Worldwide’s Dr. Murphy tackles questions on the minds of those working in drug development and clinical research.
Q: The pharmaceutical industry has been slow to uptake electronic solutions, despite their revolutionary potential. Many believe the industry is nearing a tipping point – do you agree?
A: It is said that the art of interventional clinical research is the ability to responsibly evaluate small molecules, biologics, and devices inherently unpredictable in terms of safety and efficacy, in the hopes of transforming therapy. Appropriately, the clinical trials process is one that attempts to mitigate risk, a sentiment that extends into the use of technology, making the cautious uptake of various electronic solutions a prudent business and clinical decision. Nevertheless, incremental changes in regulatory guidance and technology have converged with the need for efficient and informative trial designs, creating an imperative to re-examine the process.
Various regulatory agencies have introduced draft guidelines to remove uncertainty and shape the direction of technology, and the methods for application in the clinical trials process. These include the methods of oversight through risk-based monitoring, the use of electronic source data, and the ability to provide regulatory submissions using standardized study data. Where data acquisition platforms are not patient-facing, rules of engagement seem codified and less controversial, and adoption has been swift. For example, a cloud-based solution for data warehousing is readily suited to business documents. In contrast, similar solutions for patient-facing assessments are approached more cautiously and predicated on first addressing reliability, necessity, and utility for generating evidentiary standards for safety and efficacy. An ability to use technology intelligently in either observational or interventional research represents a challenges that the industry now fully embraces.
Q: How important is it to be able to use technological advances to share data acquired during a trial among patient participants and other collaborators, and what impact can this have on study outcomes?
A: Within the clinical trial setting, much attention is focused on the technological aspects of data acquisition and assurances of regulatory compliance. Historically, the flow of information is one-way; i.e., from the patient, to the clinician, to the database for analyses and apart from rare exceptions, the process is fully blinded to treatment group assignment. Utilization of technology to inform trial design prior to study inception or to communicate information to patients following study completion suggests possibilities that have not been fully exploited.
Prior to protocol initiation, for example, social media might be used to solicit ideas regarding protocol design before it is finalized, in order to enhance patient accrual and retention. Understanding the realities of patient management and the experience of the illness from the perspective of family and patients, assists in the resolution of conflicting data needs in the study design process. In contrast, during study conduct, technology promoting online conversation could jeopardize the integrity of the study. Anecdotes from trial participants may imply adverse events, lack of efficacy, or inconvenience, negatively impacting trial participation. These unstructured conversations, which can prove invaluable in the design phase of a project, may also inappropriately and inaccurately influence how symptoms are reported during the trial, distorting the implications derived from the resulting database.
Following product registration, quantitative study designs for quality improvement research could benefit from technology permitting distribution of information in a widespread fashion to patients receiving therapy in an adaptive process uniquely suited to that type of research. For example, stepped wedge designs sequentially rollout interventions to clinicians, physicians or organizations to monitor the impact of adoption of innovative therapy, while time series designs are useful in evaluating whether a quality improvement initiative has an effect in comparison to the secular trend relevant to this particular indication. In these settings, reciprocal exchanges of information between patients, sponsors and providers might facilitate the use of adaptive strategies determining the content and format of information most likely to influence patient and physician behavior.
The utility of sharing data, as well as information derived from those data, with study participants varies by phase of development (before, during, and after product evaluation), and the methods by which that information is structured.
Featuring insights from Michael Murphy, M.D., Ph.D., Chief Medical & Scientific Officer, Worldwide Clinical Trials.