Regulatory WOQUE-ness: Some Commentary on Patient Engagement
A recent commentary on patient engagement in drug development caught my eye.[1] Peter Pitts is a member of the Council for International Organizations of Medical Sciences (CIOMS) expert working group tasked with advancing patient engagement in drug development. His commentary comes after attending a 3-day meeting of the working group from which he had some insightful takeaways. And, while it is focused on patient engagement in drug development, his comments are broadly applicable across medical products, including devices. I thought some of his thoughts warranted further discussion here.
Pitts highlights the need for us to convert emotionally powerful anecdotes into clinically meaningful data that regulatory agencies can use. This is not a simple task. But, it is critically important if we are to make efficient and meaningful use of patient engagement with the medical products industry. It is not enough to host patient meetings if we are not going to obtain methodologically rigorous data that regulators can use. However, there are many barriers to obtaining these data for patients, advocates, and sponsors. Identifying these and addressing them is a pressing necessity.
One of the questions that Pitts raises is whether including the patient voice in medical product development is a “burden or a benefit”? This is an intriguing question. For patients and patient advocates, I believe they would consider inclusion of the patient voice as urgently critical and highly beneficial to all medical product development. But, would industry agree? Yes, we can obtain insightful benefits from engagement with patients, but is the burden of obtaining patient experience data[2] we can use too much? Having participated in the development and implementation of both qualitative and quantitative patient experience studies, I can attest to the fact that they are 1) costly, 2) time consuming, 3) methodologically complex and 4) of questionable ROI within the current regulatory framework. Pitts makes that point that executives and policy makers may see the high-level benefit of engaging patients, but does it make good business sense? It’s possible that the answer is no.
One of the most compelling points that Pitts makes is that there are institutional road blocks to taking patient engagement in medical product development into the main stream. I have experienced this first hand. As Pitts suggests, “enthusiasm is common, but commitment is rare.” This is particularly true in terms of those responsible for a sponsor’s regulatory strategy. The idea of including the patient voice in the regulatory process sounds obvious and appealing. But, when it comes to the day-to-day, regulatory managers are loath to give up their limited resource or alter their already ambitious timelines to incorporate patient experience. I’ve also come across regulatory managers who do not want to distract their FDA review teams from important questions of regulatory clearance or approval by introducing patient experience data that the review team might not find relevant or helpful. Similarly, review teams at FDA have a long way to go before we see consistent willingness to consider, much less rely upon, patient experience data in a regulatory submission for a device, drug or biologic. These roadblocks are real, the struggle is real, and it will take sophisticated change management to overcome these barriers to making patient engagement a ubiquitous reality in the medical products industry.
Pitts proposes a solution to some of these issues as a Wide-Open Quality Engagement (WOQUE) score. While somewhat tongue-in-cheek, he has a point. If we can create an ecosystem where the quality of patient engagement is measured, and the outcome of those measures matters, then we can move the needle toward greater inclusion of the patient voice across the total product life cycle. From a regulatory agency perspective, I believe some simple legislation in the next user fee cycle will go a long way to moving review teams towards more acceptance of patient experience data. I am proposing we include legislation that says, 1) FDA cannot require patient experience data, 2) if a sponsor takes the time and effort (and cost) to submit patient experience data that data MUST be considered by the review team, and 3) if the review team does not like the patient experience data or feels it cannot be considered relevant to the review decision, the review team has to tell the sponsor WHY. Simply putting these provisions into the user fee reauthorizations will help provide consistency and clarity to industry and FDA about the use of patient experience data in regulatory submissions.
This is a long-winded way of saying that I agree with Peter Pitts. We are a long way from making patients equal partners in medical product development. But, there are ways to make this happen such that the patient voice is heard throughout the product life cycle in the most impactful way possible.
[1] Peter J. Pitts. Towards Meaningful Engagement for the Patient Voice. The Patient – Patient Centered Outcomes Research. June 5, 2019
[2] For the purposes of this blog post, patient experience data is defined as all patient data including patient perspectives, preferences, experiences, reported outcomes and other data that is obtained directly from patients related to living with their disease and its treatment.