Patient interviews reveal experiences with Pompe treatment and trials
Most felt well informed and found treatment manageable in the small study
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Most adults with late-onset Pompe disease (LOPD) interviewed after participating in clinical studies of Pombiliti plus Opfolda (cipaglucosidase alfa/miglustat) found the treatment manageable, and more than half reported improvements in their quality of life, a small interview study shows.
Participants also generally felt well informed about the studies and found the time commitment manageable. The findings suggest that patient feedback could help improve future clinical trials and inform treatment decisions.
Patient feedback may help improve future clinical trials
“These exit interviews provide valuable insights into patient perspectives of [Pombiliti plus Opfolda] and study processes not captured using standard patient-reported outcome measures and should be considered in future studies,” the researchers wrote.
The study, “Patient perspectives of participation in clinical studies in late-onset Pompe disease: Structured exit interviews from cipaglucosidase alfa plus miglustat studies,” was published in Therapeutic Advances in Rare Disease. The study was funded by Amicus Therapeutics, which marketed Pombiliti plus Opfolda before being acquired by Biomarin Pharmaceutical in April. The treatment is now part of Biomarin’s commercial portfolio. Several study authors were employed by Amicus at the time of the study or are employed by Biomarin.
Pompe disease is caused by mutations in the GAA gene that lead to a shortage of a working enzyme called acid alpha-glucosidase (GAA). This enzyme helps break down glycogen, a complex sugar. Without enough working GAA, glycogen builds up in cells, especially muscle cells, causing progressive muscle weakness.
In the U.S., Pombiliti plus Opfolda is approved for adults with LOPD who are not improving on their current enzyme replacement therapy. Pombiliti delivers a working version of the GAA enzyme through an infusion into a vein, while Opfolda, taken by mouth, helps keep the infused enzyme stable.
Although clinical trials have reported improvements or stability in walking ability and breathing function, less is known about patients’ experiences.
To learn more, researchers interviewed adults in the U.S. and U.K. who had participated in one of three Pombiliti plus Opfolda clinical trials or a U.K. program that provided early access to the treatment before its approval. In all, 23 adults, including 13 men, completed the interviews. Of these, 19 had participated in clinical trials and four in the early access program.
Most participants found two-part treatment manageable
The interviews explored why patients participated, their experiences with study procedures, how convenient they found the treatment, and perceived changes in quality of life. Most participants (83%) said they had joined to explore another treatment option, contribute to drug development, or both.
Nearly all (96%) felt they had received enough information before participating, and 91% considered the time required for study visits, questionnaires, and forms acceptable. Most participants (74%) did not find the two-part treatment challenging, while the remaining 26% felt neutral about the process.
Patients must fast for at least two hours before and two hours after taking Opfolda. About half (52%) found the fasting requirement convenient or very convenient, 39% were neutral, and 9% found it mildly inconvenient.
Some participants had trouble swallowing capsules or had to fast longer than expected because of treatment delays. Scheduling infusions early in the morning helped some participants manage the fasting requirement.
Sixteen participants (70%) had received infusions at home. Of these, 11 preferred home treatment because it reduced travel and allowed them to relax or work during infusions.
However, some participants missed interacting with clinical staff. Others reported treatment delays or felt uncomfortable having unfamiliar healthcare professionals in their homes. One participant returned to clinic-based treatment.
When asked about the treatment’s overall impact, 13 participants (57%) said they felt it had helped them and improved their quality of life.
Five (22%) were unsure whether treatment had helped or changed their quality of life. Another five (22%) felt the treatment had not helped, and their quality of life was worse.
Small study highlights limits of patient-reported benefits
The findings reflected patients’ perceptions rather than objective measurements of treatment effectiveness, the researchers emphasized.
They also noted that some participants had previously received enzyme replacement therapy. Being familiar with infusions may have made the two-part treatment easier to manage.
Another limitation, the researchers noted, was the study’s small size, with only 25% of eligible patients taking part. “Given that only a small proportion of eligible patients participated in the exit interviews, we must contemplate that the positive results may have been skewed if those who responded well to the therapy were more likely to enroll and provide feedback,” the investigators wrote.
According to the researchers, ongoing studies of Pombiliti plus Opfolda now include exit interviews, reflecting a greater focus on patients’ experiences.
“These insights, which may not have been fully understood through standard clinical trial outcome measures, demonstrate the value of incorporating patient interviews into study processes to obtain key insights that influence study design, clinical practice, and prescribing decisions,” the researchers concluded.
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