Product information and disease description Place of product in therapy Disease description Burden of Erythropoietic protoporphyria or X-linked protoporphyria

Economic burden and healthcare resource utilization

7 evidence topics · 4 sources

Resource use and total cost compared with matched comparators

“Over a mean follow-up of 30 months, patients with EPP/XLP had significantly higher all-cause HRU compared with comparators, with a mean PPPY number of inpatient stays of 0.8 vs 0.2 (RR = 3.4; P < 0.001), emergency department visits of 1.5 vs 0.9 (RR = 1.7; P = 0.002), and outpatient visits of 35.2 vs 17.5 (RR = 2.0; P < 0.001). All-cause costs were also significantly higher among patients with EPP/XLP compared with comparators with a mean PPPY total cost of $71,714 vs $18,646 (ratio = 3.9; P < 0.001), driven by inpatient costs (mean = $30,909 vs $6,318; ratio = 4.9; P < 0.001) and outpatient costs (mean = $33,416 vs $7,573; ratio = 4.4; P < 0.001).” (opens the source at this quote in a new tab)

Disease-specific and comorbidity-related cost components

“Medical costs related to EPP/XLP-associated comorbidities PPPY among patients with EPP/XLP were nearly 6 times those among matched comparator patients (mean = $25,611 [SD = $173,731] vs $4,298 [SD = $30,562]; cost ratio = 5.96; P < 0.001)” (opens the source at this quote in a new tab)
“and represented almost 40% of the total all-cause medical costs.” (opens the source at this quote in a new tab)
“Among patients with EPP/XLP, the mean total EPP/XLP-specific medical costs PPPY were $17,448 (SD = $90,366), approximately one-fourth of total all-cause health care costs, and were mainly driven by EPP/XLP-specific OP costs (mean = $14,534 [$83,836” (opens the source at this quote in a new tab)

Medication use in the absence of disease-directed therapy

“Although only 7.6% of patients with EPP/XLP received treatment for EPP/XLP, most commonly afamelanotide (3.9%), most (68.4%) used medication related to EPP/XLP-associated comorbidities, including narcotics (46.3%), nonsteroidal anti-inflammatory drugs (38.2%), and antidepressants (35.1%).” (opens the source at this quote in a new tab)

Concentration of burden among adolescents

“This study found that patients with EPP/XLP have significantly higher health care use and costs compared with those without the condition. Few patients received treatment for EPP/XLP, and the economic burden was particularly high among adolescents, who currently have no approved therapies.” (opens the source at this quote in a new tab)
“The adolescent subgroup included 28 patients with EPP/XLP and 112 matched comparator patients. Descriptive results in this subgroup (not compared statistically because of the low sample size) show that these patients also incurred numerically higher HRU and costs compared with the matched comparator cohort with relative rates and cost ratios even higher than those observed in the overall sample (all-cause IP stays: RR = 14.72; days hospitalized: RR = 28.86; ED visits: RR = 1.48; OP visits: RR = 3.19; and total all-cause health care costs: cost ratio = 16.36 in the adolescent subgroup” (opens the source at this quote in a new tab)

Patient-reported visit frequency

“Adults had a mean number of eight physician visits related to EPP in the previous 12 months, and adolescents had a mean number of 3.2 visits. Adults averaged 5.5 emergency room visits annually.” (opens the source at this quote in a new tab)

Higher resource use among those with liver disease

“EPP-related HCU in the previous 12 months was higher for those with liver disease than those without liver disease, including more physician visits (mean of 16.5 versus 6.0) and emergency room visits (mean of 9.0 versus 1.9).” (opens the source at this quote in a new tab)

Encounter counts and cost in a medical-record cohort

“Within 12 months of diagnosis, the mean (SD; median) number of office visits, emergency department visits, and inpatient hospitalizations related to EPP or XLP were 4.0 (3.5; 3.0), 0.8 (1.6; 0), and 0.4 (1.3; 0), respectively.” (opens the source at this quote in a new tab)
“Inpatient hospitalizations and ED encounters specifically related to EPP or XLP were seen in fewer than 40% of patients. However, these encounters were costly, resulting in a mean cost of approximately USD2700 per patient.” (opens the source at this quote in a new tab)

Monetized indirect costs and productivity loss

No evidence found.