Science-based assessment of source materials for cell-based medicines: report of a stakeholders workshop.

Human pluripotent stem cells (hPSCs) have the potential to transform medicine. However, hurdles remain to ensure safety for such cellular products. Science-based understanding of the requirements for source materials is required as are appropriate materials. Leaders in hPSC biology, clinical translation, biomanufacturing and regulatory issues were brought together to define requirements for source materials for the production of hPSC-derived therapies and to identify other key issues for the safety of cell therapy products. While the focus of this meeting was on hPSC-derived cell therapies, many of the issues are generic to all cell-based medicines. The intent of this report is to summarize the key issues discussed and record the consensus reached on each of these by the expert delegates.

Distributive justice and regenerative medicine.

Many countries have identified regenerative medicine as a strategic priority, and have thus launched a range of initiatives to facilitate innovation in the field. This perspective paper argues that several initiatives involve resource distributions that could impinge on widely accepted egalitarian notions of fairness and justice that underpin current healthcare systems. Specifically, this paper focuses on five initiatives, and argues that these initiatives reflect a largely unacknowledged utilitarian perspective on distributive justice. The intention of this paper is not to argue against these initiatives, but rather to stimulate an open discussion on what qualifies as a just and fair system of resource distribution, so that the regenerative medicine field can responsibly deliver on its clinical potential.

Screening for mild traumatic brain injury in OEF-OIF deployed US military: an empirical assessment of VHA's experience.

BACKGROUND: VHA screens for traumatic brain injury (TBI) among patients formerly deployed to Afghanistan or Iraq, referring those who screen positive for a Comprehensive TBI Evaluation (CTBIE). METHODS: To assess the programme, rates were calculated of positive screens for potential TBI in the population of patients screened in VHA between October 2007 through March 2009. Rates were derived of TBI confirmed by comprehensive evaluations from October 2008 through July 2009. Patient characteristics were obtained from Department of Defense and VHA administrative data. RESULTS: In the study population, 21.6% screened positive for potential TBI and 54.6% of these had electronic records of a CTBIE. Of those with CTBIE records, evaluators confirmed TBI in 57.7%, yielding a best estimate that 6.8% of all those screened were confirmed to have TBI. Three quarters of all screened patients and virtually all those evaluated (whether TBI was confirmed or not) had VHA care the following year. CONCLUSIONS: VHA's TBI screening process is inclusive and has utility in referring patients with current symptoms to appropriate care. More than 90% of those evaluated received further VHA care and confirmatory evaluations were associated with significantly higher average utilization. Generalizability is limited to those who seek VHA healthcare.

Is fragmented financing bad for your health?

Americans finance health care through a variety of private insurance plans and public programs. This organizational fragmentation could threaten continuity of care and adversely affect outcomes. Using a large sample of veterans who were eligible for mixtures of Veterans Health Administration- and Medicare-financed care, we estimate a system of equations to account for simultaneity in the determination of financing configuration and the probability of hospitalization for an ambulatory care sensitive condition. We find that a change of one standard deviation in financing fragmentation increases the risk of an adverse outcome by one-fifth.

What can Medicaid data add to research on VA patients?

This article is the first to describe Department of Veterans Affairs (VA) patients' use of Medicaid at a national level. We obtained 1999 national VA enrollment and utilization data, Centers for Medicare and Medicaid Services enrollment and claims, and Medicare information from the VA Information Resource Center. The research team created files for program characteristics and described the VA-Medicaid dually enrolled population, healthcare utilization, and costs. In 1999, VA-Medicaid dual enrollees comprised 10.2% of VA's annual patient load (350,000/3,450,000); 304,000 were veterans. These veterans differed marginally from VA's veteran patients, being on average half a year younger and having 1% fewer males. Dual enrollees with mental health diagnoses and care were almost three times as numerous as long-term care patients; these two groups accounted for ~60% of dual enrollees. Dual enrollees disproportionately included housebound veterans and veterans needing aid and assistance. Half the dual enrollees had 12 months of Medicaid eligibility, and total Federal expenditures per patient not in managed care programs averaged >$18,000 (median >$6,000). Dually enrolled women veterans cost ~55% less than men. Medicaid benefits complement VA and are more accessible in many states. VA researchers need to consider including Medicaid utilization and costs in their studies if they target populations or programs related to long-term care or mental disorders.

The impact of private insurance coverage on veterans' use of VA care: insurance and selection effects.

