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Clinical Trials

Funding and publishing trials

Running large well-designed trials costs a lot of money – though as some people pointed out, trials can also save money in the long run.
 

Drugs are a huge cost to the NHS. By reducing unnecessary treatments, trials can make savings as...

Drugs are a huge cost to the NHS. By reducing unnecessary treatments, trials can make savings as...

Age at interview: 45
Sex: Female
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I guess so from a, a money point of view I know these drugs are hugely expensive and if we continue the NHS as we want it to continue, you know, any savings that can be made on these thousands and thousand of pounds into new breast cancer drugs and all sort of things, I think that’s only a good thing. And the less time you have to be on chemotherapy and go through this, because it is like putting your life on hold for six months and then it, you know, going back to work, how you’re going to feel afterwards, you know. So the less time you spend doing all of that I think would be fantastic.

Funding for trials can come from a range of sources, including:

• The NHS and the National Institute for Health Research (NIHR)
• Government-funded research councils such as the Medical Research Council (MRC)
• Major research charities such as Cancer Research UK
• Commercial companies

The patient information leaflet you are given when a trial is explained to you should always make clear who is funding the research.
 
A few people we talked to were not aware that the NHS funds trials. One person thought the trial she was in was funded by a drug company. She said, “It’s self-explanatory really... I mean, where else would the money come from?” When she was told that many trials are NHS-funded, her surprised reply was “Really? Goodness me, heavens!” (The trial she was in was in fact funded by the NHS).
 
By contrast, Marie assumed the trial she was asked about must be NHS research because she was approached through her consultant in an NHS hospital. She was worried to find it was not.
 

She had heard bad things about commercial trials. She thought a trial in an NHS hospital would be...

She had heard bad things about commercial trials. She thought a trial in an NHS hospital would be...

Age at interview: 69
Sex: Female
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The only information that I heard about clinical trials was bad information. And the few, about a year ago when all those boys had clinical trials* and they all went wrong and I know that they’re still fighting for compensation, trying to prove it with the drug companies, and I don’t agree anybody would ever be able to fight drug companies. They have too much money behind them. I knew that, but had I have known it was a foreign drug company and not my NHS research team, I wouldn’t have agreed. But I just thought that it was because I knew nothing about trials and it was put to me from an NHS hospital that it would be NHS. I don’t know why I thought that because I was totally ignorant about trials but I thought it would be done through my local hospital research.
 
*FOOTNOTE: Marie is referring to a Phase 1 trial at a commercial research unit based at Northwick Park Hospital in 2006 when 6 healthy volunteers became extremely ill. A copy of the Inquiry Report of this incident can be downloaded from the Department of Health website.
 
 
Marie referred to a Phase 1 trial at a commercial research unit based at Northwick Park Hospital in 2006, when 6 healthy volunteers became extremely ill. Many people said they were a bit suspicious of commercially run trials, and the belief that they might be more risky than publicly funded research was often given as a reason. This was certainly the view held by Leslie, who became seriously ill during a trial.
 

Leslie thinks there is too much secrecy around drug company trials, and feels they are more...

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Leslie thinks there is too much secrecy around drug company trials, and feels they are more...

Age at interview: 75
Sex: Male
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There is far too much secrecy surrounding clinical drug trials. I was not given the information that would have enabled me to make informed consent. The regulatory bodies are too closely connected with the pharmaceutical industry to exercise any control over an industry that the House of Commons Health Select Committee said is out of control. An equally supine Department of Health will not admit that the system is faulty and operates against the interests of those of the general public who are willing to put their bodies at the service of medical research.

 

So, will I ever volunteer again? No. And unless the trials are conducted more openly I can only suggest that prospective volunteers think very hard before volunteering.

 

If anything goes wrong the volunteer will be on his/her own. There will be no help from the regulatory bodies, no help from the NHS Ombudsman, no help from the Department of Health — and no legal aid, probably. I am quite sure that what happened to me was not a unique event but the drug industry very successfully hides what happens in trials — especially when things go wrong.

 

The GP/trial supervisor said that I was entitled to compensation and, some weeks after, I was offered £2000 for the ‘inconvenience’ I had sustained. I felt that ‘inconvenience’ was hardly an appropriate word to describe what had happened to me and did not accept the offer. The amount was then raised to £3000. On the advice of the GP I contacted a medical specialist solicitor. Although both the GP/trial supervisor and the drug company’s trial co-ordinator agreed that I was eligible for compensation and that Vioxx [rofecoxib] was the probable cause of my illness, it was to take me over four years before my claim came to court.

