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Michael Christel is Pharmaceutical Executive and Applied Clinical Trials' Managing Editor. He can be reached at email@example.com.
DIA’s global chief executive talks with Pharm Exec about the current trajectory of patient-centric product development and the need to establish common pathways and metrics to best harness its benefits in everyday practice.
The concept of patient-centricity may have moved beyond buzzword status, but its exact design and application in healthcare and-more so-R&D remains difficult to map out and assess.
To shed some light on this area’s course, and its prospects for delivering practical and lasting gains in product development, we sat down with Drug Information Association Global Chief Executive Barbara Lopez Kunz during the recent DIA 2018 Annual Meeting in Boston. The researcher and former scientist shared her excitement over the progress of new patient engagement approaches in industry and on the regulatory front; the continued roadblocks to convergence of ideas and best practice; and the importance of early engagement and conversation-so that ultimately we can begin to judge the patient voice/perspective impact by results and outcomes.
The following are excerpts from our discussion.
PE: At events like this one, you hear a lot about the various patient-centric initiatives in drug development. Do you see these efforts or strategies truly changing how clinical trials are conducted on a day-to-day basis?
Lopez Kunz: If you look at the history, the first step is, do people think patients should be involved in decisions around healthcare product development? Or do people, companies, regulators believe that the input of the
patient community is important? We’ve gone well past that. There’s been an enormous amount of activity-lots of research projects, lots of surveys to try to figure out the operational questions.
What we at the DIA have been doing is starting from the perspective of getting patients to understand drug development, so they can understand the how’s and the why’s and understand where they can influence. Now you have an educated patient community. I always tell people, patients are professionals, too, so many of them have their own expertise. They’re coming from perspectives, be it attorneys, for example, or other professionals in various fields. How do we get them to start leading and expressing how they can be engaged from a position of expertise? We’ve taken it from the perspective of bringing it into the conversations.
If you look at what’s actually happening, though, what you’re going to find is that there is no common pathway for where to bring patients into things like, say, clinical trial regimen or even into some topics related to value and access. We have done some work trying to look at the operational elements within companies. Many times, you find people are employed in companies to have patient roles but they’re not the same roles-they’re not reporting into the same person in the organization, there’s no common performance metrics. I believe that the future holds common roadmaps for where patients can be best engaged in clinical development-common metrics, common organizational structures-but we’re not going to get there without openly sharing what’s working today.
Going beneath that, however, where is it working well? Getting patients more involved in how clinical trial protocols are set up, getting patients more involved in adherence kind of discussions, and so on-those are places where patients obviously are having an impact today.
Patients are also very involved as advisors, even to some of the regulatory decision-making. Here in the US, the FDA just issued its first guidance on patient-focused drug development. You’re seeing all the sort of channels coming forward with insights on how it could work. But I think it’s going to be years before you really see a true integration-a systematic integration and systematic benefits that can be measured.
PE: Is it increasingly important, then, for groups like DIA to help accelerate that integration?
Lopez Kunz: I want DIA to be that organization that helps to develop that. And that’s why we’ve made patient engagement a key strategic priority for us, and we’re doing research with universities to try to establish those practical roadmaps. We’re making all of our work very specific. How do you structure an organization? Where does a patient voice come in? How do you put a clinical trial protocol together? Where does the patient get engaged? How do you take it through regulation? We’ve been trying to move those steps forward and are making some progress.
PE: At what level are your members in industry addressing those questions and taking steps to improved engagement with patients?
Lopez Kunz: Some organizations are further ahead than others. And it’s not necessarily all the big multinationals; there are some small and medium-sized companies, especially in rare disease areas, where really a lot of progress has been made in integrating the new patient voice. What we’ve been trying to do is engage and convene those organizations so they can learn from each other about what’s working. We have a program going where we’re actually trying to establish a scorecard, if you will; here are the things that you should be doing if you’re patient-centric and you’re at a best-practice level, and this is how you should be thinking about it. You monitor yourself against current best practices and then you learn from each other, and the whole organization goes up to the next level and the monitoring tools change.
