FDA Strategizes to Step Up Introduction of Lower-Cost Drugs and NGS

OncologyLive, Vol. 19/No. 12, Volume 19, Issue 12

The market for biosimilar introduction is “extremely unstable,” but the FDA is working to ease the launch of more affordable cancer medicines with a strategy that includes an attempt to reduce anticompetitive behavior.

Scott Gottlieb, MD

The market for biosimilar introduction is “extremely unstable,” but the FDA is working to ease the launch of more affordable cancer medicines, both biosimilars and generics, with a strategy that includes an attempt to reduce anticompetitive behavior, according to Scott Gottlieb, MD, commissioner of the FDA.

Gottlieb discussed the competitive landscape for oncology agents and how it is affecting the introduction of new medicines in a keynote talk at the 2018 Community Oncology Conference that the Community Oncology Alliance held in National Harbor, Maryland, in April.

There are numerous approval and marketing challenges for makers of biosimilars who seek to challenge the dominance of branded drugs. Since September 2017, 2 biosimilars have received FDA approval specifically for treatment of cancer and 2 for conditions related to cancer. Gottlieb said that pace has been a pleasant surprise. “I always thought it was going to be a slower-developing area with more hurdles to get over, but we’re right on track,” he said.

Although the FDA has no regulatory power over the prices set by manufacturers, distributors, and retailers, Gottlieb said, the FDA’s commitment to encouraging biosimilar and generic competition in the cancer drug marketplace has the potential to lower costs and improve patient access to available cancer care.

“Drug treatment costs are only a fraction of total care oncology costs, but patients [with cancer] are disproportionately shouldering the cost of oncology medicines,” stated Gottlieb. “The perverse reality of the market today is that cancer treatment comes with its own financial toxicity.”

In tandem with his discussion on improving competition in the oncology drug marketplace, Gottlieb discussed new guidelines from the FDA on the development and review of next-generation sequencing (NGS) tests.1 These tests also should help to spur innovation and the introduction of life-extending oncologics, he said.

“The FDA recognizes the tremendous potential of NGS technology to guide and improve patient outcomes,” Gottlieb said. “And we’re developing a policy approach to keep the pace with fast-moving NGS technologies that give patients and clinicians confidence in these panels’ analytical and clinical validity.”

The first breakthrough-designated NGS-based diagnostic test, F1CDx (FoundationOne CDx), was approved in November 2017. The powerful diagnostic tool can detect mutations in 324 genes and 2 genomic signatures in any solid tumor type. In March, CMS followed up with a national coverage determination in favor of NGS panel testing, expanding coverage to FDA-approved tests for patients with relapsed, refractory, or stage III cancers in addition to stage IV cancers.

Large-panel NGS tests will make it “easier and less expensive to screen patients for tumor mutations with a single test and then efficiently match them with available clinical trials,” Gottlieb said. He predicted that the information gained through this process will illuminate pathways to more effective therapies and stimulate the development of new agents. “This will increase the productivity of drug development, as drugs can then be targeted at common biomarkers across numerous tumor types.”

Gottlieb said that the FDA is striving to make NGS guidelines and current and future regulations “as nimble and sophisticated as the science driving these technologies so that clinicians and patients have access to them as soon as possible, while still providing patients with the reasonable assurance of safety and effectiveness they expect.”

The first set of guidelines addresses the design, development, and analytical validation of NGS-based panels, which can be used when diagnosing individuals with suspected genetic diseases. “For oncology, germline cancer predisposition contributes to about 5% to 10% of observed cancers. For cancer predisposition syndromes, such as Lynch syndrome, interventions like colonoscopies may be able to improve expected survival by sharply decreasing the rates of colorectal cancer,” Gottlieb noted.

These recommendations also explain what the FDA would look for in a premarket submission to assess a prospective test’s analytical and clinical validity, as well as its accuracy in detecting the presence or absence of a particular genomic change, leading to what Gottlieb said would be consensus standards for future NGS-based tests.

The second set of guidelines recognizes the availability of public human genetic variant databases, which could help to confirm the clinical utility of in vitro diagnostic (IVD) tests, particularly during premarket review. These FDA-recognized databases could serve as sources of existing evidence for the claims, such as of the clinical significance of a germline or somatic mutation, that developers of IVD tests make in the review process.

An additional piece of draft guidance explains a process that oncology trial sponsors can use to establish the risk level associated with an investigational IVD to be used in a trial of an investigational cancer drug or biological product.2 According to the guidelines, investigational IVDs are categorized as a significant risk, nonsignificant risk, or exempt from review. If a device is determined to be a significant risk, meaning that it could pose a serious risk to the health or safety of a patient, an investigational device exemption may be needed. The level of risk may disqualify a study for streamlined submission.

“This guidance reduces burdens on sponsors and on FDA staff by outlining circumstances under which sponsors may be able to include information about an investigational IVD into the investigational new drug application submission to the FDA center responsible for the therapeutic product,” Gottlieb said.

With these new policies, the FDA hopes to provide test developers with a more efficient path to market by means of efficient and accurate testing. They will encourage the innovation and adaptation of tools that can increase the productivity research and patient care.


  1. FDA finalizes guidances to accelerate the development of reliable, beneficial next generation sequencing-based tests [news release]. Silver Spring, MD: FDA; April 12, 2018. 2018. www.fda.gov/NewsEvents/Newsroom/ PressAnnouncements/ucm604462.htm. Accessed April 12, 2018.
  2. Investigational in vitro diagnostics in oncology trials: streamlined submission process for study risk determination guidance for industry. FDA website. www.fda.gov/downloads/Drugs/GuidanceCompliance- RegulatoryInformation/Guidances/UCM604441.pdf. Published 2018. Accessed May 17, 2018.