 

A volunteer may be paid for putting his/her body at the service of medical research but this does not absolve a drug company from its responsibilities should harm occur.

Volunteers should also remember that many major drug companies are now conducting their clinical trials in areas where there will be fewer questions asked should things go wrong and where they can get away with paying minimum compensation.

 

Finally, trial volunteers should be aware that they are putting their faith (and their lives) in the hands of an unscrupulous, profit-driven industry, an industry that sees volunteers as little more than test beds for their products — to be used and discarded as they see fit. The pharmaceutical industry may be ‘lethal but legal’, whilst the regulatory authorities hide behind ‘due diligence’ — ‘never explain, never apologise’ is their mantra.
However, others took the view that all trials are well regulated, whoever funds them.
 

There are strict regulations in place to protect patient safety, so the risk of something going...

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There are strict regulations in place to protect patient safety, so the risk of something going...

Age at interview: 38
Sex: Female
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There are strict ethics requirements for the research to get off the ground. It has to be checked by ethics committees. So they’re not obviously going to do anything that’s harmful to you, and there are lots of procedures in place to prevent harm. It can go wrong, like the recent botched drug trials at Northwick Park*. But I think on the whole, the huge number of trials that are carried out, whether it’s a drug or a particular type of intervention or whatever it may be, Northwick Park was one out of many thousands that are carried out, and I think the odds of something going badly wrong are just minuscule. They made some fundamental errors in their administration of the drug and the industry has hopefully learnt that lesson.
 
*FOOTNOTE: Kate is referring to a Phase 1 trial at a commercial research unit based at Northwick Park Hospital in 2006 when 6 healthy volunteers became extremely ill. A copy of the Inquiry Report of this incident can be downloaded from the Department of Health website.
 

Drug companies will of course research products in order to make money, but the UK has a good...

Drug companies will of course research products in order to make money, but the UK has a good...

Age at interview: 59
Sex: Male
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One of the challenges for trials, I think, is that it’s highly unlikely we’ll ever see dramatic results. It’s all about making improvements on the periphery. If the, you know, penicillin’s already been found. I don’t imagine, although I’m not a medical person, that there’s many more penicillins about, you know? So therefore if you’re going into a trial, you shouldn’t expect a eureka moment. It’s going to be improvements on the margins. And so your expectations should be placed like that accordingly, I think, really. I think some people get very nervous and concerned about trials sponsored by drug companies. Well, drug companies are set up to make money for their shareholders. I quite understand that. And so therefore the work they do will be about trying to find a therapy that is so good that they make a lot of money out of it. Nothing wrong with that, really, as long as they don’t take chances with wild cards that affects people’s health, on the hope that it might do something. Now that would be a fear of a lot of people. My view on that is that within the country you have sort of a very strong ethics governance and the risks of that happening are very, very low.
 
What I do think is important, though, for drug companies is if they do a trial and it doesn’t work, they publish that as well. Because you can learn as much about what doesn’t work than you can - I mean, that’s a trite phrase, but you can. And I think whilst it would be a heck of a disappointment for them, I think it’s just a civil duty to share that with everybody. 
Lester raises two further concerns affecting levels of trust in commercial research: the profit motive, and the question of whether trial results are fully published. Taking the profit motive first, several people expressed worries about the pharmaceutical industry’s need to make money out of drugs and how this affected the kind of research carried out. Celia wanted more government research funding instead, whereas Julian felt this was unlikely to be achieved.
 

Celia argues for more government research funding. She feels drug companies tend to fund research...

Celia argues for more government research funding. She feels drug companies tend to fund research...

Age at interview: 67
Sex: Female
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Well, yes, I mean I think more money does need to be put into research definitely. When you think of all the money that they waste on Trident and wars and all the rest of it. You know, I just think there’s so many more important things in life. And more money does need to be put - and, you know, they, they rely so much on the charitable organisations, Cancer Research and all the different charities - which are great and we need them, and that’s all right, because that also spreads the word to ordinary people about research, when they hear about what the charities are doing. But I still think it’s the government’s responsibility. And they have the access to the really big amounts of money.
 
What about drug company-funded trials? Do you have any worries about that?
 
Yes, I mean obviously, because they’re only interested in making money, as far as I’m concerned, which is okay, again, if they do get a breakthrough and, and, and come up with some really good treatment. But the trouble with them is that they put money into providing treatments for the same diseases. In other words, they just change, you know - for instance look at high blood pressure. There’s so many different treatments for high blood pressure, and, and we don’t need them all, do we? As far as I know we don’t need all of these different treatments, and they only go in for new treatments for the same disease because they can make more money out of it. 
 

