Q&A: Outgoing FDA drugs director Jenkins talks 'real world' data, Cures

By Sarah Chacko

After 25 years with the Food and Drug Administration, John Jenkins, director of the Office of New Drugs, retired Friday.

His tenure spans from starting out as a medical officer in the Division of Oncology and Pulmonary Drug Products in 1992 to his appointment in his most recent position 10 years later.

The Pink Sheet presented Jenkins with a parting portrait — formed from a word cloud using the headlines from their coverage of him over the year — at the FDA/CMS Summit last month. “You often loomed large in our story meetings,” said editor M. Nielsen Hobbs.

On his way out, Jenkins shared with The Hill Extra his thoughts on the regulatory road ahead for FDA, patient involvement in drug development, and how the agency can maintain its “gold standard.”

He said he was not worried about some safety standard fears raised about the 21st Century Cures act and talked about the agency's current use of so-called "real world" data.

Q: Looking back on your 25 years at the FDA, is there one accomplishment of which you are most proud?

Jenkins: When I started, in 1992, was the year that the first Prescription Drug User Fee Act was passed. That was passed in response to concerns about the FDA falling behind, compared to other regulatory agencies in the world. ... Twenty-five years later, we now lead the world in first approval of new novel medications. Last year, 83 percent of the novel medications we approved, we approved first in the United States. 

That’s been the most important public health turnaround as far as the performance of the agency. We went from being kind of a laggard in performance to being what I view as the gold standard for the rest of the world to look up to for regulatory review of new drugs and approval of new drugs. ... We’ve done it in a very balanced manner of improving our efficiency, improving our science, while maintaining standards and balance between faster approvals and safety.

Q: The new 21st Century Cures law encourages FDA to expand its use of “real world evidence” in its review of drugs and medical devices. How do you respond to concerns over the dependability of that data and its impact on safety?

Jenkins: I don’t share those fears. I don’t read the new legislation in that light. I read it as encouraging the agency to make use of the newly emerging data from electronic health records and large managed care databases to seek out high-quality data that can serve as the basis for regulatory action. I don’t see it as lowering the standards. 

In fact, in several places in the Cures legislation, Congress was explicit in stating that they weren’t lowering the standards. They were offering the agency new tools to use even greater flexibility and to modernize how we look for data to support that drugs are safe and effective.

If the provisions are implemented appropriately, it will move us into a new era of expanding the range of ways that we can collect data on the use of drugs to make regulatory decisions.

Q: How far along is the FDA in ensuring the "real world" data they collect is accurate and useful?

Jenkins: That’s very much a work in progress. These new data sources have great potential, but we also need to work through how to ensure that the quality of the data is high, that the data are reliable. ... It doesn’t mean we’re just going to take whatever we get. We’re still going to demand that it meet rigorous standards so it’s reliable and interpretable. It takes some time. 

There [are] some commitments that the agency’s made in the new user fee agreements that will be discussed in Congress this year that make clear that we need to go through a program to understand the sources of the data, their reliability, their accuracy, and how to use this data.

Keep in mind, we already use real world data ... primarily for drug safety issues. But now we’re seeing the potential to expand into using this data as a way to demonstrate the effectiveness of drugs.

It’s a journey. We’re at the beginning of that journey. We shouldn’t assume that we can jump to the end overnight and not need randomized controlled clinical trials anymore.

Q: How has the role of patient groups evolved in your decades at the agency? Do they offer helpful advice or added pressure?

Jenkins: It’s overall a positive for the drug development process. There’s a tremendous amount of work and collaboration that can occur between the agency and patient advocacy groups on better understanding the natural history of disease, the impacts they have on patients. ... Clearly, they are the experts on their disease.

What needs work over time is how to incorporate that information into the regulatory process. ... You have to think of ways to collectively look at that information and make use of it.

The place where we’re going to face continued struggles is in the area of advocacy for a specific drug. We’ve seen that, for example, with flibanserin, we saw that with eteplirsen, where the patients become very strong advocates for a particular drug and, of course, we have to make sure that the drug approval process meets the statutory standards.

Q: Does that mean the agency will look for more opportunities to get drugs into patient hands faster and rely more on post-market surveillance to ensure effectiveness?

Jenkins: Patient input has been very valuable to the agency for a long time, going all the way back to the AIDS crisis in the 1980s that led to the accelerated approval program in the early ‘90s. That was an example of the agency being responsive to public health issues and changing societal expectations.

As we’re learning more about these diseases, particularly the rare diseases, it is going to open up more opportunity for accelerated approval for some of those situations. That’s a good thing. And that will mean there will need to be work after approval to confirm clinical benefit, but I don’t see that as lowering the standards for approval. I see that as being flexible and responding to serious unmet medical needs.

Another area where the agency has responded to patient needs for access to drugs is our expanded access program for investigational drugs. We recognize that some patients with serious life-threatening diseases need access to investigational drugs before they’ve meet the standard for approval. 

The agency has a history of responding to these changing societal expectations, using the flexibility we have under the law, and working with patient groups to try to get to the right outcome while maintaining our standards for approval.

Q: Cures included provisions to improve the hiring process by removing some of the problematic qualification and pay barriers. Will that be enough to address chronic staffing shortages at the agency?

Jenkins: To me personally, that’s one of the more exciting aspects of the Cures legislation — the possibility to make FDA more attractive for young scientists and the best and the brightest in their field to work in government and contribute to the public health. That’s why we work here.

But unfortunately, historically, the agency had been at a severe disadvantage, not only in the hiring process but also in the levels of compensation that can be offered to recruit and retain those highly trained staff we need to do our work. I’m hoping that the new administration will make use of those new authorities under Cures to basically restructure the FDA pay scale to make us much more competitive with the private sector.

Q: So why retire now?

Jenkins: This is not something I decided recently. This is something I’ve been contemplating for several years.

I had originally planned to retire in the summer of 2015. That’s when I first became fully eligible for retirement. But at that time, the agency was just getting ready to start the next round of user fee negotiations. I had been the agency lead for the premarket aspects of the Prescription Drug User Fee program for PDUFA III, IV and V, and I didn’t really see that we had someone ready to step in and take that role at that time.

I then decided to postpone it again because ... we had some vacancies that we needed to fill, and I didn’t feel like I was leaving the organization on a good footing. Now the organization is on a solid footing as far as staffing for senior positions. It feels more comfortable now that I’m leaving the place in good hands.

And I’ll be 60 this summer. For me, I still have five or 10 working years left before I decide to fully retire to pursue other opportunities and try new things.

Q: In a blog post summing up the past year’s new drug approvals, you wrote, “While I am leaving FDA, FDA will not leave me; its principles and high standards will help to guide me in my future work.” What will your future work entail?

Jenkins: I’ve had some conversations with potential opportunities after I leave FDA, but they’ll become more serious after Friday. I don’t know exactly yet what it’s going to be. It’s probably going to be very similar work. But the principles that guided me while I’ve been at FDA, as far as public health service, public health commitment, will guide my decision making on what position I may eventually take in the private sector.

Q: As they look for a new director for the Office of New Drugs and potentially a new FDA commissioner, do you have any parting advice for the next administration?

Jenkins: The track record for the FDA is very strong in the new drugs program. We are the gold standard for the world. Hopefully the new administration will recognize that and take action to continue our leadership in that arena.

This interview has been edited for brevity and clarity.

To subscribe to The Hill Extra, please visit extra.thehill.com or email Jessica Falborn at jfalborn@thehill.com.