Chapter

Industrial Economics of Cord Blood Banks

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Abstract

Cord blood banks represent a major step in the advent of tissue economies in the twenty-first century. Cord blood units (CBUs) are becoming biological assets imported and exported worldwide, and valued according to a pricing logic based on their level of quality and compatibility. The controversy over public and private banking is based on a confusion between access to storage and access to a graft. In this chapter, we analyze the financial difficulties that allogeneic banks face and their dependency on the limited public funding available, which results in hindered growth. The economic analysis of commercial banks remains unexplored in the literature because of a lack of available data on their management practices and their financial performance. In 2013, out of 310 commercial banks identified in 86 countries, six banks were listed on European, Asian, and American stock exchanges. We found from their annual reports that, over the 2009–2012 period, their compound annual revenue growth rate was 9.18% despite a global economic crisis. Their average gross profit margin was 67.3% with only 1.1% of their revenue invested in R&D. In terms of cost–utility, in other words, the medical service provided for the price paid, we found that the total inventory of four leading commercial banks was 1.58 times larger than that of the combined inventories of all 137 public banks worldwide, yet their number of released CBUs—i.e., their therapeutic usefulness for patients—was 269 times lower. Given this medico-economic impasse, commercial banks probably need to rethink their business model, move away from therapeutic storage and develop into scientific banking.

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... In 2014, France decided to shut down half of its operating public banks just two years after launching a national development plan to support public banking. Without new banking strategies, many more public banks are likely to close in the coming years [6].The economic vulnerability of public banks requires that selective criteria be established to help decide which CBUs are eligible for cryopreservation. Composite scoring systems have been proposed to measure the biological quality of collected units before storage and after thawing to help transplant physicians obtain the best possible grafts for their patients [7]. ...
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Engraftment failure and delays, likely due to diminished cord blood unit (CBU) potency, remain major barriers to the overall success of unrelated umbilical cord blood transplantation (UCBT). To address this problem, we developed and retrospectively validated a novel scoring system, the Cord Blood Apgar (CBA), which is predictive of engraftment after UCBT. In a single-center retrospective study, utilizing a database of 435 consecutive single cord myeloablative UCBTs performed between January 1, 2000, to December 31, 2008, precryopreservation and postthaw graft variables (total nucleated cell, CD34+, colony-forming units, mononuclear cell content, and volume) were initially correlated with neutrophil engraftment. Subsequently, based on the magnitude of hazard ratios (HRs) in univariate analysis, a weighted scoring system to predict CBU potency was developed using a randomly selected training data set and internally validated on the remaining data set. The CBA assigns transplanted CBUs three scores: a precryopreservation score (PCS), a postthaw score (PTS), and a composite score (CS), which incorporates the PCS and PTS values. CBA-PCS scores, which could be used for initial unit selection, were predictive of neutrophil (CBA-PCS ≥ 7.75 vs. <7.75, HR 3.5; p < 0.0001) engraftment. Likewise, CBA-PTS and CS scores were strongly predictive of Day 42 neutrophil engraftment (CBA-PTS ≥ 9.5 vs. <9.5, HR 3.16, p < 0.0001; CBA-CS ≥ 17.75 vs. <17.75, HR 4.01, p < 0.0001). The CBA is strongly predictive of engraftment after UCBT and shows promise for optimizing screening of CBU donors for transplantation. In the future, a segment could be assayed for the PTS score providing data to apply the CS for final CBU selection.
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Genomic biobanks—repositories of human DNA and/or associated data, collected and maintained for biomedical research—present ethical challenges different from those traditionally associated with medical research. Historically, when researchers obtained and used tissue samples, it was for defined purposes and the nature of the research was disclosed to contributors, who were asked to consent to the specific research project. With genomic biobanks, in contrast, the entity holding the samples may not be involved in the research, and future uses of the samples may be unknown. Although traditional research promised confidentiality and/or anonymity to participants, advances in DNA technology may render these safeguards meaningless (1–3). As a consequence, many ethicists argue that traditional informed consent may be illusory if not impossible (1, 4) and that different approaches to the ethics of genetic sample collections are needed (5, 6).
