Pre-screened patients on standby for early-stage clinical trials

Highlights

  • CHDR’s biomarkers and test batteries provide proof-of-pharmacology results in the early stages of drug development. Because pharmacodynamics and pharmacokinetics profiles can differ between patients and healthy subjects, early testing in patients is essential.
  • To resolve the bottleneck created by patient recruitment, CHDR establishes pre-screened groups of patients who are ready and willing to participate in a study.
  • As soon as a sponsor is ready to test a product in this patient population, CHDR is ready with preselected patients.
  • By combining Ready-for-Research with our novel Monocentre approach, all patients participate at one central location, thereby minimising variability by using validated test batteries and biomarkers.
  • CHDR collaborates closely with patient advocacy groups and physicians, ensuring that everyone benefits from this innovative approach.

Summary

Rather than recruiting patients for a given clinical study on an as-needed basis, Ready-for-Research establishes a pool of well-defined patient groups who are ready and willing to participate in new trials, significantly reducing recruitment time and patient screening.

With Ready-for-Research, we recruit patients who have specific medical conditions and are interested in participating in drug studies. We then screen these patients using specific protocols that have been approved by an ethics committee. With Ready-for- Research, patients are invited to our facility at CHDR. After providing written informed consent and a full medical history, patients receive a comprehensive physical examination, including blood work, and we test their performance on some of our test batteries (for example, NeuroCart®). The experience is like visiting an outpatient clinic, and we try to make the patients as comfortable as possible – after all, they are making the effort to come to us, with no tangible benefit to themselves aside from a modest payment for their time. Depending on their specific medical condition and disease stage, patients can return to CHDR on a regular basis (for example, annually or semiannually) for re-screening in order to assess their condition, general health, and other possible factors that may affect their ability to participate in an upcoming study.

Once a sizable cohort of patients with the same diagnosis has been established, this cohort is now ready for research. Sponsors with an interest in studying this condition can approach CHDR to discuss a clinical trial. And of course, CHDR can approach potential sponsors who are developing drugs for this target patient group. We can offer sponsors a relatively large cohort of patients with a well-defined set of symptoms. All of the patients are available for participation at the same facility using the same procedures. In this respect, patients are recruited even before the trial exists. This method is an important addition to our Monocentre approach.

Practical answers to important research questions

  • Why patients?

    At CHDR, we believe that patients should be included as early as possible in clinical drug research. Prof van Gerven explains the underlying philosophy. ‘You can never be absolutely certain that patients will have the same pharmacodynamics and pharmacokinetics profiles that you measure in healthy volunteers. Indeed, the very system that the drug is designed to target is often altered by the disease.’ He adds with an emphasis that CHDR focuses on what he calls proof-of-pharmacology, showing that the drug affects the pharmacological mechanism, not necessarily the disease itself. ‘A drug is designed to have a specific pharmacological activity,’ adds van Gerven. ‘This is a clear prerequisite for a therapeutic effect. Unfortunately, many drugs fail in clinical trials because their pharmacological activity cannot be ensured. When you interpret the outcome of a failed trial, it makes a large difference if you can demonstrate whether the drug had – or did not have – the expected pharmacological effect in patients. So we need to develop better ways to measure these effects in patients.

    In many ways, conducting research with patients is more challenging than using healthy volunteers. For example, patient cohorts often have more variability and can be more difficult to recruit. ‘Once you have the approved protocol,’ says van Gerven, ‘you –and your sponsor – want to proceed with the study as soon as possible. So you usually don’t have much time to recruit the required number of patients. That is why even in the early phases of development, most studies are multicentre trials, which increases patient numbers and the likelihood of achieving sufficient recruitment. On the other hand, the selection criteria often require a compromise, resulting in patient characteristics that do not necessarily reflect clinical reality. In addition, it’s often impractical – or even impossible – to provide all participating centres with the more complicated tests needed for proofof- pharmacology. For example, even though our NeuroCart test battery is designed to be fully portable, we may not be able to provide all participating centres with the necessary training. Thanks to our Ready-for-Research programme combined with our Monocentre approach, NeuroCart can be used on all patients in a single study, as they will all be at the same location.’

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