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The American Journal of Managed Care October 2019
Inflammatory Bowel Disease Readmissions Are Associated With Utilization and Comorbidity
Shirley Cohen-Mekelburg, MD, MS; Russell Rosenblatt, MD, MS; Beth Wallace, MD, MS; Nicole Shen, MD, MS; Brett Fortune, MD, MSc; Akbar K. Waljee, MD, MSc; Sameer Saini, MD, MS; Ellen Scherl, MD; Robert Burakoff, MD; and Mark Unruh, PhD
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The Long-term Social Value of Granulocyte Colony-Stimulating Factors
Alison Sexton Ward, PhD; Mina Kabiri, PhD; Aylin Yucel, PhD, MSc, MBA, MHSA, PharmD; Alison R. Silverstein, MPH; Emma van Eijndhoven, MS, MA; Charles Bowers, MD; Mark Bensink, PhD, MSc, MEd; and Dana Goldman, PhD
Variation in US Private Health Plans’ Coverage of Orphan Drugs
James D. Chambers, PhD; Ari D. Panzer, BS; David D. Kim, PhD; Nikoletta M. Margaretos, BA; and Peter J. Neumann, ScD
Ease of Ordering High- and Low-Value Services in Various Electronic Health Records
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Patients’ Expectations of Their Anesthesiologists
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En-Ju D. Lin, PhD, MPH; Jennifer L. Hefner, PhD, MPH; Xianlong Zeng, MS; Soheil Moosavinasab, MS; Thomas Huber, PhD, MS; Jennifer Klima, PhD; Chang Liu, PhD; and Simon M. Lin, MD, MBA
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Junqing Liu, PhD; Fern McCree, MPH; Doug Kanovsky, BA; Patricia Santora, PhD; Kazi Ahmed, PhD; Chirag Bhatt, MBA; and Sarah H. Scholle, DrPH

The Long-term Social Value of Granulocyte Colony-Stimulating Factors

Alison Sexton Ward, PhD; Mina Kabiri, PhD; Aylin Yucel, PhD, MSc, MBA, MHSA, PharmD; Alison R. Silverstein, MPH; Emma van Eijndhoven, MS, MA; Charles Bowers, MD; Mark Bensink, PhD, MSc, MEd; and Dana Goldman, PhD
Although currently underutilized, granulocyte colony-stimulating factor prophylaxis as supportive cancer care provides substantial value to society. Aligning utilization with clinical guidelines would increase this value considerably.

Objectives: Febrile neutropenia (FN) is a life-threatening complication of chemotherapy that can lead to hospitalizations, chemotherapy dose reductions or delays, and mortality. Granulocyte colony-stimulating factor (G-CSF) prophylaxis reduces the incidence of FN, enabling patients to undergo and remain on myelosuppressive chemotherapy. We estimate the benefits of continuing current G-CSF use patterns and an alternative that aligns prophylactic G-CSF use with guideline recommendations.

Study Design: Using The Health Economics Medical Innovation Simulation microsimulation, we estimated lifetime social value (SV) of prophylactic G-CSF for a nationally representative US population with breast, lung, and gynecological cancers and non-Hodgkin lymphoma.

Methods: SV estimates included the cost of G-CSF, FN, chemotherapy relative dose intensity (RDI) less than 85% (RDI<85%), medical spending, and deaths for 3 scenarios: current use (current G-CSF use), targeted use (100% G-CSF use among patients with high FN risk), and reduced use (current G-CSF use reduced by 20% across all FN risk categories).

Results: Over 10 years, current use, compared with no G-CSF use, would decrease cases of FN by 3.3 million, prevent 354,000 cases of RDI<85%, and generate $96 billion in SV. Compared with current use, targeted use would decrease cases of FN by an additional 3.3 million, prevent 355,000 more cases of RDI<85%, and generate another $119 billion in SV. Reduced use would increase FN and RDI<85%, lowering SV by $18 billion compared with current use.

Conclusions: Current use of G-CSF prophylaxis would provide $96 billion in SV over the next 10 years. Targeting G-CSF prophylaxis to align with guidelines would more than double SV, highlighting the substantial value of appropriate FN risk assessment and targeted G-CSF prophylaxis.

