Value-Based Care in Hematopoietic Cell Transplantation and Cellular Therapy: Challenges and Opportunities
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Purpose of Review
Improved tolerability and outcomes after hematopoietic cell transplantation (HCT), along with the availability of alternative donors, have expanded its use. With this growth, and the development of additional cellular therapies, we also aim to increase effectiveness, efficiency, and the quality of the care provided. Fundamentally, the goal of value-based care is to have better health outcomes with streamlined processes, improved patient experience, and lower costs for both the patients and the health care system. HCT and cellular therapy treatments are multiphase treatments which allow for interventions at each juncture.
We present a summary of the current literature with focus on program structure and overall system capacity, coordination of therapy across providers, standardization across institutions, diversity and disparities in care, patient quality of life, and cost implications.
Each of these topics provides challenges and opportunities to improve value-based care for HCT and cellular therapy patients.
KeywordsValue-base care Hematopoietic cell transplantation CAR-T cell Cellular therapy
Compliance with Ethical Standards
Conflict of Interest
Gunjan L. Shah, Nandita Khera, and Sergio Giralt declare no conflict of interest. Navneet Majhail is a consultant for Anthem, Inc.
Human and Animal Rights and Informed Consent
This article does not contain any studies with human or animal subjects performed by any of the authors.
Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance
- 2.Brown M, Brown G, Sharma S. Evidence-based to value-based medicine. Chicago: AMA Press; 2005.Google Scholar
- 5.Hashmi SK, Bredeson C, Duarte RF, et al. National Institutes of Health Blood and Marrow Transplant Late Effects Initiative: The Healthcare Delivery Working Group Report. BBMT. 2017;23:717–25.Google Scholar
- 6.Denzen EM, Majhail NS, Stickney Ferguson S, et al. Hematopoietic cell transplantation in 2020: summary of year 2 recommendations of the National Marrow Donor Program’s System Capacity Initiative. BBMT. 2013;19:4–11.Google Scholar
- 7.Majhail NS, Murphy EA, Denzen EM, et al. The National Marrow Donor Program’s Symposium on Hematopoietic Cell Transplantation in 2020: a health care resource and infrastructure assessment. BBMT. 2012;18:172–82.Google Scholar
- 8.Neumann JL, Mau L-W, Virani S, Denzen EM, Boyle DA, Boyle NJ, et al. Burnout, moral distress, work–life balance, and career satisfaction among hematopoietic cell transplantation professionals. BBMT. 2017; https://doi.org/10.1016/j.bbmt.2017.11.015.
- 9.Majhail NS, Murphy EA, Omondi NA, Robinett P, Gajewski JL, LeMaistre CF, et al. Allogeneic transplant physician and center capacity in the United States. BBMT. 2011;17:956–61.Google Scholar
- 10.Majhail NS, Mau LW, Chitphakdithai P, et al. National survey of hematopoietic cell transplantation center personnel, infrastructure, and models of care delivery. BBMT. 2015;21:1308–14.Google Scholar
- 11.Nivison-Smith I, Milliken S, Dodds AJ, Gottlieb D, Kwan J, Ma DDF, et al. Activity and capacity profile of transplant physicians and centers in Australia and New Zealand. BBMT. 2018;24:169–74.Google Scholar
- 12.Merten JA, Shapiro JF, Gulbis AM, et al. Utilization of collaborative practice agreements between physicians and pharmacists as a mechanism to increase capacity to care for hematopoietic stem cell transplant recipients. BBMT. 2013;19:509–18.Google Scholar
- 13.Komanduri KV. Pharmacists and physicians in hematopoietic stem cell transplantation: advances and opportunities to use collaborative practice agreements to improve care. BBMT. 2013;19:505–8.Google Scholar
- 14.Medicare and Medicaid Programs. Reform of hospital and critical access hospital conditions of participation. Fed Regist. 2012;77:29034–76. To be codified at 42 CFR x482 and x48Google Scholar
- 15.Vulaj V, Hough S, Bedard L, Farris K, Mackler E (2018) Oncology pharmacist opportunities: closing the gap in quality care. JOP JOP2017026666.Google Scholar
- 16.Stickney Ferguson S, Randall J, Dabney J, Kalbacker ME, Boyle N, Thao V, et al. Perceived workforce challenges among clinical social workers in hematopoietic cell transplantation programs. BBMT. 2017; https://doi.org/10.1016/j.bbmt.2017.12.793.
