Advancing Therapeutics

Remote Digital Monitoring for Medical Product Development Workshop

The BC’s Remote Digital Monitoring for Medical Product Development Workshop held in February 2020 resulted in key considerations and recommendations which were incorporated into FDA’s Innovative Science and Technology Approaches for New Drugs (ISTAND) pilot program and guidance to support use of novel drug development tools – Digital Health Technologies for Remote Data Acquisition in Clinical Investigations.

21st Century Cures Act

The 21st Century Cures (Cures Act) has been updated with newly published Final Guidance for Qualification of Drug Development and Tools (DDT) that will act as an essential tool and roadmap for industry and all BC projects moving forward.


FDA Biomarker Qualification Milestones

Qualified Biomarkers

Clinical Evaluation and Qualification of Translational Kidney Safety Biomarkers (Kidney Safety) Project. First ever qualification by the FDA of a clinical safety biomarker and the first qualification for the Biomarkers Consortium. Composite measure biomarker can be used to detect acute kidney injury for drugs being tested in normal volunteers in Phase 1 clinical trials.

Plan Approval and Progress

Bone Quality Qualification Plan (QP) for the use of treatment-related change in bone mineral density (BMD) as a surrogate endpoint for fractures in future trials of new anti-osteoporosis drugs. This marks the first ever approval of a surrogate endpoint QP by FDA.

NIMBLE imaging workstream received a high score and funding from the last FDA U01 announcement for imaging QP development.

Kidney Safety Project Qualification Plan for the individual safety biomarker panel to indicate response to drug-induced injury to the renal tubules was submitted in September 2022.

Letter of Intent

Submitted an LOI in December 2022 for the use of Neurofilament (NF)-Light chain as a susceptibility/risk biomarker to select individuals with familial progranulin (GRN) mutations at high risk of converting to symptomatic familial Frontotemporal Degeneration f-FTD within two years for inclusion in disease modifying clinical trials.


Consensus Statements and Impact on Regulatory Decisions

ILSA Collaborative Community Charter

The International Liquid Biopsy Standardization Alliance (ILSA) Collaborative Community Charter submitted to FDA focused on promoting the global use of liquid biopsy and common reference standards in oncology and beyond.

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Sarcopenia as a Valid Biomarker for Identifying Individuals at Risk of Disability (Sarcopenia 2 Project)

Position statements defining sarcopenia and evidence-based diagnosis criteria and cut-offs were approved by an international expert team.

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Scientific Publications


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