Collecting high-quality clinical utility data is a requisite for coverage and reimbursement from payers, which often has begun too late, is non-experimental, or takes too long at a high cost. This underscores the need for better, less risky, and more principled strategies to generate evidence of impact and determine clinical utility. Mr. Ouenes will share innovative new approaches to collect high-quality clinical utility data that is both cost effective and rapid.

Immunotherapy has become a key element in the arsenal of cancer treatments. To predict outcomes of immunotherapy and guide treatment decisions, information on both the tumor and patient immune response is essential. Biodesix, Inc. will share: (1) A case study on combining genomic and proteomic tests to better target patients for ICI monotherapy, and (2) How proteomics, paired with explainable AI, can help identify new therapeutic targets guiding combo-therapy strategies.

Precision Medicine therapies have demonstrated benefits for cancer patients but are dependent on biomarker results to enable personalized treatment selection. There are four key barriers to optimal precision oncology treatment: Awareness of the test & its clinical utility, Availability of sample & test, Adoption of testing & delaying treatment until results are returned, and Access through reimbursement and reasonable out-of-pocket cost.  Each of these hurdles will be examined through the lens of how liquid biopsy tests in today's healthcare system are well suited to overcome some, but disadvantaged in other barriers.

Precision medicine is transforming treatment paradigms using complex biomarkers in the interpretation of clinical data. Incorporating MRD status as a biomarker in clinical trials can expedite clinical development by demonstrating depth and durability of clinical response thus providing supportive evidence of clinical benefit. The subsequent goal of establishing MRD as a surrogate endpoint can enable faster access to therapies for patients. The current MRD landscape and applications will be discussed.

ecDNA are large circles of DNA outside the cells’ chromosomes that can be found in nearly half of all solid tumor cancers and are thought to be a fundamental driver of cancer growth, resistance, and recurrence. Boundless Bio’s approach to develop treatments specifically targeting cancers with ecDNA gene amplification presents a unique challenge for companion diagnostic development.

The Somatic Reference Sample initiative is a public-private partnership convened by Medical Device Innovation Consortium (MDIC) guiding the development of reference samples that can be used to develop and validate NGS-based oncologic tests. Ensuring that oncology patients receive accurate results is imperative; however, lack of agreed upon well-characterized, and community-validated reference samples and data benchmarks creates potential challenges for efficient development of these tests and for understanding their results. A pilot project has been fully funded by industry and philanthropic organizations to develop, manufacture, and create validated data sets for an initial set of 10 reference samples. Participating members of the SRS Initiative include FDA, NIH, diagnostics manufacturers, funding organizations, and payors.

The current MRD testing landscape shows tremendous potential for improving the care of cancer patients, including adjuvant therapy guidance, recurrence monitoring, and treatment selection and monitoring. However, the clinical utility of MRD testing is still limited due to technical and clinical challenges, highlighting the need for more accurate ctDNA detection technologies.  This presentation will describe a highly sensitive ctDNA assay that may provide a better MRD solution.

There are many considerations that go into the development of a companion diagnostic, including platform selection, pathway approaches, regulatory approvals, and commercial alignment. This presentation will focus on the various factors and decisions that can affect how well your companion diagnostic will ultimately be adopted by the market. Learn how a top commercial lab takes an assay to market.

The convergence of technologies necessary to inform clinician and patient decision making in oncology requires a holistic view of how payers and other stakeholders assess the value of testing. The presentation will focus on the continuum of oncology patient assessment tools, from early detection through testing for minimal residual disease and will highlight evolving payer and clinician value perspectives reflected in the payer coverage and payment landscape and implications for innovators.

Precision medicine has been propelled by technological advances enabling the development of novel treatments tailored to an individual patient. Companion diagnostics (CDx) play a vital role in precision medicine, and there is a growing number of regions with CDx policies. This presentation will provide an overview of the evolving global regulatory landscape for diagnostics. Potential strategies for navigating the changing environment with considerations for co-development programs will also be discussed. 

Facilitating the coordinated development and review of targeted therapies and companion diagnostics for rare populations can be challenging. This presentation will discuss opportunities to improve patient access to clinical trials of biomarker-driven investigational products for rare diseases and expand and expedite the development of companion diagnostic tests.

The detection of circulating tumor DNA (ctDNA) has tremendous potential in oncology to enable patient selection for therapy, monitor disease progression, and improve timely access to new therapeutics. This presentation will review regulatory considerations for liquid biopsy assays, explore the current utility of liquid biopsy assays as companion diagnostics and as clinical trial assays, and highlight future directions of the field including the detection of molecular residual disease.

As Europe's long-awaited in vitro diagnostic regulation finally comes into force, certain provisions reach beyond the EU to laboratories conducting biomarker testing on EU patient samples. This talk will untangle the legislation and help pharma and diagnostic sponsors understand what they must do and by when to fully comply with the regulations.

Over the last decade, biomarker-driven companion diagnostics accompanied by molecular-targeted drugs or biologics have been developed and introduced in cancer care, leading to significant improvements in patients' treatments and outcomes. Extensive clinical research on "Liquid Biopsy," such as circulating tumor DNA (ctDNA), has recently demonstrated the advantages of blood over tissue, leading to the intensive investigation of ctDNA for the measurement of predictive biomarkers and companion diagnostics, for a range of therapeutics across different cancer types. In this presentation, I will highlight the potential of ctDNA-driven genomic and epigenomic companion diagnostics, as well as their future prospects in cancer care management.

Most cancer treatments work though activation of the cell suicide program of apoptosis. Cells that more readily die in response to various stimuli are referred to as “primed”. Hence, the degree of priming of a tumor sample can be predictive of responses to anti-cancer drugs and can be established by measuring this priming. We previously established Clinical Laboratory Improvement Amendment (CLIA) tests that use a proprietary modification of a method called “BH3 profiling” to indirectly measure priming. Our next generation predictive diagnostic tests directly measure priming in a way that overcomes liabilities of the indirect measurements of priming. We have found that this new approach allows more accurate and precise measurements. The new method (PRIMABTM) relies on proprietary complex specific antibodies and is seen to be simpler, more robust, and much more amenable to clinical use. This novel approach could have broad applications in personalized medicine. The PRIMABTM platform has been developed in Eutropics’ CLIA labs and is being made available.

We describe the discovery, validation, and mechanistic insight into a blood-based 5-microRNA risk (miRisk) score that predicts survival of non-small cell lung cancer (NSCLC) patients receiving immunomonotherapy of either pembrolizumab or nivolumab and performs better than the gold-standard of PD-L1 (Rajakumar et al., 2022, npj Precision Oncology. In press.).

NIST, in collaboration with the Early Detection Research Network (NCI, NIH) is developing references materials for methylated genomic DNA. Methylated DNA is promising biomarker for many cancer types. We utilized genomic DNA from reference cell line to developed 5 different percentage of methylated DNA as candidate methylated DNA reference materials and performed interlaboratory studies.