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Gutierrez Leaving FDA After Leading Dx Regulatory Efforts During Precision Medicine Revolution

This article has been updated to correct Don St. Pierre's title. He is currently deputy director in the Office of In Vitro Diagnostics and Radiological Health, not associate director. 

NEW YORK – Alberto Gutierrez, director of the US Food and Drug Administration's Office of In Vitro Diagnostics and Radiological Health, is leaving the agency after 25 years of public service.

Gutierrez joined the agency in 1992 as a researcher at the FDA's Center for Biologics Evaluation and Research working on methods of determining the purity and structure of vaccine components, and eventually became director of OIVD within FDA's device center in 2009. He will retire from the FDA at the end of September to explore other opportunities in the healthcare sector, but hasn't yet decided what his next steps will be.

Don St. Pierre, currently deputy director at OIVD, will be acting director of the office until the agency can hire Gutierrez's replacement.

Gutierrez's time at the FDA was a period of breakneck advancement in molecular diagnostics, and in response to the growing impact of genetic testing on the public health, OIVD also expanded. In dealing with a market that today includes 70,000 genetic testing products, FDA has at times responded with aggressive regulatory action. However, the agency also sought to advance the field of precision medicine by fostering better collaboration between its drug and device centers and by finding ways to reduce the regulatory burden on companies developing next-generation sequencing tests.

Gutierrez led OIVD during "a revolution in precision medicine," said Lakshman Ramamurthy, who worked at the FDA from 2007 to 2013. Ramamurthy, who considers Gutierrez a mentor and a good friend, said, "He is someone who always cared very deeply about serving the public."

When Gutierrez was a "fledgling reviewer," Steven Gutman, the first director of OIVD (then called Office of In Vitro Diagnostic Device Evaluation and Safety), recalled a "huge fight" they had over the clearance of a product. Gutman felt strongly that it should be cleared, and Gutierrez maintained that it shouldn't be.

The director got the final word and the product got a green light. As punishment for disagreeing with him, Gutman quipped that he made Gutierrez deputy director of the office in 2007. "I was impressed with how good he was," he said.

Gutman created OIVD in 2002 to harmonize pre-market and post-market regulatory activities for in vitro diagnostics into one multi-disciplinary office. Previously these regulatory aspects of IVDs were siloed across different centers, which created inefficiencies when communicating and making decisions about products.

"That initiative has my name on it," said Gutman. "But what I'm most proud of … is he's taken what I've started and grown it." OIVD is three times larger now than it was when Gutman ran the office. The office not only regulates IVDs, but radiology medical devices as well, and implements the mammography quality program.

A testament to how Gutierrez ran OIVD is the fact that the so-called "total product life cycle" model piloted by the office — where pre-market and post-market oversight of a certain product type are performed by an integrated unit — is now being considered by other sectors in the agency. "It's an administrative and scientific feat of no small consequence that he's pulled off," Gutman said.

After Gutman's departure from the FDA, Gutierrez became its director in 2009. Shortly thereafter, the agency cracked down on direct-to-consumer marketing of health-related genetic tests, reasoning that companies marketing such products without FDA review were putting the public health at risk. The agency's regulatory actions against companies like 23andMe would for many years restrict the sale of genetic tests that gauged risks for Alzheimer's and other serious diseases without a doctor's prescription. 

However, in an attempt to balance public health protections with access, Gutierrez's OIVD never barred consumers from having direct access to their raw, uninterpreted genomic data. The agency also has not hindered DTC access for recreational genomics, such as ancestry testing. However, proponents of DTC genomics have continued to accuse the agency of paternalism in this regard.

Perhaps the biggest controversy while Gutierrez was OIVD director was the agency's decision to regulate LDTs, which the agency felt were becoming technologically complex and being marketed too broadly. The FDA had historically left the Centers for Medicare & Medicaid Services to regulate LDTs according to the Clinical Laboratory Improvement Amendments, but as the molecular diagnostics market grew, the agency felt there were critical gaps within those regulatory standards placing the public health at risk.

After holding several meetings, the agency released a legally and politically controversial draft guidance on its plan to regulate LDTs in 2014. Throughout, the lab industry and pathologists pushed back against FDA oversight as cumbersome and a threat to innovation. 

Roger Klein, chair of the Association for Molecular Pathology's professional relations committee, acknowledges he had differences of opinion with Gutierrez over the regulation of LDTs, and supported an AMP proposal to update existing CLIA regulations instead of subjecting lab tests to FDA oversight. But Klein also served with Gutierrez on the Clinical Laboratory Improvement Advisory Committee, an expert group that provides scientific and technical guidance to the US Department of Health and Human Services, and said Gutierrez has "sincere concern for the public health," and noted his departure as "a significant loss to the agency." 

Despite the clashes with the lab community over LDT regulations, Gutierrez said he's most proud of building a team that came up with creative ways to apply regulations so they wouldn't be too burdensome on industry. Those who knew and worked with Gutierrez attest to the fact that he and his team at OIVD were cognizant of not stifling the nascent fields of genetics and precision medicine with heavy-handed oversight.

Gutierrez helped advance a number of programs that refined the agency's approach and thinking around precision medicines. Officials in the diagnostics sector worked with the drugs center to streamline procedures that enabled speedy approval of personalized treatments and companion tests that identify which patients would respond to those therapies.

According to the non-profit Personalized Medicine Coalition, precision treatments accounted for more than 20 percent of new molecular entities approved by the FDA for the past three years. "Although they may not know it, patients are deeply indebted to FDA for its … long-standing commitment to advancing precision medicine, which has led to a new era of drugs that are guided by biomarker strategies," PMC President Edward Abrahams said. "Elizabeth Mansfield and Alberto Gutierrez are among the leadership team at FDA that has brought about this change." 

