A panel of regulatory experts convened at DeviceTalks Minnesota this year to chat with MassDevice.com editor Sarah Faulkner. The group of regulatory experts included Danelle Miller, VP of regulatory policy & intelligence at Roche Diagnostics; Megan Brandt, VP of regulatory affairs and quality assurance at CHF Solutions (NSDQ:CHFS); and Mac McKeen, regulatory science fellow at Boston Scientific (NYSE:BSX).
Faulkner: We have a new president in the White House and a new FDA chief. What does a change in the guard mean for your job and what are some of the things you’ve noticed from Dr. Gottlieb’s first public statements?
Miller: Now that we have a commissioner who’s actually confirmed and in the office, the FDA can start working on its priorities and getting some things out that have been on hold for a number of months. I think perhaps, more importantly, is that what we see in Dr. Gottlieb is a shift in the approach, a very innovative, forward leaning approach in terms of how can we get products to market that are safe and effective. We’re finding a much more open agency to talk about creating regulatory pathways.
Brandt: One thing I’ll be curious to see how it pans out in terms of initial public statements related to the safety and effectiveness early in the development process is really, how that is going to affect performance goals. From an industry perspective there’s been a lot of chat about, “How do we get devices to market faster?” I think, understanding the synergies between his initial public comments and how that will unfold in reality will be really interesting to watch as the months and years proceed.
McKeen: I think Doctor Gottlieb is a, maybe ideal candidate is an overstatement, but the fact that he was a leader within FDA and is now coming back. He’s got the insights as to how the agency functions. As with any new commissioner, there’s always big issues on the horizon. He’s come out and said he wants to solve the opioid crisis. He, like President Trump, thinks drugs cost too much so we’ll see if they can have any influence there. I don’t know that the DA has any real influence there but I think the safety and efficacy data will be a part of that.
He also did a nice piece on digital health and that’s refreshing that he is enlightened and that he wants to lean forward with the agency and really make things more efficient.
Faulkner: Are you guys seeing an attempt to harmonize regulations across countries?
Brandt: There is a body called the IMDRF – the International Medical Devices Regulatory Forum. They are meeting frequently to try to standardize the different regulations from a global perspective. You can hardly keep up with the global regulatory landscape because weekly there’s new things that are being published.
Miller: What we’re dealing with is not just the potential global convergence, which I think is something that’s actually more positive where you can see more regulation from countries, but I think we’ve got different legislative paradigms. I’ll give you an example: TGA, which is the Australian [inaudible 00:12:51] recently issued a draft saying that they would consider accepting overseas authorizations by other countries, essentially, that are of a similar nature. That’s a positive because that means that if they trust another country to do an authorization, they can accept that or do a group review that’s going to get those products to their patients faster.
Faulkner: Clearly the global regulatory landscape is a little overwhelming. Even for regulatory policy experts. What can companies do to best prepare themselves for what seems to be a constantly changing landscape?
Brandt: From a smaller company perspective, there’s a couple of things. First is just to stay informed. To just be aware of what is changing, to kind of have it in the back of mind as you’re developing your strategy. Another thing more from a tactical perspective, is to change over a lot of our documentation to the STUD format, which is the globally harmonized regulatory format for small organizations.
Then, lastly, I think is don’t be afraid to ask for help. Reach out to the respective folks in the geographies that you’re going after. You know, my last organization we actually were pursuing approval in Japan.
We actually came across a program called Harmonization by Doing, which was a partnership between FDA and Japan. It brought in industry, academia, and then both PMDA and FDA from a regulatory perspective. Being part of that group we were actually able to “fast track” both our clinical study and our subsequent regulatory applications in parallel in both the US and in Japan. Just doing a bit more digging and checking all the different options that you have available to you are some of the key things to do as things continue to evolve.
Miller: Regulatory intelligence – it’s a buzz word, but it’s absolutely critical. We have to know what’s going on in various countries.
We have to look about five plus years out. We look at our pipeline and look at, “What is the regulatory paradigm?”
McKeen: You have to consider the unique requirements that you have for those markets outside of CE Mark and FDA. Address that early on and budget and have that at the ready as you begin to do your submission work. It’s working with people and the process. Building relationships with the regulatory authority, whether it’s FDA or notified body.
