Next-generation sequencing (NGS) is becoming a commonly used tool in cancer treatment. Transparency regarding the clinical performance and utility of different NGS-based tests available will aid in clinical decision-making and facilitate improvements in patient care. The 7th Annual Blueprint for Breakthrough Forum, held by Friends of Cancer Research (Friends) and Alexandria Real Estate Equities, Inc., showcased innovative reimbursement mechanisms that can help facilitate and encourage the development of evidence over time with the ultimate goal of ensuring maximum benefit to patients and the healthcare system overall.
The meeting opened with remarks from Lynne Zydowsky, Alexandria Real Estate Equities, Inc. and Ellen Sigal, Friends of Cancer Research, who introduced the keynote speaker, FDA Commissioner Dr. Scott Gottlieb. Dr. Gottlieb addressed efforts that the FDA is taking to modernize diagnostic test regulations including referencing the technical assistance submitted to Congress and collaboration with the Medical Device Innovation Consortium to develop matched tumor-normal reference samples to support the development and validation of next generation sequencing-based tests.
“The FDA has an obligation to regulate diagnostics, and I believe we should do so in a way that advances opportunities for innovation, while maximizing protections for patients” – Scott Gottlieb, FDA
The keynote address was followed by presentations from Katherine Szarama, CMS Coverage and Analysis group and Jon Hirsch, Syapse. Dr. Szarama provided an overview of CMS’ Medicare National Coverage Determination for Diagnostic Laboratory Tests using Next Generation Sequencing, and Jon Hirsch presented mechanisms for collecting real-world evidence and outcomes to support reimbursement highlighted in the meeting whitepaper. Jon Hirsch emphasized that evidence collection to support the utility of a diagnostic should be pragmatic, utilize retrospective and prospective data, reduce the burden to the care team, and preserve access to the diagnostic test.
The first panel, moderated by Kavita Patel of the Brookings Institution, discussed collection and sharing of evidence to support clinical implementation and reimbursement for diagnostic tests. Alex Lazar set the stage by describing hierarchical levels of evidence to support follow-on diagnostics with randomized clinical trials (RCT) representing the gold standard for evidence collection to prospective trials with clinical sample analysis and real-world evidence. The panel walked through outcomes measurable by real-world evidence that could support follow-on diagnostics when RCTs are infeasible or unethical and Girish Putcha of Palmetto GBA opined that the differential reimbursement policies between LDTs and FDA-reviewed diagnostics by payors dis-incentivizes pursuit of regulatory review. The panel was comprised of various stakeholders in the diagnostic space including FDA, diagnostic test developers, real-world data vendors, and payors.
The second panel was composed of individuals representing patients, industry, diagnostic developers, CMS, FDA, and private payors. The discussion was moderated by Kate Rawson of Prevision Policy, and the panelists discussed policy priorities for supporting development of high-quality diagnostic tests including how to incorporate real-world evidence to support clinical utility of tests and better align CMS and commercial payor reimbursements. Michael Berger from Memorial Sloan Kettering noted that there is no real incentive under the current CMS payment structure to pursue a 510(k) clearance while Timothy Stenzel, Director of Office of In Vitro Diagnostics and Radiological Health at the FDA, urged diagnostic developers to pursue dialogue with the FDA earlier in the development process to facilitate better regulatory review. Representing the patient perspective during this conversation, patient advocate Florence Kurttila acknowledged the importance of questionnaires, such as the survey developed for the whitepaper, for patients to gather information about their tests and emphasized that patients should have the assurance that they are receiving well-validated diagnostic tests.
“Patients don’t know what they don’t know about their tests” – Florence Kurtilla, Patient Advocate
The meeting ended with an acknowledgement from Friends of Cancer Research’s President and CEO Jeff Allen to the meeting panelists, working group members, and meeting attendees for their engagement on this complicated but incredibly important topic.