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On 31 May 2010, 14 072 567 bone marrow/apheresis donors registered in 44 countries and 426 501 cord blood units banked in 26 countries for public use were available to treat candidates to haemopoietic stem cell transplant lacking a family related compatible donor. Despite these impressive numbers, additional efforts are required to ensure that all patients, including those from ethnic minorities, can promptly find a suitable donor. Governments, clinicians, scientists, patients and stakeholders should share the responsibility to develop haemopoietic stem cell donation and cord blood banking models able to fully match all patient needs. In this regard, current scientific evidence and prevalent opinions among expert clinicians support solidaristic cord blood donation for public use against the alternative option of commercial autologous cord blood storage.
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This paper examines an emerging bioeconomy centred on the international banking and trade in cord blood. Since the late 1980s cord blood has been used in an expanding range of treatments and as an alternative to the use of bone marrow stem cells. This is particularly the case in treating ethnic minority populations who have historically been under-represented in bone marrow registries. The paper explores the mobilisation and commercialisation of an increasingly important bioeconomic resource with cord blood units trading internationally at high prices. This is a market mediated through a sophisticated global network of immunologically typed and matched bodily matter in which immunity has become a form of 'corporeal currency'. Based on recent international figures we reflect upon the balance of trade between imports and exports across the world's cord blood bioeconomy. Theoretically, this case is, we suggest, an extension of what Roberto Esposito (2008) has termed an 'immunitary paradigm' in which immunity has become the basis for new forms of bioeconomic flow, circulation and exchange. Esposito (2008). Bios: Biopolitics and Philosophy. Minnesota, MN: University of Minnesota Press.
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The controversy surrounding private banking of umbilical cord blood units (CBU), as a safeguard against future malignancy or other life-threatening conditions, raises many questions in pediatric clinical practice. Recent favorable experiences with autologous transplantation for severe aplastic anemia using privately stored CBU, suggested a possible utility. While private banking is difficult to justify statistically or empirically, there may exist rare cases where autologous transplant of stored umbilical CBU could be beneficial. The reality of privately banked CBU and the possibility for future discovery of additional indications for autologous cord blood transplant, motivated us to re-examine our attitudes towards private cord blood banking.
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Once considered biological waste, umbilical cord blood (UCB) has become an accepted source of haematopoietic stem cells (HSCs). With initial success in the pediatric setting, UCB transplantation continues to gain favor in the adult patient population. Novel approaches to UCB transplantation include use of two units and a variety of graft manipulations. Additional uses for UCB are currently being explored and include applications in regenerative medicine and immunotherapy. © 2010 The Author(s). Vox Sanguinis
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SUMMARY: BACKGROUND AND METHODS: As a source of hematopoietic stem cells, cord blood (CB) is an alternative to bone marrow or peripheral blood stem cells (PBSC). The Mannheim Cord Blood Bank has currently stored about 1,750 allogeneic CB units. Here we report our experiences and discuss future perspectives of CB banking. We analyzed CB units for nucleated cell (NC), mononucleated cell (MNC) and CD34+ cell count, volume, colony-forming units (CFU-GM) as well as ethnic background of the donor. Transplanted CB units were analyzed for patient and transplant characteristics and compared to stored CB units. RESULTS: Only 25% of all collected CB units met storage criteria. Main reasons for exclusion were: i) insufficient volume (57.7%), ii) delayed arrival at the processing site (19.2%) and iii) little cell count (7.2%). Up to now 36 CB units have been released for transplantation mainly to children (62%). Transplant indications were hematological diseases, immune deficiencies and metabolic diseases. Transplanted CB units showed significantly higher cell counts compared to stored units (NC: 12.5 vs. 7.2 x 10(8), MNC: 4.7 vs. 2.9 x 10(8), CD34+ cells: 3.3 vs. 1.8 x 10(6), mean; p < 0.001 each) and were found more often in ethnic minority groups (36 vs. 20%; p = 0.04). CONCLUSIONS: Even though cell count and volume are key parameters for the eligibility of CB units, our data indicate that the ethnic background of the donor also plays a major role. Collection and processing of CB should be optimized in order to gain maximum volume and cell counts.
