Assessing the Effect of Injectable or Oral Routes of Treatment Administration on Patient-Reported Outcomes and out-of-Pocket Costs Among Patients with Multiple Myeloma
Authors: Gupta Shaloo , Abouzaid Safiya , Liebert Ryan , Parikh Kejal , Rosenberg Aaron S 
Published: November 2019

Abstract
Introduction: Healthcare resource use and patient preference play a major role in treatment decisions for multiple myeloma (MM); therefore, it is important to understand the impact of cancer treatment on patient-reported outcomes in a real-world setting. This study aimed to characterize the impact of MM treatments by oral or injectable routes of administration on work productivity, healthcare resource use, and out-of-pocket costs, from the patient perspective.
Methods: This was a cross-sectional survey of 137 US MM patients identified from the Lightspeed GMI global panel and its partners, aged ≥18 years who were currently on a treatment regimen comprised of either oral therapy only (oral users) or on injectable therapy with or without oral medication (injectable users). Patients completed a web based self-administered survey and the study assessed patient's experiences using the Work Productivity and Activity Impairment (WPAI) questionnaire and patient-centric questions related to MM. Patient characteristics, treatment history, healthcare resource use and patient-reported outcomes were summarized and compared between oral and injectable users. Multivariable analyses estimated the association between oral vs. injectable route of administration and outcomes of the WPAI, the number of oncologist/hematologist visits in the past three months, hours spent at appointments related to MM treatment in the past month, feeling upset or frustrated while at the doctor's office (not at all/rarely vs. somewhat often/often/very often; 5 point Likert scale), out-of-pocket costs for oncologist/hematologist visits in the past three months, and financial burden regarding all MM related out-of-pocket expenses (no burden/some burden vs. moderate/high/extremely high burden; 5 point Likert scale). Analyses were adjusted for patient characteristics that differed significantly between oral and injectable users (sex, line of therapy, number of treatments, length of time of current regimen, number of days since last treatment dose, prior radiation treatment, education, BMI, and performance status).
Results: A total of 137 respondents participated in the study, of which 43.1% reported currently being on an oral only regimen and 56.9% on an injectable regimen. Patient characteristics were generally similar between treatment cohorts with an average age of 55.7 years (SD=13.7), mean time since diagnosis of 4.3 years (SD=4.3), currently employed (44.5%), having health insurance coverage (97.1%) and mean number of comorbidities (mean=0.3 (SD=0.9), adjusted Charlson comorbidity index). Oral users were less likely to report prior radiation treatment (1.7% vs. 12.8%), and more likely to be on 1st line therapy (64.4% vs. 42.3%, p=0.024) than injectable users. On average oral users reported being on less MM prescriptions in their current regimen than injectable users (1.2 vs. 1.7, p<0.001). In univariate analysis of the WPAI, among the employed sample, oral users reported significantly lower overall work impairment than injectable users (52.4% vs. 68.3%, p=0.049). This was primarily driven by decreased impairment while at work (presenteeism: 46.8% vs. 62.3%, p=0.044) while no difference in absenteeism from work due to impairment (17.9% vs. 21.8%, p=0.446) was found. In multivariable analysis, these differences were no longer significant.
After adjusting for patient characteristics, oral users reported fewer oncologist/hematologist visits in the past three months (3.0 vs. 4.7, p=0.027) and lower out-of-pocket costs for all these visits ($130.47 vs $329.49, p=0.025). Oral users were numerically more likely to report no burden/some financial burden regarding all MM related out-of-pocket expenses (45.7% vs. 30.9%, p=0.221), and spend less time spent at appointments related to MM treatments in the past month (5.4 vs. 7.2 hours, p=0.145). Oral users were more likely to report not at all/rarely experiencing frustration while at the doctor's office (80.6% vs. 57.1%, p=0.042).
Conclusions: Findings suggest that an all-oral treatment regimen was associated with less healthcare resource use and financial burden than an injectable treatment regimen, and potentially less work productivity loss. However, the latter should be interpreted with caution given the small sample size of employed respondents. These findings should be taken into consideration while making treatment decisions with MM patients.

Disclosures
Gupta: Celgene Corporation: Consultancy, Research Funding; Kantar Health: Employment. Abouzaid:Celgene Corporation: Employment, Equity Ownership, Research Funding. Liebert:Kantar Health: Employment; Celgene Corporation: Consultancy, Research Funding. Parikh:Celgene Corporation: Employment, Equity Ownership, Research Funding.