The primary motivation of oncology patients for entering a trial is the possibility of tumor shrinkage. The expectations are high, with 47% of participants expecting tumor shrinkage and 14% expecting a cure. However, with a typical phase I response rate of 4 % to 20 %, there is a huge gap between expectation and reality.


Drug development is a long and expensive path for pharmaceutical companies. After rigorous experimentation on isolated cells and animal models (pre-clinical studies), the very best drug candidates are evaluated in clinical trials. A phase I clinical trial means that the treatment is given to a small number of human participants for the first time. This essential process allows to determine the dose and schedule of the treatment and to evaluate the toxicity and rate of elimination of the drug by the human body. Phase I clinical trials are essential for the improvement of therapeutic options for the patients. Advantages and drawbacks are associated with participation in those trials and it is mandatory for the physician to be completely transparent in informing the patient about risks and benefits of their participation. But what exactly motivates participants to accept entering a trial and what do they expect from it?

A group of researchers from the UK evaluated motivations and expectations of more than 300 participants in phase I clinical trials in oncology. Their results, published in the journal Cancer, showed that the motivations for participation were: the possibility of tumor shrinkage (84%), the absence of alternative treatments (56%), the physician’s recommendation (44%) and the belief that this research could help others (38%). Overall, 84% of patients were willing to enter the phase I clinical study. While 47% of participants actually expected tumor shrinkage and 14% expected to be cured, the therapeutic reality is very different. Over the last 20 years, the typical response rate of phase I trials in oncology is between 4% and 20%, with a median survival of 6 months. This short survival is due to the fact that only patients with advanced malignant disease, refractory to standard therapy, are included in those trials.

The discrepancy between expectations and therapeutic reality is not necessarily negative and keeping fate in the treatment is always a good thing.


Written By: Jean-Michel Bourget, PhD

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