August 5, 2014
The authors of an article published in Value for Health regret that classical clinical trials generally do not represent patient interests and rarely inform participants of results during the study. Under such conditions, patients often lose interest and drop out of trials. Experience shows that participants’ active involvement in the design and progression of clinical trials results in greater patient retention and more meaningful results. Informed patients are more willing to engage in time-consuming and effort-requiring studies as they feel valued, empowered and capable of assessing therapeutic options.
Mullins et al. suggest methods of designing trials from a patient’s point of view rather than the investigator’s. Pragmatic, Bayesian statistics and adaptive trials can improve patient safety and increase recruitment and retention. Pragmatic trials are designed to result in outcomes that are most relevant and beneficial to participants. Bayesian statistics draw on collected evidence to update knowledge on the treatment and outcome probabilities. This approach aims to produce results that are easily interpreted and reflect the reality of medical decision making. Adaptive trials also evolve on the basis of data accumulated during the trial and allow patients to be transferred to a more effective treatment if it becomes available once the trial has begun.
The three trial designs are particularly relevant for rare diseases as they are more likely to retain already limited numbers of patients whilst potentially offering them early benefits. Nevertheless, the authors highlight several limitations: pragmatic trials are designed to reflect a ‘real world’ situation which is difficult to quantify and qualify; Bayesian statistics are resource intensive; and adaptive trials might not offer the required evidence for regulatory approval. To truly qualify trial designs as patient-centered, Mullins et al. emphasize the primary need for sustained efforts to inform and involve patients and advocates at all stages of clinical studies.