Abstract
PurposeTo share better practice in establishing data monitoring committees (DMCs) for observational, retrospective safety studies with joint‐industry sponsorship.MethodsA DMC model was created to monitor data from an observational, retrospective, post‐authorization safety study investigating risk of medullary thyroid cancer in patients treated with long‐acting glucagon‐like peptide‐1 receptor agonists (LA GLP‐1RAs) (NCT01511393). Sponsors reviewed regulatory guidelines, best practice and sponsors' standard operation procedures on DMCs. Discussions were held within the four‐member consortium, assessing applicability to observational, retrospective, real‐world studies. A DMC charter was drafted based on a sponsor‐proposed, adapted DMC model. Thereafter, a kick‐off meeting between sponsors and DMC members was held to receive DMC input and finalize the charter.ResultsDue to this study's observational, retrospective nature, assuring participant safety – central for traditional explanatory clinical trial models – was not applicable to our DMC model. The overall strategy and key indication for our real‐world model included preserving study integrity and credibility. Therefore, DMC member independence and their contribution of expert knowledge were essential. To ensure between‐sponsor data confidentiality, all study committees/corporations and sponsors, besides the DMC, received blinded data only (adapted to refer to data blinding that revealed the specific marketed LA GLP‐1RA/sponsor). Communication and blinding/unblinding of these data were facilitated by the contract research organization, which also provided crucial operational oversight.ConclusionsTo our knowledge, we have established the first DMC model for joint industry‐sponsored, observational, retrospective safety studies. This model could serve as a precedent for others performing similar post‐marketing, joint industry‐sponsored pharmacovigilance activities.
Highlights
The importance of real-world evidence to support clinical trial data is increasingly recognized and requested by stakeholders, for example, health regulatory agencies, health policy decision-makers, patient groups, public funders and academia
Aligned with Lillenfeld et al,[14] we found that responsibility of a Data monitoring committees (DMCs) is to patients, physicians, payers, health policy regulators and others who are impacted by having the pharmaceutical approved
Several challenges were met during model set-up, including a lack of DMC precedents for retrospective, observational studies and necessary multi-sponsor collaboration, each with respective standard operation practices (SOPs)
Summary
The importance of real-world evidence to support clinical trial data is increasingly recognized and requested by stakeholders, for example, health regulatory agencies, health policy decision-makers, patient groups, public funders and academia. Results from highly selected and controlled environments provide high internal validity but are not necessarily generalizable to real-world settings (i.e. failure to provide external validity).[1,2,3,4,5] the potential existence of unforeseen safety signals/safety concerns (unknowns) may persist, despite evaluation in well-performed clinical trials and following marketing authorization. Industry sponsors with marketing authorizations for same-class drugs are encouraged, by regulatory authorities, to conduct joint collaborative real-world studies.[6] inconvenience to patients, physicians and registries is minimized while data exposure is maximized. The DMC model we share monitors data from an observational, retrospective, real-world study. This model accommodated additional challenges resulting from one DMC serving four industry sponsors within one study
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Free) 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
