Currently Viewing:
The American Journal of Managed Care October 2016
Cost-Effectiveness of a Statewide Falls Prevention Program in Pennsylvania: Healthy Steps for Older Adults
Steven M. Albert, PhD; Jonathan Raviotta, MPH; Chyongchiou J. Lin, PhD; Offer Edelstein, PhD; and Kenneth J. Smith, MD
Economic Value of Pharmacist-Led Medication Reconciliation for Reducing Medication Errors After Hospital Discharge
Mehdi Najafzadeh, PhD; Jeffrey L. Schnipper, MD, MPH; William H. Shrank, MD, MSHS; Steven Kymes, PhD; Troyen A. Brennan, MD, JD, MPH; and Niteesh K. Choudhry, MD, PhD
Benchmarking Health-Related Quality-of-Life Data From a Clinical Setting
Janel Hanmer, MD, PhD; Rachel Hess, MD, MS; Sarah Sullivan, BS; Lan Yu, PhD; Winifred Teuteberg, MD; Jeffrey Teuteberg, MD; and Dio Kavalieratos, PhD
Patients' Success in Negotiating Out-of-Network Bills
Kelly A. Kyanko, MD, MHS, and Susan H. Busch, PhD
Connected Care: Improving Outcomes for Adults With Serious Mental Illness
James M. Schuster, MD, MBA; Suzanne M. Kinsky, MPH, PhD; Jung Y. Kim, MPH; Jane N. Kogan, PhD; Allison Hamblin, MSPH; Cara Nikolajski, MPH; and John Lovelace, MS
A Call for a Statewide Medication Reconciliation Program
Elisabeth Askin, MD, and David Margolius, MD
Postdischarge Telephone Calls by Hospitalists as a Transitional Care Strategy
Sarah A. Stella, MD; Angela Keniston, MSPH; Maria G. Frank, MD; Dan Heppe, MD; Katarzyna Mastalerz, MD; Jason Lones, BA; David Brody, MD; Richard K. Albert, MD; and Marisha Burden, MD
Mortality Following Hip Fracture in Chinese, Japanese, and Filipina Women
Minal C. Patel, MD; Malini Chandra, MS, MBA; and Joan C. Lo, MD
Currently Reading
Estimating the Social Value of G-CSF Therapies in the United States
Jacqueline Vanderpuye-Orgle, PhD; Alison Sexton Ward, PhD; Caroline Huber, MPH; Chelsey Kamson, BS; and Anupam B. Jena, MD, PhD
Does Medicare Managed Care Reduce Racial/Ethnic Disparities in Diabetes Preventive Care and Healthcare Expenditures?
Elham Mahmoudi, PhD; Wassim Tarraf, PhD; Brianna L. Maroukis, BS; and Helen G. Levy, PhD

Estimating the Social Value of G-CSF Therapies in the United States

Jacqueline Vanderpuye-Orgle, PhD; Alison Sexton Ward, PhD; Caroline Huber, MPH; Chelsey Kamson, BS; and Anupam B. Jena, MD, PhD
Granulocyte-colony stimulating factors (G-CSFs) reduce the risk of febrile neutropenia in patients with cancer. This study evaluates the clinical and nonclinical value associated with G-CSFs.
This study estimates that 314,442 patients with cancer were potential candidates for prophylactic G-CSFs in the United States in 2014. Based on the parameters and assumptions used in our calculations, G-CSFs generated a total of almost $8.5 billion in SV. More than 97% of this value was attributable to estimated improvements in clinical outcomes. Importantly, reductions in overall mortality from both reduced FN hospitalizations and chemotherapy dose reductions avoided accounted for $10 billion in SV. Since we found few studies that investigated improvements in nonclinical outcomes associated with G-CSFs, our SV estimate of $232 million may be a conservative lower bound for the total nonclinical value.

By considering all possible benefits of G-CSFs, our study results suggest these therapies provide substantial value for society, particularly for patients. The portion of value accruing to manufacturers in the form of profits is approximately 15%. These estimates are similar to previous work that suggests that between 5% and 19% of the value generated by gains in cancer survival have been appropriated by manufacturers in the form of profits.47 Our analysis further demonstrates the need to evaluate the cost-effectiveness of medical innovations in a way that incorporates broader impacts to society, such as nonclinical benefits.  Failure to include these components can significantly underestimate the economic value of medical innovations and the value to patients. 

