Workshops | Day 1 | Day 2

September 12, 2018
Time: 1:00 PM – 5:00 PM

Moderator: Felix Frueh, Executive Partner, Opus Three
Fees: Free for paid event attendees, $250 for others
Completion Credit: No CME but provide Certificate of Completion

Discussion Leaders:

  • Felix Frueh, Executive Partner, Opus Three
  • Federico Goodsaid, Associate Partner, Opus Three
  • Edmund Pezalla, Founder & CEO, Enlightenment Bioconsult

Workshop Objectives:

This four-hour workshop is intended to be an interactive educational experience focused on discussing the frontiers of regulatory and reimbursement aspects for the development of novel biomarkers and companion diagnostics.

Part 1.    Do I need a Companion Diagnostic? (Felix Frueh)
Companion diagnostics are required where the diagnostic has been integral for the development of the therapeutic. This is the case with biomarkers for the selection of patients with an increased likelihood of response to therapy. However, not all uses of biomarkers or diagnostics associated with drug therapies have such a requirement. Information derived from the diagnostic may simply provide additional insights to evaluate or adjust a particular therapy, for example, the diagnostic may inform about potential dose adjustments, confirm disease, or for other uses.  An accurate context of how the diagnostic test is used during drug development is critical: while companion diagnostics may significantly improve the efficacy rate (e.g. by exclusion of likely non-responders), they may also limit the market for a new therapy. Other diagnostic classifications such as “complementary” could be more appropriate. An exhaustive evaluation of which type of use is appropriate needs to be considered early in development and should be discussed with regulators to assure a smooth development of the therapy in question and to avoid surprises at the time of market approval.  This presentation will discuss different factors to consider in a decision about whether or not a companion diagnostic classification is accurate and whether alternative classifications are appropriate.

Part 2.  An In-depth Look At Regulatory And Reimbursement Aspects Of Companion Diagnostics (Felix Frueh, Federico Goodsaid, Edmund Pezalla)

Companion diagnostics have evolved in the past few years, moving from one-marker/one-drug paradigm to more complex models including multi-gene, multi-panel products. Both regulators and payers are adapting and developing new guidance and policies.  The regulatory response to this change is reflected in a sequence of approvals from CDRH over the past two years. It is of particular interest to evaluate where and how these tests are run, how they are interpreted, and the claims included in the product label. These case studies illustrate a nuanced new regulatory approach to companion diagnostics, and a redefinition of the product development and regulatory submission criteria for their success. Similarly, payers are adapting and have been developing new coverage and reimbursement policies for both diagnostics and pharmaceuticals to streamline the process without hindering patient access. Workshop participants will walk away with a better understanding of the different regulatory pathways along with an appreciation of the payer landscape both of which drives better decision making in clinical development and are necessary for a successful commercialization strategy.