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FDA Moves to Regulate Thousands of Diagnostic Tests

Mary K. Caffrey
Thousands of tests that give doctors guidance on how to treat cancer or other progressive, chronic illnesses will face FDA scrutiny for the first time, according to a July 31, 2014, announcement from FDA Commissioner Margaret A. Hamburg, MD.

The FDA issued final guidance on its clearance policies for companion diagnostics, which are used in cancer treatment to identify those patients who may benefit from a specific therapy. These tests, often based on genetics, are frequently developed in tandem with the therapy and were already subject to FDA clearance.1

The agency had also exercised oversight of all direct-to-consumer tests as well as those whose reliability had to be guaranteed across multiple laboratories, such as those in hospitals. What’s new is the FDA’s movement into the realm of laboratory developed tests, known as LDTs, which are designed, manufactured and used within a single laboratory; tests are performed with a patient’s blood or tissue samples.1

“Ensuring that doctors and patients have access to safe, accurate and reliable diagnostic tests to help guide treatment decisions is a priority for the FDA,” Hamburg said in a statement. “Inaccurate test results could cause patients to seek unnecessary treatment or delay and sometimes forgo treatment altogether. Today’s action demonstrates the agency’s commitment to personalized medicine, which depends on accurate and reliable tests to get the right treatment to the right patient.”1

The number of tests that could be affected by FDA’s action could be as high as 11,000, and the number of labs involved as high as 2000.2 Forthcoming guidance documents will spell out how manufacturers will provide information about their LDTs, which they can continue to sell and perform pending new rules. For now, the FDA will not move to regulate LDTs for rare diseases and certain tests for which there is no FDA-cleared option.

FDA’s move is not a surprise, as the agency has had its eye on the industry for some time. FDA issued a report in October 2013, “Paving the Way for Personalized Medicine,”3 outlining its possible role in nurturing molecular diagnostics. Some industry experts like Bruce Quinn, MD, PhD, of Foley Hoag LLP, have highlighted the contrast between the FDA’s view of the potential of molecular diagnostics compared with CMS,4 which is being sued by the California Clinical Laboratory Association over reimbursement disputes with Medicare contractors.5

The agency has also been pressed by some members of Congress to close the regulatory gap exposed by the sharp rise in genetic and molecular testing, particularly in cancer care. US Rep. Louise M. Slaughter, a New York Democrat who is a microbiologist, hailed the FDA’s move, which came just over a year after she wrote to the Office of Management and Budget calling on the agency to exercise its authority to bring “transparency and accountability” to the field. “Diagnostic testing is an incredible medical breakthrough that holds the key to personalized medicine, but patients can only realize the benefits of diagnostic testing if the tests are safe and effective,” Slaughter said in a statement.6

In her June 2013 letter, Slaughter noted that LDTs outside FDA oversight not only lack pre-market approval, but also are not subject to any post-market surveillance or recalls, despite their “interchangeable” nature in clinical settings. The current system, she said, offers “little transparency for patients or even doctors regarding what test has been used.”6

There has been debate within the testing industry whether more FDA oversight helps or hurts. While regulation would bring higher costs to enter the market, some believe it could pave the way for fewer reimbursement disputes with insurers and, especially, with CMS.

One testing company contacted by Evidence-Based Oncology said officials would review FDA’s plan before reacting. In an e-mailed statement, Myriad Genetics said the company “looks forward to collaborating with policymakers and stakeholders to provide input and focus on the future of the US regulatory process for LDTs.”

Myriad, which has a reimbursement application pending with CMS for its Prolaris prostate cancer test, also said in its statement, “Myriad has a strong track record of scientific excellence that includes conducting numerous clinical studies in support of the analytical and clinical validity of our diagnostic tests.”

Nanostring CEO Brad Gray said a riskbased system of regulation could work as long as the reimbursement process recognized its value. He discussed the clearance process that Nanostring’s Prosigna breast cancer assay went through, which Gray said “required multiple clinical and analytical validation studies run to the FDA’s exacting standards across thousands of samples. We believe that the results are impressive, with the Prosigna assay outperforming the leading Laboratory Developed Test, Oncotype Dx, in a 1,000+ patient comparative effectiveness study.” (Gray cited a study that appeared in the Journal of Clinical Oncology.7)

“As a result, we believe the key to making the system work will be recognition by payers of the substantial time and investment required to meet the FDA requirements,” Gray continued. “Many industry participants are watching closely to see how payers react to the Prosigna assay in terms of setting both medical policy and establishing reimbursement. It will be important for them to see that the investment in FDA clearance is worthwhile if others are to follow in our footsteps.”

Gray noted that already, a large national payer has started offering Nanostring reimbursement for Prosigna. EBO
References

1. FDA takes steps to help ensure the reliability of certain diagnostic tests [press release]. Silver Spring, MD: FDA Newsroom; July 31, 2014. http://www.fda.gov/NewsEvents/Newsroom/ PressAnnouncements/ucm407321.htm

2. Herper M. FDA to regulate thousands of cancer, genetic and other diagnostics. Forbes website. http://www.forbes.com/sites/matthewherper/2014/07/31/fda-to-regulate-thousands-ofcancer-genetic-and-other-diagnostics/. Published July 31, 2014. Accessed August 4, 2014.

3. Simoncelli T. Paving the way for personalized medicine: FDA’s role in a new era of medical product development. http://www.fda.gov/downloads/scienceresearch/specialtopics/personalizedmedicine/ucm372421.pdf. Published October 2013. Accessed March 7, 2014

4. Caffrey MK. When science outpaces the payers: reimbursement in molecular diagnostics. Am J Manag Care. 2014;20(SP7):SP234-SP236.

5. Overly J. Medicare sued over erratic payment policies. Law360.com. http://www.law360.com/articles/530713/medicare-sued-over-erratic-payment-policies. Published April 22, 2014. Accessed April 25, 2014.

6. FDA heeds Rep. Slaughter’s call for transparency in laboratory diagnostic testing [press release]. Washington, DC: US House of Representatives; July 31, 2014. http://www.louise.house.gov/press-releases/fda-heeds-rep-slaughters-call-fortransparency-in-laboratory-diagnostic-testing/.

7. Dowsett M, Sestak I, Lopez-Knowles E, et al. Comparison of PAM50 risk of recurrence score with Oncotype DX and IHC4 for predicting risk of distant recurrence after endocrine therapy. J Clin Oncol. 2013;31(22):2783-2790.
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