The costs of developing new drugs are high, so Julian thinks investment from drug companies is...

The costs of developing new drugs are high, so Julian thinks investment from drug companies is...

Age at interview: 61
Sex: Male
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Some people worry about trials being funded by drug companies. Is that an issue for you or does it not worry you at all?
 
No, I don’t think there’s any way of avoiding that. I mean, the sums involved in developing a new drug are enormous, so the estimate is for every drug that succeeds - I mean you must know this better than I do - every drug that succeeds and makes it to market, the cost is something like half a billion pounds, depending a little bit.
 
So that’s an enormous cost which is paid by the drug company that develops the drug. And I suppose you could have government financing that, but as things are, without enormous restructuring of the way that medical services and medical research are funded, it’s unavoidable that drug companies must foot the bill.
 
I suppose the thing that I do worry about, that again is being tackled now in various ways, is the unfairness of allocation of resources. So there are drugs which are desperately needed to treat infectious disease in the third world, for instance, which could benefit enormous numbers of people, which, until recently, have not been developed because the rewards from developing drugs for poor people in poor countries are rather small, even though the benefit of the drug may be huge. But I think foundations like the Gates Foundation are beginning to remedy that problem by providing incentives for drug companies to develop those drugs. 
However, Caroline noted that funding priorities are influenced not only by drug companies but also by powerful charities with interests in specific conditions. (See also ‘Under-researched topics/priorities for other research’).
 

Caroline argues that the research agenda is partly driven by the interests of drug companies and...

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Caroline argues that the research agenda is partly driven by the interests of drug companies and...

Age at interview: 42
Sex: Female
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I do think there are important issues to do with the funding and where it comes from, because obviously there are forces at work, the drug company, who have an enormous vested interest in people being ill, because if nobody’s ill, nobody takes any drugs. So they are not interested in funding preventative healthcare, because for them they’d go out of business. They’re the big players when it comes to funding healthcare research. And it’s very difficult to find funding from other bodies for stuff that isn’t to do with drug intervention. And, you know, it falls to the government to fund that kind of thing, and charitable organisations. Government, yeah, great, they could potentially be quite even handed. Charities are not. So for example if a charity is a particularly sexy charity that attracts a lot of money, and organisations that deal with children typically do that, organisations that deal with old people typically don’t. So those charities have lots of money to spend, the successful, well-known, sexy charities, and obviously they want to spend it on their particular thing that they deal with. They’re not going to fund research on, you know, something that’s nothing to do with what they support. So that is an immense problem, because those particular charities don’t necessarily serve the needs of the biggest number of people. I mean if you looked, I don’t know what the demographics are, but you could argue the case that the healthcare needs for old people are the biggest number, the biggest group of people requiring healthcare, and that’s where the money should be poured. But it doesn’t work like that, and so there are lots and lots of funding issues for that kind of research.
 
Whereas things like research into a new drug treatment for stroke, you know, obviously lots of money there. Things like research into drug and drug therapies for MS [multiple sclerosis], lots of money there, because organisations like the MS Society are huge and strong and attract lots of funding because they’re very efficient at fund-raising. So, yes, there are lots of issues to do with funding. 
Some people argued that investment from drug companies was vital, and said they had few concerns about their involvement, or at least wanted to keep an open mind. Anthea commented particularly on Phase 1 trials, “If the companies weren’t funding them, they wouldn’t be being done. There’s no way that the National Health Service or anybody else could afford to do these trials.”
 

Tony thinks there are conspiracy theories about the role of the pharmaceutical industry, but he...

Tony thinks there are conspiracy theories about the role of the pharmaceutical industry, but he...

Age at interview: 74
Sex: Male
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I know that there is a great, shall we say, conspiracy theory [laughs] about big pharma, and up to a point I keep an open mind about this. It is big business. There are multi millions of pounds involved. And I don’t for one second begrudge the profits that the big pharmaceutical companies make, because I know how much it costs them to start new drugs. Whether they sometimes abuse the relationship they have with some medical practitioners, I’m in no position to judge or pass comment. I can see that it could happen.
Another concern raised by Lester (above) is the issue of publishing trial results. There are concerns that research teams are less likely to publish the results of trials which show the intervention did not have the effects they hoped it would have. (These results are sometimes called ‘negative findings’). Many people think drug companies have an extra reason to keep negative findings quiet, because it may affect their sales and profits. There was a lot of support for regulations to insist that all results are made public.
 

Amanda worries that commercial trials may suppress ‘negative’ findings. Good research is not...

