Meet the Expert: Carrie M. Kuehn, MA, MPH, LPD, RAC

Principal Consultant & Founder, Evergreen Strategic Consulting LLC

Carrie M. Kuehn, MA, MPH is the founder and principal consultant of Evergreen Strategic Consulting LLC. In May 2019, she spoke at the 10x Medical Device Conference on the topic of FDA’s Patient Preference Initiative – what does it mean for the medical device industry? FDA guidance defines Patient Preference as “qualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes that differ among alternative health interventions.” We spoke with Carrie several months ago about what this is and why device companies should care.

Can you give us a brief overview of your professional background prompting this specialized focus?

My background is in public health with degrees in anthropology and epidemiology. After getting my master’s degrees, I worked at the University of Washington in observational research, primarily in rehab medicine. I then had the opportunity to go into industry at Amgen in their pharmacoepidemiology group. From there, I went into consulting at Exponent for nearly ten years where I immediately started working on medical device work as a project manager. That introduced me to the whole FDA regulatory environment, particularly medical device regulatory.

In 2015, I went back to school as a 40-year-old single mom to get a doctorate at Northeastern University College of Professional Studies. My doctoral research focused on how the FDA got patient data into their regulatory decision-making. That really opened up the entire area of regulatory policy and patient engagement to me, which at the time was pretty new. FDA wasn’t really mandated to engage the patient until the 2012 when the Food and Drug Administration Safety and Innovation Act (FDASIA) came out. At the time, I was working on my dissertation and there was a lot happening. I talked to some amazing people at the Agency and really got interested in the whole area. I finished my doctorate research in 2017 and then worked in patient engagement at a medical device company, which gave me the opportunity to manage patient preference studies as well as engage with policy advocacy groups on behalf of industry. It was a good, in-depth experience from an industry perspective–the idea of getting patient engagement and getting the patient data into regulatory policy. Now I’m on my own and still working on it.

How would you describe FDA’s Patient Preference initiative?

For the purpose of medical devices, which are my focus, the Patient Preference Initiative is specific to Center for Devices and Radiological Health (CDRH). It’s their implementation of legislative changes that have happened since 2012 when FDASIA was passed that empower them to include the patient voice in regulatory decision-making.

CDRH’s reference to “patient preference” is unfortunate because it implies something way more simplistic than what it actually means. Patient preference is not about patient satisfaction. What CDRH is really focused on is patient benefit-risk tolerance. Patients provide a unique voice and perspective about living with their medical conditions. This initiative is a systematic way to elicit, measure and incorporate patient preferences through the total product life cycle of a device. The idea is to use patient preference studies to gain understanding of the benefit-risk trade offs that patients are willing to make to gain access to a new device.

We’re talking about things like willingness to tolerate risk giving the benefits of a new treatment; priorities for treatment characteristics; ranking of treatment outcomes; willingness to pay; and the amount of lifetime patients are willing to trade off to receive treatment. FDA created an Infographic that explains the initiative through the total product lifecycle.

The trajectory of where we are today perhaps started with the the founding of HIV patient advocacy groups. Is this initiative being driven by life-threatening diseases or chronically ill patients? Are we talking about risk benefit because these patients are going to endure the most risk to achieve the most benefit?

You’re on point. It’s more applicable though in the biologic and drug sections as far as patient policies—what is called patient-focused drug development. In doing so, twenty different disease areas were picked in which to host meetings and get patient feedback, which is a completely different approach than what CDRH has done.

Patient engagement with FDA really came from the HIV activists in the late 80s. They’d had enough. They were fed up with lack of access to potentially life-saving treatment and didn’t think the existing clinical trial paradigm was going to save the lives that needed saving. What they did was incredibly important. They became very smart about clinical trials and regulatory frameworks, the statistics, recruitment, randomized trials, and they got very conversant about all these things. Activists came to the table with regulators and were able to speak their language and negotiate with them on equal footing. It was groundbreaking and led to significant changes in investigational drug laws, the way clinical trials were conducted, and new drug applications – which fundamentally changed regulatory policy.

