By Ann Neuer
May 17, 2010 | Evidence is mounting that the Food and Drug Administration (FDA) wants to hear the voice of the patient in clinical trials. In December 2009, FDA put forth a new guidance—Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims—to present how the agency will review and evaluate patient-reported outcomes (PRO) instruments and what constitutes a well-designed PRO tool.
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| Doug Engfer |
“The net takeaway from the guidance is that the level of rigor has been increased on the part of the regulator because they value the voice of the patient. They want to make sure PRO data are collected in a reliable way so they can make reasonable judgments based on those data,” says Doug Engfer, president and CEO of invivodata, maker of DiaryPRO and SitePRO, two well-known ePRO solutions. Engfer comments further that the days of patients filling out paper diaries retrospectively as they sit in the parking lot prior to their study visits are hopefully numbered as sponsors will be required to show that their PRO instruments comply with the protocol and are fit for the task.How to make a fit-for-the-task PRO tool is what the guidance is all about. It states that tools must contain several components that prove their usefulness, such as content validity, construct validity, and ability to detect change. In simple terms, content validity refers to the extent to which the instrument measures the concept of interest. “In other words, does the tool measure what you think it measures?” explains Jean Paty, co-founder and chief regulatory advisor for invivodata’s PRO consulting division.
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| Jean Paty |
Construct validity, the next pivotal concept, measures the instrument’s ability to determine whether the documented relationships between results gathered using the instrument and results gathered through other means are consistent with pre-existing hypotheses concerning those relationships. Finally, ability to detect change refers to whether the instrument can identify that a change has taken place. For example, if the patient’s pain reduces, the instrument should detect this change, and if the instrument doesn’t work this way, it could change the outcome of the clinical trial.“The key point is that these concepts represent a strategic change. If you can’t defend your PRO instrument or measurement selection, there’s no way for anyone to believe your drug works. This is not casual. We’ve seen people get turned away because no one believed in the measurement they were using,” Paty remarks.
To help companies face the daunting task of complying with the new guidance, invivodata is offering a free 60-minute PRO GAP analysis for a limited time period. The analysis enables pharmaceutical and medical device researchers to speak with senior PRO scientists via teleconference to assess what will be expected of them in their clinical research and PRO data from a regulatory standpoint. For invivodata, ePRO is an integral part of the GAP analysis. Topics to be discussed run the gamut from the best way to develop a PRO tool that will support novel labeling claims to whether a study should use interactive voice response (IVR) or a personal digital assistant (PDA) to collect PRO information. To help customers make the latter decision, invivodata has the ePRO Modality Decision Tool that it co-developed with Perceptive Informatics.
Since release of the final guidance, Engfer says, “Our phone has been ringing off the hook. It and the fact that we formed a collaborative agreement in January with Quintiles as primary preferred partner for ePRO, have caused us to increase our growth targets for the year substantially.”