Remarks by Dr. Amy Abernethy to the FDA/CMS Summit
Speech by
Amy Abernethy, MD, PhD.
Principal Deputy Commissioner - Office of the Commissioner
(Remarks as prepared for delivery)
Good morning.
I’m delighted to be with you for this year’s FDA/CMS Summit.
I thought I might focus my remarks today on several interrelated topics that have been of particular interest and concern to me, both during my tenure at the FDA, and throughout my career before coming to Washington.
This involves the importance of developing better, more sophisticated, and more efficient ways to use and build data and technology solutions to strengthen patient care and enhance the development of new treatments and cures. It involves both the innovation that is part of the development process and the process itself – making sure we have the data and technology systems and tools we need to support our scientific work.
As some of you may know, I was a clinician, as well as a professor of medicine at Duke Medical School, where I ran an organization called the Center for Learning Healthcare. In our work there we were very focused on these questions and closely studied ways that we could better organize data to strengthen health care opportunities.
In addition, I spent some time outside of the “ivory tower” of academia and joined a tech start up. I realized we needed to get some unconventional partners involved in these processes.
My immersion in these new worlds enabled me to gain expertise in some areas, that I never would have anticipated, such as learning the in’s and out’s of venture funding, business development, agile software product design, data lakes and pipelines, and advanced analytics.
But through it all – in both my academic work, my private sector work, and now at in my work in the public sector – there has always been one guiding star, one preeminent focus… the patient.
There’s an old saying that I’m sure some of you have heard, that “A good physician treats the disease, the great physician treats the patient who has the disease.” But today, we know that these two thoughts are not mutually exclusive, and in fact reinforce each other.
Our focus at the FDA is always to be learning from every patient. We want their knowledge to inform our work in the best possible ways.
There’s a simple reason for that -- no one understands the effects of a disease and the effectiveness (or for that matter, ineffectiveness) of a treatment more than the patient who is living with that disease.
Today, we have more options for treatments than ever, thanks in part to the promise of personalized medicine. But this only goes so far. It’s only by listening to patients that we can shape these treatments to unveil new and better solutions.
In short, to harness the full potential of new therapies to transform care, we need to be able to learn from every patient. The challenge is applying this growing amount of information in a way that effectively matches the right treatment with the right patient.
To do this, we must—and do--become more efficient, more collaborative, and more data-driven.
The irony is, we don’t lack for data. Everyone here has heard and reheard those supposed magic words “big data.”
But it’s not just getting or having data that poses the challenge. The focus must be on how to make this data available and useful … how to integrate the increasing wealth of available data into effective regulatory decision-making.
The data needs to be of better quality and we need the ability to mine it in new ways, using the most modern analytical and technological capabilities.
We need to know what types of data are best for the application we’re working on, and which are the most efficient, cost-effective, and respectful of a patient’s privacy and other needs.
By doing this, we can effectively use the data to connect cutting-edge scientific discoveries to the real-world products and solutions that make a meaningful difference in people’s lives.
I learned first-hand the importance of having the right kind of health care data for a particular patient and the challenges that can involve.
Her name was Janet, and she was an emergency room nurse, so she already had a pretty good understanding of the challenges we faced in fighting her disease. When she came in we worked together to try and figure out the best treatment. As she told me, “take a piece of my tumor and map it out.” She also enrolled in a new clinical trial, but soon after, received a call from the company with the disappointing news that trial had been stopped.
Like any smart, industrious, and curious person searching for a cure, Janet asked the company for her data. But they refused, saying that they “owned it.”
But Janet wouldn’t give up. She asked, “shouldn’t the patients be the ones to drive the conversation? Shouldn’t they have access to their information?”
Through her treatment, we started to reframe the broader issue of how information gets out there – and worked to develop a mechanism that allowed us to aggregate and share it.
At the academic setting, my team engaged in a number of efforts to support this approach. We studied point-of-care data collection solutions embedded in Electronic Health Records or on a patient’s computer, and the quality of data routinely collected in the clinic and methods to improve data quality. We learned, for instance, how to harness these datasets for clinical trials, participant recruitment, new statistical analyses, and healthcare quality monitoring. We organized datasets anchored by longitudinal patient journeys and patient-defined outcomes.
Our goal was to test the different types of components needed to build a successful patient-centered learning healthcare system. And in treating Janet, I learned how to be better equipped to find and use this information.
Given the focus of my work in academia and in the tech industry, it seemed a natural next step for me to come to the FDA, and to support the critical work being done here in this area.
As an agency where every meaningful decision is premised on and reinforced by the best available science, the FDA plays an essential and unique role in helping chart the course for nothing less than the future of medical science.
Our agency serves as both a compass and, to some degree, a barometer (and, as long as we’re talking about measuring, maybe even a speedometer) for the medical and scientific community and the progress we can and should be making.
But the FDA not only sets the direction and charts the course for scientific innovation, it also helps ensure, through its focus on regulatory science, that this journey will be as orderly and responsive as possible.
To put it simply, regulations are the guideposts for industry. By clarifying what to do – and what not to do -- regulations help keep us from getting distracted and allow us to focus our energy on the most effective tools and methods where the impact will be felt the most.
