Industry Perspective on Biomarker Development and Qualification

Abstract

Pharmaceutical and biotechnology companies routinely use biomarkers to obtain quantitative metrics for drug exposure, efficacy, and safety and to inform clinical trial design with regard to patient selection, treatments, and outcomes. Biomarker science has the unique capability to catalyze precompetitive collaborations between academia, industry, regulatory agencies, and other stakeholders with the ultimate goal of accelerating the delivery of safe and effective medicines to patients, particularly in areas of high unmet need.

Safety biomarker applications in drug development

“…While much progress has been made towards the qualification of biomarkers for some organ injures including liver, kidney, and muscle, significant gaps remain for other target organs such as biomarkers of vascular injury in humans, sensitive biomarkers of pancreatic injury, circulating biomarkers of testicular toxicity, and biomarkers of injury to the central nervous system. Because the process of biomarker development is complex and requires considerable resources (Gerlach et al, 2018), these efforts will best be pursued by consortia such as PSTC and IMI. Prior to widespread acceptance, each biomarker must undergo a rigorous assay validation; demonstrate relevance to humans and an association with clinical endpoints reproducibly in multiple studies and gain consensus regarding the level of evidence needed to support a qualification for the stated COU.…”

Next‐Generation DILI Biomarkers: Prioritization of Biomarkers for Qualification and Best Practices for Biospecimen Collection in Drug Development

“…These challenges may be overcome through collaborations between pharmaceutical companies, academicians, and regulators. 5 As an example, 12 out of 14 successful qualifications of markers for different renal or other organ system effects in the nonclinical and clinical space were outcomes of a consortium, alliance, or partnership. 6 From the list of Current Biomarker Qualification Submissions that are under review or undergoing consultation and advice, 15 of 19 submissions (~80%) are from consortia or partnerships.…”

Process Improvement for Maximized Therapeutic Innovation Outcome

“…Translation and ultimately adoption into the clinic can only be accelerated if we begin to streamline clinical trial processes . Greater rates of success in clinical development will be achieved by innovation in the development of biomarkers that can predict responses, outcomes, and adverse events that advantage novel clinical trial designs . Regulatory decisions about the relative value of developing therapeutics will reflect new paradigms in assessing relative risk and benefit .…”

“…Similarly, biomarkers have transformed healthcare management paradigms across a broad spectrum of diseases . These include biomarkers that forecast who will develop a disease, those that prognose whose disease will advance, and those that predict who will respond to therapy . Nowhere is the impact of biomarkers more apparent than in the management of patients with cancer .…”

“…These biomarker‐driven approaches to molecularly segmenting tumors by mutational identity is revolutionizing the development of novel biological therapies and the associated transformation of clinical trials paradigms to accelerate their approval and availability to patients who need them . In turn, this acceleration of discovery and development is reciprocally driving biomarker innovation, to identify more sensitive and specific diagnostic paradigms for predicting therapeutic results and eliminating adverse events of novel therapeutics . Development of the paradigm for model‐informed proarrhythmic risk assessment of drugs is an example of innovation in the design of biomarker paradigms that minimize the risk of adverse events .…”