Defense Date

Fall 9-6-2017


One-year Embargo

Submission Type


Degree Name



Pharmacy Administration


School of Pharmacy

Committee Chair

Khalid M Kamal

Committee Member

Kumar Mukherjee

Committee Member

Jordan R Covvey

Committee Member

Vincent Giannetti


MEPS, economic burden, healthcare resource utilization, incremental costs, PMPM, PTMPM, ICER, ACER, budget impact analysis, cost effectiveness analysis


Objectives: (1) To estimate the burden of illness in cystic fibrosis (CF) using 2010-2014 Medical Expenditure Panel Survey (MEPS) data, and (2) to conduct a pharmacoeconomic evaluation of lumacaftor/ivacaftor in the treatment of CF.

Methods: The study was conducted in two parts. Part 1 involved a retrospective analysis using individuals in MEPS database with an International Classification of Diseases, Ninth Revision, Clinical Modification (ICD-9-CM) diagnosis code of 277.00 for a principal diagnosis of CF. Dependent variables were cumulative and out-of-pocket (OOP) expenditures and independent variables included patient demographics and clinical characteristics. Unweighted and weighted estimates of expenditure in CF group, and an incremental cost burden in CF group compared to non-CF group was estimated. In Part 2, static decision models were developed using MS Excel® to evaluate the cost effectiveness and budget impact of lumacaftor/ivacaftor over a 1-year time frame from a payer perspective. Model inputs included drug costs (wholesale acquisition costs from 2015 Redbook), drug administration/monitoring costs from package inserts, Centers for Medicare and Medicaid 2016 Physician Fee Schedule and published literature, percentage predicted forced expiratory volume (FEV1% predicted)and pulmonary exacerbation values from TRAFFIC and TRANSPORT clinical trials and cost to treat pulmonary exacerbations from published literature. The outcomes in the CEA included total cost of therapy, average cost effectiveness ratio (ACER) defined as cost per FEV1% predictedand incremental cost effectiveness ratio (ICER) defined as difference in the ratio of cost per FEV1% predictedof lumacaftor/ivacaftor and placebo. Outcomes in the BIA included total budget impact, cost per member per month (PMPM) defined as total budget impact per hypothetical plan population, and cost per treated member per month (PTMPM) defined as total budget impact per target CF population. All costs were adjusted to 2016 dollars and one-way sensitivity analyses were conducted to test the model robustness given uncertainty in model inputs and study assumptions

Results: The average annual cost of CF care in the US between 2010-2014 was approximately $44,600, with prescription medications being the highest cost contributor. The mean cumulative expenditure in children with CF was $13,990 compared to $3,231.27 non-CF group, whereas in adults with CF it was $16,975.80 compared to $8,859.87 in non-CF group. The annual cost of therapy per patient due to lumacaftor/ivacaftor was $379,780.11 in 2016. The ACER in patients treated by lumacaftor/ivacaftor was $151,912.044 while the ICER compared to a placebo was $95,016.28 per FEV1% predicted. The annual total budget impact due to inclusion of lumacaftor/ivacaftor on the health plan formulary was $266,045.59. The PMPM cost was $0.022 and the PTMPM cost was $6.2067.

Conclusion: The systematic assessment of incremental CF costs and its impact on the society is essential in increasing the awareness of decision makers to implement intervention strategies that are effective in lowering the disease incidence and the overall cost of disease management. In CF patients, lumacaftor/ivacaftor has demonstrated better clinical effectiveness compared to placebo alongside an increased drug acquisition cost. However, the therapy may be a viable alternative to existing standard therapy over a short-time horizon. Healthcare payers, both private and public, need to evaluate the cost effectiveness and the financial impact when considering expansion of new drug coverage in CF management.



Available for download on Thursday, December 13, 2018