The ROI of ePRO Phil Lee, President and CEO The power of listening to the patient voice by using e-diary systems to collect the best PRO data possible and manage subject enrollment, compliance, and safety in real time may still be overlooked by some sponsors, who are hesitant to switch to ePRO because they view it as an additional expense. These sponsors may be missing out on a larger opportunity to reduce costs and shorten trial timelines. Often-overlooked paper costs that are eliminated or minimized by ePRO: • Queries on diary/questionnaire data • Double data entry • Storing and archiving physical records • Site and CRA time reviewing diaries • Larger-than-needed sample sizes • Faxing fees • Manual calculations • On-site CRA monitoring • Data cleanup delays to diary database lock Doing More with Less More importantly, however, is ePRO’s proven ability to show safety and efficacy by using smaller sample sizes compared with paper-based trials. By capturing only complete, logical, and legible records, ePRO provides higher quality data with less data variance. Missing data are also greatly reduced because of the site’s enhanced ability to proactively manage subject compliance and enrollment on a real-time basis. In addition, ePRO’s ability to ensure that patients’ responses are timely and logical leads to less recall bias and a resulting reduction in data variance in some indications. The resulting decrease in standard deviation allows sponsors to achieve study power with fewer patients — thereby saving significant money and time. For example, in Merck’s randomized trial to compare the relative capabilities of paper and electronic patient diaries to show efficacy, patients with chronic primary insomnia were treated with an approved safe and effective insomnia medication. Although the mean change in sleep time was statistically equivalent for the two arms, the e-diary data exhibited a 35% smaller standard deviation with a more normal distribution. The team calculated that the same study power could have been achieved with a sample size reduction of 56% simply by using an e-diary system. In another example, Novartis collected endpoint data on an e-diary and was able to prove efficacy with only 31% of the estimated number of randomized patients necessary for study power; the trial was interrupted due to external circumstances. Adaptive Sample Sizing Adaptive sample sizing is an innovative clinical-design methodology that uses actual data variance to enable biostatisticians to adjust the required number of patients necessary to achieve study power. E-diary systems allow clinical studies to be designed so patients can move to different arms of the trial automatically in accordance with the protocol rules, while the reduced data variance and up-to-the-minute data transfers increase the value of prescheduled interim analyses. The Ethics Effect Decreasing a trial’s sample size has additional benefits beyond improving the bottom line. It allows biopharmaceutical companies to bring beneficial new treatments to the people who need them faster, and it exposes fewer patients to investigational therapies. The Bottom Line The ultimate ROI is an approved drug, treatment, or therapy developed rapidly and marketed before the competition. With ePRO, sponsors take advantage of reduced data variance and adaptive sample sizing to run smaller, faster, and less costly trials. In pivotal studies, the clean, complete, and reliable data sets ePRO provides offer more conclusive evaluations of therapy safety and efficacy. PHT Corp. PHT Corp., Charlestown, Mass., is a provider of electronic patient reported outcome (ePRO) solutions used in more than 280 clinical trials around the world. For more information, visit phtcorp.com. June 2007 VIEW on Clinical Services E-Clinical solutions

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