Historical trends in health care-related financial holdings among members of Congress.

BACKGROUND: Revelations that some members of Congress, including members of key health care committees, hold substantial personal investments in the health care industry have raised concerns about lawmakers' financial conflicts of interest (COI) and their potential impact on health care legislation and oversight. AIMS: 1) To assess historical trends in both the number of legislators holding health care-related assets and the value and composition of those assets. 2) To compare the financial holdings of members of health care-focused committees and subcommittees to those of other members of the House and Senate. METHODS: We analyzed 11 years of personal financial disclosures by all members of the House and Senate. For each year, we calculated the percentage of members holding a health care-related asset (overall, by party, and by committee); the total value of all assets and health care-related assets held; the mean and median values of assets held per member; and the share of asset values attributable to 9 health asset categories. FINDINGS: During the study period, over a third of all members of Congress held health care-related assets. These assets were often substantial, with a median total value per member of over $43,000. Members of health care-focused committees and subcommittees in the House and Senate did not hold health care-related assets at a higher rate than other members of their respective chambers. CONCLUSIONS: These findings suggest that lawmakers' health care-related COI warrant the same level of attention that has been paid to the COI of other actors in the health care system.

Allocating scarce life-saving resources: the proper role of age.

The COVID-19 pandemic has forced clinicians, policy-makers and the public to wrestle with stark choices about who should receive potentially life-saving interventions such as ventilators, ICU beds and dialysis machines if demand overwhelms capacity. Many allocation schemes face the question of whether to consider age. We offer two underdiscussed arguments for prioritising younger patients in allocation policies, which are grounded in prudence and fairness rather than purely in maximising benefits: prioritising one's younger self for lifesaving treatments is prudent from an individual perspective, and prioritising younger patients works to narrow health disparities by giving priority to patients at risk of dying earlier in life, who are more likely to be subject to systemic disadvantage. We then identify some confusions in recent arguments against considering age.

Association Between Financial Incentives and Participant Deception About Study Eligibility.

Importance: Offers of payment for research participation are ubiquitous but may lead prospective participants to deceive about eligibility, jeopardizing study integrity and participant protection. To date, neither the rate of payment-induced deception nor the influence of payment amount has been systematically studied in a nationally representative randomized survey experiment. Objectives: To estimate payment-associated deception about eligibility for an online survey and to assess whether there is an association between payment amount and deception frequency. Design, Setting, and Participants: Randomized, 7-group survey experiment. Data were collected in March 2018 and analyzed from March to August 2018. The setting was a nationally representative online survey among US adults drawn from the GfK KnowledgePanel. Interventions: Varying payment amounts for participation ($5, $10, or $20 cash equivalent) and direction of eligibility criterion (having received or not having received an influenza vaccination in the past 6 months). Main Outcomes and Measures: Proportion of respondents reporting recent influenza vaccination. Results: In total, 2275 individuals participated in the survey, a 59.4% (2275 of 3829) response rate; 51.8% (1108) were female, and 21.1% of respondents (399) were aged 18 to 29 years, 24.9% (532) were aged 30 to 44 years, 26.0% (601) were aged 45 to 59 years, and 28.0% (738) were 60 years or older. For participants offered a $5 incentive, the reported frequency of recent influenza vaccination was 16.6% higher (95% CI, 9.1%-24.1%) among those told that eligibility (and thus payment) required recent vaccination than among those told that eligibility required no recent vaccination. The corresponding differences were 21.0% (95% CI, 13.5%-28.5%) among those offered $10 and 15.4% (95% CI, 7.8%-23.0%) among those offered $20. Estimated proportions of ineligible individuals who responded deceptively regarding eligibility ranged from 10.5% to 22.8% across study groups. There was no evidence that higher payment was associated with higher frequency of deception. Conclusions and Relevance: In a nationally representative randomized survey experiment to evaluate whether and to what extent payment is associated with participants misleading investigators about their research eligibility, this study found evidence of significant deception. However, no association was observed between payment amount and frequency of deception. Further research is needed to extend these findings to clinical research. These data suggest that, when possible, investigators should rely on objective tests of eligibility rather than self-report.

Beyond financial conflicts of interest: Institutional oversight of faculty consulting agreements at schools of medicine and public health.

IMPORTANCE: Approximately one-third of U.S. life sciences faculty engage in industry consulting. Despite reports that consulting contracts often impinge on faculty and university interests, institutional approaches to regulating consulting agreements are largely unknown. OBJECTIVE: To investigate the nature of institutional oversight of faculty consulting contracts at U.S. schools of medicine and public health. DESIGN: Structured telephone interviews with institutional administrators. Questions included the nature of oversight for faculty consulting agreements, if any, and views about consulting as a private versus institutional matter. Interviews were analyzed using a structured coding scheme. SETTING: All accredited schools of medicine and public health in the U.S. PARTICIPANTS: Administrators responsible for faculty affairs were identified via internet searches and telephone and email follow-up. The 118 administrators interviewed represented 73% of U.S. schools of medicine and public health, and 75% of those invited to participate. INTERVENTION: Structured, 15-30 minute telephone interviews. MAIN OUTCOMES AND MEASURES: Prevalence and type of institutional oversight; responses to concerning provisions in consulting agreements; perceptions of institutional oversight. RESULTS: One third of institutions (36%) required faculty to submit at least some agreements for institutional review and 36% reviewed contracts upon request, while 35% refused to review contracts. Among institutions with review, there was wide variation the issues covered. The most common topic was intellectual property rights (64%), while only 23% looked at publication rights and 19% for inappropriately broad confidentiality provisions. Six in ten administrators reported they had no power to prevent faculty from signing consulting agreements. Although most respondents identified institutional risks from consulting relationships, many maintained that consulting agreements are "private." CONCLUSIONS AND RELEVANCE: Oversight of faculty consulting agreements at U.S. schools of medicine and public health is inconsistent across institutions and usually not robust. The interests at stake suggest the need for stronger oversight.