Amanda worries that commercial trials may suppress ‘negative’ findings. Good research is not...

Age at interview: 54
Sex: Female
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Well, most trials, drug trials, are funded by the pharmaceutical industry, and I find that very worrying because they own the data then, and they can control the data. There was one rheumatoid arthritis drug called anakrina, for example, that we systematically reviewed, looked at the scientific evidence, and the drug company had suppressed some of the evidence. There were three trials that they used to get the drug licensed with about a thousand people in, which suggested it worked, and then after it was licensed for use there was a great big trial, bigger than the other three trials put together, a safety trial, in which they wouldn’t publish whether it worked or not, the efficacy data. In fact they claimed they hadn’t measured it, but we did have the protocol so we knew that wasn’t true. And it turned out, you know, they, they just suppressed that data. They claimed it hadn’t been measured. They said, “Oh well, you couldn’t, this was a trial done in a different population. This was now done in a standard rheumatoid arthritis population instead of a highly selected population, and therefore it was too confounded to be usable.” But it was a randomised controlled trial, so the two groups were comparable. So my conclusion was that actually this didn’t work, if you use it in ordinary people, a drug that they were trying to promote for use in ordinary people. So yeah, the fact that trials are published by the pharmaceutical industry our defence has to be that the data is made public domain. But I, I think the government should be funding more trials. I think participation in research should be just built in to the health service, in such a way that it becomes very cheap and painless. You know, there’s not huge costs because part of the way we routinely collect data anyway.
 
What are the obstacles to that at the moment, do you think?
 
Research is not cheap. It costs. I mean, it isn’t cheap because it has to be done well. I think there’s absolutely no point doing research badly. I mean, I’ve seen people put together protocols and then cut down their sample size* calculations because it’s coming out at too costly. But I actually think what’s, you know, £2 million sounds like a lot of money, but if you’re preventing a lot of deaths it actually becomes quite trivial in terms of health costs. So no, I think it should be nationally funded, and I think it, more should be funded.
 
*FOOTNOTE' The number needed for each trial (the ‘sample size’) is proposed by statisticians doing ‘power calculations’. In some cases a small number of participants may be enough, but if you are looking for fairly small changes in outcome you will need large numbers of participants to pick these up reliably. 
 

Amanda was a member of a research ethics committee and was surprised it was not seen as not part...

Amanda was a member of a research ethics committee and was surprised it was not seen as not part...

Age at interview: 54
Sex: Female
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I think it’s desirable that people should know the results, I mean, that’s why we go into it. I personally feel worse, what’s worse, and is for a trial to come out and then not get published at all, you know, rather than the individuals being informed. And interestingly enough on the research ethics committee I’ve sat on and I’ve tried to bring up, “They have no publication policy, they have no strategy for publishing this trial” and being told that we’re not allowed to discuss that. Whereas I always think it ought to be built in that this data will be put into the public domain. I mean, I have a colleague who works in another country who was part of a multicentre trial for a major pharmaceutical company, and in the trial protocol it said results will be published within, I think it was 24 months of the close, after recruitment of the last patient. And they added into their, their centre, “Should the results not have been published within 24 months of the closure of the trial, our centre reserves the right to publish the results for this centre”, and they were excluded from the trial, because they insisted on that. They were told that could not be put in. Well, if they’re going to be published within 24 months, why could that not be put in? And so they refused to do the trial without that in. And they, and they - so that’s, that’s an insight. So who owns the data? I think the data ought to be owned by the people who are in the trial.
 
So I like the new strategies that are coming out, that data has to be put into the public domain and it should be a prerequisite, certainly for funding from government bodies. You know, if our government’s going to fund a trial, well, let’s have the results, and I - more than just the results, let’s have the data, suitably anonymised to protect patient confidentiality, in the public domain to be re-analysed, because even in the analysis there could be bias. So, I’m a great believer in transparency.
However, Kate, who works for a healthcare charity, thinks that drug companies have acquired an unfair reputation. Most research teams will want to publish their findings as articles in academic medical journals, which get independent experts to review the articles before they are accepted (a system known as peer review). Kate thought sometimes peer reviewers were more likely to find fault with drug company research because there is “a bit of a stigma attached to pharmaceutical companies”.
 

Kate's charity worked on a study with a drug company, and they found it really difficult to get...

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Kate's charity worked on a study with a drug company, and they found it really difficult to get...

