If my patient Jimmy had been diagnosed with cancer back in the 1980s, his prognosis would have been grim. Jimmy is eight months old and has hepatoblastoma, a rare type of liver cancer; in the past the only hope of cure was surgery (if possible), together with old, ineffective chemotherapy treatments.However, new drugs, tested in a series of clinical trials throughout Europe involving more than 500 children, have dramatically boosted survival rates in less than 10 years. Jimmy’s chances of recovery are now 75 per cent, up from 30 per cent.
As a consultant who has treated children with cancer for more than two decades, I have seen the difference trials have made. Every year, 1,500 children are diagnosed with cancer in Britain. 40 years ago, a quarter were expected to survive – now four in five will do so, as my co-authors and I reported in the Annals of Oncology last week. But my concern is that unwieldy EU regulation is now threatening the advances we have made – and future progress.
A clinical trial compares current treatment with a modification of that treatment or with a new one that doctors and scientists hope will work better. Survival rates can be boosted – as we see in cases like Jimmy’s. It also means that we can develop treatment regimes with less severe side effects.
I’ve just completed a 10-year international clinical trial into Wilms’ tumour (a kidney cancer), in which more than 4,000 children took part. One of the drugs used to treat it can cause heart failure in later life. We’ve been able to show that an additional 25 per cent of children with Wilms’ tumour don’t need this drug. We also discovered a new sub-type of Wilms’ tumour that benefits from stronger treatment; we are now doing more molecular tests to see how many sub-types of tumour there are so that we can personalise each child’s treatment further.
But the 2004 EU Clinical Trials Directive is putting such breakthroughs at risk. It was brought in with the laudable aim of harmonising trial methods and setting standards for administration. The impact is different; the number of new clinical trials in Europe between 2007 and 2011 dropped by a quarter.
The reasons? First, the directive applies the same standards of safety reporting to drugs that have been used for decades as drugs never used in humans before. So every time a child enrolled in a trial is admitted to hospital – even if it is nothing to do with the trial – this must be reported, generating a huge amount of paperwork.
The directive has also made it mandatory for each country to ensure participants get anti-cancer drugs free of charge. In Britain, the NHS provides standard drugs for free, and we work with drug companies to provide new drugs for children’s cancer trials. But in Poland or Hungary, for example, the institution leading the trial is expected to pay treatment costs, which most cannot afford.
Added to this is the EU’s insistence on insurance. In Britain we have NHS indemnity insurance but in other countries the cost has gone up 100-fold.
Bureaucracy has slowed everything down. Yet pharmaceutical companies with drugs to trial want partnerships with institutions that can get a study running in 60 days. We’re a long way off that. So instead such companies are going to North America, China and India. That means children in Britain may not get new drugs for 10 years or more because we won’t have early access to information that shows which treatments are working.
I wholeheartedly support current efforts to reform the directive. We need to ensure a more proportionate approach to risk, more flexibility and less paperwork. Most of all, drugs that are in habitual use should no longer be subject to the full force of regulation.
If this doesn’t happen, we’ll be stuck using standard treatments rather than finding new ones. We’ll see too many children with rarer cancers continuing to die, and others enduring serious side effects such as hearing problems and infertility.
I’m not sure the trial I’ve just completed would have gone ahead if the directive had been in place a few years earlier. It certainly would have taken much longer to set up. But it’s already making a big difference to many children with kidney cancer – and has given us vital information for the future.
Kathy Pritchard-Jones is professor of paediatric oncology at Great Ormond Street Hospital and the University College London Institute of Child Health
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