Patient-Reported Outcome Measures in Hypogonadism
Patient-Reported Outcome Measures in Hypogonadism
Aim The aim of this qualitative analysis was to establish the content validity of two new patient-reported outcome (PRO) measures: Sexual Arousal, Interest, and Drive Scale (SAID) and Hypogonadism Energy Diary (HED).
Methods Four separate qualitative studies were conducted with 125 men with hypogonadism (mean age: 53 years, 85% adult onset). Study 1 used focus groups/interviews to identify important and relevant concepts related to the experience of hypogonadism and its treatment in men primarily with adult-onset hypogonadism. Study 2 tested items generated for assessments of low sex drive and low energy. Study 3 used interviews to confirm in men with early-onset hypogonadism that low sex drive and low energy were also important and relevant symptoms. Study 4 tested final versions of the two PROs and determined equivalency of paper-based and electronic versions of the two PROs.
Results Of the concepts emerging in Studies 1 and 3, low sex drive and low energy were the symptoms most often spontaneously mentioned. Coding of transcripts from Studies 1 and 3 led to the generation of items for the SAID and HED. After item testing (Studies 2 and 4), the final SAID included five items pertaining to arousal, interest in sex and sex drive with a 7-day recall period and the final HED included two items (energy, tired/exhausted) to be administered three times per day.
Conclusion The SAID and HED have content validity established according to regulatory guidance and, therefore, the potential to provide the patient perspective of treatments for hypogonadism.
Signs and symptoms associated with male hypogonadism may include decreased sexual interest or drive [with or without erectile dysfunction (ED)], decreased energy, mood changes, decreased lean body mass and increase in visceral fat, decreased body hair, skin alterations, regression of secondary sexual characteristics and osteoporosis. Some of the signs and symptoms can be measured clinically (e.g., lean body mass and bone mineral density), but other important symptoms are best measured by patient report (e.g., sex drive, energy level, erectile function and mood).
To provide the patient perspective of treatments for hypogonadism, it is essential to assess those symptoms that are best measured by patient report. Of those symptoms, both qualitative and quantitative studies suggest that decreased sexual functioning (sex drive and erectile functioning) and decreased energy have the greatest impact on men's lives. There is an existing patient-reported outcome (PRO) instrument that assesses EF—the Erectile Function Domain of the International Index of Erectile Function (IIEF-EF). The IIEF-EF has both clinical and regulatory acceptance as a primary end-point in trials of treatments for ED. There are also existing multi-domain PRO instruments that include assessments of sex drive and energy, such as the Psychosexual Daily Questionnaire (PDQ). However, the PDQ, as with other existing instruments previously used in the evaluation of hypogonadism treatment, has demonstrated evidence of reliability and validity but lacks evidence of the level of patient input in item development required to establish content validity. As stated in the Food and Drug Administration's guidance for industry entitled Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims, 'Testing other measurement properties will not replace or rectify problems with content validity.'
Given the significance of levels of sexual drive and energy in men with hypogonadism, the absence of content valid and psychometrically sound assessments of these two symptoms for the evaluation of both new and existing hypogonadism therapies represents an unmet measurement need. The overall goal of this study was to establish the content validity of two new PRO instruments, one that assesses the level of sex drive in men with hypogonadism and one that assesses the energy level of these men. The intent of these measures is to provide a brief assessment of the two concepts while meeting current regulatory standards for PRO measure development.
Abstract and Introduction
Abstract
Aim The aim of this qualitative analysis was to establish the content validity of two new patient-reported outcome (PRO) measures: Sexual Arousal, Interest, and Drive Scale (SAID) and Hypogonadism Energy Diary (HED).
Methods Four separate qualitative studies were conducted with 125 men with hypogonadism (mean age: 53 years, 85% adult onset). Study 1 used focus groups/interviews to identify important and relevant concepts related to the experience of hypogonadism and its treatment in men primarily with adult-onset hypogonadism. Study 2 tested items generated for assessments of low sex drive and low energy. Study 3 used interviews to confirm in men with early-onset hypogonadism that low sex drive and low energy were also important and relevant symptoms. Study 4 tested final versions of the two PROs and determined equivalency of paper-based and electronic versions of the two PROs.
Results Of the concepts emerging in Studies 1 and 3, low sex drive and low energy were the symptoms most often spontaneously mentioned. Coding of transcripts from Studies 1 and 3 led to the generation of items for the SAID and HED. After item testing (Studies 2 and 4), the final SAID included five items pertaining to arousal, interest in sex and sex drive with a 7-day recall period and the final HED included two items (energy, tired/exhausted) to be administered three times per day.
Conclusion The SAID and HED have content validity established according to regulatory guidance and, therefore, the potential to provide the patient perspective of treatments for hypogonadism.
Introduction
Signs and symptoms associated with male hypogonadism may include decreased sexual interest or drive [with or without erectile dysfunction (ED)], decreased energy, mood changes, decreased lean body mass and increase in visceral fat, decreased body hair, skin alterations, regression of secondary sexual characteristics and osteoporosis. Some of the signs and symptoms can be measured clinically (e.g., lean body mass and bone mineral density), but other important symptoms are best measured by patient report (e.g., sex drive, energy level, erectile function and mood).
To provide the patient perspective of treatments for hypogonadism, it is essential to assess those symptoms that are best measured by patient report. Of those symptoms, both qualitative and quantitative studies suggest that decreased sexual functioning (sex drive and erectile functioning) and decreased energy have the greatest impact on men's lives. There is an existing patient-reported outcome (PRO) instrument that assesses EF—the Erectile Function Domain of the International Index of Erectile Function (IIEF-EF). The IIEF-EF has both clinical and regulatory acceptance as a primary end-point in trials of treatments for ED. There are also existing multi-domain PRO instruments that include assessments of sex drive and energy, such as the Psychosexual Daily Questionnaire (PDQ). However, the PDQ, as with other existing instruments previously used in the evaluation of hypogonadism treatment, has demonstrated evidence of reliability and validity but lacks evidence of the level of patient input in item development required to establish content validity. As stated in the Food and Drug Administration's guidance for industry entitled Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims, 'Testing other measurement properties will not replace or rectify problems with content validity.'
Given the significance of levels of sexual drive and energy in men with hypogonadism, the absence of content valid and psychometrically sound assessments of these two symptoms for the evaluation of both new and existing hypogonadism therapies represents an unmet measurement need. The overall goal of this study was to establish the content validity of two new PRO instruments, one that assesses the level of sex drive in men with hypogonadism and one that assesses the energy level of these men. The intent of these measures is to provide a brief assessment of the two concepts while meeting current regulatory standards for PRO measure development.