How Biotechs Can Future‑Proof Phase I Trials for Regulatory Approval (Part 1)

*Full transcript available below

In an interview with BioPharm International®, Ben Edwards, chief operating officer at Avance Clinical, emphasizes that the future of early development hinges on specialist contract research organization (CRO) partnerships. He frames Phase I development not as a standalone safety exercise, but as the foundation of a quantitative, exposure–response package that can credibly drive Phase II.

At the core, Edwards explains, is scientific and regulatory design, including selecting the right population—whether healthy volunteers or targeted patients—and defining endpoints that capture pharmacokinetics (PK), pharmacodynamics (PD), safety, and early activity. Equally crucial are project managers who understand the cadence of Phase I and can keep data flowing to biotech sponsors in near real time, enabling them to secure the next funding tranche and refine their Phase II strategy.

Why do regulators now expect quantitative exposure–response evidence from Phase I?

Edwards highlights the shift reflected in recent FDA guidance. As he notes, “The regulators really do want a quantitative package using Phase I data and pre-clinical data, which are characterized by the dose-exposure response, rather than just selecting a minimum or maximum tolerated dose.” That expectation makes specialist biometrics teams indispensable, ensuring PK and PD outputs are rapidly available and analytically robust, he adds.

The consequence of misaligned trial design is severe, Edwards warns. “So, it's critical to choose the right CRO and the right teams to really guide you in the design of those trials—to collect the right exposure data, the right endpoint data,” he states. “If you don't do that, and you're not collecting the data the regulator wants to see, you might need to restart again.”

For biotechs, investing early in expert CRO partners is now a scientific and strategic necessity, Edwards stresses.

About the speaker

Ben Edwards, Chief Operating Officer, Avance Clinical

Working closely with the CEO, Edwards leads business growth and aligns operations with future goals. With more than 24 years of industry experience, including over 19 years in CROs, he has supported global and Asia-Pacific clients in achieving pharmaceutical market registrations, including approvals from FDA, the European Medicines Agency, Japan’s Pharmaceuticals and Medical Devices Agency, and China’s National Medical Products Administration. Edwards champions an action-oriented, ethical, and accountable, team-first culture focused on delivering client value and improving patient access to treatment.

Transcript

Editor's note: This transcript is a direct, unedited rendering of the original audio/video content. It may contain errors, informal language, or omissions as spoken in the original recording.

Speaker 1

My name is Ben Edwards. I'm the Chief Operating Officer for Avance Clinical. My role at the business is to really be the architect for designing and delivering our operations, for our clinical CRO business. I've been in the CRO sector for over 25 years, working globally to support biotechs to deliver their drug development to international regulators.

So it's really important to select specialist CRO teams to really accelerate drug delivery. And in particular, I see three sort of categories of specialist teams that can really support that. Firstly, scientific and regulatory teams to really expertly design a trial select the right patient population, whether it be healthy volunteers or a certain patient population, even in phase one adaptive design trials, and really select the endpoints for the trials in an expert way. That's critical. Secondly, it's really critical to have highly trained project managers that really understand the cadence and pace of phase one trials so that they can work with the client, understand the client's needs and how to give them access to data. And then finally, access to data for biotechs is critical. They really do need that in order to be able to get their next tranche of funding or support the development of their phase two program. So having a specialist biometrics team that really understands the data requirements is really critical. So what data do they need? Is it PK data, PD data? How can they access that quickly, in real time after the patients have been dosed? In order to select the phase two dose. The regulators really do want a quantitative package using phase one data and pre clinical data, which is characterized by the dose exposure response, rather than just selecting a minimum or maximum tolerated dose. I should say so it really the FDA released some guidance documents in 2024 and that really shows that the phase two enabling packages should include a concise data package which really talks about pharmacokinetic data, pharmacodynamic data, safety data and early efficacy data, or early activity data. And so therefore phase one design should be really conducted very carefully to generate that exposure response data required for phase two. So it's critical to choose the right CRO and the right teams to really guide you in the design of those trials to collect the right exposure data, the right endpoint data, if you don't do that and you're not collecting the data the regulator wants to see, you might need to restart again. So having that real strong scientific advice to get that exposure and response data, and potentially even patient data at phase one is critical

in order to select the phase two dose, the regulators really do want a quantitative package using phase one data and pre clinical data, which is characterized by the dose exposure response, rather than just selecting a minimum or maximum tolerated dose, I should say so It really the FDA released some guidance documents in 2024 and that really shows that the phase two enabling packages should include a concise data package which really talks about pharmacokinetic data, pharmacodynamic data, safety data and early efficacy data, or early activity data, and so therefore phase one design should be really conducted very carefully to generate that exposure response data required for phase two. So it's critical to choose, you know, the right CRO and the right teams to really guide you in the design of those trials to to collect the right exposure data, the right endpoint data. If you don't do that and you're not collecting the data the regulator wants to see, you might need to restart again. So having that real strong scientific advice to get that exposure and response data, and potentially even patient data at phase one is critical.