Shouldn’t the FDA already be talking to their stakeholders—the patients?

Yes, it seems so obvious. I think what regulators, industry and the public forget is that FDA operates as an administrative agency overseen by Congress. What is forgotten is that FDA has to operate within the guidelines of their congressional mandate. What needs to stay uppermost is that if we want FDA changes, there needs to be legislative changes as well. That’s why FDASIA is so important as well as user fee reauthorization acts, and 21st Century Cares Act. All of those legislative actions essentially enabled FDA to give a greater voice to patients.

Were legislative changes driven by other group activists or did society start to recognize the need for more patient engagement in healthcare?

I am most familiar with the 21st Century Cares Act because of my doctorial research. If you look at the supporters, Fred Upton was the congressional chair and champion for that act. Supporters came from across-the-board: patient groups, the National Health Council, and AdvaMed. These advocacy groups supported this act, enabling FDA to give a greater voice to patients. Some patient groups are bigger and more sophisticated than others. There’s the Michael J Fox Foundation, which is a highly sophisticated, large group representing Parkinson’s patients. Then there are smaller “mom and pop” groups that connect over Facebook. They tend to funnel up to organizations such as NORD – National Organization of Rare Diseases – that advocates on behalf of these small groups. It’s a whole process.

Looking at FDA Infographic, the initiative seems to involve comprehensive patient monitoring throughout the product lifecycle. That is arguably costly and potentially complicated. Is this actually happening?

The FDA’s ideological target for patient engagement is to engage the patient throughout the total life cycle. I don’t think it’s happening enough though. There are logistical and financial barriers for industry to fully implement this type of engagement in their product lifecycles. It’s unclear how to fully overcome those barriers.

Can you talk more about these barriers?

There are definitely problems with legal and regulatory costs, paying patients for their time, potentially running afoul of anti-kickback statutes, and advertising and promotion laws. Patient preference studies can be very expensive depending on the specific disease area. They are not simple to do and require a certain level of expertise. Companies are concerned about the numbers necessary for a study, how much data is needed and what will be the cost. Medical device companies lack the budget for this kind of expense compared to pharma. Time is also a factor because regulatory agencies expect adherence to the specified timeline. The biggest issue for medical device companies is that there is no proof that patient engagement practices are going to help.

Why would medical device companies go through patient engagement unless it was for a risky device that needed patient preference data to get it through regulatory?

Exactly. What is the ROI? Unfortunately, at this time, if we submit these data to FDA as part of a regulatory submission, there is no requirement that FDA look at it. They can consider it and are encouraged to do so, but if they don’t look at it, they don’t need to give a reason for not doing so. To spend $200,000 or more on a risk-benefit study with no guarantee that the FDA will consider it in its regulatory review is a real problem.

If correctly implemented, this initiative seems to bode well for the patients, who are the ultimate consumers of the therapies. What if this new data is not understood? What in your mind needs to happen for this to start gaining some traction?

The Medical Device Innovation Consortium (MDIC) can help with best practices, but most importantly, I think there are provisions that could go in the next user fee reauthorization legislation that would help. For example, the provisions could state that the FDA can’t require patient preference data but if it is submitted, the FDA has to consider it in the review and provide reasons provided if they don’t like the data. This would give industry comfort knowing that if they go through the process and costs to get this patient data, then at least the FDA is going to look at it, and provide feedback to the sponsor if they don’t like it and why. Right now, there is no leverage.

It sounds like what’s needed is more refinement via legislation.

Yes! There are small device companies who could benefit from this data – even simple data. You have a company that could benefit from just asking patients what their preferred clinical outcomes are and use it as input into the company’s first IND. Until then, there’s going to be limited uptake by industry because they don’t have to do it, and there’s no guarantee that if they put it in the effort to do it that it will get considered. The only benefit then is that patient input was included.