Medical Imaging Emerges in Clinical Trials: A 2007 Assessment
CenterWatch 2007
The FDA, in an effort to increase new drug approvals, has identified imaging as an important technology for assessing new therapeutics and accelerating drug development in the future.
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James Pierce, PHD MSPH
Scientific Advisor
The migration from paper based questionnaires to electronic formats for the collection of patient reported outcomes (PROs) represents a significant advancement for improving the accuracy and timeliness of PRO data. There are two common forms of electronic PRO (ePRO) data collection: handheld electronic devices that patients use to enter their outcomes information; and interactive voice response (IVR) systems that utilize standard telephones to record patient data entries.
Although there are clear advantages to collecting data electronically, sponsors must remember that the most important consideration for the migration to ePRO is not the benefits for the sponsor, but the impact on the patients involved in the trials. The U.S. Food and Drug Administration draft guidance states that sponsors must demonstrate evidence (usually through either a cognitive debrief or a small scale validation study) that the electronic modality they select for a specific trial delivers comparable results to the original paper based version. In addition, it is critical that the ePRO assessment be appropriate for the patient population, the drug/device product, and the type of data being collected in the study. However, there is very little peer-reviewed or publicly presented data available that would help sponsors evaluate a trial and select the right ePRO system for a given set of circumstances.
To address this issue and help sponsors select the most appropriate ePRO solution for a specific trial, Perceptive Informatics, a PAREXEL company, in conjunction with our partner invivodata, has developed a set of decision-making criteria based on our real-world experience implementing ePRO systems for hundreds of clinical trials. It is important to recognize there is no single factor that makes one ePRO solution better than the other. The best solution is the one that fits the specific circumstances of each trial.
Using four identified categories, the findings below describe the factors that should be carefully weighed when evaluating which ePRO approach to implement for a specific study.
Instrument and study measurement characteristics
PRO measures vary greatly in length and complexity, and care is needed to avoid an excessive burden to subjects independent of ePRO mode, whether handheld electronic devices or IVR systems. Lengthy or complex measures can be problematic for either mode and it is important to think through patient burden, memory load, and user acceptability when deciding on the appropriate system.
Another important consideration when deciding between handheld devices or IVR systems is whether the study protocol requires the use of graphics, such as body images, numeric rating scales (NRS) or visual analog scales (VAS) for patient-reported measurements. The classical VAS consists of a line with the ends marked with anchors, but with no subdivisions or numbers along the line. In contrast, for NRS subjects enter a specific number to describe a threshold range for a given health outcome (i.e. an 11-point scale with 0 = no pain, and 10 = worst possible pain). Because the term VAS is often used interchangeably with NRS, there is some confusion about whether VAS can be effectively implemented in an ePRO format. Increasingly, there is a movement to reclassify many VAS measures without descriptive anchors or markers as NRS. NRS questions can be implemented using either IVR or device-based ePRO. In determining which ePRO solution is most appropriate for a “VAS” measure, it is important to decide what kind of scale is to be used because the implications for each can vary.
Study protocols
The requirements of the study protocol can have a significant impact on an ePRO decision. There is no silver bullet for sponsors when deciding on a modality for protocols that require frequent assessments. It is most important to think through patient access and compliance. For example, if patients are asked to enter data at specific times of day this may necessitate reminders or alarms to maintain compliance. When studies require such alarms, ePRO systems offer varied flexibility and efficiency in generating those reminders. One must consider the profile of the patients enrolled in the study and what would be most likely to increase compliance.
Patient population characteristics
Although age, physical limitations, literacy and language issues, study size, and geographic distribution have sometimes been cited as barriers to the implementation of ePRO technology, experience has shown that these issues can be overcome by careful study design that is based on the perspective of the patients to be enrolled in a study. For example, patient age is often used to preclude ePRO solutions, however, there is no evidence to support this idea. In fact, trial experience shows that older patients – including those who are unfamiliar with computer systems – are willing to use either type of ePRO system, generally prefer them to paper, and are more compliant.
Data integration and access issues
For both IVR and device-based ePRO systems, convenient data access is an important benefit. Access to real-time data allows patient compliance to be closely monitored and quickly corrected when needed. For many sponsors integrating ePRO systems with patient randomization, medical management, and other electronic systems is critical for the success of a trial. Based on study specific factors, ePRO systems confer advantages to the sponsor.
Conclusion
While this is a simplified examination of the ePRO evaluation process, there are no simple answers to questions about which ePRO solution is the right one. Every study situation is unique, and all of these factors must be considered in order to reach a logical solution. In addition to the factors listed above, cost is obviously a major factor in any ePRO decision. Sponsors should not consider the factors above in isolation from the costs associated with study start up, implementation, logistics, and volume.
Note: The result of this initiative is a science-based tool that, when used properly by clinical trials sponsors, results in an objective comparison of the relative merits of the two most commonly used ePRO modalities.
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