Addressing Gaps in Bringing New Medicines to Patients 

Amy M. Miller, PhD

Throughout my career, I’ve focused on addressing barriers to health care innovation that hamper patients’ ability to access cutting-edge treatments. A big part of what attracted me to the PhRMA Foundation was its track record in helping tackle such barriers. Through its grants and fellowships to promising researchers, the Foundation builds academic capacity in biopharma to examine the knowledge gaps that hold back innovation.

Last week I moderated a conference hosted by the Association for Molecular Pathology exploring barriers to patient access to molecular diagnostic testing, which is critical to the advancement of precision medicine. This is an area close to my heart as a former executive of the Personalized Medicine Coalition. The day gave me fresh insight on how challenges facing the diagnostics industry are also challenges for the biopharmaceutical industry, and the importance of thinking beyond industry silos in addressing barriers to innovation.

Precision medicines account for about 35% of new medicines approved in the U.S. each year. To prescribe a precision medicine, clinicians will order a diagnostic test to ensure the patient has the biomarkers that the medicine targets. For example, before prescribing a lung cancer patient a drug targeting a mutation in the EGFR gene, a clinician will first use a diagnostic test to confirm that the patient has that mutation. When patients are not able to access the appropriate diagnostic test, their access to the corresponding medicine is jeopardized.

This is not a new model. Medicines with genomic testing requirements have been on the market for about 25 years. Yet the diagnostics industry is still challenged by practical considerations around clinicians ordering the right tests, medical coding issues, and appropriate value-based reimbursements for molecular tests by insurers. I realized during the conference that failure to address these challenges has created a system of workarounds that deepen health care disparities and hinder the ability of researchers (like those we fund at PhRMA Foundation) to draw on good health claims data.

The problems begin when clinicians need to order diagnostic testing. Should they order a test that looks for a single biomarker associated with a therapy, or should they order a more comprehensive test that looks for a wider range of biomarkers? For instance, should the lung cancer patient receive a test for EGFR only, a panel looking at many relevant genes, or complete genomic sequence of the solid tumor that can identify other potential mutations? Which option will be reimbursed by the patient’s health insurance and with what cost-sharing arrangement? If the clinician orders the single gene test and it’s negative, will there be enough tumor-tissue sample for additional testing? The list of considerations for clinicians is long, and the answers are not always clear.

Unfortunately, once the test is ordered, the path is not necessarily more straightforward, as complex insurance coverage and reimbursement issues come into play. Some insurers will not cover certain diagnostic tests or may require medical billing codes to be submitted in a specific way in order for labs to receive reimbursement. As such, labs may end up using medical billing codes for diagnostic tests such as single gene tests even though the lab performed a panel or comprehensive genomic sequence of the tumor. The labs do this to guarantee reimbursement, but they are eating the cost difference between the test that provides more limited data and the test that was performed. While this practice may be possible in academic medical settings where state-of-the-art treatments are expected and testing serves as a route to clinical trials, it is not possible in many of the community health care settings where patients are treated. As a result, this workaround widens the health disparities gap that already exists between patients treated in these two settings.

What’s also highly concerning to me is the degree to which this practice makes health care claims data incorrect, potentially muddling health economics and outcomes research (HEOR) models attempting to understand the relative value of diagnostics and therapeutics within the health care system. If HEOR researchers are basing models of cost on real-world data that incorrectly captures diagnostic test costs, value, and associated outcomes, then we’re not setting appropriate industry benchmarks for the future.

Clearly, we must look beyond our own industry to examine the broader challenges that impact pharmaceutical innovation – including the issues that underpin the data we use to conduct research and make health care policy decisions. I left the meeting with a renewed sense of the importance of PhRMA Foundation’s work to break down silos between fields, advance the frontiers of science and research, and build a talent pipeline for the future.