Patient-Reported Outcomes (PROs) were either aimed at assessing and monitoring symptoms or quality of life measurements in later stages of product development or in post-marketing, real-world settings. Only recently have PROs found their way into a patient-centric product development approach even in early stages of clinical trials, i.e. phases I and II, respectively. In phase I, when the maximally tolerated dose of an oncology product candidate is being assessed, physician-recorded outcomes have shown to be less reliable than if patients themselves report the tolerability of a drug candidate.

Fig. 1 Study design for the application of tablet-based PROs in Phase I Clinical Trials

AEs = Adverse Events, PRO-CTCAETM = Patient-Reported Outcomes Version of the Common Terminology; Mid-C1Mid-C2 = mid-cycle 1, 2

(Figure taken from 3)

The use of PROs, especially PRO-CTCAETM, in early phase clinical trials can provide data which are more reliable when the aim is to objectively determine symptom-toxicity. Thanks to technological advantages, assessing PROs in real time by, for instance, Wearables and Apps has become much more feasible and convenient.  

Source:

  1. Basch, E. and C. Yap, Patient-reported Outcomes for Tolerability Assessment in Phase I Cancer Clinical Trials. JNCI: Journal of the National Cancer Institute, 2021. https://doi.org/10.1093/jnci/djab017 °

2. Acquadro, C., et al., Incorporating the patient’s perspective into drug development and communication: an ad hoc task force report of the Patient-Reported Outcomes (PRO) Harmonization Group meeting at the Food and Drug Administration, February 16, 2001. Value Health, 2003. 6(5): p. 522-31.   https://doi.org/ 10.1046/j.1524-4733.2003.65309.x °

3. Veitch, Z.W., et al., Underreporting of symptomatic adverse events in phase I clinical trials.JNCI: Journal of the National Cancer Institute, 2021. https://doi.org/10.1093/jnci/djab015 °

4. Kennedy, F., et al., Online monitoring of patient self-reported adverse events in early phase clinical trials: Views from patients, clinicians, and trial staff. Clin Trials, 2020: p. 1740774520972125. https://doi.org/10.1177/1740774520972125  

5. Chen, A.P., et al., Chapter 13 – Incorporating Patient-Reported Outcomes Into Early-Phase Trials, in Novel Designs of Early Phase Trials for Cancer Therapeutics, S. Kummar and C. Takimoto, Editors. 2018, Academic Press. p.