Martha Bonino is the Director of Strategic Accounts at Clinipace Worldwide.

My colleague, Dr. Lee Schacter, MD, Executive Medical Director of Oncology, and I, along with Dr. Afshin Safavi, Ph.D., founder and Chief Scientific Officer at BioAgilytix, a provider of bioanalytical testing services, were thrilled with the interest we received during our latest webinar on Cancer Drug Development Using Molecular Pathology.

In our first Q&A post, we addressed some of the questions we received around enabling technologies. Up next, we’re going to address questions around the future direction of molecular profiling. If you have any additional thoughts or related questions to ask our experts, please share below in the comments.

Q #1: Will we really see approvals based on mutation markers instead of by traditional disease? If so, will trials be done across various traditional cancer types, and how will dosing be determined, assuming it would be different for different cancers?

Dr. Schacter: I think we will see it eventually. What we’re likely going to see is an evolutionary process whereby we document that targeted therapy works in disease X and provides a clinical benefit, preferably survival. So, you’ve shown that you’ve met the standard criteria. Now, you show that mutation is a driver in other diseases, and you may be able to register not only on survival or progression pre-survival, but on response. While that might be the first step down the road for the FDA to get comfortable, it’s very likely that once you show response, you’re going to get off-label use, and there will be pressure for approval for reimbursement purposes outside the US. It may well be European agencies that drive some of the change, whereby once you’ve shown a survival or progression pre-survival in disease X, response alone in disease Y may get you extended label and reimbursement.

So I think it’s going to be a progression. We’re just now understanding the concept that molecular pathology is really what’s going on. It will take a while, but I think it’s coming, and we need to start thinking about it.

Martha Bonino: I agree with Dr. Schacter. I think we will see approvals based on mutation markers, but it will take time. Assuming we are correct and, in the not too distant future, tumors will be defined by the oncogenic driver rather than the histology, then trials and approvals based on the driver will become standard. I think the transition will be approval using standard end points in one disease with a given driver, and secondary approval for other diseases with the same driver using limited numbers of subjects based on response.

Q #2: How will biomarkers be commercialized? Should the company whose drug is indicated using the markers promote them alongside the drug?

Dr. Schacter: A great example to share is the package insert for Crizotinib, which reads “XALKORI is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are anaplastic lymphoma kinase (ALK)-positive as detected by an FDA-approved test.” The test is part of the package insert, so it can be promoted alongside the drug, but whether or not it is promoted would be a marketing decision. It is necessary to include the need for the test in promotional material about the drug, but whose “FDA approved test,” if any, is promoted will doubtlessly depend on the value to the company.

Be sure to listen to our webcast in its entirety to hear more about Cancer Drug Development Using Molecular Pathology. Thanks for tuning in, and check back soon for the third part of our Q&A series!