Am J Manag Care. 2019;25(10):486-493
Takeaway Points

We modeled the benefits of prophylactic granulocyte colony-stimulating factor (G-CSF) use among patients with breast, lung, and gynecological cancers and non-Hodgkin lymphoma.
  • Over 10 years, current use of G-CSF prophylaxis provides substantial benefit, reducing cases of febrile neutropenia (FN) by 3.3 million and cases of chemotherapy reduced dose intensity (RDI) less than 85% (RDI<85%) by 354,000, resulting in $96 billion in social value (SV).
  • Targeting G-CSF prophylaxis to patients at high risk of FN would increase these benefits over that period, resulting in 6.6 million fewer cases of FN, 709,000 fewer cases of RDI<85%, and an SV gain of $215 billion.
  • Appropriate FN risk assessment and targeted G-CSF prophylaxis use in patients presents an opportunity to provide substantial value to society.
Over the past 3 decades, medical and pharmaceutical innovations have made substantial strides in many disease areas that have increased both longevity and quality of life (QOL). However, these technological advancements are often met with criticism over high prices, and, despite research showing that the benefits can outweigh the costs of new therapies, these treatments may often be underutilized.1-3 For example, statins represent a huge technological breakthrough for the treatment of cardiovascular disease, and a large literature of clinical trial data and cost-effectiveness modeling demonstrates their wide-ranging cost-effectiveness, yet data still suggest that this breakthrough therapy is greatly underused.3

Similarly, prophylactic administration of granulocyte colony-stimulating factor (G-CSF) is underutilized despite guideline recommendations and literature on the cost-effectiveness of varying options.4-6 G-CSF use is well established as the standard of supportive care among patients receiving myelosuppressive chemotherapy to prevent the onset of febrile neutropenia (FN; fever and infection),7-9 which subsequently can lead to hospitalizations, dose reductions, and increased risk of mortality.10,11 National Comprehensive Cancer Network (NCCN) and American Society of Clinical Oncology guidelines recommend primary prophylactic G-CSF in patients receiving a chemotherapy regimen associated with more than a 20% risk of FN. The guidelines also suggest that G-CSF therapy should be considered for patients receiving a chemotherapy regimen with a 10% to 20% risk of FN and who have additional risk factors (eg, age ≥65 years, poor performance status, history of FN, comorbid conditions).12 Despite these clear and well-respected guidelines and evidence from meta-analyses supporting the favorable benefit–risk profile of G-CSFs,13 real-world use is lower than recommended. For example, only 51% of patients with breast cancer and 28% of patients with lung cancer with high risk of FN have been documented as receiving G-CSF prophylaxis.14

G-CSF prophylaxis continues to be the subject of much debate due to the rising costs of cancer care. Although previous research has noted the clinical benefits of these therapies and has demonstrated their cost-effectiveness, it has failed to completely account for their potential indirect benefits in the form of improved productivity and QOL.7,15,16 FN can be very debilitating and often leads to inpatient hospital stays and potentially death. Although some previous studies have accounted for reductions in QOL, they have not included lost work or reductions in productivity. There has been much discussion recently about the importance of including these indirect benefits in any value calculation, and both the Second Panel on Cost-Effectiveness17 and the International Society for Pharmacoeconomics and Outcomes Research’s Special Task Force on Value Assessment Frameworks18 recommended doing so where possible. Our study builds upon the current literature by including these indirect sources of value in our model and estimating the accumulation of value over patients’ lifetimes.

Additionally, our model builds on the current literature by estimating the social value (SV) both at the US population level of current G-CSF prophylaxis use and at a level of use better aligned with NCCN guidelines. SV quantifies the resources that society would be willing to give up in order to achieve the health, QOL, and productivity benefits associated with G-CSF prophylaxis. This study represents the first comprehensive estimate of the benefit to society from G-CSF prophylaxis use that better aligns with guidelines.


We used The Health Economics Medical Innovation Simulation (THEMIS), an established microsimulation model based on the Future Elderly Model,19,20 to predict the lifetime value associated with use of G-CSF consistent with NCCN guidelines.12 A patient-level microsimulation like THEMIS allows costs and benefits to be estimated over individuals’ lifetimes within a variety of subgroups while capturing the heterogeneity that would be lost in a cohort simulation. Our model focused on 4 patient populations with high rates of use of myelosuppressive chemotherapy regimens categorized as high and intermediate FN risk,12 and it documented use of G-CSF prophylaxis that is not consistent with NCCN guidelines—overuse in the population at low risk of FN and/or underuse in the population at high risk of FN. Specifically, we focused on patients with breast, gynecological, and lung cancers and non-Hodgkin lymphoma (NHL) who were 25 years or older, and we simulated their health status, health spending, and mortality experience over their lifetimes.

THEMIS is based on data from the Panel Study of Income Dynamics (PSID)21 and the Health and Retirement Study.22 Both data sets are nationally representative panel surveys that have been ongoing since 1968 and 1992, respectively. The breast, gynecological, and lung cancer cohorts were modeled using the PSID data; however, the survey does not differentiate patients with NHL from patients with other lymphomas. Thus, we built a synthetic cohort for the NHL population based on Surveillance and Epidemiology End Results (SEER) data. A raking procedure, which adjusts the weights of the PSID cancer population to match the distributions of the NHL population in SEER, was combined with SEER NHL prevalence and incidence data and the National Cancer Institute lymphoma incidence projections to compute a population of prevalent and incident NHL cases.23-27

Copyright AJMC 2006-2020 Clinical Care Targeted Communications Group, LLC. All Rights Reserved.
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