On February 19, Friends of Cancer Research (Friends) hosted a public webinar examining the role of advocacy in advancing AI-enabled diagnostics tools and companion diagnostic (CDx) tests for rare biomarkers. Titled “Advancing the Future of Diagnostics and Regulatory Innovations Meeting Recap: Sharing Next Steps and Opportunities with Advocates,” the webinar built upon discussions from the “Advancing the Future of Diagnostics and Regulatory Innovations” public meeting held on February 4. This session aimed to extend educational outreach within the oncology advocacy community, highlighting key insights, challenges, and opportunities in AI-driven diagnostics and regulatory innovation to enhance diagnostic accuracy and advance drug development.
Evaluating Digital Pathology and AI in Diagnostics & Validating Diagnostic Tests for Rare Biomarkers
The webinar delved into the role of AI-enabled digital pathology in improving accuracy, reproducibility, and efficiency in oncology diagnostic testing and development. By addressing variability in test interpretation and supporting pathologists in high-volume workflows, AI-enabled tools have the potential to enhance patient stratification and treatment selection.
George Green, GA Green Consulting LLC, described how AI enables standardized digital imaging analyses, offering more consistent and interpretable results— ultimately helping to refine diagnostic precision and optimize patient health outcomes. He cited examples of how AI-enabled diagnostic tools can streamline work, freeing-up time for pathologists to focus on complex and difficult-to-interpret cases. Alain Silk, Tempus AI, noted that while use of AI-enabled diagnostics is growing, only about 5-10% of FDA-authorized AI tools are applied in oncology, with most being used in radiology. This underscores the need for continued validation and regulatory refinement to expand their impact in cancer care.
Ensuring the reliable integration of AI in diagnostics requires clear regulatory frameworks, diverse and representative data sets, and ongoing performance evaluation to maintain scientific rigor and patient safety.
The discussion also addressed key challenges in validating CDx tests for rare biomarkers, which are critical tools in targeted therapy development. Megan Doyle, Eli Lilly & Company emphasized that trial enrollment often depletes available samples, limiting those available for diagnostic validation studies. This can delay companion diagnostic development and approval. Joan Mancuso, Friends Advisory Advocate, stressed that without accessible and reliable biomarker testing, patients may miss out on targeted therapies that could improve survival. She also highlighted concerns about racial and ethnic disparities in biomarker expression, noting that AI-enabled diagnostics must be validated on diverse datasets to ensure accuracy across populations. Without representative data, these technologies risk misclassifying biomarker status, limiting patient access to appropriate treatments and reinforcing existing disparities.
Audience Q&A
The audience Q&A session explored key challenges in AI-enabled digital pathology, validating CDx for rare biomarkers, and ensuring equitable patient access to diagnostics. Panelists addressed data privacy concerns, emphasizing that AI-driven pathology relies on de-identified data sets and regulatory safeguards to ensure compliance with the Health Insurance Portability and Accountability Act (HIPAA) and General Data Protection Regulation (GDPR). Discussions also highlighted barriers to obtaining clinical samples for rare biomarker validation. Panelists explored potential alternative approaches, such as using post-mortem samples, though logistical and ethical considerations remain due to rapid tissue degradation and challenges obtaining informed consent. The session also examined financial toxicity and other access barriers to biomarker testing, especially in community healthcare settings where advanced diagnostics, such as next-generation sequencing (NGS), may not be available or reimbursed. Panelists underscored the critical role of patient advocacy in clinical trial planning and design, regulatory decision-making, and reimbursement policies. Advocate participation in research can help ensure that novel diagnostics reach patients without delays or disparities.
Implications for Patient Advocacy and Policy
Advocacy plays a critical role in shaping the future of diagnostic innovations, ensuring that advancements, including AI-enabled tools and biomarker-driven diagnostics, are developed, validated, and implemented effectively. Advocates can help drive efforts to enhance transparency, promote scientific rigor, and support regulatory policies that facilitate timely diagnostic development while maintaining high validation standards. Ensuring equitable access remains a key priority, alongside addressing technical, regulatory, and clinical challenges that impact the adoption of new technologies.
Collaboration between patients, industry leaders, regulators, and researchers is key to integrating AI-enabled diagnostics into clinical practice, improving patient outcomes and ensuring that innovations benefit all patients. Advocacy is also crucial in navigating issues such as data privacy, diagnostic reimbursement, and the generation of high-quality evidence to support regulatory decision-making. Moving forward continued engagement from the advocacy community will be key in accelerating innovation while ensuring that novel diagnostics reach patients in a timely, effective, and equitable manner.