- 17.LeMaistre CF, Farnia S, Crawford S, McGuirk J, Maziarz RT, Coates J, et al. Standardization of terminology for episodes of hematopoietic stem cell patient transplant care. BBMT. 2013;19:851–7.Google Scholar
- 18.Majhail NS, Giralt S, Bonagura A, Crawford S, Farnia S, Omel JL, et al. Guidelines for defining and implementing standard episode of care for hematopoietic stem cell transplantation within the context of clinical trials. BBMT. 2015;21:583–8.Google Scholar
- 20.Khera N, Gooley T, Flowers MED, Sandmaier BM, Loberiza F, Lee SJ, et al. Association of distance from transplantation center and place of residence on outcomes after allogeneic hematopoietic cell transplantation. BBMT. 2016;22:1319–23.Google Scholar
- 21.Morrison CF, Martsolf DM, Wehrkamp N, Tehan R, Pai ALH. Medication adherence in hematopoietic stem cell transplantation: a review of the literature. BBMT. 2017;23:562–8.Google Scholar
- 22.• Battiwalla M, Hashmi S, Majhail N, Pavletic S, Savani BN, Shelburne N. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: developing recommendations to improve survivorship and long-term outcomes. BBMT. 2017;23:6–9. First in series of papers describing the National Institutes of Health HCT Late Effects Initiative process and recommendations. Google Scholar
- 23.Gea-Banacloche J, Komanduri KV, Carpenter P, et al. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: the Immune Dysregulation and Pathobiology Working Group Report. BBMT. 2017;23:870–81.Google Scholar
- 24.Armenian SH, Chemaitilly W, Chen M, et al. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: the Cardiovascular Disease and Associated Risk Factors Working Group Report. BBMT. 2017;23:201–10.Google Scholar
- 25.Morton LM, Saber W, Baker KS, Barrett AJ, Bhatia S, Engels EA, et al. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: the Subsequent Neoplasms Working Group Report. BBMT. 2017;23:367–78.Google Scholar
- 26.Bevans M, El-Jawahri A, Tierney DK, et al. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: the Patient-Centered Outcomes Working Group Report. BBMT. 2017;23:538–51.Google Scholar
- 27.Hashmi SK, Bredeson C, Duarte RF, et al. National Institutes of Health Blood and Marrow Transplant Late Effects Initiative: the Healthcare Delivery Working Group Report. BBMT. 2017;23:717–25.Google Scholar
- 28.Shaw BE, Hahn T, Martin PJ, Mitchell SA, Petersdorf EW, Armstrong GT, et al. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: the Research Methodology and Study Design Working Group Report. BBMT. 2017;23:10–23.Google Scholar
- 30.Baker KS, Davies SM, Majhail NS, et al. Race and socioeconomic status influence outcomes of unrelated donor hematopoietic cell transplantation. BBMT. 2009;15:1543–54.Google Scholar
- 31.Rao K, Darrington DL, Schumacher JJ, Devetten M, Vose JM, Loberiza FR. Disparity in survival outcome after hematopoietic stem cell transplantation for hematologic malignancies according to area of primary residence. BBMT. 2007;13:1508–14.Google Scholar
- 32.Majhail NS, Nayyar S, Santibañez MEB, Murphy EA, Denzen EM. Racial disparities in hematopoietic cell transplantation in the United States. BMT. 2012;47:1385–90.Google Scholar
- 33.Hamilton BK, Rybicki L, Arai S, et al. Association of Socioeconomic Status with chronic graft-versus-host disease outcomes. BBMT. 2018;24:393–9.Google Scholar
- 36.Pidala J, Craig BM, Lee SJ, Majhail N, Quinn G, Anasetti C. Practice variation in physician referral for allogeneic hematopoietic cell transplantation. BMT. 2013;48:63–7.Google Scholar
- 37.Preston E, Chinapen S, Borrill T, et al. Disease progression is main barrier to allogeneic hematopoietic stem cell transplantation (HCT) in patients with newly diagnosed and relapsed acute leukemia. BBMT Abstract. 2018; 465.Google Scholar
- 41.Lawitschka A, Güclü ED, Varni JW, Putz M, Wolff D, Pavletic S, et al. Health-related quality of life in pediatric patients after allogeneic SCT: development of the PedsQL Stem Cell Transplant module and results of a pilot study. BMT. 2014;49:1093–7.Google Scholar
- 48.de Wreede LC, Watson M, van Os M, Milligan D, van Gelder M, Michallet M, et al. Improved relapse-free survival after autologous stem cell transplantation does not translate into better quality of life in chronic lymphocytic leukemia: lessons from the randomized European Society for Blood and Marrow Transplantation-Intergroup study. Am J Hematol. 2014;89:174–80.CrossRefPubMedGoogle Scholar
