Tag Archives: medicaltrials

There will never be enough trials – additional innovation is needed


By Professor David Walker

Professor of Paediatric Oncology Faculty of Medicine Health Sciences, University of Nottingham

There will never be enough trials – additional innovation is needed I was at a meeting recently with clinical academics, charity chief execs, health regulators, representatives of the pharmaceutical industry – and one lone voice that cut to the heart of the matter.

That voice was Debbie Binner, whose teenage daughter had died of a rare bone cancer, Ewing’s Sarcoma. Debbie was the reason we were there. When her daughter, Chloe, died she had just turned 18. A few months short of her 18th birthday, the chance of a drug trial was offered.

But Chloe needed to be 18 to be eligible and was refused entry. Our meeting, held in the House of Commons with Paul Burstow MP in the Chair, was to discuss how we, the experts, could ensure that younger people had access to more trials of new therapies for their life-threatening conditions.

Many speakers spoke about new ways to reform the regulatory system so that it could work better for young patients. We heard about changes in the EU Clinical Trials Directive that is being enacted in Brussels, that may help.

Listening to my colleagues, I was struck by one thought: that if you put people in a room who are expert on a regulatory process they want to make better, but inevitably more rules.

What is needed is a feeling that we can work for the patients’ benefit using our established professional values and standards rather than a raft of ever more complex rules. Research does require regulation to introduce new drugs and treatments safely. That is true. But trials cannot be the only answer.

There will never be enough trials, they take time and money, there are rarely trials for the less common diseases. So when patients are in a situation with “nowhere else to go” and if there is no trial for which they are eligible, they need to be able to try treatments that might work, based upon the best judgment of their medical advisors. We need to allow them to try such new drugs that may be applicable and collect that experience to inform the next generation of trials.

The Saatchi Bill would do this, by protecting individual doctors who try new, licensed but untrialled treatments, on patients who have consented to such treatment outside of a formal trial. This cannot be – and isn’t – a license for the maverick doctor acting alone.

The doctor would also need to share the decision with peers as part of the multi-disciplinary teams that already exist in modern health systems. The Saatchi Bill would meet the needs of pharma and academia – but most importantly the patients and their families who need to be given additional choice – and experience the associated hope.

If organised with light touch this could offer valuable information to researchers whilst sustaining hope for families that “no stone is being left unturned”.

The Saatchi Bill offers a framework where practitioners would be supported in exploring innovative therapies, whilst building evidence of their applicability and effectiveness as part of the process of acquiring the evidence necessary to launch the next generation of trials. This is why I commend the Saatchi initiative.

→READ Professor Walker’s biog

→READ the full story in the Telegraph

→WATCH Lord Saatchi explain the Bill