PurposeThe purpose of this study was to identify patient beliefs as well as clinical realities about insulin that may be barriers to type 2 diabetes patients initiating insulin treatment when recommended by their physician. This information was then used to develop a clinically relevant, cross-culturally valid patient education tool with the goal of providing unbiased, medically informative statements addressing these barriers.MethodsThirteen focus groups were conducted in five countries (Germany, Sweden, The Netherlands, UK, and USA) to collect qualitative data on attitudes about insulin therapy from type 2 diabetes patients aged 18 or older whose physician had recommended initiating insulin treatment in the past 6 months (n = 87). Additionally, a panel of four clinical experts was interviewed to ascertain obstacles they experience in initiating insulin with their patients.ResultsOn the basis of the interview data, the ten questions that asked about the most important barriers were generated. The clinical expert panel then generated clinically accurate and unbiased responses addressing these concerns, and the educational tool “Questions about Starting Insulin: Information on the Myths, Misconceptions and Clinical Realities about Insulin” was drafted. The draft tool was pilot tested in a group of patients and finalized.ConclusionsPatient misconceptions, as well as some clinical realities, about insulin treatment and diabetes can influence the decision to initiate insulin treatment and ultimately impact disease management. The educational tool developed through this study was designed to help patients who are deciding whether or not to initiate insulin therapy as recommended by their physician, and facilitate patient–health-care provider interactions.

ObjectiveGrowth hormone deficiency (GHD) treatment for children requires growth hormone injections, typically administered daily until the child reaches adult height. Child GHD treatment burden is not well understood and no disease-specific measures exist to assess this burden. The purpose of the study was to explore GHD treatment burden for children and their parents by conducting concept elicitation interviews supporting a theoretical model of the impact of GHD treatment.MethodsFour focus groups (in Germany) and 52 telephone interviews (in the UK and USA) were conducted with children/adolescents with GHD aged 8 to <13 years and parents of children with GHD aged ≥4 to <13 years. The purpose of the interviews was to understand the experience of GHD treatment from the child’s perspective, and for parents, the impact of their child’s treatment on themselves. Interview transcripts were analyzed thematically based on modified grounded theory principles.ResultsInterviews with 70 respondents who produced descriptions (n = 73) of patients experiences with GHD treatment (three parents spoke for two children each) were conducted. Analysis identified three major areas of GHD treatment burden for children: physical; emotional well-being; and interference. Parent burdens identified were: emotional well-being and interference. Modifiers such as treatment efficacy and duration, which may impact the degree of treatment burden severity, were identified.ConclusionsOverall treatment burden of child GHD is considerable for children and their parents. The concept elicitation and theoretical model can be used to develop a disease-specific outcome measure, which adequately reflects the burden of GHD treatment for children and their parents.

PurposeResearch demonstrates that children and adolescents with growth hormone deficiency (GHD) are impacted in multiple ways beyond their short stature; however, there are no disease-specific measures to assess these impacts. The purpose of this study was to examine the burden of GHD on children and adolescents, and to conduct concept elicitation to develop a model of the impact of GHD to support a disease-specific outcome measure.MethodsFour focus groups and 52 telephone interviews were conducted with children with GHD and parents/guardians of children with GHD to understand the experience and impacts from the child’s perspective, reported by children or parent-observers about the impact on the child. The interviews and focus groups were conducted in Germany, the United Kingdom, and the United States. Interview transcripts were analyzed thematically based on modified grounded theory principles.ResultsThere were 73 descriptions of patient’s experiences elicited from 70 respondents, as three respondents spoke for two children each. A majority of GHD descriptive narratives refer to boy children (n = 51, 69.9%) and a majority of children had taken GHD treatment (n = 64, 89%). Analysis identified four major areas of GHD impact: Signs and Symptoms (beyond short stature), Physical Aspects of Daily Life, Social Well-Being, and Emotional Well-Being.ConclusionsThe burden of GHD in children and adolescents is considerable and not limited to short stature. The severity of GHD impact on children and adolescents appears to be variable and individualized, but these data indicate that early identification and growth hormone treatment may lead to fewer impacts.

