THOUGHT LEADERSHIP

Home visits as part of clinical research have accelerated in recent years as we strive to find the right balance between facilitating study participation while accommodating participant’s busy everyday lives. Clinical trials in rare diseases are even more challenging than trials in other diseases due to a number of factors:

• Small number of eligible trial participants
• Complicated by heterogeneity among rare disease patients
• Most have no cure and manifest at a young age
• Less than 10% of rare diseases have a specific treatment
• Many have other debilitating conditions / physical limitations making it difficult to attend frequent study visits

The combination of home study visits and the right technology removes barriers to optimal patient recruitment, compliance and retention.

All clinical data managers (CDMs) are familiar with the difficulties of ensuring that laboratory values are compared to the local laboratory normal ranges that were valid at the time of the measurement. In studies with many local laboratories, and in global studies, the same test may be reported in different units across the study. To help alleviate these problems, some studies use central laboratories. Yet there are some problems with this approach as well. Shipping samples from study sites to the central laboratory incurs extra cost and runs the risk that the sample will be unusable when it arrives. 

Due to the worldwide pandemic, the CDISC European Interchange, originally scheduled to take place in person in Berlin, Germany, was presented completely online. Susan Boquist, Associate Director, Statistical Programming, took advantage of the accessibility and logged in at 3:00 am her time both mornings. Though it may have become an April Fool’s Day to remember, with only a few weeks’ notice, the organizers were able to make the format transformation flawlessly. The conference schedule was consolidated into one track. A networking app was employed to allow for attendees to easily communicate with each other, ask questions of the presenters, and view and vote on the poster presentations. Training and workshops were rescheduled for another time. It was so successful that everyone hopes the organizers will consider adding a virtual option for future conferences. 

In recent years, efforts to streamline clinical research activities while improving subject safety and data integrity have included risk based monitoring (RBM). As recently as May 2019, the International Council for Harmonisation (ICH) proposed revisions to its General Considerations for Clinical Studies (E8) guideline. Section 3 of E8 (R1) stresses the importance of managing the quality of safety measures and data collection as part of an entire Quality Plan in a clinical program and its studies. This effort is called Risk-Based Quality Management (RBQM).