- 52.Deng G, Giralt S, Chung DJ, Landau H, Siman J, Search B, et al. Acupuncture for reduction of symptom burden in multiple myeloma patients undergoing autologous hematopoietic stem cell transplantation: a randomized sham-controlled trial. Support Care Cancer. 2018;26:657–65.CrossRefPubMedGoogle Scholar
- 53.Anderson KO, Giralt SA, Mendoza TR, Brown JO, Neumann JL, Mobley GM, et al. Symptom burden in patients undergoing autologous stem-cell transplantation. BMT. 2007;39:759–66.Google Scholar
- 54.Bevans MF, Mitchell SA, Barrett JA, et al. Symptom distress predicts long-term health and well-being in allogeneic stem cell transplantation survivors. BBMT. 2014;20:387–95.Google Scholar
- 55.Dueck AC, Mendoza TR, Mitchell SA, Reeve BB, Castro KM, Rogak LJ, et al. Validity and reliability of the US National Cancer Institute’s Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE). JAMA Oncol. 2015;1:1051–9.Google Scholar
- 56.Shah N, Shi Q, Williams LA, et al. Higher stem cell dose infusion after intensive chemotherapy does not improve symptom burden in older patients with multiple myeloma and amyloidosis. BBMT. 2016;22:226–31.Google Scholar
- 57.Khera N, Chang Y, Hashmi S, et al. Financial burden in recipients of allogeneic hematopoietic cell transplantation. BBMT. 2014;20:1375–81.Google Scholar
- 58.Farnia S, Ganetsky A, Silver A, Hwee T, Preussler J, Griffin J, et al. Challenges around access to and cost of life-saving medications after allogeneic hematopoietic cell transplantation for Medicare patients. BBMT. 2017;23:1387–92.Google Scholar
- 62.• Maziarz RT, Farnia S, Martin P, Komanduri KV. Optimal benefits for hematopoietic stem cell transplantation: a consensus opinion. BBMT. 2014;20:1671–6. Recommendations by the Financial Working Group of the National Marrow Donor Program-sponsored System Capacity Initiative on what to include in a bundled insurance payment. Google Scholar
- 64.Preussler JM, Mau L-W, Majhail NS, et al. Administrative claims data for economic analyses in hematopoietic cell transplantation: challenges and opportunities. BBMT. 2016;22:1738–46.Google Scholar
- 65.Preussler JM, Denzen EM, Majhail NS. Costs and cost-effectiveness of hematopoietic cell transplantation. BBMT. 2012;18:1620–8.Google Scholar
- 66.Majhail NS, Mau LW, Denzen EM, Arneson TJ. Costs of autologous and allogeneic hematopoietic cell transplantation in the United States: a study using a large national private claims database. BMT. 2013;48:294–300.Google Scholar
- 68.Shah GL, Winn AN, Lin P-J, et al. Cost-effectiveness of autologous hematopoietic stem cell transplantation for elderly patients with multiple myeloma using the surveillance, epidemiology, and end results-Medicare database. BBMT. 2015;21:1823–9.Google Scholar
- 70.Worcester S (2017) CAR T-cell therapy: moving from cost to value. In: Hematol. News. https://www.mdedge.com/hematologynews/article/152563/all-acute-lymphoblastic-leukemia/car-t-cell-therapy-moving-cost-value. Accessed 15 Jan 2018.
- 72.LeMaistre CF, Farnia SH. Goals for pay for performance in hematopoietic cell transplantation: a primer. BBMT. 2015;21:1367–72.Google Scholar
- 73.Farnia S, Shah J, Rinkle V, Gajewski J. ASBMT autologous CAR-T coding guidance. 2017Google Scholar
- 74.•• Tice JA, Walsh JM, Otuonye I, Chapman R, Kumar V, Seidner M, Ollendorf DA, Pearson SD (2017) Chimeric antigen receptor T-cell therapy for B-cell cancers: effectiveness and value. Inst Clin Econ Rev 1–120. Draft evidence report by the Institute for Clinical and Economic Review summarizing currently known clinical data on CAR T cells and presenting economic models. Google Scholar
- 75.Stranges E, Russo C, Friedman B. Procedures with the most rapidly increasing hospital costs, 2004–2007 [HCUP statistical brief 82]. Rockville, MD. 2009Google Scholar
- 76.Gajewski JL, McClellan MB, Majhail NS, et al. Payment and care for hematopoietic cell transplantation patients: toward a specialized medical home for complex care patients. BBMT. 2018;24:4–12.Google Scholar
- 77.Transplantation C for B and M (2017) CIBMTR report of survival statistics for blood and marrow transplantation. https://www.cibmtr.org/ReferenceCenter/SlidesReports/StatReport/Pages/index.aspx. Accessed 15 Jan 2018.