Age at interview: 38
Sex: Female
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I’m kind of on the fence when it comes to drug companies, because we’ve done quite a lot of work with one particular drug company with one particular drug that was shown to have benefit. But because the, the drug company did the research, there’s all these question marks about, “Is it just because it’s that company?” You know, they’ve got a bad reputation for hiding bad results and only publishing positive things and possibly skewing their results. So we did an independent audit of this particular drug and we found it did confer benefit. But getting that study published in the scientific journals has been an absolute nightmare. It’s taken about nine months of constantly responding to very minor reviewers’ comments, until eventually the editor of the journal just went, “Oh, just publish it.” Because it had the association with the pharmaceutical company, even though it was completely independent, they had nothing to do with the study, we really struggled to get the work published. And it is shown to be of benefit. So there is, yeah, a bit of a stigma attached to pharmaceutical companies.
 
That’s interesting, that it’s actually thereby preventing results from coming out, rather than the company trying to suppress results.
 
Yes. I think that there was, there have been a few dodgy dealings, I think, in the pharmaceutical industry, and I think it’s really tainted their image and made things difficult for themselves, which is unfortunate. Because I think, you know, they do good. But it’s just the whole, “They’re there to make a profit” that makes it a little bit uncomfortable. 
Problems in publishing ‘negative’ findings may not be simply to do with who funds the research, however. Publicly funded researchers may also find it difficult to be honest about ‘negative’ findings, as they may feel it will affect their career and their academic reputation. They may feel very passionate about a new treatment they have developed and be unwilling to admit that their hopes for it have been disappointed.
 

It takes a long time for most trial results to be published. Sometimes when results seem...

It takes a long time for most trial results to be published. Sometimes when results seem...

Age at interview: 59
Sex: Male
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The trial that I was involved with lasted for three years and it’s finished now. And what happens is that all the data is collected. It’s anonymised, that’s to say none of us ever know who the individuals were. It’s just on numbers and averages. And that goes through an independent committee. And then the results are published, really.
 
Now, there are quite a lot of issues with all of that. Number one, it takes a long time for data to be published in a trial. And that’s one of my disappointments, if you like, that because people quite rightly are frightened to jump to early conclusions, they say, “Oh, we’ve got to get all the data in before we publish.” Well, sometimes if you’ve got a three-year trial or a four-year trial, you might know after a year what’s really going on, but people very rarely publish it. And that then puts the people going into the trial at years two, three and maybe four at a disadvantage, because more is known about the likelihood of success than what they may be told. So I wish there were ways in which data could be more freely available. I think - and I’m not talking necessarily about my own experience - but I think one of the risks is, if you’ve got a group of people who are absolutely passionate about a trial for a therapy that might work, if in the early part it isn’t giving them what they expect, there was a very natural, understandable reluctance to publish that or to tell anybody. You just sort of keep on, “Well, maybe this is a bit of a tweak. Maybe it will get better.” I feel almost guilty saying that, because that’s making them feel that they’re less than honest. They’re not. They’re just human. And I think it’s absolutely understandable. I could sort of take it back to my time in the bank. If we were trying to sell a particular - if my team, my colleagues were trying to sell a particular mortgage and it wasn’t selling, you wouldn’t sort of very often look back and say, “Gosh, is the mortgage right?” You’d just try and keep selling it a bit harder and hope it would go through and ignore the customer complaints. You know, you’d set up a call centre. It’s human nature. You don’t do it out of nastiness. It’s just how it is.
 
FOOTNOTE' Researchers are right to be cautious about jumping to conclusions too early in a trial. Large trials can take a long time to get enough people and enough data to draw reliable conclusions. However, sometimes results are so obvious that a trial has to be stopped early, either because the one of the treatments is strikingly better than the other, so it becomes unethical to withhold it, or because risks are revealed that are too great.
(There is a dilemma here – researchers are right to be cautious about jumping to conclusions too early in a trial. Large trials can take a long time to get enough people and enough data to draw reliable conclusions. However, sometimes results are so obvious that a trial has to be stopped early, either because one of the treatments is strikingly better than the other, so it becomes unethical to withhold it, or because risks are revealed that are too great).
 
Even if researchers do submit reports of ‘negative’ findings, academic journals are sometimes less likely to accept them for publication compared to articles reporting ‘positive’ findings. New breakthroughs may be seen as more exciting reading than a report that a treatment does not have the effects hoped for and is not worth pursuing.
 
But ‘negative’ findings are just as important and valuable as ‘positive’ findings. They can mean that future patients are not exposed to unnecessary risks from treatments which do not work, and that money can be better spent elsewhere. In recent years there have been a lot of efforts to encourage researchers and journals to think differently about ‘negative’ findings.
 
See also ‘Feedback of trial results’.

Last reviewed September 2018
Last updated July 2011

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