Author Affiliations: Precision Health Economics (JV-O, ASW, CH, CK), Los Angeles, CA; Harvard Medical School, Department of Health Care Policy (ABJ), Boston, MA.

Source of Funding: This study was supported by Amgen, Inc.

Author Disclosures: Drs Vanderpuye-Orgle and Sexton, Ms Huber, and Ms Kamson are employees of Precision Health Economics (PHE), which was compensated by Amgen to conduct the study. Dr Jena is a consultant at PHE.

Authorship Information: Concept and design (ABJ, ASW, JV-O); acquisition of data (CH, ABJ, CK, ASW); analysis and interpretation of data (CH, ABJ, ASW, JV-O); drafting of the manuscript (CH, ABJ, CK, ASW, JV-O); critical revision of the manuscript for important intellectual content (CH, ABJ, JV-O, ASW); statistical analysis (ABJ, ASW); provision of patients or study materials (ASW, JV-O); obtaining funding (ASW, JV-O); administrative, technical, or logistic support (CK); and supervision (ABJ, JV-O).

Address Correspondence to: Anupam B. Jena, MD, PhD, Harvard Medical School, Department of Health Care Policy, 180 Longwood Ave, Boston, MA 02115-5899. E-mail:

1. Aarts MJ, Grutters JP, Peters FP, et al. Cost effectiveness of primary pegfilgrastim prophylaxis in patients with breast cancer at risk of febrile neutropenia. J Clin Oncol. 2013;31(34):4283-4289. doi: 10.1200/JCO.2012.48.3644.

2. Wang X-J, Lopez S-E, Chan A. Economic burden of chemotherapy-induced febrile neutropenia in patients with lymphoma: a systematic review. Crit Rev Oncol Hematol. 2015;94(2):201-212. doi: 10.1016/j.critrevonc.2014.12.011.

3. Caggiano V, Weiss RV, Rickert TS, Linde‚ÄźZwirble WT. Incidence, cost, and mortality of neutropenia hospitalization associated with chemotherapy. Cancer. 2005;103(9):1916-1924.

4. Schilling MB, Parks C, Deeter RG. Costs and outcomes associated with hospitalized cancer patients with neutropenic complications: a retrospective study. Exp Ther Med. 2011;2(5):859-866.

5. De Naurois J, Novitzky-Basso I, Gill M, Marti FM, Cullen MH, Roila F; ESMO Guidelines Working Group. Management of febrile neutropenia: ESMO clinical practice guidelines. Ann Oncol. 2010;21(suppl 5):v252-v256. doi: 10.1093/annonc/mdq196.

6. Rajan SS, Lyman GH, Carpenter WR, Stearns SC. Chemotherapy characteristics are important predictors of primary prophylactic CSF administration in older patients with breast cancer. Breast Cancer Res Treat. 2011;127(2):511-520. doi: 10.1007/s10549-010-1216-1.

7. Shayne M, Crawford J, Dale DC, Culakova E, Lyman GH; ANC Study Group. Predictors of reduced dose intensity in patients with early-stage breast cancer receiving adjuvant chemotherapy. Breast Cancer Res Treat. 2006;100(3):255-262.

8. Kuderer NM, Dale DC, Crawford J, Cosler LE, Lyman GH. Mortality, morbidity, and cost associated with febrile neutropenia in adult cancer patients. Cancer. 2006;106(10):2258-2266.

9. Smith TJ, Bohlke K, Lyman GH, et al; American Society of Clinical Oncology. Recommendations for the use of WBC growth factors: American Society of Clinical Oncology clinical practice guideline update. J Clin Oncol. 2015;33(28):3199-3212. doi: 10.1200/JCO.2015.62.3488.

10. Crawford J, Ozer H, Stoller R, et al. Reduction by granulocyte colony-stimulating factor of fever and neutropenia induced by chemotherapy in patients with small-cell lung cancer. N Engl J Med. 1991;325(3):164-170.

11. Aapro MS, Cameron DA, Pettengell R, et al; European Organisation for Research and Treatment of Cancer (EORTC) Granulocyte Colony-Stimulating Factor (G-CSF) Guidelines Working Party. EORTC guidelines for the use of granulocyte-colony stimulating factor to reduce the incidence of chemotherapy-induced febrile neutropenia in adult patients with lymphomas and solid tumours. Eur J Cancer. 2006;42(15):2433-2453.

12. Lieschke GJ, Burgess AW. Granulocyte colony-stimulating factor and granulocyte-macrophage colony-stimulating factor. N Engl J Med. 1992;327(1):28-35.

