Earlier this month, Janet Woodcock, Director of the Center for Drug Evaluation and Research at the US Food and Drug Administration (pictured below), said that the clinical trials system in the US is ‘broken’.
“I personally believe, like many other people, that the clinical trial system we have right now is broken. It isn’t working to meet the objectives that we need it for. Our ability to generate the needed evidence efficiently and in a cost-effective matter will continue to be a barrier to innovation and to the quality of care around the world.”
Inefficiency in clinical trials is well documented; we have discussed problems with recruitment, retention, and feasibility studies before. What Janet Woodcock is saying though, is that the clinical trials system more generally, is simply not working as we need it to.
Q: What’s missing?
A: Real-World Evidence
The FDA has recently been praised for embracing Real-World Data (RWD), but this statement from Woodcock suggests that things are not moving fast enough. Woodcock made her remarks during a 2-day workshop organised by the National Academies and sponsored by the FDA. The workshop focusses on examining the impact of Real-World Evidence on medical product development.
“FDA’s committed to exploring the use of real-world evidence in regulatory decision making,” Woodcock said.
The remarks were echoed by other senior members of the FDA, and demonstrate that the movement to harness existing data sources such as electronic health records to make decisions about the safety and efficacy of medical products is here to stay. The move will not only ensure that the side-effects of new products are reported accurately, but it’s also an initiative that aims to curb the cost and time of developing new drugs. Latest figures from the US suggest that a new drug takes an average of 12 years, at a cost of $2.5 billion. The rising expense is in large part due to expensive and increasingly complex randomised clinical trials.
Routine integration of Real-World Evidence into the FDA’s regulatory decisions was described by Jane Woodcock as the “big kahuna”, adding that this process will require a cohesive, hybrid approach from multiple departments within the agency.
Whilst regular use of RWD and RWE to make healthcare decisions in the US is certainly a challenge, it’s success has already been demonstrated by the NIH Collaboratory. The NIH Collaboratory has established pragmatic clinical trials, with research taking place directly in the hospital, clinic, or other everyday healthcare setting – instead of being isolated in a randomised clinical trial.
We’ll be watching closely to see how the FDA’s decision-making changes with the integration of RWE. Of course, the next step for us is learning from their processes and integrating RWE into decision-making within the UK’s health systems and regulatory bodies.