Are clinical trials designed to benefit patients, or are they primarily intended to facilitate the registration of new medicines? Why are trials initiated by the pharmaceutical industry, physicians, and researchers—but not with patients?

Patients and patient advocates can offer invaluable insights into the real-world value of a medicine. Of course, overall survival is crucial – patients want to live to 87 and beyond. However, quality of life is just as important. So why not consistently include quality of life as an endpoint in trials? Why not let patients themselves provide feedback on their quality of life during and after treatment?

Another major issue in trials is the lack of real attention given to side effects. Why do they occur? Can we not design and develop medicines without them? Of course, we understand that many medicines are inherently toxic and may therefore have side effects. However, has the industry ever genuinely prioritised the development of treatments that eliminate or minimise these adverse effects?

Imagine if patients were involved in drug design from the outset and said: “We want a medicine that extends our lives by at least one year (on average) and has no side effects at all. If you cannot achieve this, we do not want your medicine.” Wouldn’t this lead to a fundamentally different approach to drug development? Of course, we might ultimately accept some side effects, recognising that medicines affect individuals differently. What is tolerable for one patient might be unbearable for another. But at the very least, let’s aim for medicines without side effects and see where that takes us.

Currently, clinical trials are primarily conducted to secure regulatory approval for new medicines, with minimal patient involvement. If patients were integrated into the design phase – helping to define the desired outcomes of a medicine – fewer drugs would likely reach the market, simply because patients would reject those that offer only marginal benefits at exorbitant costs. Quality of life would rightfully become a key outcome measure, as it always should have been. This shift would not only benefit patients but also serve society as a whole.

A powerful example of how multiple outcomes can be incorporated into trials is the One2Treat initiative. I encourage you to explore and support it.

Peter Kapitein
Patient Advocate Inspire2Live

One2Treat also has been a guest at our World Campus. You can watch their talk and presentation on ‘Shifting the paradigm of drug development with more patient-centric clinical trials’ >>