13. Dale DC. Colony-stimulating factors for the management of neutropenia in cancer patients. Drugs. 2002;62(suppl 1):1-15.

14. Freyer G, Ligneau B, Trillet-Lenoir V. Colony-stimulating factors in the prevention of solid tumors induced by chemotherapy in patients with febrile neutropenia. Int J Antimicrob Agents. 1998;10(1):3-9.

15. Barnes G, Pathak A, Schwartzberg L. G-CSF utilization rate and prescribing patterns in United States: associations between physician and patient factors and G-CSF use. Cancer Med. 2014;3(6):1477-1484. doi: 10.1002/cam4.344.

16. Waters GE, Corrigan P, Gatesman M, Smith TJ. Comparison of pegfilgrastim prescribing practice to national guidelines at a university hospital outpatient oncology clinic. J Oncol Pract. 2013;9(4):203-206. doi: 10.1200/JOP.2012.000662.

17. Flynn TN, Kelsey SM, Hazel DL, Guest JF. Cost effectiveness of amphotericin B plus G-CSF compared with amphotericin B monotherapy. treatment of presumed deep-seated fungal infection in neutropenic patients in the UK. Pharmacoeconomics. 1999;16(5, pt 2):543-550.

18. Whyte S, Cooper KL, Stevenson MD, Madan J, Akehurst R. Cost-effectiveness of granulocyte colony–stimulating factor prophylaxis for febrile neutropenia in breast cancer in the United Kingdom. Value Health. 2011;14(4):465-474. doi: 10.1016/j.jval.2010.10.037.

19. Silber JH, Fridman M, Shpilsky A, et al. Modeling the cost-effectiveness of granulocyte colony-stimulating factor use in early-stage breast cancer. J Clin Oncol. 1998;16(7):2435-2444.

20. Wildiers H, Reiser M. Relative dose intensity of chemotherapy and its impact on outcomes in patients with early breast cancer or aggressive lymphoma. Crit Rev Oncol Hematol. 2011;77(3):221-240. doi: 10.1016/j.critrevonc.2010.02.002.

21. Aarts MJ, Peters FP, Mandigers CM, et al. Primary granulocyte colony-stimulating factor prophylaxis during the first two cycles only or throughout all chemotherapy cycles in patients with breast cancer at risk for febrile neutropenia. J Clin Oncol. 2013;31(34):4290-4296. doi: 10.1200/JCO.2012.44.6229.

22. Myeloid growth factors. National Comprehensive Cancer Network website. Accessed September 4, 2013.

23. Eldar-Lissai A, Cosler LE, Culakova E, Lyman GH. Economic analysis of prophylactic pegfilgrastim in adult cancer patients receiving chemotherapy. Value Health. 2008;11(2):172-179. doi: 10.1111/j.1524-4733.2007.00242.x.

24. Dulisse B, Li X, Gayle JA, et al. A retrospective study of the clinical and economic burden during hospitalizations among cancer patients with febrile neutropenia. J Med Econ. 2013;16(6):720-735. doi: 10.3111/13696998.2013.782034.

25. Braithwaite RS, Meltzer DO, King Jr JT, Leslie D, Roberts MS. What does the value of modern medicine say about the $50,000 per quality-adjusted life-year decision rule? Med Care. 2008;46(4):349-356. doi: 10.1097/MLR.0b013e31815c31a7.

26. Weinstein MC. How much are Americans willing to pay for a quality-adjusted life year? Med Care. 2008;46(4):343-345. doi: 10.1097/MLR.0b013e31816a7144.

27. Calhoun EA, Chang C-H, Welshman EE, Fishman DA, Lurain JR, Bennett CL. Evaluating the total costs of chemotherapy-induced toxicity: results from a pilot study with ovarian cancer patients. Oncologist. 2001;6(5):441-445.

28. Fortner BV, Tauer KW, Okon T, Houts AC, Schwartzberg LS. Experiencing neutropenia: quality of life interviews with adult cancer patients. BMC Nurs. 2005;4:4.

29. Consumer Price Index—all urban consumers [2014]. Bureau of Labor Statistics website. Accessed November 17 2014, 2014.

30. SEER stat fact sheets: cancer of any site. National Cancer Institute website. Accessed November 5, 2014.

31. Howard J. WTC Health Program: myeloid malignancies. CDC website. Published February 1, 2014. Accessed September 2016.

