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Applying regulatory experience in the technology space: Q&A with Lowell Schiller, Chief Legal and Regulatory Officer at Aetion



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Lowell Schiller, J.D., recently joined Aetion as Chief Legal and Regulatory Officer, building on a career of working on policies related to real-world evidence (RWE) and accelerating innovation at the U.S. Food and Drug Administration (FDA) and on Capitol Hill. 

He most recently served as the FDA’s Principal Associate Commissioner for Policy. In this role, Lowell led the FDA’s Office of Policy, which is responsible for agency-wide development of regulations and guidance, and provided leadership on issues across the FDA’s jurisdiction. 

Prior to that, Lowell served as the FDA’s Acting Chief Counsel and as the Senior Counselor to the FDA Commissioner. Before his time at the FDA, he worked on the Senate Health, Education, Labor, and Pensions Committee, where, among other things, he contributed to developing the 21st Century Cures Act. 

Now at Aetion, Lowell looks forward to continuing to advance important goals like accelerating innovation and helping deliver cutting-edge advancements to patients. Read on as he discusses how the FDA’s stance on RWE has evolved over time, shares his thoughts about the agency’s future direction, and explains how science and policy must innovate together to advance RWE.

Responses have been edited for clarity and length. 

Q: What attracted you to the RWE and technology space?
A: I’ve long believed that one of the keys to innovation in medical product development—and in the broader health care ecosystem—is in better harnessing the power of real-world data. There is so much diffuse information about how products are used, about the impacts of different diseases and conditions, and about different possible interventions. Leveraged the right way, these data can help us figure out how to get innovative, safe, and effective treatments to the right patients faster, while broadening access and reducing burden on the health care system.

To achieve this, we must navigate a range of cutting-edge issues—scientific, technical, legal, data privacy, and others. But these are issues we need to navigate; there is so much promise in real-world data, and I am delighted to work alongside the Aetion team to explore and solve these challenges. 

Q: How has the FDA’s stance on RWE evolved over time?
A: FDA clearly recognizes the power of real-world evidence, and the agency has been working hard to ensure that the regulatory framework keeps pace with innovation. On the surveillance side, FDA’s been using a variety of real-world data for quite some time—for example, through the Sentinel System for monitoring medical product safety. We’re also seeing real-world evidence play an increasingly important role beyond safety surveillance, including in product approvals, such as to improve the efficiency of clinical studies or to supplement clinical trial data.

In the future, I think we’re going to see an increased reliance on real-world evidence in regulatory submissions. FDA has been working hard to prepare for, and hopefully accelerate, that reality. For example, FDA published a thoughtful framework on real-world evidence and put out draft guidance documents on key topics, including the use of real-world evidence in regulatory submissions. These actions are responsive to mandates like those of the 21st Century Cures Act, but also I think they reflect a recognition within the agency of the increasing importance of real-world evidence and the need to make regulatory expectations as clear as possible, as early as possible, to help inform strong development programs.

FDA has also been doing a lot of work to modernize the agency’s IT infrastructure. This anticipates increasingly sophisticated uses of large data, in regulatory submissions and otherwise. It is critical work that will pay real dividends going forward. 

Q: What can industry expect to learn from the FDA’s coming RWE-related guidance?
A: I think we’ll continue to see robust guidance development in this space. Developing guidance on RWE has been a priority for the agency, and I expect that it will continue to be a priority going forward. As FDA gains more experience and sees more submissions that rely on real-world evidence in more ways, agency staff will hopefully be able to leverage this experience to provide more detailed recommendations. I’d hope to see, for example, more in-depth discussion of statistical and methodological considerations, and more about what would make data more or less fit for purpose in various scenarios. 

Bear in mind that these are the kinds of recommendations that come from programmatic experience. That’s not developed overnight; it requires a sustained partnership and cooperation between government and non-government stakeholders. 

Q:What do you expect to see in the future in terms of regulatory policy and standards around RWE?
A: First of all, I expect we’re going to see a lot of activity, to meet statutory mandates and otherwise. I also expect that future policy development is going to draw on lessons learned thus far. For example, the COVID-19 pandemic has brought new urgency to the need to learn about what’s happening in real-world settings in terms of disease progression and impacts of diagnostics and treatments on different populations. Aetion has been doing great work partnering with FDA in this effort. I expect that FDA and other regulators will be able to draw on this experience in thinking about real-world evidence in the COVID-19 setting and beyond. Because ultimately, whether you’re dealing with a pandemic or a rare disease, or something else, we want to be able to harness the best tools and use them to drive innovation and help to ensure timely patient access.

I also expect that future policy development will draw on ongoing work, like the RCT DUPLICATE project, to help inform how RWE can support regulatory reviews and potentially offer insights that historically have required large randomized controlled trials.

Q: What key learnings have you gained from the FDA’s stakeholders in public forums, like public comments, with regard to RWE?
A: One thing you learn from listening to public commentary in this space is that there’s lots of excitement around potential uses for real-world evidence. This has generated a lot of buzz, which is great, but it also carries certain challenges. For example, it’s important not to get carried away by the excitement and start applying the label of “real-world evidence” to things that don’t meet that definition. As more stakeholders talk about real-world evidence, and ultimately seek to rely on it, it’s important to make sure that we’re aligned on definitions with a clear understanding of what real-world evidence is, what it isn’t, and what regulatory-grade real-world evidence looks like. 

Another thing I’ve seen is a great hunger for in-depth guidance from regulators. I think that kind of regulatory guidance, and clearer rules of the road, will be incredibly helpful. However, it’s important to bear in mind that in areas of rapid innovation such as the real-world evidence space, there’s a risk that if the agency provides recommendations that are too detailed too early on, before the agency has enough experience, the recommendations might not be a great fit and could end up stifling innovation. So there has to be a balance. 

Q: What advice would you give to biopharma organizations as they use RWE in regulatory submissions?
A: First of all, review the available guidance closely, and make sure you have a clear and detailed understanding of what regulatory-grade real-world evidence looks like, including what it would look like for the submission at hand, which will differ depending on the development program. 

Also, biopharma should try to leverage available opportunities to communicate with the agency relatively early in the development program, if possible. Not everything that you need to consider is spelled out in guidance, and novel questions abound in this space. If there is an early opportunity to consult with a regulator, that insight can be critical. 

Q: In your opinion, what will it take to advance RWE? Does policy need to catch up with science, or science with policy?
A: It’s both, they need to evolve together. Good regulatory policy is informed by experience. When that experience is lacking, you run the risk that the policy won’t hold up in practice, or could even be a hindrance. When it comes to policies relating to real-world evidence, particularly in regulatory submissions, that experience is gained by reviewing regulatory submissions. I think regulators understand that this effort needs to be a dialogue and a partnership. It doesn’t work to wait for a critical mass of submissions before providing meaningful guidance, because having meaningful guidance in place is going to be critical to help spur investment in developing quality regulatory submissions. The science and the policy need to move together and push each other forward, hopefully in close partnership and communication.

Q: Now working in the technology space, which challenges are you most eager to tackle?
A: I’m excited to continue to help advance many of the same goals as I did in previous roles, just from a somewhat different perspective. I think there’s a shared set of goals among public and private stakeholders in the RWE space, which includes leveraging cutting-edge science to help accelerate innovation and drive more efficient development programs, and ultimately to help patients access the right interventions. I look forward to continuing that work from a new perspective. 

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