But more than just setting the stage or making (and enforcing) rules, more than just approving or not approving new treatments, the FDA’s unique and vital role – and the core of our regulatory decision-making -- is designed to support and meet the Agency’s mission to protect and promote the health of the American people, to generate support for the development of products and tools that provide real world answers and meet the needs of individual consumers and patients. In doing this, we serve as a gateway for scientific promise.
We do this by continuing to speed development of effective therapeutics through the promotion of innovative clinical trial designs such as platform trials, basket studies, adaptive trials, and pragmatic randomized controlled trials.
We do this though our use of expedited programs, such as Fast Track and Breakthrough, Accelerated Approval, and Priority Review, which were established to help develop and speed the review of drugs, biologics, and devices that treat serious conditions and fill unmet medical needs.
And we do this while balancing timely patient access with the FDA’s high standards for safety and effectiveness; balancing the desire and need for innovation and for speedier development of new treatments with the need to meet required standards for the safety and efficacy of those treatments … what has come to be known as “the FDA gold standard.”
But at the center of this work and at the core of our ability to make these types of informed evaluations – is the effective generation and application of necessary data, anchored by the patient’s needs and patient-defined outcomes.
We depend on high-quality data to support scientific innovation and fulfill our unique role to help scientists and developers turn their vision for scientific advancement into reality for patients and consumers, and to make sure the products that are being developed for patients are safe and effective for their intended use.
It’s why we work with researchers to build linkages between complex and diverse data sets, to aggregate data across institutional boundaries; to use novel analytical approaches, and (as I said in the beginning) to harness real world data.
To give you just one example, our Center for Devices and Radiologic Health (CDRH) has been working through a public/private partnership to develop the National Evaluation System for Health Technology (or NEST) that merges several disparate sets of health systems data to allow for studies of device safety and efficacy in real-world use.
But advances in information technology and data will not reach their potential without a modern FDA ¬ an FDA with a modernized technical infrastructure and new ways of analyzing diverse streams of data and of harnessing the human expertise of our regulatory scientists and physicians.. This infrastructure and expertise will give FDA the foundation to support and evaluate work in precision medicine, genome sequencing; biomanufacturing, microbial genomics, in silico imaging for clinical trials, and so many other areas that were once deemed futuristic, but today are the reality of medical research and practice.
In short, to operate at world class levels, we must stay ahead of the science. And in this day of rapid scientific advances, that can be a challenge.
We live in a time of extraordinary scientific progress – when our growing understanding of the underlying biology of disease offers us enormous, transformational opportunities. As scientists, doctors, and regulators, we regularly witness unprecedented innovation and scientific breakthroughs.
One way that FDA is working to ensure full participation in rapidly evolving technologies, and that I have been very involved with, is our recently announced Technology Modernization Action Plan (or in the world of FDA acronyms, TMAP). This is one of the areas I’ve been particularly focused on as FDA’s Principal Deputy Commissioner and Acting Chief Information Officer.
TMAP is an important first step towards modernizing FDA’s approach to using technology for its regulatory mission, including the review of medical product applications.
Importantly, it helps provide a technological foundation for development of FDA’s ongoing strategy for the stewardship, security, quality control, analysis, and real-time use of data. This will allow us to smooth? the path to better therapeutic and diagnostic options for patients and clinical care providers, and better tools to enhance and promote public health.
The plan has three components:
First, modernization of FDA infrastructure to make sure we can support emerging uses of artificial intelligence, blockchain, and other technologies. By advancing our FDA infrastructure, we will be able to more quickly and effectively evaluate these proof-of-concept projects.
Second, use cases. There are three categories of use cases that are intended to be living examples so people can wrap their heads around what’s possible. These include things that are already happening, for example, precision medicine, our PREDICT system, and machine learning to update to a smarter system and approach.
A good example of an early success story in FDA’s modernization is a recently published draft guidance outlining the FDA’s intent to implement a digital framework for the electronic submission, review, and tracking of certain investigational new drug (IND) safety reports in FAERS. In a constrained environment of the 7- and 15- day safety reporting process, it attacks a simple problem with a simple tech fix.
Rather than submitting safety reporting data with inflexible PDFs, which cannot be manipulated and take a significant amount of FDA staff effort and time, submitting data via the FAERS system will enable us to more nimbly manage, visualize, and analyze the data. This results in a lower burden on industry, increased safety of patients in clinical trials, and reduced workload on FDA staff.
The third component of the plan is improved communication with the data and tech industry. Public engagement is critical, and we will be seeking input from the public on the deployment of modern technology and data strategies at FDA, and also how we can be part of a larger technology community that understands where FDA is going.
To enable this “learning by doing”, FDA has created a new, forward-looking project called CoInnovate@FDA. Designed to be an incubator of new ideas and a means of building future technical and organization capacity for smart data, CoInnovate@FDA will connect work from across FDA and with research and clinical work from across the health care system.
These new efforts are consistent with and help advance the goals of the 21st Century Cures Act to spur medical product innovation and ensure that patients get access to safe and effective treatments as quickly as possible. And they are an essential piece of FDA’s broader strategic policy.
With protecting and promoting the public health as our mission, one of our jobs as an Agency is to always ask the question: how can we perform our mission as effectively and efficiently as possible? The answer lies in FDA’s commitment to make regulatory decisions based on sound science and data. And we are constantly looking at better ways to collect and analyze the data that can help FDA make crucial and often time-sensitive decisions.
