Search

Data silos are proliferating while research and development activity explode following genetic and immunological advances for many clinically described disorders with previously unknown etiologies. The latter event has inspired optimism in the patient, clinical, and research communities that disease-specific treatments are on the way. However, we fear the tendency of various stakeholders to balkanize databases in proprietary formats, driven by current economic and academic incentives, will inevitably fragment the expanding knowledge base and undermine current and future research efforts to develop much-needed treatments. The proliferation of proprietary databases, compounded by a paucity of meaningful outcome measures and/or good natural history data, slows our ability to generate scalable solutions to benefit chronically underserved patient populations in ways that would translate to more common diseases. The current research and development landscape sets too many projects up for unnecessary failure, particularly in the rare disease sphere, and does a grave disservice to highly vulnerable patients. This system also encourages the collection of redundant data in uncoordinated parallel studies and registries to ultimately delay or deny potential treatments for ostensibly tractable diseases; it also promotes the waste of precious time, energy, and resources. Groups at the National Institutes of Health and Food and Drug Administration have started programs to address these issues. However, we and many others feel there should be significantly more discussion of how to coordinate and scale registry efforts. Such discourse aims to reduce needless complexity and duplication of efforts, as well as promote a pre-competitive knowledge ecosystem for rare disease drug development that cultivates and accelerates innovation.

Background Scrolling is a perceived barrier in the use of bring your own device (BYOD) to capture electronic patient reported outcomes (ePROs). This study explored the impact of scrolling on the measurement equivalence of electronic patient-reported outcome measures (ePROMs) in the presence and absence of scrolling. Methods Adult participants with a chronic condition involving daily pain completed ePROMs on four devices with different scrolling properties: a large provisioned device not requiring scrolling; two provisioned devices requiring scrolling – one with a “smart-scrolling” feature that disabled the “next” button until all information was viewed, and a second without this feature; and BYOD with smart-scrolling. The ePROMs included were the SF-12, EQ-5D-5L, and three pain measures: a visual analogue scale, a numeric response scale and a Likert scale. Participants completed English or Spanish versions according to their first language. Associations between ePROM scores were assessed using intraclass correlation coefficients (ICCs), with lower bound of 95% confidence interval (CI) > 0.7 indicating comparability. Results One hundred fifteen English- or Spanish-speaking participants (21-75y) completed all four administrations. High associations between scrolling and non-scrolling were observed (ICCs: 0.71–0.96). The equivalence threshold was met for all but one SF-12 domain score (bodily pain; lower 95% CI: 0.65) and two EQ-5D-5L item scores (pain/discomfort, usual activities; lower 95% CI: 0.64/0.67). Age, language, and device size produced insignificant differences in scores. Conclusions The measurement properties of PROMs are preserved even in the presence of scrolling on a handheld device. Further studies that assess scrolling impact over long-term, repeated use are recommended.

A growing number of participants in clinical trials are sharing information about their health online. It's time that the drug development community starts to examine how this social media use might compromise the integrity of research studies and how it might also offer new opportunities.