Executive Summary

Abernethy comes to FDA from Flatiron Health, which develops oncology-specific electronic health record software and real-world evidence for cancer research. She succeeds FDA veteran Rachel Sherman, who will retire from the agency in January.

Incoming US FDA Principal Deputy Commissioner Amy Abernethy will bring extensive experience in real-world evidence and health information technology to the number-two position at FDA under Commissioner Scott Gottlieb in early 2019. She will succeed long-time FDA official Rachel Sherman, who is retiring from the agency.

Flatiron Health Inc., where she is chief medical officer, chief scientific officer and senior vice president, oncology. Flatiron develops oncology-specific electronic health record software and real-world evidence for cancer research. The firm was acquired by Roche in March 2018.

Gottlieb announced the personnel change in a Dec. 17 email to FDA staff. “Dr. Abernethy is a gifted oncologist and a specialist in palliative care medicine who has dedicated her career to patient care and medicine. She’s a highly regarded thought leader who has held numerous positions of leadership in her fields of interest and distinguished herself for her intellect, her passion for patient care and science, and her collegiality.”

The appointment also provides a potential succession plan, for Gottlieb, should the need arise. (See sidebar for related story.)

Abernethy will succeed FDA veteran Rachel Sherman, who will retire in January. Sherman first left FDA in 2014 after 25 years but returned a year later to serve under then-Deputy Commissioner for Medical Products and Tobacco Rob Califf, and became his number-two when he became commissioner in 2016. She stayed on in essentially the same role under Gottlieb. (Also see “Gottlieb Adds A No. 2: Sherman Is US FDA Principal Deputy Commissioner” – Pink Sheet, 21 Aug, 2017.)

Sherman provided an important sense of stability and continuity during the transition after the 2016 elections, and hands-on leadership for several key Gottlieb initiatives, including the agency’s work on developing real-world evidence and patient-focused drug development activities. She was also put in charge of stickier policy issues, like the opioid response and heading the internal “Right-to-Try” working group.

While Sherman brought unsurpassed institutional memory and regulatory experience to the role, Abernethy is likely to be much more comfortable in the public-facing aspects of the position. In her public appearances, Abernethy presents with an assertiveness and business-first attitude, while also conveying a warm bedside manner. Her style is captured in a 2013 TEDMED talk, “Can Patient Data Have a Second Life?”

Sherman’s future plans are not yet known. During her 18 months away from FDA, she worked at Greenleaf Health, LLC, a Washington-D.C. based consulting firm.

While Abernethy’s official start at FDA awaits final clearances CDER Deputy Director for Operations Patrizia Cavazzoni will serve as acting principal deputy commissioner. Like Abernethy, Cavazzoni is a former industry executive (joining FDA from Pfizer Inc.) with a strong focus on real-world evidence. (Also see “US FDA Rethinking Post-Market Safety Function As Part Of New Drug Review ‘Modernization'” – Pink Sheet, 9 May, 2018.)

Deep Expertise in Real-World Evidence

Abernethy joined Flatiron in 2014 and helped build the health IT firm’s vision of converting oncology electronic health records into a useable source of real-world data that can support regulatory decision-making. She brings that expertise to FDA at a time when the agency is establishing a framework for real-world evidence; her presence at the agency could be critical to accelerating that process. (Also see “US FDA Is Hesitant About Using Observational Studies In Real-World Evidence Framework ” – Pink Sheet, 6 Dec, 2018.)

Abernethy travels in a tight circle of big names and principal organizations in real-world evidence, clinical effectiveness research and patient-centric care. She was a central figure in establishing the Patient-Centered Outcomes Research Institute; leads the PCORnet Patient-Reported Outcomes Task Force; serves on the advisory group to the Duke Margolis Real World Evidence Collaborative; and previously ran the Center for Learning Health Care in the Duke Clinical Research Institute.

Through PCORI, Abernethy has worked closely with FDA and stakeholder groups like the Reagan-Udall Foundation, Friends of Cancer Research and the Personalized Medicine Coalition. Flatiron participates in Reagan-Udall’s “Big Data for Patients” program, and she has collaborated with Friends on a number of real-world evidence meetings, the most recent in July 2018. She serves as a PMC board member.

She also speaks regularly at the National Academies, most recently on immune checkpoint inhibitors, and how RWE could support the development of I/O combination therapies. That is a solid, cutting-edge subject for a top FDA official, and it hits on one of the agency’s (and specifically Center for Drug Evaluation & Research Director Janet Woodcock’s) main themes: getting more data on new cancer treatments from patients in the community setting to support regulatory decisions.

From the outside, Abernethy has encouraged FDA to adopt broader use of real-world evidence to support regulatory decisions. At a meeting in 2016 hosted by Friends and Alexandria Summit, she presented an approach that would rely on an assessment of patient records to define an endpoint similar to progression-free survival in a clinical trial. (Also see “Clinical Trial Endpoints May Not Fit Real-World Studies” – Pink Sheet, 27 Jun, 2016.)

At the time, that idea was greeted somewhat skeptically by FDA Office of Oncology & Hematology Products Director Richard Pazdur, who noted that progression-free survival is already a problematic endpoint for clinical studies. He urged suggested instead a focus on different endpoints for real world studies, like “time to next treatment,” rather than try to “shoehorn” clinical trial endpoints “into the real-world setting.”

Abernethy is credited with coining the term “financial toxicity” as a side effect of high-priced oncology treatment. That dovetails well with Gottlieb’s willingness to speak openly on drug pricing.

Given Roche’s recent acquisition of Flatiron Health, it would appear to be a good time for Abernethy to make a pivot to the public sector. However, it may raise questions about Roche’s large investment ($2.1 billion) in the real-world evidence effort.

Abernethy’s departure would seem to indicate that she views the potential to advance the field as greater in the public sector than in the private sector. At the same time, she will technically be joining FDA directly from a pharmaceutical company. While she can hardly be painted as a career industry official like Cavazzoni or the newly named Office of New Drugs Director Peter Stein (a former Merck & Co. Inc. exec), the agency will need to be careful not to fuel criticism that it is replacing career FDA staff with new recruits from industry. 

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