Pragmatic, real-world trials will bring treatments to patients
Translational research has succeeded in moving molecules from the laboratory into the clinic and further improvements would benefit patients and biopharm. Conducting studies in real-life settings to get real-life results, known as pragmatic research, is proposed to optimise the process.
“It is about bringing the right intervention to the right patient at the right time, and that is the essence of “pragmatic research””, says a piece published in the journal of Pragmatic and Observational Research.
To achieve this “long overdue” goalnew, innovative approaches must be adopted alongside expansion and improvements of current methods, says Abdul Rahman Jazieh, of King Saud Bin Abdulaziz University for Health Sciences, Riyadh, Saudi Arabia.
For instance, use of expanded-access programmes, which make investigational new drugs available to patients outside of clinical trials, should be increased. Using expanded-access could tap into a larger patient pool and provide data from a more real-world setting than trials.
Widespread use of expanded-access should be complemented by innovative study designs. These should produce results that are relevant to real-world patients and be conducted as early as possible in the regulatory process to support systemic collection of safety and efficacy data.
Particular efforts should be made to test medicines in different ethnic groups to assess the impact of pharmacogenomic variability prior to widespread use of a medicine. Globalisation of the pharmaceutical and clinical trial sectors has increased focus on pharmacogenomic variability.
Finally, Jazieh says research infrastructure should be revamped through increased use of practice-based trials. Using a network of doctors’ practices will increase the accessible patient population and “may help to identify the gap between recommended care and actual care”.
Implementing these changes is intended to increase the validity of extrapolating clinical trial findings across the broad heterogeneous patient populations that receive marketed therapeutics.
