Core Principles for a Successful Registry.
Research institutions, patient advocacy groups, and biopharmaceutical companies, especially those conducting research in rare or orphan diseases, are launching patient registries at an ever-increasing pace. Registries are valuable research tools, as illustrated by the diversity in their sponsors, objectives, and target audiences. They can span the research continuum from a simple collection of patients who share a common health issue, to a regulatory post-marketing commitment to confirm the long-term safety and effectiveness of a new drug or device. Among this diversity, however, my experience has revealed that project teams can benefit from a few, common registry design principles.
Have a vision for the future, but take care of your current needs.
A registry may run for many years and it is important to create a roadmap to guide its long-term evolution. But deciding registry design elements to fulfill a potential post-marketing commitment when your investigational drug candidate is just completing Phase 1, for example, may not be the best use of time and resources. During a long clinical development program, there will be unexpected detours – new clinical findings, shifts in physician perceptions or regulatory guidance, and emerging competitive technologies – that may influence a future post-marketing commitment. In early phases of drug development, focusing a registry to inform the design of planned randomized clinical trials, support regulatory discussions, and engage key opinion leaders (KOLs) and patient advocacy groups may provide a better pay-off. Similar assessments in each phase of your research program will focus your team’s efforts on the current needs and promote the long-term success of your registry program.
Understand your target audiences and key stakeholders.
Before a research institution, patient advocacy group, or biopharmaceutical company embarks on a patient registry, they should be aware that other stakeholders with similar research interests might have long-established registries or research databases. Biopharmaceutical companies in particular can have a difficult time launching a new registry in support of a drug development program because KOLs and patient advocacy groups with their own registries may be reluctant to support a company-sponsored program for fear of losing control of their own projects. Collaboration is key! Understanding the objectives, processes, and data output of research conducted by other stakeholders, can help you to work toward a common goal. Regardless of the registry’s sponsor, rallying your target audiences and key stakeholders around a common cause, exploring data sharing agreements, and coordinating research efforts – which often rely on the same limited resources – will go a long way to create a positive research atmosphere. Develop sensible communication plans and encourage positive engagements. Remember: It’s all about the patients!
Pick the technology. Don’t let the technology pick you.
There has been an explosion of patient-facing, health-related, mobile applications over the past several years that can be adapted to support research studies through the use of tablets and smartphones. Just because a technology CAN do something, does not mean it’s best to incorporate that application into your registry. For example, a “chat room” application with which a patient can discuss health issues with other patients may introduce a confounding factor to a registry study that is evaluating patient outcomes in response to routine medical care. Design your registry – its objectives, the data elements, and processes – THEN pick the technology that will best support the successful implementation of your study.
By adhering to these core principles, you can guide the development and dynamic growth of your registry to meet your long-term research objectives!