Naturalistic drug effect studies. Trial@Home
Traditional relationships between doctors and patients have changed. Patients are better educated than ever before, and want to take responsibility for their own lives. Although most patients value the relationship with their doctor, many now want to be informed and coached about health issues, rather than ‘treated’ by a benevolent and knowledgeable, yet somewhat paternalising physician. These changes will also affect clinical research.
Many patients with untreatable conditions are looking for alternative therapies. From the patient’s perspective, the risks associated with an unproven treatment understandably become less important if the disease is severe enough. The internet is a ready source of all types of compounds, often of dubious origin and quality. Access to experimental therapies is subject to strict regulations, and it is still difficult for a patient to be entered into a trial. Some patients now claim their right to decide for themselves, how much (unknown) risk they are willing to take with new unproven treatments. Organizations like MyTomorrows offer experimental drugs to patients who cannot be helped with regular treatments – outside of a clinical trial. Unfortunately it is not in the patients’ benefit if new therapies are used in practice, without first being properly studied. Nonetheless, it is understandable that patients are looking for ways to accelerate the availability of new treatments – and researchers will have to respond.
Patients and healthy people are also taking responsibility for monitoring of their own health. An increasing number of genetic screens and laboratory diagnostics are offered to patients, often with medical advice. Miniature medical applications are increasingly sensitive, and large companies like Apple already offer apps and portable devices, which are able to constantly monitor physiological or behavioural indicators of healthy and performance in daily life. Unfortunately, the reliability of many of these applications is still unclear.
Over the decades, CHDR has devoted much of its time and resources to the development of tests for drug effects and disease. We use this experience in the collaboration with a number of technical parties in the joint development and validation of applications that patients can use at home, during the conduct of a clinical trial. Some examples include smartphone apps to photograph skin lesions, for new drugs in dermatology; small portable devices that continuously measure vital functions, like the Vital Connec®; and the Mini-NeuroCart®, which is based on CHDR’s drug-sensitive multimodal CNS test battery, made suitable for studies ‘in the field’. In addition, CHDR also develops tablet-based apps for ambulant patient instructions and effect measurements. And this is just part of our Trial@Home initiative, which aims to perform highly informative, data intensive studies under naturalistic conditions – during attacks of recurrent diseases, or for chronic drug effects in the comfort of the patient’s home, in their own bed at night, in the office, or any other situation that is affected by the compound – not just in the clinical research unit. The time has come that patients and researchers can team up to improve clinical research.
by Joop van Gerven