With the US Food and Drug Administration (FDA) shifting its clinical trial regulations in recent years, working closely with regulators at the early stages of trial design is becoming increasingly important for successfully executing an oncology trial.
Speaking at the Clinical Trials in Oncology (CTO) East Coast 2025 conference, taking place on 8-9 July in Boston, US, AstraZeneca’s oncology, regulatory, strategy executive director Shaily Arora noted that FDA initiatives such as Project Optimus for dose optimisation are “fundamentally grounded” in providing better care to patients.
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“There had been a lot of accelerated approvals in oncology, but a small percentage of them did not translate into clinical benefit when confirmatory trials read out,” Arora explained.
Initiatives such as Project Optimus emerged from the need for more rigorous dose-finding studies early in drug development. Recent tightening of regulatory requirements for accelerated approvals, particularly around confirmatory trials, has been driven by delays and issues in verifying clinical benefit. Underlying all of this is the goal that we want medicines that truly provide benefit to our patients.
Arora also stressed the value of close collaboration with regulators, sharing that working together to shape fit-for-purpose study designs ensures effective utilisation and optimisation of early phase trials.
Speaking on the same panel, Fatima Scipione, vice president of global patient affairs at Blueprint Medicines, called for sponsors to consider patient-centric outcomes where possible.
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By GlobalDataScipione said: “If we don’t anchor there, and truly look to change that with their experiences between medicines and other clinical development programmes, then in the end, we’re not going to be successful with regulatory.
“These ‘anchoring aspects’ provide the strength and conviction necessary to talk in depth with our regulators about what we need to do to ensure our trial designs are appropriate.”
The value in patient advocacy groups
The panel discussion moved on to what approaches can be taken to mitigate the risks associated with the current regulatory environment.
Scipione underscored the critical importance of engaging advocacy partners early in the trial planning process. She shared a real-world example from her previous role where recruitment stalled in Brazil due to a misalignment with the local patient landscape, despite full operational readiness.
“We had sites selected, feasibility done, contracts in place – and we still weren’t enrolling,” she recalled.
“It wasn’t a question of operational readiness. It was that we hadn’t asked the right local voices early enough.”
“I remember an advocacy leader telling us, ‘These sites don’t see the patients you’re trying to recruit.’ That stopped me cold. Because no amount of site activation can fix the wrong assumptions.
“It was a wake-up call: if you’re not working with the advocacy community from day one – especially in global trials – you’re not just risking slow recruitment. You’re risking trial failure. It’s that simple.”
