies' retrospective analysis because the data "plausibly" showed that EGFR-inhibiting drugs were not efficacious in patients with mutant KRAS. The companies replicated this finding, showing "consistent differences" in objective response between wild-type and mutated KRAS in six pooled single-arm studies. The companies also presented a statistical plan ahead of conducting genomic analysis for data collection and retrospective analysis.

— Turna Ray

PGx & Molecular Dx Notes

Quidel and BioHelix announced that they will jointly develop and commercialize in vitro molecular diagnostic tests for infectious pathogens. The tests will use BioHelix's isothermal amplification technology and will be done using a handheld device.

The molecular diagnostics developer BioMarker Strategies received a fast-track SBIR grant from the National Cancer Institute. With its initial award of $254,000, BioMarker will develop a prototype of its live-tumor cell testing system.

Decode Genetics closed its Woodridge, Ill., facility and eliminated about 60 positions,
according to a filing with the US Securities and Exchange Commission.


$3.4 million
Amount the FDA gave Biosearch Technologies to develop rapid, multiplexed fluorescent pathogen and bioterror detection assays.

Funded grants

$1,990,198/FY 2009
Comparative Effectiveness in Genomic Medicine
Grantee: Katrina Armstrong, University of Pennsylvania
Began: Sep. 30, 2009; Ends: Aug. 31, 2011

Armstrong and her colleagues plan to perform observational and experimental studies of clinical effectiveness including four pilot studies on the pharmacogenomics of nicotine addiction treatment, SNP panels in breast cancer risk screening and prevention, personalized treatment for non small cell lung cancer, and CDKN2A/p16 testing and adherence to melanoma prevention behaviors.

$961,148/FY 2009
CTRIP: Genetic Testing to Individualize Management of Common Heart Diseases
Grantee: Gerald Dorn, Washington University
Began: Sep. 30, 2009; Ends Aug. 31, 2011

Dorn and his collaborators will be researching genetic risks, genetic modifiers, and pharmacogenomic interactions involved in heart failure. First, they will resequence associated genes and loci to find possible causal variants and then they'll create an online data coordinating center.

The Scan

Two J&J Doses

Johnson & Johnson says two doses of its SARS-CoV-2 vaccine provides increased protection against symptomatic COVID-19, CNN reports.

Pfizer-BioNTech Vaccine Response in Kids

The Pfizer-BioNTech SARS-CoV-2 vaccine in a lower-dose format appears to generate an immune response among children, according to the Washington Post.

Chicken Changes to Prevent Disease

The Guardian writes that researchers are looking at gene editing chickens to help prevent future pandemics.

PNAS Papers on Siberian Dog Ancestry, Insect Reproduction, Hippocampal Neurogenesis

In PNAS this week: ancestry and admixture among Siberian dogs, hormone role in fruit fly reproduction, and more.