The head of the FDA Center for Drug Evaluation and Research, Dr Janet Woodcock, has described the current system for conducting clinical trials as "broken" and questioned the use of 'real-world evidence'.
In a speech to the US National Academies of Sciences, Engineering and Medicine, Dr Woodcock called for a new way to collect and utilise patient data.
She raised the future prospect of more investment in master protocols and the related development of clinical trial networks.
A master protocol involves more than one or two treatments in more than one patient type or disease within the same overall trial structure. It is an overarching protocol designed to answer multiple questions, for example multiple therapies targeting a particular biomarker-defined population or disease subtype, and would inevitably require more collaboration between companies.
They differ from the current approach for clinical trials where a single therapy is studied in a single disease.
In a recent article in The New England Journal of Medicine, Dr Woodcock said a master protocol, "may involve direct comparisons of competing therapies or be structured to evaluate, in parallel, different therapies relative to their respective controls."
According to Dr Woodcock, a key advantage of a master protocol is that it can leverage existing infrastructure and similarities between trials involving new treatments, and more than the small number currently underway are inevitable given the growth in personalised medicine and the use of biomarkers.
She also acknowledged the challenges of master protocols, including issues surrounding data use, publication rights, and the timing of regulatory submissions.
On real-world evidence, which is likely to play a much bigger role in FDA decision-making given new laws expanding its use in the drug approval process, Dr Woodcock said it has not been used extensively to date and that it may not even be effective at uncovering the true efficacy of a new treatment.
She did acknowledge the new laws will provide a significant incentive for industry to improve the collection of real-world evidence because it allows companies, in some cases, to base submissions on observational studies, insurance claims data, patient input, and other anecdotal data rather than full clinical trial results.