Mansfield, who was deputy director of the personalized medicine group within OIVD, worked closely with Gutierrez on many initiatives to streamline review and approval of diagnostic products. However, she left the agency earlier this year to join Grail, a company that aims to develop a pan-cancer early detection blood test.  

While at the agency, Mansfield worked with Gutierrez to advance mechanisms that would enable NGS-based tests to go quickly through regulatory review and come to market. For example, with the clearance of Illumina's MiSeqDx system, the agency published special requirements that other labs could follow to launch tests that run on the same system, with the same intended use, without submitting for premarket review. 

NGS is an area where the FDA is "trying to be creative, think out of the box, and not be a barrier to new technologies," said Gutman, who is a strategic advisor at Myraqa, a regulatory consulting firm owned by sequencing platform provider Illumina.

Under Gutierrez's leadership at OIVD, the agency put out draft guidelines on how labs could demonstrate analytical validity for germline NGS tests and encouraged data sharing by proposing the use of public variant databases to establish clinical validity of NGS tests. More recently, the agency approved the first NGS-based companion diagnostic and the first panel CDx that can identify best responders to multiple lung cancer drugs. 

"They clearly get it that NGS is an important technology that can transform modern-day medicine," Gutman said.

Ramamurthy, now the global regulatory lead at cancer genomic analysis firm Foundation Medicine, highlighted Gutierrez's involvement in the Parallel Review program, through which test providers can apply to have FDA and CMS simultaneously review the safety and efficacy of a diagnostic and whether the product meets the criteria for Medicare coverage.

As a reviewer at the FDA, Ramamurthy also worked on this program, and he is now leading efforts at Foundation Medicine as the company pursues parallel review of its NGS test FoundationOne. While Gutierrez's departure is certainly a loss, Ramamurthy doesn't expect his departure to impact innovative programs like Parallel Review.

Gutierrez's departure, however, comes during a period of transition for the agency as it aligns with the priorities of a new administration. President Trump has promised to scale back government regulations, and much to the relief of the lab industry, following the presidential elections last year, the FDA said it would not finalize its controversial LDT guidance.

"It's very much unknown how laboratories are going to be regulated, if at all, on LDTs," Gutierrez said. "I would like to see some resolution to that. It doesn't necessarily have to be through the agency, but the community has to come to an idea of what is the best way to do this."

The debate over the agency's oversight of LDTs has been going on for as long as Gutierrez has been at the agency. During 17 years within FDA's Center for Devices and Radiological Health, he spent much time on the LDT regulatory issue. While Gutierrez would have liked to see this resolved before his departure, he felt that the agency had advanced in its discussions with the lab community and brought to light the regulatory gaps within CLIA.

In Gutman's view, although FDA didn't finalize the LDT guidance, the issue isn't dead. "You might be able to escape it for a couple of years during the Trump administration, but it's a compelling discrepancy in the way we regulate lab tests," he said. "It's got to be addressed at some point."

For what it's worth, the FDA this year published a discussion paper on its current thinking on LDTs, which the agency drafted with significant input from the lab industry. The American Clinical Laboratory Association, a group that has been staunchly against FDA's oversight of LDTs, responded more favorably to the discussion paper and sees it as an opportunity to advance a diagnostic-specific legislative solution through Congress. 

The consumer genomics industry also got a boost this April, when the agency allowed 23andMe to begin offering a handful of disease-risk tests more than three years after the company had to stop marketing such tests following an FDA warning letter. The agency also hasn't gotten in the way of low-cost genetic testing companies, such as Color Genomics, offering hereditary cancer and high cholesterol risk tests that consumers can order online and quickly have a doctor in an independent network review and approve. 

Meanwhile, the new FDA commissioner, Scott Gottlieb, will likely continue to find areas where the agency can take a light regulatory touch so consumers have access to innovative technologies and products. Before becoming commissioner, Gottlieb recommended FDA take a staged regulatory approach where a diagnostic can be fully validated in the post-market setting. 

In line with this rationale, one of Gottlieb's first efforts since becoming commissioner is the so-called Pre-Cert for Software Pilot Program. Within this program, companies can voluntarily have FDA review and certify their systems for software design, validation, and maintenance. The certification may allow some firms to commercialize low-risk digital health technology products without premarket review if they collect and submit post-market data. 

Describing this program, Gottlieb wrote, "When people think about personalized medicine, they often think of genetic testing and sequencing of the human genome. But the concept of personalized medicine is much broader. It includes the re-imagination of healthcare delivery. It includes empowering consumers to take more control of their own healthcare information to make better informed decisions about their medical care and healthy living."

The launch of this pre-certification pilot aligns with recent trends in the genomics space. In July, a software-focused company called Helix launched the first ever genomics marketplace, where consumers can have their exome sequenced once and buy apps that query that genomic data in different contexts, such as family planning, weight management, or carrier screening. 

Despite the loss of Gutierrez and Mansfield's personalized medicine leadership at the FDA, proponents of the space feel reassured by Gottlieb's comments and by recent approvals, such as the first pan-cancer, tissue-agnostic cancer drug. "While it is of concern that they are leaving the agency, we can expect that Scott Gottlieb will continue to build on the [personalized medicine] foundation of the past decade," PMC's Abrahams said. 

As the field continues to advance personalized medicines through greater understanding of the role of biomarkers in drug response and adverse events, the FDA will continue to play a crucial role, Gutierrez reflected. "I don't believe that's going to change."