Clinical studies in paediatric oncology

Few topics in medical ethics divide laypeople and medical professionals into two camps as quickly as the discussion about clinical trials involving children. They often raise the following questions:

Are clinical studies in children necessary?

Do children benefit from clinical trials?

Or do children need to be protected from the experimental use of medication?

A conflict of interest between protection and care

This highlights the conflict of interest between ensuring the protection and care of patients who are unable to make autonomous decisions and the right of children, in particular, to also benefit from the development of new medications.

Two-thirds of all drug applications in inpatient paediatrics are prescribed without reliable data on dosage, tolerability or efficacy and without formal approval regarding age, dosage and dosage form, and are therefore prescribed under the personal responsibility of the treating physician.

One in ten medicines used in outpatient care is prescribed off-label. The proportion of unlicensed medications is highest in neonatology, at around 90 per cent. This situation led to the term ‘therapeutic orphans’ being coined in 1968.

These essential aspects of paediatrics have long been neglected. It was only the developments of recent decades that led to a new awareness, which is reflected in changes to the legal framework governing clinical research. This also meant that the framework conditions for clinical trials, particularly those involving children and adolescents, were adapted.

Today's survival rate is over 85 per cent

Therapy optimisation studies have formed the basis of clinical oncology research in paediatrics for decades and are largely responsible for today's average survival rate of over 85 per cent.

Cutting-edge treatment option

Patients treated in clinical trials benefit from

• Cutting-edge treatment options,
• A patient insurance policy,
• An ethical assessment of the rationale behind a study,
• Collective knowledge gathered in study centres and, above all,
• A so-called reference assessment, which almost completely rules out misdiagnosis.

A resounding ‘Yes’ to clinical trials

In summary, it can be said that children not only benefit from greater safety in the use of clinical trials, but also gain access to innovative therapeutic measures that would otherwise be reserved for adult patients.