Innovating in Oncology Drug Development: 9 Takeaways from the FDA & NCI Panel at ASCO

At the recent ASCO congress, a panel chaired by Dr Julie Gralow featuring both Dr Richard Pazdur & Dr Paul Kluetz from the FDA Oncology Centre of Excellence as well as colleagues from NCI & MSK on the topic of overcoming challenges in oncology trials through innovation, made for interesting listening.

Below are nine takeaways from the panel and implications for drug developers.  

There remains strong interest in using pragmatic trial designs in Oncology from the FDA. 


‍
The agency representatives discussed Project Pragmatica, an FDA initiative investigating how elements of pragmatic trial design could lead to efficiencies in oncology drug development.
While there was general encouragement for the industry to undertake pragmatic trials in the right context, challenges related to trial endpoints and data integrity will require sponsors to think beyond traditional trial design elements to realise the potential opportunities pragmatic trials provide. A pragmatic trial design potentially suited to accelerated approval is discussed here.
‍

The panel highlighted that oncology trials have grown in complexity.

‍‍

While the need for drug developers to simplify protocols was a dominant theme in the discussions, there was an acknowledgement that the reasons for trial complexity are multifactorial, including the development of targeted therapies, an increase in biomarker-driven trials, and the incorporation of new technologies into trial conduct.
‍

Creativity around site selection could be a solution to trial recruitment challanges.
‍‍

The discussions on trial recruitment were framed in the US context, with some panellists expressing their preference for sponsors to expand the number of US sites instead of undertaking multinational studies. However, countries aside, the panellists encouraged sponsors to think beyond running trials in the same large centres and instead explore other care settings, including decentralised approaches. For sponsors, however, the challenges of how to ensure the integrity of both trial conduct and data in these settings remain and will require new partnerships & ways of working to overcome.
‍

The panel highlighted that ICFs have dramatically grown in complexity.
‍

Panellists emphasised that they thought ICFs had become unnecessarily complex & cumbersome, which can lead to patients not fully understanding the trial or intervention, ultimately harming trial recruitment.
‍

Reducing both site & patient burden was a key theme.
‍

‍There was ample discussion on the need for sponsors to design trials with both patients and clinical sites in mind. Overly burdensome visit schedules or invasive procedures can lead to suboptimal recruitment and increased withdrawals. Trial burden could be reduced by streamlining protocols, incorporating patient-centric endpoints, leveraging Telehealth and incorporating technology to simplify data capture.
‍

Emphasis was placed on learning from running trials during the pandemic.
‍

The COVID-19 pandemic spurred the rapid adoption of virtual healthcare technologies, demonstrating that remote patient monitoring, visits, and data collection could be effectively integrated into clinical trials. While these approaches will require validation in the regulatory context as well as optimised trial designs, they provide an avenue to accelerate trial recruitment & potentially significantly reduce both patient and site burden.
‍

Randomised studies remain the preferred approach in Oncology.
‍

While it was recognised that there are instances where randomisation isn’t possible or unethical, randomised studies remain the gold standard in Oncology trials.
‍

Real-world evidence was acknowledged as having an important role to play.
‍

RWE was recognised as having a key role to play in both supporting the approval of new treatments as well as ensuring optimal trial design & patient selection. This sentiment links closely to the aspects discussed around pragmatic trials.
‍

‍Sponsors were reminded to avoid over-collecting data.
‍

The panel emphasised the importance of sponsors restricting data collection to essential elements that align with the study's main goals. Excessive data collection can result in avoidable burdens on trials, escalating expenses, and prolonging timelines.

‍

‍

‍