What does FDA really think about Patient Preference Information (PPI)?

Let’s talk about FDA guidance. Most of us in the regulatory affairs field would agree that guidance documents from FDA are meant to reflect what FDA is thinking about a particular topic. Though they do not carry the weight of law or regulation, and every single one of them says the contents are non-binding, FDA guidance documents are treated as requirements imposed by FDA. At a minimum, these guidance documents are interpreted as FDA’s thinking with regard to how they want to see the actual regulations implemented by industry. 

Given that setup, let’s talk about FDA’s guidance document Patient Preference Information - Voluntary Submission, Review in Premarket Approval Applications, a Humanitarian Device exemption Applications, and De Novo Requests, and Inclusion in Decision Summaries and Device Labeling, Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders, affectionately known as the “PPI Guidance.” The original PPI guidance was disseminated in 2015 following a flurry of policy activity aimed at including the patient voice in regulatory decision-making. For the Center for Devices and Radiologic Health (CDRH) at FDA, the PPI guidance described the flagship program for patient input into medical device regulatory review. PPI was the means by which CDRH implemented the Science of Patient Input, and the PPI guidance was supposed to help industry navigate this new program. 

Fast forward 8 years, and it’s hard to see the positive impact the PPI Guidance had on regulatory decision-making at CDRH. Over this time period, only a handful of medical devices were cleared by CDRH using a patient preference study in the consideration of the risk-benefit profile of the device. Maybe this is why, earlier this year, CDRH requested public comments on proposed updates to the PPI guidance, which were due to the docket on June 6th. In their request for comments, CDRH suggested that five new questions be added to the guidance with answers from FDA to help guide industry. And while nearly all commenters agreed that these questions were helpful, most of us (your esteemed author submitted comments too) agreed that even more needed to be done to modify the guidance so the medical device industry can truly understand what FDA really thinks about PPI and how it can be used to support a device submission. 

I will get into the specific areas of commentary in a future post. But, the general idea that came across from the commenters was that we don’t know what FDA wants when it comes to PPI. The current guidance contains vague language about how the patient preference field is evolving and changing. It also mentions qualitative PPI along with very specific methods for quantitative PPI studies. But, experience has shown that FDA doesn’t like qualitative PPI data and won’t accept it to support a regulatory submission. Those of us who have worked in this field for the last few years have observed inconsistencies with what the guidance suggests and what FDA will accept. This has caused industry to falter with regard to submitting PPI with their device submissions. Medical device companies are either spending a lot of money on studies that FDA won’t accept, or they just aren’t doing the studies at all. The promise that the original PPI program offered simply hasn’t manifested itself. 

So, what can be done? We need FDA to tell us what they want from PPI studies in the updated guidance. We need specific direction on the types of data they will accept, sample size calculations, how they want the data presented and interpreted, whether highly rigorous quantitative experimental choice studies are the only thing that will be accepted, and what do we do if conducting such a study is prohibitively expensive (as many of them are for the medical device industry). We need less cheerleading about a cool idea for generating patient input, and more specificity about what FDA’s thinking is when it comes to PPI in support of medical devices submissions. If FDA can give us more transparency into what they want, I believe the device industry will be better equipped to provide meaningful PPI data that represents patients’ preferences and meets the needs of regulators.