• Aligning Coverage with Transformative Value: Given the rapid evolution of diagnostics science, how can Medicare's "reasonable and necessary" coverage standard evolve to more flexibly and proactively incorporate transformative diagnostics to reshape care pathways rather than reacting to established evidence?
• Addressing the "Paradox" for Prevention and Early Detection: For innovative diagnostics focused on early disease detection or risk stratification (e.g., blood-based neurobiomarkers in early Alzheimer’s Disease detection, host response to infection assays to determine the need for antimicrobial therapy), how can CMS better capture the short and long-term value in improved outcomes and avoidance of more costly downstream interventions? How could CMS “reward” manufacturers and other developers for these critical diagnostics innovations?
• Predictability vs. Flexibility: Striking the Right Balance for Innovation: How should Medicare's national and local coverage and payment determination processes offer greater predictability for diagnostic innovators to plan R&D investments, while simultaneously maintaining enough flexibility to adapt to breakthrough technologies and rapidly evolving clinical value?
• Adapting RCT Requirements for Diagnostic Paradigms: Given the unique characteristics of diagnostics—often impacting decision-making rather than direct intervention, coupled with the longer time horizon to demonstrate ultimate outcomes in Medicare populations—how can Medicare’s general reliance on prospective randomized clinical trials be adapted to foster innovation without imposing unfeasible burdens? What alternative or complementary evidence generation approaches (e.g., real-world evidence, virtual clinical utility studies) should CMS and its Contractors accept when evaluating coverage for novel diagnostics when traditional RCTs are impractical or unethical?​

• The Five Questions for Coding: Who, What, When, Where and How for Innovative Diagnostics
• Why Coding Matters and What Role it Plays in Medicare Payment
• Key Steps for Innovators Before Beginning the Coding Journey​

The evolving global landscape of early-stage development in drug trials presents new challenges and opportunities for companion diagnostics in precision medicine. This session will explore how precision medicine trials are adapting to dynamic global changes. Topics include the increasing regulatory requirements of the EU IVDR for pharma-diagnostic combined trials, the uncertain future of LDTs in the U.S., and the complexities of navigating China’s NMPA regulations, geopolitical pressures and evolving policies. With expert talks followed by a panel discussion, attendees will gain critical insights into navigating these global challenges in early-stage trial development effectively.

The shared goal of bringing new drugs and associated CDx to market to treat patients is the driving force of the partnership between diagnostic developers and biopharma companies. The key to a successful collaboration is teamwork, support and trust. Open and transparent communication allows for adaptation to changes in regulatory environments, and the ability to adhere to a joint strategy for bringing novel therapeutics and diagnostics to patients in need.

CDx submission requires a set of extensive analytical validation studies. Study design, data analysis plan, and acceptance criteria are often challenging decisions. Guidelines (e.g. FDA, CLSI) are indeed available. However, an appropriate interpretation is almost always case-dependent. In addition, ‘surprise’ situations are often encountered but textbook suggestions are not available. For example, how to determine LOD when the hit rates are mostly 0% and 100%? How to demonstrate the suitability of contrived samples? How to determine the acceptance criteria? This talk will provide a statistician’s view on the design and analysis of CDx analytical validation studies.

This presentation aims to explore the co-creation of companion diagnostics (CDx) through early collaboration between development and business teams, emphasizing the critical role of early commercial input for future success. We will cover the entire lifecycle of CDx development, from biomarker selection to test commercialization, providing valuable insights and addressing various challenges and considerations.

The In Vitro Diagnostic Regulation (IVDR) introduces a significant paradigm shift in the regulatory landscape for biomarker tests used in pharmaceutical clinical trials. This roundtable aims to:

•  Foster an open discussion among attendees, focusing on unique and challenging scenarios encountered during clinical trials under the IVDR framework
• Explore potential solutions, share insights, and collaboratively navigate the complexities to ensure compliance and that the data generated can support subsequent regulatory submissions.

Medulloblastoma is the most common pediatric brain and spine malignancy. Currently, no biomarkers are known. We detect whole tumor cells in microcirculation within peripheral blood and cerebrospinal fluid and monitoring levels throughout treatment, correlating findings with patient outcomes.

Explore the emerging role of computational pathology in refining predictive biomarker testing, specifically for antibody-drug conjugates (ADCs). Leveraging advancements in digital and computational pathology may significantly improve patient care by enhancing the potential for treatment response prediction. Quantitative Continuous Scoring (QCS), a state-of-the-art image analysis technique, offers an objective and precise assessment of biomarker expression at the single-cell level, identifying patients who may benefit most from ADC treatments.

This talk will highlight the insight on genetic testing for HRR for eligibility for PARP inhibitor therapy, including the efficacy data of Niraparib + Abirateron (AKEEGA) in prostate cancer patients. The main highlight is to share the advantages and disadvantages of tissue and liquid assays and show both of them are complimentary to each other.

Novel technologies are aiding biomarker assessment to advance precision medicine. Artificial intelligence (AI)-enable diagnostics are transforming cancer drug development and care, offering tools to improve reproducibility, accuracy, and scalability. These technologies hold significant promise for addressing challenges such as variability and inefficiencies in traditional assessments. This session will explore unique approaches to evaluate consistency in results to support new test development and utilization.

Partnering for AI-enabled diagnostics in an age of innovation is evolving traditional RX and DX constructs. The emergence of digital and AI-powered technologies are providing more opportunities yet creating more complexities for deal-making in the industry. This discussion includes examples of how these technologies have transformed the industry and will continue to change the status quo.

Due to challenges in the clinical delivery of molecular testing and targeted therapies, many patients do not receive the most effective personalized treatments. Personalized medicine strategies reach only 36 percent of potentially eligible advanced non-small cell lung cancer (aNSCLC) patients. Although these findings are made in aNSCLC, they likely reflect similar gaps in all biomarker testing-driven indications. Clear practice strategies are needed to improve implementation across personalized medicine. To help catalyze clinical and operational practice and policy reforms, here we describe an implementation roadmap that establishes and promotes best practices and policies for implementing precision oncology.