This is a data driven process. Sure, relationships matter – you don’t want to lie to these agencies because that is going to create a problem for you downstream, but you need to understand the requirements. Whether it’s a startup, mid-size or large company, to navigate regulatory you need to be well read and understand the requirements.
I think in the last five years the FDA CDRH has been a part of or published over 150 guidance documents. It’s almost a weekly event. In some cases more, right? We have whole staff dedicated to [guidance documents] at Boston Scientific.
Faulkner: The medical world is changing thanks to innovations in digital health. How has the FDA responded to a rapidly-evolving medical practice?
McKeen: Digital health is the buzz right now. The FDA really leaned forward, and I like to recognize them and applaud the efforts of Bakul Patel who’s really heading up the digital mobile apps part of FDA.
In 2015 they published the mobile medical apps guidance document that was good and bad.
There was this visual of a triangle in there and the top part was, this is a medical device if your digital health app was telling you to go to doctor or take your insulin. In the bottom part of the pyramid was health and fitness – Fit Bits and things of that nature. But then 80% of this pyramid was enforcement discretion. That’s kind of scary when the FDA says, “enforcement discretion.” It’s a big gray space and now the FDA is further defining that.
I think it was very valuable to the industry and to developers to have that first cut at the guidance and as more guidance comes out, this enforcement discretion is becoming less mysterious.
Brandt: I think it’s going to be something that continues to evolve. Especially with innovation, because it’s going to be new things that the agency isn’t used to seeing. As the first company goes in with their submission, there’s likely going to be a steep learning curve but hopefully, on the back end it’ll come out better for the rest of the industry. I think, one example is the 23andMe approval. That really set a precedent and the FDA’s reduced that footprint for consumer genetics.
Miller: 23andMe was absolutely groundbreaking. They actually looked at 10 different diseases and conditions that were all cleared at the same time. They made it clear that 23andMe would not have to come back with any additional diseases and conditions unless they use the intended use that is for diagnosis.
McKeen: It’s similar to TSA pre-check. This first approval was under a 510K and so long as you stay in good compliance with the regs and apply your special controls, you can expand your label following these rules.
They’ve developed a road map and pathway for industry to follow. I think that’s the first and not the last.
Faulkner: What are you guys keeping an eye on in terms of regulatory policy?
Miller: We are looking for movement on 21st Century cures. We were very pleased with some of the changes that were made there with respect to accessories, with respect to digital health and a number of other things. From our perspective though, we’re really looking at: what is FDA going to do with respect to global data?
Also the health IT, digital health space is very important to see where that goes. From the diagnostic perspective, there is a bill floating in the House right now. The Energy and Commerce committee has released a draft that would treat diagnostics as diagnostics and not as devices. Those are probably the three primaries that we’re really watching this year.
Brandt: One of my biggest focuses is on the real world evidence. I think there’s a lot of good work that’s going on right now to help really bring that forward.
I’m really hopeful that, that will gain some traction because I think it’s going to be a much better way for us to harness all the data that we’re currently collecting with electronic health record. I think being able to work smarter with the data that we’re capturing is something that’s going to be really important as we look to the future.
Faulkner: What are your best practices for companies to sustain good regulatory standing with the FDA and if you’re a young startup, how do you get your foot through the door at the FDA?
McKeen: I think pre-submission meetings are a very effective tool. I had one last month, I have one next month, and then we have one in September. I get my international colleagues tell me that they’re jealous, sometimes, of how FDA has all these tools to engage and interact whereas they don’t.
Brandt: Getting in front of FDA early is going to be very important so that they know who you are and what you’re trying to accomplish. Pre-submission is the perfect vehicle for that and the important thing is that you go into those meetings prepared. Don’t go in and ask FDA what you should do – go in with a plan and say, “This is what we’re proposing to do and why.”
Miller: One of the things that I think we all agree to is to meet early. You can do an information meeting and say, “This is where we’re going in the future. These are what we think the challenges will be. These are some of the types of solutions that we see. Can we collaborate with you to try and get there.”
Don’t be afraid to go out there and share your ideas.
This conversation has been edited and condensed for length.