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Clinical trials with first- and second-generation epidermal growth factor receptor tyrosine kinase inhibitors (EGFR-TKIs) reported severe adverse events (SAEs) in 6%-49% of patients with EGFR-mutated non-small cell lung cancer. This study describes incremental healthcare resource utilization (HRU) and costs associated with real-world management of AEs in this population, with a focus on SAEs.Patients receiving erlotinib, gefitinib, or afatinib as first-line (1L) monotherapy were identified from IQVIA™ Real-World Data Adjudicated Claims-US database (04/01/2012-03/31/2017). Relevant AEs were selected from corresponding prescribing information; SAEs were identified from hospitalization claims. HRU and cost per-patient-per-month (PPPM) were assessed during 1L treatment and compared for patients with and without each AE using multivariate Poisson and linear regression, respectively, adjusting for baseline characteristics.Of 1646 patients, 86.9% were treated with erlotinib, 12.1% with afatinib, and 1.0% with gefitinib. In 1L, 12.2% of patients had ≥1 acute SAE (220.1/1000 patient-years). Patients with any SAE had higher PPPM costs than patients without SAEs (cost difference = $4700, p 0.001). Incremental costs ranged from $2604 PPPM for diarrhea to $10,143 PPPM for microangiopathic hemolytic anemia (MAHA), and were statistically significant for all SAEs (all p 0.001) except MAHA (p 0.0528). Patients with any SAEs had higher rates of HRU relative to patients without SAEs (hospitalization rate ratio = 6.15; outpatient visits rate ratio = 1.21; all p 0.001).More than one-tenth of patients experienced SAEs, resulting in sizeable economic burden with respect to HRU and costs. EGFR-TKIs with more favorable safety profiles may reduce the burden of managing this population.

Context Real-world data on treatment patterns and outcomes in patients with RRMM resistant to multiple drug classes are limited. Objective To assess clinical treatment patterns and refractory status in patients with RRMM. Methods This longitudinal retrospective cohort study utilized the COTA de-identified real-world database derived from US electronic medical records of partnered healthcare providers from Q3 1988 through Q1 2020; 650 adults with active RRMM previously exposed to ≥1 proteasome inhibitor (PI) and 1 immunomodulatory drug (double-exposed) and who received ≥3 prior lines of therapy (LOTs) were identified. Patients were further categorized as refractory to a PI and an immunomodulatory drug (double-class refractory [DCR]; n=381) or additionally to an anti-CD38 monoclonal antibody (i.e., daratumumab; triple-class refractory [TCR]; n=173). Index LOT was the fourth (double-exposed) or new LOT after evidence of DCR or TCR status following ≥3 or ≥4 prior LOTs (DCR or TCR, respectively). Median follow-ups from index LOT initiation through end of data availability/death were 24 (double-exposed), 14 (DCR), and 8 (TCR) months. Assessments included therapies received before/during the index LOT, duration of treatment (DOT), and treatment discontinuation reasons. Results Patient characteristics included age ≥65 years (49% DCR; 53% TCR), International Staging System stage III (14%; 16%), and high-risk cytogenetics (56%; 66%); >62% had prior autologous stem cell transplantation. Patients had a median of 3 (DCR) and 6 (TCR) prior LOTs. Retreatment percentages among DCR patients were 60% (PI) and 69% (immunomodulatory drug); 30% were exposed to daratumumab. For TCR patients, retreatment percentages were 65% (PI), 60% (immunomodulatory drug), and 29% (daratumumab). Median DOTs were 3.3 (DCR) and 2.8 months (TCR). Patients discontinued index therapy most commonly due to disease progression (59% DCR; 60% TCR) or toxicity (23%; 24%). After index LOT, 70% DCR and 58% TCR patients continued to a subsequent LOT. Conclusions Treatment options are limited for US patients with heavily pretreated RRMM. DCR/TCR patients were frequently retreated with a PI, immunomodulatory drugs, or daratumumab (TCR patients) despite refractoriness to these agents. Many DCR/TCR patients stopped active MM treatment after discontinuing index treatment. Funding GlaxoSmithKline (212953).

Presented at the ISPOR 20th Annual International Meeting, Philadelphia, PA, USA, May 16–20, 2015 • As prenatal testing for Down syndrome (DS) evolves, expectant parents must make decisions about their pregnancy options with limited time and information1 • Cuckle et al. estimated the incremental lifetime cost of raising a child with DS to be as high as $900,000 (2013 USD)2; this study used estimates from Waitzman et al.3 which included direct costs and indirect costs over the entire lifespan for 558 individuals born in California in 1998 with DS • Boulet et al. have also estimated that the annual average incremental medical costs incurred by insurers for children with DS under 5 years of age was approximately $33,347 (2004 USD)4; their study does not address individual out-of-pocket costs to parents • Expectant couples making pregnancy decisions are more interested in knowing what their individual out-of-pocket expenses for raising a child with DS will be rather than societal costs or medical costs incurred by their insurance company • Little information about costs from the individual parental perspective exists in the current literature To estimate the out-of-pocket health care costs associated with raising a child with DS between birth and 18 years of age from the perspective of commercially insured parents BACKGROUND STUDY OBJECTIVE