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A pilot study was conducted to determine the safety and feasibility of intravenous administration of autologous umbilical cord blood (CB) in young children with acquired neurologic disorders. Most CB units (CBUs) were electively stored in private CB banks. Unlike public banks, which utilize specific criteria and thresholds for banking, private banks generally store all collected CBUs. CBUs of eligible patients containing more than 1 × 10⁷ cells/kg were shipped to Duke from the banks of origin after confirming identity by HLA typing. On the day of infusion, CBUs were thawed and washed in dextran-albumin and infused intravenously. Patients were medicated with acetaminophen, diphenhydramine, and methylprednisolone before transfusion. Data regarding patients, infusions, and CBUs were collected retrospectively. Characteristics of CBUs were compared to existing data from CBUs publicly banked at the Carolinas Cord Blood Bank. From March 2004 to December 2009, 184 children received 198 CB infusions. Three patients had infusion reactions, all responsive to medical therapy and stopping the infusion. Median precryopreservation volume (60 mL vs. 89 mL, p < 0.0001), total nucleated cell count (4.7 × 10⁸ vs. 10.8 × 10⁸, p < 0.0001), and CD34 count (1.8 × 10⁶ vs. 3.0 × 10⁶, p < 0.0001) were significantly lower than publicly stored CBUs. Postthaw sterility cultures were positive in 7.6% of infused CBUs. IV infusion of autologous CB is safe and feasible in young children with neurologic injuries. Quality parameters of privately banked CBUs are inferior to those stored in public banks. If efficacy of autologous CB is established clinically, the quality of autologous units should be held to the same standards as those stored in public banks.
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Paradoxically, France is one of the leading exporters of cord blood units worldwide, but ranks only 17th in terms of cord blood units per inhabitant, and imports 64% of cord blood grafts to meet national transplantation demands. With three operational banks in 2008, the French allogeneic cord blood network is now entering an important phase of development with the creation of seven new banks collecting from local clusters of maternities. Although the French network of public banks is demonstrating a strong commitment to reorganise and scale up its activities, the revision of France's bioethics law in 2010 has sparked a debate concerning the legalisation of commercial autologous banking. The paper discusses key elements for a comprehensive national plan that would strengthen the allogeneic banking network through which France could meet its national medical needs and guarantee equal access to healthcare.
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To investigate the cost-effectiveness of private umbilical cord blood banking. A decision-analytic model was designed comparing private umbilical cord blood banking with no umbilical cord blood banking. Baseline assumptions included a cost of $3,620 for umbilical cord blood banking and storage for 20 years, a 0.04% chance of requiring an autologous stem cell transplant, a 0.07% chance of a sibling requiring an allogenic stem cell transplant, and a 50% reduction in risk of graft-versus-host disease if a sibling uses banked umbilical cord blood. Private cord blood banking is not cost-effective because it cost an additional $1,374,246 per life-year gained. In sensitivity analysis, if the cost of umbilical cord blood banking is less than $262 or the likelihood of a child needing a stem cell transplant is greater than 1 in 110, private umbilical cord blood banking becomes cost-effective. Currently, private umbilical cord blood banking is cost-effective only for children with a very high likelihood of needing a stem cell transplant. Patients considering private blood banking should be informed of the remote likelihood that a unit will be used for a child or another family member. III.
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Using processed consensus investment research, AVOS Life Sciences has analysed the performance of the top 14 pharmaceutical companies as a group for the period 2008–2013E (Supplementary information S1 (box)). The analysis focuses on the major Rx drug portfolio (MRDP; the collection of branded drugs, each of which is predicted to achieve at least US$500 million in annual sales) of each company as a measure of its overall performance.
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To determine risk factors of umbilical cord blood transplantation (UCBT) for patients with lymphoid malignancies. We evaluated 104 adult patients (median age, 41 years) who underwent unrelated donor UCBT for lymphoid malignancies. UCB grafts were two-antigen human leukocyte antigen-mismatched in 68%, and were composed of one (n = 78) or two (n = 26) units. Diagnoses were non-Hodgkin's lymphoma (NHL, n = 61), Hodgkin's lymphoma (HL, n = 29), and chronic lymphocytic leukemia (CLL, n = 14), with 87% having advanced disease and 60% having experienced failure with a prior autologous transplant. Sixty-four percent of patients received a reduced-intensity conditioning regimen and 46% low-dose total-body irradiation (TBI). Median follow-up was 18 months. Cumulative incidence of neutrophil engraftment was 84% by day 60, with greater engraftment in recipients of higher CD34(+) kg/cell dose (P = .0004). CI of non-relapse-related mortality (NRM) was 28% at 1 year, with a lower risk in patients treated with low-dose total-body irradiation (TBI; P = .03). Cumulative incidence of relapse or progression was 31% at 1 year, with a lower risk in recipients of double-unit UCBT (P = .03). The probability of progression-free survival (PFS) was 40% at 1 year, with improved survival in those with chemosensitive disease (49% v 34%; P = .03), who received conditioning regimens containing low-dose TBI (60% v 23%; P = .001), and higher nucleated cell dose (49% v 21%; P = .009). UCBT is a viable treatment for adults with advanced lymphoid malignancies. Chemosensitive disease, use of low-dose TBI, and higher cell dose were factors associated with significantly better outcome.
