From Study-by-Study to Portfolio Recruitment: Q&A with Gaynor Anders, Trialbee

The FDA's move toward single pivotal trials is compressing the margin for error in patient recruitment in ways the industry has never had to contend with before—and exposing how ill-equipped most sponsors are to operate without the safety net of parallel studies.

To explore this further, Applied Clinical Trials recently caught up with Gaynor Anders, chief delivery officer at Trialbee, about what one-shot recruitment planning looks like in practice, why siloed study teams and budgets are the core organizational barrier to programmatic approaches, and what true partnership between sponsors, CROs, and recruitment partners needs to look like to make this model work.

ACT: How is the FDA's shift toward single pivotal trials changing recruitment planning and execution for sponsors?

Anders: This is an interesting one. The news came out in February of this year, and I've been speaking to some sponsors about it—mixed feelings, really. On one hand, people understand it could be good: approvals may come through quicker, and there are potentially lower costs for sponsors because they only have to run one pivotal study with confirmatory evidence to back up whether their compound does what they think it will do. But what sponsors think they'll save in some areas, they may have to spend in others—namely in patient recruitment. Recruitment will be a lot less forgiving than it has ever been before. You get one shot at it. That's the phrase I keep hearing from sponsors.

There are a few specific areas our clients have been speaking about. Recruitment now hinges on one study. Traditionally, if recruitment on a study was failing or underdelivering, there might be another study running at the same time that could help pick up some of the pieces—that safety net is gone. Feasibility is going to need to start earlier and be more precise, because there's no wiggle room. Diversity and truly representing patient groups will be under the microscope—with one study, every study has to have those factors built in. The quality of patients is going to be as important as just filling the study, because high-quality referrals increase the chance those patients are retained, and retention is going to be as big a challenge as recruitment itself.

We're also finding that some recruitment decisions are now being escalated to board level. More money is going to be spent on the recruitment piece to get it right, and senior executives are telling us this is now within their remit—because they know they're going to be spending more on patient identification through data, digital marketing, trial finder websites, and technology infrastructure, all trying to get the greatest return on investment from that one pivotal study.

ACT: What are the practical consequences of rebuilding patient pipelines from scratch for each individual study, and why has that model persisted for so long?

Anders: Starting with why it's persisted—I've been doing this role for coming on 30 years, and traditionally studies were very protocol centric. A good number of years ago we all talked about patient centricity, but what we're seeing now with this shift is a move toward patient centricity within a relationship-building context. Sponsors building relationships with groups of patients, enabling them to sign up to communities where they can receive information about the work a sponsor is doing, and thinking about studies programmatically—looking across a whole therapeutic area, regardless of whether it's in the same indication or across compounds, and thinking about where they can maximize the chance to talk to patients, build those relationships, and enable them to want to take part in research.

We know that about 32% of budgets are spent on patient recruitment for any clinical trial, so it's about being clever with what is a large ticket item. We are definitely seeing—and encouraging—sponsors to move away from spending money on one study, screening patients, having patients who don't meet the criteria disengage entirely, and then putting the burden on those patients to find another study on their own. Instead, keep those patients engaged for the next study coming along or for an alternative study, and make sure they feel valued.

The minute a patient voices interest in research as a treatment option, we should be encouraging that. If we can't match them to one study, we should be trying to match them to another—and putting the burden on us as industry professionals rather than on the patient.

ACT: How does program-level recruitment actually work in practice, and what does it require from sponsors in terms of infrastructure and data sharing?

Anders: The first thing it requires is study teams to stop working in silos—which I know is much easier said than done. I was at a conference a couple of weeks ago where that was a hot topic on a panel I was part of. Different study teams know what's happening on their own study but don't necessarily know what their peers are working on. That has to change.

It also means sponsors need a way of funding a programmatic view of studies—whether that's splitting patient recruitment budgets across a number of studies or having an enterprise-level budget holder responsible for signing off on budgets that can support multiple studies. Study budgets are traditionally as siloed as the study teams themselves. What you want is efficiencies that can be shared across therapeutic areas, compound leads, and study managers, and the only way to do that is to have those individuals working together with a shared pool of funding.

The second piece is technology. If you're going to have programmatic approaches with trial finder websites and sponsor-led patient matching capabilities, you need integrations built into any patient recruitment program that enable end-to-end analytics tracking. Your patient recruitment platform needs to integrate with your CTMS, and everything has to be linked and talk to each other. If you can do that, it minimizes the burden on study teams, enables data to flow through, and gives study teams the end-to-end analytics they need to demonstrate value upward to senior management—and to make the case for doing it again on the next program.

You also need to support sites. Sites can be overburdened with patients, so you need to screen patients in a way that minimizes the burden on sites until those patients are ready to enter a study. And we do work with sponsors to help shape data privacy, communication, opt-in consent language so that they feel comfortable storing patient data and matching those patients to other studies they may have coming.

ACT: What are the biggest adoption challenges when sponsors try to shift from study-by-study recruitment to a portfolio-level approach across a therapeutic area?

Anders: Some of it is how sponsors are organized. Some of it is a genuine mindset shift—thinking about things differently, thinking beyond your own study and asking what this means for patients. We have sponsors where we sit down and talk with them about what studies they have coming down the line—studies that may not be on ClinicalTrials.gov yet, that may not even be firmed up as the next studies they're going to run—because you need to think far enough ahead to map this out effectively.

We do a lot of protocol mapping with sponsors to help them understand how their studies could be brought together to form a program. It may not be an obvious program at first, but there's always a program there if you look for it. Then it's about encouraging those teams to work together, addressing the budget constraints of doing that, and making sure the technological side of things is in place.

ACT: For CROs trying to build infrastructure around this model, what does success look like and how is it measured?

Anders: It's a tricky one. CROs have always been measured on the completion of the study—patients flowing into the study. What we find sometimes is that there's a challenge around whose responsibility patient recruitment actually is: the CRO, the sponsor, or the patient recruitment partner that's been brought in. The number one thing I would say is that success for a CRO is true partnership. When we can work hand in hand with the CRO from the patient recruitment side of things, we can help the CRO succeed, which in turn helps the sponsor succeed. It's a triangle partnership where everyone is working toward the same aim.

For CROs, a lot of it comes down to visibility into data. They want analytics at their fingertips—how studies are performing within a program, how they can be successful on the next studies coming down. Most CROs have sponsors they work with time and time again as preferred providers, so having access to the data they need while working in partnership really enables them to plan for future programs with those sponsors. And then working with us as a patient recruitment provider to support sites with engagement, making sure sites are doing what they need to do and patients are actually flowing all the way through the funnel.