A panel of diverse experts, moderated by Wendy Selig of the Melanoma Research Alliance, came together at Partnering for Cures to discuss the realities and opportunities in the world of molecular diagnostics. Several themes emerged during the discussion, including:
- Regulatory gaps – for example, laboratory developed tests – are creating uncertainty, particularly when it comes to reimbursement.
- Managing next-generation data in a productive way will be key to optimizing the potential of molecular diagnostics.
- Payers are fundamentally optimistic about personalized medicine but cynical because of experience.
- Though we have a lot of information, we don’t always know what it means.
Michael Pellini of Foundation Medicine underscored this point by highlighting the practical challenges that need to be addressed. “If we continue to think about each molecular test for a targeted therapy as a single test in which the clinician needs to be brilliant enough to single out the markers that he or she will test for in that patient and be right, that is challenge number one,” he explained. Pellini also pointed to other practical challenges, including the question of whether there will be enough tissue from a biopsy to conduct each test and how push-back from payers on reimbursement could impact use.
Jeffrey Trent of The Translational Genomics Research Institute (TGen) added that although technology has led us to a place where we can make informed treatment decisions based on molecular genetics, it is important that we do not overlook patients who do not have genetic alterations that can be specifically targeted by drugs. This is a challenge that TGen is addressing by conducting clinical trials in patient populations who cannot be stratified and treated based on genetic lesions.
Alberto Gutierrez of the FDA discussed some of the challenges with respect to the regulatory environment. He explained that the regulatory environment in this area is not clear and that a lot of the molecular testing that goes into diagnostics are laboratory-developed tests, which are not regulated by the FDA. Despite this gap, Gutierrez said that the FDA is more interested in clinical validity than clinical utility when it comes to molecular diagnostics. He explained the importance of ensuring that these tests provide physicians with meaningful data that they can understand and use confidently when making treatment decisions.
Sean Tunis of the Center for Medical Technology Policy provided the payer perspective. “Payers are fundamentally optimistic about personalized medicine, but cynical by experience because they know that they aren’t going to get exactly what they were promised in terms of savings,” he said. Tunis explained that payers are most concerned that the smaller populations of patients as a result of stratification by genetic subtype will lead to higher cost due to low demand for each individual test. He also said that payers are worried that in cases where risk outcome is low, clinicians may decide to continue with a more aggressive treatment path. In these cases, not only is the test reimbursed, but payers are also paying for a therapy that may not be necessary for the patient.
In conclusion, Selig asked the panelists what they think is the most important change that we need to pursue right away. Everyone agreed that first and foremost, we need not become overly engulfed in exploratory technology, and instead continue to make sure that we can treat the patients that need treatment today. Parkinson and Tunis highlighted that important changes need to be made with respect to reimbursement for diagnostic tests. As Tunis put it, “Whenever we get the regulatory framework figured out, we then need to tie it to reimbursement.”