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The recent Perspective by Sullivan (Sullivan, M. J. Banking on cord blood stem cells. Nature Rev. Cancer 8, 555–563 (2008)
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To develop consensus-based recommendations guiding the conduct of cost-effectiveness analysis (CEA) to improve the comparability and quality of studies. The recommendations apply to analyses intended to inform the allocation of health care resources across a broad range of conditions and interventions. This article, first in a 3-part series, discusses how this goal affects the conduct and use of analyses. The remaining articles will outline methodological and reporting recommendations, respectively. The Panel on Cost-Effectiveness in Health and Medicine, a nonfederal panel with expertise in CEA, clinical medicine, ethics, and health outcomes measurement, was convened by the US Public Health Service (PHS). The panel reviewed the theoretical foundations of CEA, current practices, and alternative procedures for measuring and assigning values to resource use and health outcomes. The panel met 11 times during 2 1/2 years with PHS staff and methodologists from federal agencies. Working groups brought issues and preliminary recommendations to the full panel for discussion. Draft recommendations were circulated to outside experts and the federal agencies prior to finalization. The panel's recommendations define a "reference case" cost-effectiveness analysis, a standard set of methods to serve as a point of comparison across studies. The reference case analysis is conducted from the societal perspective and accounts for benefits, harms, and costs to all parties. Although CEA does not reflect every element of importance in health care decisions, the information it provides is critical to informing decisions about the allocation of health care resources.
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A large number of institutions have started programs banking umbilical cord blood (UCB) for allogeneic unrelated-donor and related-donor transplantation. However, limited information is available on the financial issues surrounding these activities. The aim of this study was to determine the fee per UCB unit released for transplantation that would allow cost recovery after 10 years. Three organizational models were considered suitable to provide units for five UCB transplants per 1 million population per year, a figure that would translate into an annual need for 280 units in Italy. Models A, B, and C included, respectively, seven networked banks, each with an inventory of 1,500 units; two networked banks, each with an inventory of 5,000 units; and one bank with an inventory of 10,000 units. It was estimated that it would take 3 years to develop the cryopreserved inventory and that approximately 3 percent of the inventory could be released and replaced each year during the 7-year interval between the fourth and tenth years of activity. The data on the costs of labor, reagents and diagnostics, disposables, depreciation and maintenance, laboratory tests, and overhead, as well as the operational data used in the analysis were collected at the Milano Cord Blood Bank in 1996. Fees of US $15,061, $12,666, and $11,602 per unit released during the fourth through the tenth years of activity allow full cost recovery (principle and interest) under Models A, B, and C, respectively. Although UCB procurement costs compare favorably with those of other hematopoietic cell sources, these results and the current fee of US $15,300 used in some institutions show that UCB is an expensive resource. Therefore, judicious planning of banking programs with high quality standards is necessary to prevent economic losses. The advantages of lower fees associated with the centralized banking approach of Model C should be balanced with the more flexible collection offered by Model A.
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Pregnant patients have the option of storing their infant's cord blood with a private/commercial company for possible future use by the child or other family members. Some patients also have the option to donate the cord blood to a public bank for anyone to use. We evaluated patient understanding about cord blood banking in a cohort of patients with access to both options. Anonymous questionnaires were collected from 325 pregnant patients seen in our Antepartum Testing Unit. Compared to those donating to a public bank, women planning on storing with a private/commercial company were less likely to believe that a suitable donor could be found from a public cord blood bank. Women had a strikingly poor understanding regarding the current uses for cord blood therapy. When asked whether cord blood has been used successfully to treat Alzheimer's disease, Parkinson's disease, and spinal cord injury only 28%, 24%, and 24%, respectively, correctly knew that it had not. Obstetricians should assume that pregnant women are poorly informed about cord blood banking. The decision making process should be conducted with the goal of ensuring every pregnant woman the opportunity to make a well informed decision about cord blood banking.