OBJECTIVE: To examine private insurance coverage and its impact on use of Veterans Health Administration (VA) care among VA enrollees without Medicare coverage. DATA SOURCES: The 1999 National Health Survey of Veteran Enrollees merged with VA administrative data, with other information drawn from American Hospital Association data and the Area Resource File. STUDY DESIGN: We modeled VA enrollees' decision of having private insurance coverage and its impact on use of VA care controlling for sociodemographic information, patients' health status, VA priority status and access to VA and non-VA alternatives. We estimated the true impact of insurance on the use of VA care by teasing out potential selection bias. Bias came from two sources: a security selection effect (sicker enrollees purchase private insurance for extra security and use more VA and non-VA care) and a preference selection effect (VA enrollees who prefer non-VA care may purchase private insurance and use less VA care). PRINCIPAL FINDINGS: VA enrollees with private insurance coverage were less likely to use VA care. Security selection dominated preference selection and naïve models that did not control for selection effects consistently underestimated the insurance effect. CONCLUSIONS: Our results indicate that prior research, which has not controlled for insurance selection effects, may have underestimated the potential impact of any private insurance policy change, which may in turn affect VA enrollees' private insurance coverage and consequently their use of VA care. From the decline in private insurance coverage from 1999 to 2002, we projected an increase of 29,400 patients and 158 million dollars for VA health care services.

Health care resource utilization and expenditures associated with the use of insulin glargine.

BACKGROUND: Newer insulins, such as long-acting analogues, offer promise of better glycemic control, reduced risk for diabetes complications, and moderation of health care use and costs. OBJECTIVE: We studied initiation of insulin glargine to evaluate its association with subsequent health service utilization and estimated expenditures. METHODS: Patients of the Veterans Health Administration, US Department of Veterans Affairs (VA) who initiated insulin glargine (n=5064) in 2001-2002 were compared with patients receiving other insulin (n=69,944), matched on prescription month (index date). Inpatient and outpatient VA care in the 12 months after a patient's index date was evaluated using Tobit regression, controlling for prior utilization, demographic characteristics, comorbidities, glycosylated hemoglobin (HbA(1c)) levels, and diabetes severity. National average utilization costs and medication acquisition costs were used to estimate the value of VA expenditures. RESULTS: Compared with other insulin users, insulin glargine initiators had higher HbA(1c) values (8.72% vs 8.16%) prior to the index date, but greater subsequent HbA(1c) reduction (-0.50% vs -0.22%). After adjustment for age, prior utilization, HbA(1c) levels, and other factors, insulin glargine initiation was associated with 2.4 (95% CI, 1.1-3.7) fewer inpatient days for patients with any hospital admission (US $820 lower costs per initiator), 1.6 (1.2-1.9) more outpatient encounters ($279 higher costs per initiator), and $374 ($362-$387) higher costs for diabetes medications. The net difference was an average lower VA cost of $166 (-$290 to $622) per patient. CONCLUSIONS: Insulin glargine use was associated with decreased inpatient days but increased outpatient care, and the value of the net change in utilization to VA offset the additional medication expenditures. Initiation of insulin glargine improves glycemic control and may reduce time in hospital without additional use of health resources.

VA-Medicare dual beneficiaries' enrollment in Medicare HMOs: access to VA, availability of HMOs, and favorable selection.

This study examined the association between Veterans Administration (VA)-Medicare dual beneficiaries' HMO enrollment and factors including sociodemographics, access/attachment to VA, self-reported health status, and characteristics of Medicare HMO markets. The results showed that availability of Medicare HMOs and less access to VA care were the major predictors of VA-Medicare dual beneficiaries' HMO enrollment. Other significant predictors of HMO enrollment were age (65-69), having no college education, VA priority status (low income; less than 50 percent service disability). There was some evidence of favorable selection measured by self-reported health status. The identified HMO enrollment profile can position VA better in attracting and managing the care of these beneficiaries and in meeting potentially large shifts in their need for VA care if Medicare benefits or policies change markedly.

Dual-system use: are there implications for risk adjustment and quality assessment?

The authors examined the implications of dual-system use for risk adjustment and quality assessment. The sample (n = 34 151) included all veterans dually enrolled in the Veterans Health Administration (VA) and the private sector in 1998 with (1) an inpatient discharge from either a VA or Medicare setting for 1 of 6 conditions/procedures and (2) inpatient and/or outpatient use in both the VA and private sector. The authors used the Diagnostic Cost Groups risk-adjustment system to obtain concurrent and prospective health status (relative risk scores) using veterans' Medicare diagnoses only, VA diagnoses only, and diagnoses from both systems. Both concurrent and prospective relative risk scores increased when diagnoses from both systems were used; the population's disease profile also was affected. The authors conclude that it is important to capture the true disease burden of the population by obtaining diagnoses from all health care systems providing care to facilitate meaningful comparisons of performance.