Commissioner Gottlieb Updates Community Oncologists on the FDA's Mission to Improve Access

From the day that he took office, Scott Gottlieb, MD, 23rd Commissioner of the FDA, has been on a mission to develop policies and implement changes that can tackle the challenges facing the agency, and healthcare in general, and the past year with him at the helm has seen the FDA approach several issues head on.

At the 2018 Community Oncology Conference hosted by the Community Oncology Alliance (COA), April 12-13 in National Harbor, Maryland, Gottlieb provided the audience with an overview of current and future plans of the regulatory agency, particularly its strides within the molecular diagnostic testing space and liquid biopsies.

In December of last year, the FDA approved the first comprehensive companion diagnostic test for solid tumors that uses next-generation sequencing (NGS) technology to examine all classes of genomic alterations in the 324 genes known to cause cancer growth—Foundation Medicine’s FoundationOne CDx test.

The rapidly falling cost of NGS, Gottlieb said, can allow whole-genome sequencing and aid precision-guided treatments. NGS, he added, is a glaring example of the innovative advances and strides in cancer research.

“FDA recognizes the importance of this, and we want to serve as a bridge to allow this innovation to come into the market,” while simultaneously ensuring patient safety.

Just last month, CMS announced that it has finalized a National Coverage Determination that covers diagnostic laboratory tests that use NGS for patients with advanced cancer.

“We want cancer patients to have enhanced access and expanded coverage when it comes to innovative diagnostics that can help them in new and better ways,” said CMS Administrator Seema Verma when making the announcement. “That is why we are establishing clear pathways to coverage, while at the same time supporting laboratories that currently furnish tests to the people we serve.”

FDA wants to be “as nimble and sophisticated as the science that drives these technologies, so clinicians and patient can have access to them as soon as possible,” Gottlieb said. This will allow NGS technology to guide clinical trial participation and allow personalized treatment options for cancer.

Gottlieb informed the audience about 3 new announcements by the FDA that can further the routine use of NGS. The first addresses the design, development, and analytical validation of NGS-based diagnostics; the second provides guidance on the use of public human genetic variant databases to confirm clinical validity of the tests; and the third makes it easier for drug and diagnostic developers to file their documents with the FDA by providing a streamlined submission process for risk determination.

“The streamlined submission process will allow an easier common filing for the drug and its companion diagnostic and avoid 2 separate applications,” Gottlieb said. This could ease some of the administrative burden on pharmaceutical manufacturers, as well as the regulators reviewing these documents, and create a more cohesive process.

Gottlieb noted that the guidelines can reduce screening time and cost. From the patient’s perspective, NGS can help avoid multiple tissue biopsies. “NGS will also improve the process of matching patients to participate in suitable clinical trials,” he said, considering the ease of the screening process and the large number of patients' genetic variations that the physicians would get a glimpse into.

Gottlieb emphasized, however, that while the recipe for oncology innovation includes researchers, clinicians, regulators, policy makers, and advocates, “public confidence in the institutions that support innovation is vital.”

Gottlieb then turned his attention to the high cost of care, especially in oncology. “Cost of care is one of the bigger challenges in oncology, and cancer patients are disproportionately shouldering these costs,” he said, via co-pays and deductibles. “While the FDA cannot regulate drug prices and it is not our primary role, Congress has provided us the ability to reduce anticompetitive behavior to allow access to products such as biosimilars.”

The other issues that influence cost are the drug development timelines and development costs, he added. Indirect financial costs of time and risk are inherent to clinical trials, which in turn impacts drug costs. NGS, Gottlieb believes, can come into focus here. “Many trials fail, often in late stages, not just because of science but trial conductance. We need better clinical trial designs,” he said.

Emphasizing the value of targeted and personalized treatments, Gottlieb said that biomarker-directed oncology trials are more likely to succeed.

Following a question by an oncologist from the audience on his prediction for the biosimilar drug market, Gottlieb said, “We are in very early stages of biosimilar development. We have challenges with physician adoption, especially for curative treatments, but not for treating chronic diseases like rheumatoid arthritis.” However, he pointed out that physicians had similar concerns with generics when the Hatch-Waxman act was instituted in 1984.

Just last month, Gottlieb told the audience at the 2018 National Health Policy Conference of America’s Health Insurance Plans that misaligned incentives in the biosimilars market—a product of contracting practices as well as consolidation across the drug supply chain—could be a barrier to patient access to biosimilar medicines.

“Market penetration for biosimilars is extremely hard, especially because of the rebate structure for branded drugs,” Gottlieb pointed out to the COA audience. “This is a huge impediment for their market entry.”