Towards consensus practices to qualify safety biomarkers for use in early drug development

Application of any new biomarker to support safety-related decisions during regulated phases of drug development requires provision of a substantial data set that critically assesses analytical and biological performance of that biomarker. Such an approach enables stakeholders from industry and regulatory bodies to objectively evaluate whether superior standards of performance have been met and whether specific claims of fit-for-purpose use are supported. It is therefore important during the biomarker evaluation process that stakeholders seek agreement on which critical experiments are needed to test that a biomarker meets specific performance claims, how new biomarker and traditional comparators will be measured and how the resulting data will be merged, analyzed and interpreted.

[1]  Amy G. Aslamkhan,et al.  Urinary biomarkers trefoil factor 3 and albumin enable early detection of kidney tubular injury , 2010, Nature Biotechnology.

[2]  Joseph V Bonventre,et al.  Next-generation biomarkers for detecting kidney toxicity , 2010, Nature Biotechnology.

[3]  A. B. Hill.  The Environment and Disease: Association or Causation? , 1965, Proceedings of the Royal Society of Medicine.

[4]  F. Sistare,et al.  Preclinical Predictors of Clinical Safety: Opportunities for Improvement , 2007, Clinical pharmacology and therapeutics.

[5]  S. Waldman,et al.  Biomarker Development, Commercialization, and Regulation: Individualization of Medicine Lost in Translation , 2007, Clinical pharmacology and therapeutics.

[6]  J. Wagner.  Strategic approach to fit-for-purpose biomarkers in drug development. , 2008, Annual review of pharmacology and toxicology.

[7]  D. DeMets,et al.  Biomarkers and surrogate endpoints: Preferred definitions and conceptual framework , 2001, Clinical pharmacology and therapeutics.

[8]  Serafino Pantano,et al.  Urinary clusterin, cystatin C, β2-microglobulin and total protein as markers to detect drug-induced kidney injury , 2010, Nature Biotechnology.

[9]  Krishna Prasad,et al.  Renal biomarker qualification submission: a dialog between the FDA-EMEA and Predictive Safety Testing Consortium , 2010, Nature Biotechnology.

[10]  J S Alpert,et al.  Myocardial infarction redefined--a consensus document of The Joint European Society of Cardiology/American College of Cardiology Committee for the redefinition of myocardial infarction. , 2000, Journal of the American College of Cardiology.

[11]  D. Ransohoff.  Bias as a threat to the validity of cancer molecular-marker research , 2005, Nature reviews. Cancer.

[12]  Frank E. Harrell,et al.  Regression Modeling Strategies: With Applications to Linear Models, Logistic Regression, and Survival Analysis , 2001 .

[13]  Dawn G Goodman,et al.  Best Practices Guideline: Toxicologic Histopathology , 2004, Toxicologic pathology.

[14]  J. Wagner,et al.  Biomarkers and Surrogate End Points for Fit‐for‐Purpose Development and Regulatory Evaluation of New Drugs , 2007, Clinical pharmacology and therapeutics.

[15]  Innovation OR Stagnation.  Challenge and Opportunity on the Critical Path to New Medical Products , 2004 .

[16]  Frank D Sistare,et al.  Research at the interface of industry, academia and regulatory science , 2010, Nature Biotechnology.

[17]  E. DeLong,et al.  Comparing the areas under two or more correlated receiver operating characteristic curves: a nonparametric approach. , 1988, Biometrics.

[18]  Viswanath Devanarayan,et al.  Fit-for-Purpose Method Development and Validation for Successful Biomarker Measurement , 2006, Pharmaceutical Research.

[19]  J D Eversham,et al.  Opportunities for improvements in flocking models , 2008 .

[20]  D. Gerhold,et al.  Kidney Injury Molecule-1 Outperforms Traditional Biomarkers of Kidney Injury in Multi-site Preclinical Biomarker Qualification Studies , 2010, Nature Biotechnology.

[21]  B. Cummins,et al.  Cardiac-specific troponin-I radioimmunoassay in the diagnosis of acute myocardial infarction. , 1987, American heart journal.

[22]  D Amakye,et al.  A Prototypical Process for Creating Evidentiary Standards for Biomarkers and Diagnostics , 2008, Clinical pharmacology and therapeutics.

[23]  J. Bonventre,et al.  Kidney Injury Molecule-1 ( Kim-1 ) : A Tissue and Urinary Biomarker for Nephrotoxicant-Induced Renal Injury . , 2003 .