32. Naeim A, Henk HJ, Becker L, et al. Pegfilgrastim prophylaxis is associated with a lower risk of hospitalization of cancer patients than filgrastim prophylaxis: a retrospective United States claims analysis of granulocyte colony-stimulating factors (G-CSF). BMC Cancer. 2013;13:11. doi: 10.1186/1471-2407-13-11.

33. Flowers CR, Seidenfeld J, Bow EJ, et al. Antimicrobial prophylaxis and outpatient management of fever and neutropenia in adults treated for malignancy: American Society of Clinical Oncology clinical practice guideline. J Clin Oncol. 2013:31(6):794-810. doi: 10.1200/JCO.2012.45.8661.

34. Crawford J, Dale DC, Kuderer NM, et al. Risk and timing of neutropenic events in adult cancer patients receiving chemotherapy: the results of a prospective nationwide study of oncology practice. J Natl Compr Canc Netw. 2008;6(2):109-118.

35. Cooper KL, Madan J, Whyte S, Stevenson MD, Akehurst RL. Granulocyte colony-stimulating factors for febrile neutropenia prophylaxis following chemotherapy: systematic review and meta-analysis. BMC Cancer. 2011;11:404. doi: 10.1186/1471-2407-11-404.

36. Kuderer NM, Dale DC, Crawford J, Lyman GH. Impact of primary prophylaxis with granulocyte colony-stimulating factor on febrile neutropenia and mortality in adult cancer patients receiving chemotherapy: a systematic review. J Clin Oncol. 2007;25(21):3158-3167.

37. Liu PH, Wang JD, Keating NL. Expected years of life lost for six potentially preventable cancers in the United States. Prev Med. 2013;56(5):309-313. doi: 10.1016/j.ypmed.2013.02.003.

38. Ko CY, Maggard M, Livingston EH. Evaluating health utility in patients with melanoma, breast cancer, colon cancer, and lung cancer: a nationwide, population-based assessment. J Surg Res. 2003;114(1):1-5.

39. Trillet-Lenoir V, Green J, Manegold C, et al. Recombinant granulocyte colony stimulating factor reduces the infectious complications of cytotoxic chemotherapy. Eur J Cancer. 1993;29A(3):319-324.

40. Havrilesky LJ, Reiner M, Morrow PK, Watson H, Crawford J. A review of relative dose intensity and survival in patients with metastatic solid tumors. Crit Rev Oncol Hematol. 2015;93(3):203-210. doi: 10.1016/j.critrevonc.2014.10.006.

41. Almenar D, Mayans J, Juan O, et al. Pegfilgrastim and daily granulocyte colony-stimulating factor: patterns of use and neutropenia-related outcomes in cancer patients in Spain—results of the LEARN Study. Eur J Cancer Care (Engl). 2009;18(3):280-286. doi: 10.1111/j.1365-2354.2008.00959.x.

42. Vogel CL, Wojtukiewicz MZ, Carroll RR, et al. First and subsequent cycle use of pegfilgrastim prevents febrile neutropenia in patients with breast cancer: a multicenter, double-blind, placebo-controlled phase III study. J Clin Oncol. 2005;23(6):1178-1184.

43. Nichol MB, Sengupta N, Globe DR. Evaluating quality-adjusted life years estimation of the Health Utility Index (HUI2) from the SF-36. Med Decis Making. 2001;21(2):105-112.

44. Stanton D. Amgen prepped to compete in US filgrastim market post Sandoz’s Zarxio launch. BioPharma-Reporter website. Published July 31, 2015. Accessed April 5, 2016.

45. Amgen Inc. 2014 annual report and 10-K. Accessed December 8, 2015.

46. Research and markets: filgrastim & pegfilgrastim biosimilars & biosuperiors 2015 - a G-CSF & GM-CSF competitor analysis [press release]. Dublin, Ireland: Business Wire; September 1, 2015. Accessed October 23, 2015.

47. Lakdawalla DN, Sun EC, Jena AB, Reyes CM, Goldman DP, Philipson TJ. An economic evaluation of the war on cancer. J Health Econ. 2010;29(3):333-346. doi: 10.1016/j.jhealeco.2010.02.006. 
Copyright AJMC 2006-2020 Clinical Care Targeted Communications Group, LLC. All Rights Reserved.
Welcome the the new and improved, the premier managed market network. Tell us about yourself so that we can serve you better.
Sign Up