As a scientist now working as a director of research responsible for overseeing a large programme of research funding, I can understand the frustration experienced by researchers who have invested years in a clinical trial, which then shows a new treatment to have significant clinical benefit but which patients can’t access because it doesn’t get approved for use on the UK’s health services. The frustration the research community must feel, surely pales into insignificance in comparison to the frustration of patients.
Time and again The National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) reject new treatments because they claim that the industry prices them above accepted thresholds or that there is insufficiently robust evidence of clinical benefit. Of course, if the treatment has little evidence of benefit, you would expect regulatory agencies to turn it down. But when new treatments are priced beyond the financial constraints of our health services, or evidence isn’t presented in a way that the regulators will accept, patients start to question whether new innovations will ever reach them.
That said, let’s not forget that there are new treatments and technologies that won’t severely compromise health service budgets. As pioneering research shows us new ways to use existing drugs and technologies, we have the potential to deliver affordable clinical benefit to thousands of patients. You’d expect that this would allow for the quick adoption of innovation across our health services; innovation that brings real benefits to patients at a price the state can afford—you’d be wrong.
This year the results of a large UK led trial called STAMPEDE were revealed to great cheers (and not a little envy from our US-based colleagues) at the American Society of Clinical Oncology annual meeting (ASCO)—the largest international cancer conference. The findings were staggering. In an age where we’ve learned to talk about new cancer treatments in terms of “incremental benefit,” these researchers showed a 22 month survival benefit from giving men newly diagnosed with metastatic prostate cancer docetaxel chemotherapy at the same time as hormone therapy, instead of after hormone therapy—the current treatment pathway. How often do you hear scientists say the word “staggering” in the same sentence as results?
Yet, despite the fact that docetaxel is an off-patent, inexpensive drug, we know that men are still being denied earlier access to this treatment. What’s more, we lack any sense of how long it will take for this to become standard practice. Results like this don’t come around very often, so we need to make sure that when they do we are ready to implement them without undue delay. So what is causing the delay?
First, docetaxel is off-patent, so there isn’t much incentive for the manufacturer to champion the cause to get it re-licensed or to promote its use. Alongside this and more importantly, the results of STAMPEDE haven’t been published in a peer-reviewed journal yet. Although it has been talked about and the execution of the trial widely praised by leaders in prostate cancer clinical research at conferences, it is publication that is considered to be the “gold standard” means of validating evidence and is what UK health services are waiting for before they implement the findings of this trial.
Knowing how important peer-reviewed publication is to the widespread uptake of research, couldn’t researchers be preparing a manuscript at the same time as undertaking the significant data analysis that is already needed for a conference presentation? At ASCO some very big names in prostate cancer research were praising the results of this particular trial very loudly. Shouldn’t their having seen the results at a conference speed up the peer review process? Or could we make a more radical change, and have a system where data are released to UK Health Bodies to organise their own peer review process whilst awaiting publication?
I ask these questions because if publishing continues to be delayed, we leave patients without access to clinically beneficial treatments that could transform their lives. There is no fast-track for research results that can make a real and tangible difference to patients, and perhaps there should be. After all, the Americans haven’t let publication delays get in their way. They included upfront docetaxel in clinical guidelines straight after the presentation at the ASCO conference.
I’m not suggesting we do away with peer-reviewed publication. It is absolutely right that any changes in clinical practice are based on a robust analysis of the results including quality of life and cost effectiveness. I also recognise that speeding up publishing processes is likely to be challenging. I am going to suggest that we need another way forward; a way to get affordable, repurposed treatments with clinical benefit to the people who need them.
If we truly want the UK to be at the forefront of medical innovation, shouldn’t we also champion our health services—who are actually leading this type of research—to become early adopters of innovation? In practice this would simply mean being aware of the research taking place in their hospitals and engaging with the research teams as soon as early trial evidence indicates promise and cost-effectiveness. This would allow for assessment of the infrastructure and resource changes that would be needed for when these findings are finally published. Forward planning could turn our health services into proactive leaders in innovation, rather than leaving them scrambling to react to old news. This is already happening in the West of Scotland—let’s learn from them
I strongly believe that the docetaxel arm of STAMPEDE can be a trailblazer for a new co-ordinated forward-planning approach across the UK’s health services. If we get it right, we could see Health Bodies across the UK being able to provide accelerated access to cost-effective and clinically beneficial treatments and technologies developed by health services based research teams. There can be no excuse for any of us not to at least try to make this work.
Iain Frame is director of research, Prostate Cancer, UK.
Competing interests: None declared.