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Background: Growth hormone deficiency treatment involves daily injections, which can be difficult to administer, incorporate into daily routines, and lead to poor treatment compliance. The Treatment Burden–Child Growth Hormone Deficiency (TB-CGHD) measure was developed according to FDA and EMA regulatory guidance to assess key aspects of GHD treatment burden experience in children. There are two versions of the measure: self-report (PRO) for children 9 to <13 years and observer-report (ObsRO) for parents/guardians of children 4 to <9 years. Items are based on qualitative interviews with 39 children and 34 parents of children with GHD. This study presents TB-CGHD validation study results. Methods : A non-interventional, multi-clinic-based study (US and UK) of pre-pubertal children diagnosed with GHD (treatment naïve [TN] or ongoing treatment) and parents/guardians of similar children was conducted. All subjects completed a baseline assessment battery in clinic, with in-person follow-up for the TN group. Psychometric analyses were conducted according to an a-priori statistical analysis plan to determine the measurement model, internal consistency, construct validity, test-retest reproducibility, sensitivity to change and minimally important difference (MID). Results : The analytic data set was comprised of 243 subjects (145 children and 98 parents/guardians). Children were on average 9.2 years, 72% male, with average child’s age at diagnosis 6.9 years. Post item reduction resulted in a 14-item measure. Factor analyses identified 3 domains (Physical (PHYS), Emotional Well-being (EWB), Interference (INT)). Internal consistency reliability was acceptable (Cronbach’s alpha >0.70) as was test-retest for the Total, PHYS and EWB domains (ICC >0.70) and slightly lower than expected for INT (0.64). At least one of the convergent validity hypotheses for total and domains were significant. For known-groups validity, TB-CGHD discriminated between length of time to administer injections (p<0.01) and a trend showing scores differed by length of time on treatment. Improvements in scores at 12-week follow-up were shown for EWB and Overall domains (range: 3.6-14.3 points). Associated effect sizes (mean change divided by the baseline standard deviation) ranged from -0.27 to -0.57, indicating that the TB-CGHD is sensitive to change at low to moderate levels depending on domain. Triangulating (averaging various MID calculation methods) found the MID for the Total scores of PHYS and INT to be 6 points, and 9 for EWB. Conclusions : The final TB-CGHD was found to be reliable and valid and is considered ready for inclusion in clinical trials and clinical practice. Accurate and reliable assessment of treatment burden can help researchers and clinicians better assess and address impacts of treatment, factors that may affect compliance, and may improve the quality of doctor-patient communications.

PurposeThe aim was to evaluate the measurement properties of the Growth Hormone Deficiency-Child Treatment Burden Measure-Child (GHD-CTB-Child), a patient-reported outcome (PRO) for children aged 9 to < 13 years; the Growth Hormone Deficiency-Child Treatment Burden Measure-Observer (GHD-CTB-Observer), an observer-reported outcome (ObsRO) version completed by parents/guardians of children with growth hormone deficiency (GHD) aged 4 to < 9 years; and the Growth Hormone Deficiency-Parent Treatment Burden Measure (GHD-PTB), a PRO that assesses the treatment burden of parents/guardians living with children with GHD aged 4 to < 13 years. Methods A non-interventional, multi-center, clinic-based study across 30 private practice and large institutional sites in the United States and the United Kingdom was conducted. The sample consisted of 145 pre-pubertal children aged 9 to < 13 years at enrollment with a physician confirmed GHD diagnosis as well as 98 parents/guardians of pre-pubertal younger children aged 4 to < 9 years at enrollment with a physician confirmed GHD diagnosis. The child sample consisted of 59 treatment-naïve children (no prior exposure to growth hormone [GH] therapy; were starting GH treatment at study start per standard of care) and 184 children already maintained on treatment for at least 6 months. At baseline, all study participants completed a paper validation battery including all measures needed to conduct the validation analyses. Follow-up assessments with children in the maintenance group and their caregiver/parent were conducted approximately 2 weeks post-baseline to evaluate test-retest reproducibility. To evaluate sensitivity to change and meaningful change thresholds, treatment-naïve participants in both child and parent/guardian populations were assessed within 1 week of report of minimal improvement between week 3 and week 11 and at week 12. Psychometric analyses were implemented following an a priori statistical analysis plan. Results Factor analyses confirmed the a priori conceptual domains and Overall score for each measure (GHD-CTB-Child and GHD-CTB-Observer domains: Physical, Emotional Well-being, and Interference; GHD-PTB domains: Emotional Wellbeing and Interference). Internal consistency was acceptable for all measures (Cronbach's alpha > 0.70). Test-retest reliability was acceptable for the Physical, Emotional, and Overall domains of the GHD-CTB versions, and the Emotional and Overall domains of the GHD-PTB (intraclass correlation coefficient above 0.70). All but one of the convergent validity hypotheses for the GHD-CTB versions and all hypotheses for the GHD-PTB were proven (r > 0.40). Known-groups validity hypotheses were significant for length of time to administer the injections in the GHD-CTB versions (p < 0.001 for Physical, Emotional, and Overall, and p < 0.01 for Interference) and whether parents/guardians versus child gave the injections more often for the Emotional domain of the GHD-PTB (p < 0.05). Associated effect sizes ranged from −0.27 to −0.57 for GHD-CTB versions an...

24% wanted HCPs to talk about factors besides HbA1c. Further, while 44% of HCPs interviewed said the easiest way to achieve control was intensifying medication, patient's often associated intensification and/or complex medication regimens as a negative aspect of control. CONCLUSIONS: The concept of "being in control" has multiple meanings to patients and these definitions are often at odds with HCP definitions. This discordance may result in suboptimal patient-HCP interactions as well as contribute to physician inertia to initiate and/or intensify treatments. Patient education and research is needed to better understand the management of uncontrolled diabetes. PDB78AnAlyzing eq-5D in PhAse 3 clinicAl triAls of tyPe 2 DiABetes mellitus (t2Dm): is meAn chAnge cAPturing PAtient imPAct?