Product information and disease description Place of product in therapy Disease description Burden of RAF-altered pediatric low-grade glioma requiring first-line systemic therapy

Economic burden and healthcare resource utilization

5 evidence topics · 2 sources

Summary: healthcare resource use and costs

No study reporting total direct medical costs of pLGG care in the United States was identified. A Day One-sponsored U.S. study of linked claims and electronic health records (Optum Market Clarity) followed 154 patients aged 18 years or younger with pLGG for 36 months: most patients (95% to 98%) had office or outpatient visits in each 6-month interval, 18% to 28% visited an emergency room, and 9% to 27% had an inpatient stay; mean inpatient stay duration increased from 5.1 days at baseline to 8.1 days during follow-up. Over the study period, 74% of patients filled prescriptions for anti-infectives, 56% for antiemetics, and 65% received pLGG-directed treatment (most often brain surgery); chemotherapy was recorded in 14%. Tumor histology and molecular status could not be characterized.

For long-term survivorship, a Taiwanese national claims study of 33,105 five-year survivors of childhood cancer or benign brain tumor (median follow-up 7 years) reported median annual medical expenses of US $614.08 for brain cancer survivors (n = 2,241) and US $336.89 for benign brain tumor survivors (n = 7,825), versus US $203.90 for 64,754 matched individuals without cancer. No publication was identified that quantifies infusion visit, central venous access, or hospitalization costs of first-line carboplatin and vincristine or vinblastine in pLGG.

U.S. linked claims and EHR study: cohort and overall healthcare resource utilization

“Optum ® Market Clarity Data were used to identify patients aged ≤18 years with an ICD-10 code for brain neoplasm, ≥1 physician notes, and with evidence of pLGG recorded between January 1, 2017 and June 30, 2018. Outcomes including health characteristics, HRU, medications, and procedures were assessed at 6-month intervals over 36 months.” (opens the source at this quote in a new tab)
“HRU, including speciality care visits, filled prescriptions, and administered medications, was common; 74% of patients had prescriptions for anti-infectives, 56% antiemetics, and 52% required pain or fever relief. Sixty-five percent of patients underwent treatment to control their pLGG, the most common being brain surgery. Little decline was observed in medication use during the study period.” (opens the source at this quote in a new tab)
“Eighty-six patients (56%) received commercial benefits and 68 patients (44%) received Medicaid benefits.” (opens the source at this quote in a new tab)
“Patients with pLGG have complex healthcare needs requiring high HRU, often over a long time.” (opens the source at this quote in a new tab)

U.S. linked claims and EHR study: sites of care, inpatient stays, and emergency room use

“Most patients (95–98% across the study period) visited an office or outpatient facility, 38–50% a diagnostic laboratory, 18–28% an ER, and 9–27% an inpatient facility in each of the 6-month intervals ( Figure 2D ).” (opens the source at this quote in a new tab)
“However, the mean inpatient stay duration increased by 59% from 5.1 days during the baseline period to 8.1 days during the follow-up period ( Figure 2E ).” (opens the source at this quote in a new tab)
“Time spent in ER visits and inpatient stays places a substantial burden on families and adds to time spent away from work, school, and normal activities, potentially adding to financial burden and overall stress.” (opens the source at this quote in a new tab)

U.S. linked claims and EHR study: pLGG-directed treatments and limitations

“In the overall study period, 65% of patients received pLGG-specific treatment, the most common being emergency brain surgery (55%) and planned brain surgery (27%) ( Figure 3D ).” (opens the source at this quote in a new tab)
“Chemotherapy was given to 14% of patients during the study, with the highest proportion of patients receiving it in the first 6-month observation interval (7%) and its use declining over the follow-up period.” (opens the source at this quote in a new tab)
“In particular, it was not possible to: characterize the tumors histologically or genomically, ascertain the patient’s history of disease, anatomical primary site, or determine their stage on the treatment journey.” (opens the source at this quote in a new tab)

Long-term survivorship: annual medical expenses of brain tumor survivors (Taiwan national claims)

“A total of 33,105 children had survived for at least 5 years after the first appearance of a diagnostic code of cancer or a benign brain tumor before the age of 18 years from 2000 to 2010 with follow-up to 2015.” (opens the source at this quote in a new tab)
“Survivors with female gender, diagnosis before the age of 3 years, and diagnosis of brain cancer or a benign brain tumor had significantly higher annual outpatient expenses (all P<.001). Moreover, the analysis of outpatient medication costs showed that hormonal and neurological medications comprised the 2 largest costs in brain cancer and benign brain tumor survivors.” (opens the source at this quote in a new tab)

Group	Median annual total expense, US $ per person (IQR)
Brain cancer survivors (n=2241)	“614.08 (267.05-1793.94)”
Benign brain tumor survivors (n=7825)	“336.89 (184.64-652.14)”
Hematologic cancer survivors (n=3934)	“268.48 (159.50-475.08)”
No cancer individuals (n=64,754)	“203.90 (118.98-347.55)”

Direct medical costs of first-line chemotherapy administration, central venous access, and survivorship care in U.S. pLGG

No evidence found.