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International Clinical Trials

Brave New World

“Houston, we have a problem.” Famous words from the Apollo 13 manned space mission that are now a cliché for an understated disaster. Applying this to the clinical trials world, many in the industry would agree that we do, indeed, have a problem.

The difficulties we face are complex; there are various moving parts and multiple causes. Sites are poorly resourced; trials are ultra-regulated; companies are bureaucratic; sites regard trial work as low priority; Western costs are high; data quality can be inadequate; patients are less well motivated than we hope; study designs are unrealistic; and company resources are limited.

However, despite widespread acknowledgement of these problems, the industry remains slow-moving, with action and overhauls hard to come by. This is probably because there is so much at risk. Trials cost tens and, occasionally, hundreds of millions of pounds to run, not to mention the lost opportunity costs incurred if they fail or are delayed. Such high stakes make experimentation rare.

The industry’s urge is to tackle these problems one by one as they arise, to get trials to go as planned. Metaphorically, we look down at our boots, seeing just the next step to make sure we do not trip. This is understandable perhaps, but we need to lift up our heads and look at the horizon – to give proper, high-level consideration to solving problems holistically, with a strategic and commercial mindset.

Greatest Challenge

Recruitment and retention of patients is our greatest challenge. According to widely held belief, only six per cent of medics take part as principal investigators in studies in the West and, of those, at least a third barely manage to recruit any patients. In short, we get every Western patient from just four per cent of the population; however, according to data from the Medical Research Network, 25 per cent of medics report that they would like to take part in research, but cannot due to lack of previous experience or resources.

If expertise and resources are the only problem, surely we have to give it to them. This is quite possible – we have done it in the past – and it is not expensive in the larger scheme of things. We can keep them as key opinion leaders (KOLs), in focus groups and on publication committees.

There are no excuses for keeping poorly recruiting sites in our trials. But that is not the big prize. The ideal result is a small number of really interested sites each recruiting lots of patients, maximising the site performance through interest, greater training and focus, and thereby improving data quality and enhancing cost effectiveness – all in one go.

Reaching Out

We have to increase the reach of sites, enabling them to recruit many more patients. Here, the internet is key to success: health-related issues are one of the most common searches by far, internet penetration in all age groups is now high, and patients are actively looking for information and help, which they often see as a clinical trial.

Some argue that virtual trials might be the answer. But, while these studies reach out to patients directly, they then attempt to care for and assess the patient through a computer. This does not work – and people do not like it. They want expert advice, but also care and attention, while the industry and regulators want safety – all of which means very few trials could ever be done virtually.

We should replace the computer with the community. Patients are either in hospital or in the community, after all. We have been looking after patients in their homes for decades, and only for one day a month is a patient at a site during a trial. For the other 27 to 30 days, they are in their own home. Patients are taken to sites because that is where the medic is, not because it is safer there.

Medics on the Move

Most of the medics are in the trial just to provide access to the patient, not because they are KOLs or because they are driving the science. So, if they are only being used each month to check the basic safety of the patient, and otherwise all they provide is patient access, why not fi nd the patients through the internet and social media, and use a medic or nurse – or both – in the community to look after safety?

However, this raises a dilemma: what about specialist assessments? The issue here is not equipment and experts to use it – most local hospitals are equipped with MRI scanners, radiography of all types, lung function labs, ultrasound assessments and a radioisotope capability. The issue is access to specialist medical experience and knowledge – the rheumatologists, cardiologists or neurologists that are all in local hospitals may not be interested.

We should not look to reduce interaction with the principal investigator to zero, but we could cut it to, say, 50 per cent. Occasionally, the patients will need to go to the site, but if it is half as often, it may be possible to get twice the consent rate.

Alternatively, medics could be sent to the patient. A travelling medic who does only one specifi c study in a country could be much cheaper than a host of unproductive sites with all their overheads and lack of interest. In addition, we can and should look at telemetry and other technology solutions, but these do not generally replace a trained medic, so are not likely to be mainstream tools for the foreseeable future.

New Model

In summary, the clinical trials world faces problems: access to insufficient numbers of patients through sites, poor quality work from too many sites, and patients who do not want to take part as much as before.

There are specialist research sites that really want to do the research and do it well, treating their own patients and those found through the internet in large numbers. Much of the work will be done in the community with these patients, who are now more distant from the principal investigator – leaving perhaps just a third of visits to be conducted at the site. This is not necessarily a radical step, and the technology is not untested. We do all of this today – just not in the same study and not routinely.

Furthermore, moving to this kind of new model need not be expensive. Without the overheads and large numbers of sites, and with faster recruitment and improved retention, costs would go down, not up. It is also a safe approach but, importantly, community care needs to be considered from the start in trial design, to ensure the right assessments are made at the right time by the right people.

Taking the Risk

Such a system will work, but there are no guarantees. Restrictive protocols will still have very few patients; alternative treatments may continue to make a trial unappealing; competitive trials may still abound; the science may remain uninteresting; and the pay for the sites may be too low. In all these cases, the trial is either dead anyway, or it will at least improve. After all, if you make the trial patient-centric by seeing the patients in their own home – and if you can reach out to ten times the number through the internet than you can get through the sites themselves – even tough recruitment will improve.

The real concern is who is authorised to take the risk of doing something different? Not many project managers, directors or even vice presidents of clinical research will be prepared to. When you are looking down at your boots, anyone can authorise the same old lowrisk solutions. When you look up at the horizon, you need leaders to step beyond the risk-averse mindset and truly take the plunge.

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Dr Graham Wylie is Chief Executive Officer of the Medical Research Network and is responsible for company strategy and making it stand out from the competition. With a BSc in Pharmacology, an MBBS from St Bartholomew’s College London, and more than 21 years of experience in the pharma industry with names such as Pfizer, PAREXEL and Healthcare at Home, Graham has successfully grown the MRN from a small UK start-up to the leading provider of nursing focused patient recruitment and retention solutions.
Dr Graham Wylie
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