Clinical Trials – proportionate regulation is necessary but not sufficient

Posted on May 2, 2013 by


Last week I gave evidence to the House of Commons Science and Technology Committee enquiry on clinical trials, alongside Cancer Research UK, Wellcome Trust and the Medical Research Council. Our earlier consultation response is here.

The exam question set by the Committee was a pretty complex one, going something like this “What are the barriers to conducting trials in the UK and will the proposed new regulations make things any easier? And how can we create a more open and agile research environment that is both more productive and where public involvement is greater and public confidence is higher?”

How to do more clinical trials

This is a really importance topic to many AMRC members. Over a third of our member charities are currently funding clinical research – that might be drug trials, new surgical techniques, diagnostics or other interventions – and AMRC members fund 32% of 2600 studies in the NIHR portfolio. Of these, 14% (361 studies) are CTIMPS (clinical trials of investigational medicinal products), which are regulated by EU clinical trials legislation and so getting the regulation right is important – but even more important are the other practical things we can do within the UK to ease the progress of clinical research and trials.

We know for example that in some areas of research there is simply insufficient research capacity in the UK for charities to fund – 44% of Action on Hearing Loss research budget was  spent overseas in 2011 because of a lack of researchers in the UK working on hearing loss. And we know that research is an international business and that charities want to fund the best science for the benefit of patients and sometimes that is overseas. So we need to make sure that the environment in the UK really does help research to happen in our institutions and that we can attract the best talent to come to British universities.

Finding good researchers is perhaps half the battle but a clinical trial is nothing without participants.  Almost three quarters of the public have told us they want to take part in the research but they often have no idea how to become involved. And this is borne out by a recent NIHR “mystery shopper” survey, which found that found that 91% of hospitals did not have any public information about trials occurring there and of the 40 Patient Advice and Liaison Services (PALS) only 3 had any information on research.

There is a fantastic resource in the UK Clinical Trials Gateway – when I Googled clinical trials as a bit of my own research last night this great site came up in 0.14secs. But another NIHR Patient Survey showed that that 80% had not heard of UKCTG. So clearly the website needs more promotion and patients need to know that UKCTG is open for business.

Regulations are important but the world is changing

We hope that both the proposed EU Clinical Trials Regulation going through EU Parliament and the HRA and its NHS regulatory pilot currently underway make trials easier to conduct without compromising safety. But it is clear that the regulatory framework needs to be more agile and flexible to cope with emerging novel therapies, for example the combination of drugs and devices or biomarkers and the smaller sample sizes and more personalised medicines associated with stratified medicine and pharmacogenetics.

The outcomes of research should be public

The committee were very interested in the issues of what happens to research once it is completed. My fellow panellists and I all agreed that trials should be registered. In fact it has been long standing AMRC advice that charity funders should require in their T&Cs the publication of the findings of research within a suitable time frame (such as 1 year after completion – see our guidance Charities and medical research). In our recent survey we found that 80% of our members who conduct clinical research told us that they include a requirement in their T&Cs that research results should be published and 62% follow this up to check. We are looking at further ways to help charities monitor and enforce this.

And we know that negative findings can also really illuminate research questions – but we don’t yet have an obvious way to do that. But just publishing findings won’t be enough; making the outcomes of research public means also making those outcomes easily understandable to a lay reader. There are some fantastic examples of this, such as CancerHelpUK.

Are CSRs the answer?

The committee asked if wider use of Clinical Study Reports (CSRs) to the regulator would be helpful. CSRs are formal reports by the drug trial sponsor, usually a pharmaceutical company, university or hospital, which are a requirement when the study leads to drug licensing . These confidential reports contain detailed information right down to individual patient health records. Glenis Willmott, the MEP leading the new Clinical Trials Regulation through the EU Parliament, has recommended that CSRs should be a requirement for all studies covered by the legislation. We have some concerns about this:

  • It places a huge burden and cost on academic researchers, especially those conducting small-scale or early trials (which do not currently require a CSR), as they do not have the resources or expertise to produce them
  • These are vast documents running to many many volumes. They really are unlikely to be a helpful tool in practice for making research findings more easily or widely understood
  • And there is a big question about how to protect the identifiable personal data these reports hold.

I believe that it is important that the outcomes of research are shared more openly. The public has often funded these projects and they should be able to see the results. More open dissemination of outcomes including negative findings might also reduce study duplication and encourage collaboration. And  providing researchers with access to underlying data for further research can also speed medical advance. But we have to make sure that public information is in a clear and readable form, and that data is shared without compromising patient confidentiality.

Posted in: Policy, Research