FDA requirements obviously play a big role in setting the table and the timelines, and there’s probably going to be another three more guidances coming out. But I think industry is very involved and anxious to make moves in the right direction. Clearly, there’s been investment and, clearly, there is a requirement and an expectation that there will be a return on that investment. I would say three to five years from now, it’s going to look very, very different.
The issue with the research that’s been going on in this area is that it’s pretty fragmented. People have bitten off small pieces, but there’s no common stream. We’re hoping to do that. One of the benefits that DIA brings to the fore is that we are in a position around the world, so we have a global platform. In Europe, the Innovative Medicines Initiative (IMI) has put a number of patient programs together. We’ve been very involved with EUPATI, which is the European Patients' Academy on Therapeutic Innovation. We’ve developed a portfolio with it, as part of a consortium, that’s content knowledge and that has trained the patient community. We’ve been able to take the learnings from Europe and translate that knowledge around the world. As patients are getting educated, there’s a need to educate everybody in the ecosystem about how you do more patient engagement.
PE: How can some cultural touchpoints that experts have brought up here, such as “diversity” and “community,” help advance this effort?
Lopez Kunz: When people think about diversity, they tend to think about race and gender and sexual preference. Diversity is really all about that, plus the diversity of thought and perspectives that need to be brought in to be successful. Having the various components-the regulatory perspective, the benefit-risk view-and all that diverse thought and conversation early on, including from the people who are making value and pricing decisions, is critical.
And then putting the patient view on top of that. As a patient, if you go to the doctor and the doctor says you have a certain condition, so go do this, it might not be in line with your preference in terms of symptoms that you want to affect or your lifestyle decisions about how you want to be treated. Patient engagement should come first. Who’s thinking about what a pediatric patient really wants or what a geriatric patient really wants? Who’s engaging them? It’s one drug, one device fits all. [But] you might have great manual dexterity or not, you might be able to self-inject or not. Those kinds of things need to be brought in really early so that at the end of the day, we’re optimizing toward a certain outcome.
You’re not going to have patients involved in the science behind the clinical trial, the protocol development, but their needs and priorities need to be built in.
As far as “community,” the people that end up in healthcare, I think we all have our stories, and I see us as one large community. I do believe that, somehow, we’re connected up-patients, providers, etc. I think it’s a wonderful, compassionate community. Wherever I go around the world, it’s all the same conversation. I could be listening to it through simultaneous translation in China or Japan or in India or wherever-the community is all the same, carved out of the same mindset.
What I’d like to see [in patient-centricity] is a converging. It seems when we start, everybody is diverging and there’s a whole bunch of small fragmented things happening. I’d like to figure out how do we start pulling it together so that the resources that we have to try to move the system along are rowing in the same direction. That’s what’s going to happen next-people are going to start seizing on things and there will be some evidence that cause/effect is happening; you have a patient voice in these particular areas in clinical development, and look what happens? You get a much more comprehensive output of a clinical trial or you get better patient adherence, for example. Then people are going to say, “that’s what can be best practice.”
PE: You mentioned FDA’s push for patient-focused R&D. Since Commissioner Gottlieb has taken over, what’s your view on the agency becoming noticeably more transparent itself, and lending its voice to wider healthcare issues like drug pricing?
Lopez Kunz: I will say, number one, I think he’s doing a great job. I think he’s established a level of urgency around some really important issues like the opioid crisis and drug pricing, which, of course, is necessary. But I also think he’s done a lot to create an organization where there’s clear leadership and accountability. The general sentiment is that-obviously, people who came before him set it up to keep going in that direction-people are happy with the focus. There’s been a number of new guidances that have come out. The focus in digital health and data sciences is really important. The Oncology Center of Excellence is super important. And, of course, the patient-focused drug development guidances that are coming out.
I was invited to the National Academies’ session on patient-centric initiatives [recently], and the patient groups there were talking about how much more inclusive the FDA is being in engaging them and getting their perspectives on guidances. Of course, there’s work to be done. I’m not going to pretend like we solved it, but I think that it feels different, it feels a lot more open and refreshing.
I like that the FDA is starting to comment on the pricing issue. It’s actually important for the regulators that they’re considering and collaborating with people early on who make those pricing decisions and reimbursement decisions. Who wants to spend time putting a drug through clinical trials and regulatory, only to find that patients can’t access it?