The US Food and Drug Administration (FDA) has announced plans to revitalise clinical research in the USA, as part of a US Department of Health and Human Services (HHS) initiative titled ‘Operation TrialBlazer’. The FDA has proposed multiple actions to achieve this aim, targeting both early- and late-stage clinical development. The HHS Roadmap calls for coordination across multiple HHS components to maintain the USA’s competitiveness in global pharmaceutical development and ensure US patients have early access to innovative therapies, by eliminating ‘unnecessary delays, redundant requirements and regulatory ambiguity’.
The report cites competition from China in particular, noting that in 2024 the number of clinical trial registrations in the country surpassed that of the US, with concerns that companies are moving investment abroad. Projections based on current trends suggests that 35% of FDA approvals will be from Chinese biotech companies. The report highlights that speed and efficiency are key factors driving companies to conduct clinical research in other markets, such as China and Australia, which offer quicker pathways.
A key action the FDA is looking to introduce is an Expedited Investigational New Drug (IND) pilot programme, which aims to establish a network of Qualified Research Institutions (QRIs), such as medical centres, contract research organisations or healthcare networks, to partner with sponsors on developing and reviewing IND submissions for first-in-human clinical trials. To support this, a real-time, rolling submission platform is proposed, allowing the FDA to review QRI recommendations and IND submission components, and to communicate securely with sponsors to provide guidance. The aim is that this collaborative approach, combined with more targeted use of FDA reviewer resources, will reduce the number of clinical holds and accelerate sponsor’s path to IND readiness. The proposal is open for public consultation, until 22July.
The strategy also involves updating guidance documents, including a revised draft guidance on ‘Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products’, open to comments until 22 September, and ‘ Master Protocols for Drug and Biological Product Development’, which is open to comments until 24 August, both documents aiming to increase efficiency. A new draft guidance document on ‘Quantitative Systems Pharmacology-Based Dose Selection for Minimum Anticipated Biological Effect Level in First-in-Human Trials’ has been published, to support developers in selecting initial dose for FIH trials using quantitative systems pharmacology (QSP)-based approaches.
Further work is underway to clarify expectations for CMC data for Phase 1 IND sponsors, an area which can delay the development timelines when more data is produced than is necessary. The CMC IND webpage for CDER-regulated drugs has been updated to clarify these requirements, supplemented by new Phase 1 Contact Centre, where specialists will quickly respond to questions from developers.
The Operation does not rest solely with the FDA, delivering change to the US clinical research ecosystem will require work from other federal entities as well. Time will tell whether these changes shift the increasingly competitive clinical research landscape, particularly as other economies, such as the European Union and the UK, roll out their own initiatives to make research more attractive for developers. In the press release, HHS Secretary, Robert F. Kennedy Jr said:
’Under President Trump’s leadership, HHS is launching a coordinated department-wide effort to restore America’s leadership in clinical research, remove unnecessary barriers, and bring more clinical research and investment back to the United States. America led the world in medical innovation before. We will lead again.’
