Discussion
’, pGHD, GHD
GHD
To our knowledge, this is the first global survey comparing physicians’ experiences and perspectives of the once-weekly somatrogon regimen with the current standard of care – once-daily rhGH – in children with GHD. The findings of this study indicate that the once-weekly somatrogon regimen was generally preferred and was easier for physicians to monitor compared with the once-daily rhGH regimen. Overall, the majority of physicians preferred once-weekly somatrogon vs once-daily rhGH (75.0% vs 8.3%, respectively) and reported the once-weekly somatrogon regimen to be more convenient (79.2% vs 4.2%, respectively) and less burdensome (79.2% vs 12.5%, respectively). More than half of the physicians (62.5%) reported that less effort is required to monitor adherence in patients with once-weekly somatrogon compared with once-daily rhGH. Half of the physicians felt that once-weekly somatrogon was more beneficial to their patients, and most of the physicians (62.5%) perceived no difference between the regimens in terms of being more likely to support positive long-term growth outcomes or reduce healthcare utilization. Overall, a very large proportion of physicians were “very satisfied”/“satisfied” with both once-weekly somatrogon (95.8%) and once-daily rhGH (87.5%).Willingness to prescribe once-weekly somatrogon in the future was reported by the majority of physicians compared with once-daily rhGH (83.3% vs 4.2%, respectively). This finding aligns with the previously highlighted feedback around convenience and burden, but the results of our study also highlight other aspects of the once-weekly somatrogon regimen that support this perspective. Based on the open-ended free-text responses, physicians felt that the use of once-weekly somatrogon may reduce the burden of chronic daily injections for children. Physicians’ positive responses to once-weekly somatrogon were also driven by reasons related to decreased caregiver burden, better patient acceptance, a higher level of convenience, and improved adherence, indicating that these factors, in addition to efficacy, are important determinants of physicians’ perceptions of pGHD treatment. This study highlights the relevance of physician and patient factors in the preference for, and selection of, pediatric GH therapy as well as physicians’ beliefs that once-weekly somatrogon has the potential to address the long-standing unmet need for increased treatment adherence, and hence improved treatment outcomes, with GH therapy.To date, few, if any studies have assessed long-term patient adherence and treatment outcomes with a LAGH analog, likely because LAGH analogs in pGHD, such as lonapegsomatropin and somatrogon, have only recently received approval (In order to optimize pGHD therapy, pediatric endocrinologists use their clinical judgment and previous experience with GH treatment, in addition to being receptive and responsive to patients’ and caregivers’ perceptions of and concerns with the therapy. According to the open-ended responses to the survey, other reasons physicians favored somatrogon included patient acceptance of the treatment; reduced patient and caregiver burden, including decreased injection frequency; increased freedom that results from a simplified treatment regimen, such as the flexibility of a sleepover, traveling without the restrictions of a daily injection, patient care within split households, and the freedom to administer somatrogon on a weekend, with no interference on school days. These findings are consistent with previous studies demonstrating that GH product selection decisions were based on physicians’ perceptions of patients’ treatment acceptance (From the patient perspective, a less frequent injection schedule has previously been indicated as a preferred factor among adult and pediatric patients with GHD in a discrete choice experiment study in the United States (Based on the studies described above, it appears that ease of use of the treatment and patient comfort are key considerations when physicians make prescribing decisions. In our survey, most physicians indicated that there was no difference between once-weekly somatrogon and once-daily rhGH in terms of the time and effort required to explain the injection device IFU and the treatment regimen to patients and caregivers. However, these findings should be viewed in context of the limited use of somatrogon to date. It is possible that differences between the two treatment regimens may be more discernible with continued use of, and familiarity with, once-weekly somatrogon. A few physicians expressed their concern related to the non-physiologic (elevated and non-pulsatile) GH exposure with somatrogon relative to daily rhGH. The physiologic secretion pattern of GH is episodic and pulsatile, exhibiting a diurnal rhythm, with approximately two-thirds of the total daily GH secretion occurring in major pulses during slow-wave sleep in the night (One of the strengths of our study is its international scope, with information collected about the experiences of physicians operating in different settings from over 10 different countries. The response and completion rates were also high (70.3% and 80.0%, respectively). Another strength is that physicians’ responses were based on their direct experience, as the study was limited to physicians with significant prior experience of treating children with GHD with once-daily rhGH and with once-weekly somatrogon in the phase III study. Given the cross-sectional nature of the study, another strength is the ability to quickly obtain and compile information about the study physicians’ experiences with once-weekly somatrogon.Our study has several limitations. First, our study relies on physicians’ recall and opinion. There was variability in the length of time between a physicians’ last treatment experience and their completion of the survey. Physicians who treated patients at a more distant time point may have had less accurate recall compared with those with more recent experiences. Second, the study enrolled physicians who treated children with GHD in the pivotal phase III study, restricting their experience with once-weekly somatrogon to a clinical trial setting in a few patients as compared with the significant experience they had gained with once-daily rhGH, the standard of care for many years. It is worth noting that at the time this survey was conducted, somatrogon (NGENLA™) had not been marketed, and consequently, it remains to be assessed whether real-world experience with once-weekly somatrogon differs from that of a clinical trial setting. Third, since completion of the survey was voluntary, these study results may not reflect the experience of all the eligible physicians in the phase III trial. Fourth, due to the limited sample size and the small number of participating physicians in each country, statistical comparisons were not performed, and country trends were not analyzed or presented. Fifth, since the physicians participating in the survey were investigators in the somatrogon clinical trial, they may have a bias toward the potential benefits of LAGH therapy.In conclusion, the results of our survey study demonstrate that participating physicians had a favorable perception of, and positive experience with, treating children with GHD with once-weekly somatrogon. Compared with once-daily rhGH, physicians found once-weekly somatrogon to be more convenient and less burdensome for patients. Most physicians had a clear preference for once-weekly somatrogon and were likely to prescribe somatrogon if it were available to them. Almost all physicians were “very satisfied”/“satisfied” with the once-weekly somatrogon regimen. Results from this study provide initial insights into physicians’ experience and perspectives of once-weekly somatrogon treatment. Future clinical practice studies are needed to characterize the real-world, long-term treatment experience with the once-weekly somatrogon injection regimen.