Table 2.

List of drug development tools endorsed by the FDA with examples of data/knowledge sharing initiatives

(Disease) Area	Endorsement	Data resource	Supporting information
Biomarker qualification
Nonclinical	Urinary biomarkers: Albumin, β2‐Microglobulin, Clusterin, Cystatin C, KIM‐1, Total Protein, and Trefoil factor‐3	Short‐term rat GLP toxicology studies by Merck and Novartis	Guidance document: https://www.fda.gov/media/82532/download
Nephrotoxicity	Urinary nephrotoxicity biomarkers as assessed by immunoassays	Short term rat GLP toxicology studies conducted at AstraZeneca, Bayer, Biotrin, BMS, GSK, and Sanofi‐Aventis	FDA review document: https://www.fda.gov/media/113664/download
Cardiac troponins	Serum/plasma cardiotoxicity biomarkers as assessed by immunoassay	Data from 20 publications as critical to the qualification of troponins for use in the rat	FDA review document: https://www.fda.gov/media/87774/download
IS	Diagnostic biomarkers used with other clinical and host factors to identify patients with IS	The data to support qualification were obtained with the use of the Bio‐Rad Platelia Aspergillus enzyme immunoassay	FDA Guidance Document: https://www.fda.gov/media/94480/download
COPD	Prognostic biomarker used with other characteristics to enrich for COPD exacerbations	The COPD Biomarkers Qualification Consortium Database	FDA Guidance Document: https://www.fda.gov/media/92782/download
PKD	Total kidney volume as assessed by magnetic resonance imaging, computed tomography, and ultrasound	Three patient registries (University of Colorado‐Denver, Mayo Clinic, and Emory University) and two longitudinal cohort studies (CRISP1 and CRISP2 on the natural history of autosomal dominant PKD	FDA Guidance Document: https://www.fda.gov/media/93105/download
Nephrotoxicity	Urinary nephrotoxicity biomarker panel as assessed by immunoassays	Normal healthy volunteers	Qualification Determination Letter: https://www.fda.gov/media/115635/download
CHMI	Monitoring biomarker informs initiation of treatment with antimalarial drug following CHMI with P. falciparum sporozoites in healthy subjects in clinical studies for vaccine and/or drug development	Nonclinical and clinical data from multiple sources including the University of Washington	Qualification Determination Letter: https://www.fda.gov/media/119374/download
FFP initiative
AD	FFP Disease Progression Model: Placebo/Disease Progression	ADNI, and CPAD databases	Determination letter: https://www.fda.gov/media/98856/download
Multiple	FFP Statistical Method: MCP‐Mod	–	Determination letter: https://www.fda.gov/media/99296/download

AD, Alzheimer’s disease; ADNI, AD Neuroimaging Initiative; CHMI, Controlled human malaria infection; COPD, chronic obstructive pulmonary disease; CPAD, Critical Path for Alzheimer's Disease; CRISP, Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease; FDA, US Food and Drug Administration; FFP, Fit‐for‐Purpose; GLP, Good Laboratory Practice; IS, invasive aspergillosis; MCP‐Mod, Multiple Comparison Procedure – Modeling; PKD, Polycystic Kidney Disease.