Addressing Financial Barriers to Patient Participation in Clinical Trials: ASCO Policy Statement.

Research conducted through clinical trials is essential for evaluating new treatment modalities, establishing new standards of cancer care, and ultimately improving and prolonging the lives of patients with cancer. However, participation in trials has been low, and this is attributable to various factors including patient financial barriers. Such financial barriers include the rising cost of cancer care; a lack of transparency in coverage policy; and the perception of ethical, compliance, or institutional impediments to patient financial support. ASCO convened a roundtable discussion with a variety of stakeholders to define the scope of the problem, as well as to identify clinical practice and policy solutions applicable at the institutional and system-wide levels. This statement summarizes key discussions from the ASCO Roundtable, as well as findings from the literature, and provides ASCO's recommendations for overcoming financial barriers that may otherwise prevent participation in clinical trials. These recommendations broadly address the following key areas: (1) improving the policy environment for coverage of clinical trials; (2) facilitating transparency among providers, patients, and payers for trial-related out-of-pocket costs; (3) refuting the specter of inducement to enable targeted financial support for patients; and (4) improving the available data on costs of cancer clinical trials.

Characteristics Associated With Preferences for Parent-Centered Decision Making in Neonatal Intensive Care.

Importance: Little is known about how characteristics of particular clinical decisions influence decision-making preferences by patients or their surrogates. A better understanding of the factors underlying preferences is essential to improve the quality of shared decision making. Objective: To identify the characteristics of particular decisions that are associated with parents' preferences for family- vs medical team-centered decision making across the spectrum of clinical decisions that arise in the neonatal intensive care unit (NICU). Design, Setting, and Participants: This cross-sectional survey assessed parents' preferences for parent- vs medical team-centered decision making across 16 clinical decisions, along with parents' assessments of 7 characteristics of those decisions. Respondents included 136 parents of infants in 1 of 3 academically affiliated hospital NICUs in Philadelphia, Pennsylvania, from January 7 to July 8, 2016. Respondents represented a wide range of educational levels, employment status, and household income but were predominantly female (109 [80.1%]), white (68 [50.0%]) or African American (53 [39.0%]), and married (81 of 132 responding [61.4%]). Main Outcomes and Measures: Preferences for parent-centered decision making. For each decision characteristic (eg, urgency), multivariable analyses tested whether middle and high levels of that characteristic (compared with low levels) were associated with a preference for parent-centered decision making, resulting in 2 odds ratios (ORs) per decision characteristic. Results: Among the 136 respondents (109 women [80.1%] and 27 men [19.9%]; median age, 30 years [range, 18-43 years]), preferences for parent-centered decision making were positively associated with decisions that involved big-picture goals (middle OR, 2.01 [99% CI, 0.83-4.86]; high OR, 3.38 [99% CI, 1.48-7.75]) and that had the potential to harm the infant (middle OR, 1.32 [99% CI, 0.84-2.08]; high OR, 2.62 [99% CI, 1.67-4.11]). In contrast, preferences for parent-centered decision making were inversely associated with the following 4 decision characteristics: technical decisions (middle OR, 0.82 [99% CI, 0.45-1.52]; high OR, 0.48 [99% CI, 0.25-0.93]), the potential to benefit the infant (middle OR, 0.42 [99% CI, 0.16-1.05]; high OR, 0.21 [99% CI, 0.08-0.52]), requires medical expertise (middle OR, 0.48 [99% CI, 0.22-1.05]; high OR, 0.21 [99% CI, 0.10-0.48]), and a high level of urgency (middle OR, 0.47 [99% CI, 0.24-0.92]; high OR, 0.42 [99% CI, 0.22-0.83]). Conclusions and Relevance: Preferences for parent-centered vs medical team-centered decision making among parents of infants in the NICU may vary systematically by the characteristics of particular clinical decisions. Incorporating this variation into shared decision making and endorsing models that allow parents to cede control to physicians in appropriate clinical circumstances might improve the quality and outcomes of medical decisions.

Biomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act Coverage.

Aaron Kesselheim and colleagues examine orphan-designated drugs approved between 2009 and 2015 in the United States.

Patient Advocacy Organizations, Industry Funding, and Conflicts of Interest.

Importance: Patient advocacy organizations (PAOs) are influential health care stakeholders that provide direct counseling and education for patients, engage in policy advocacy, and shape research agendas. Many PAOs report having financial relationships with for-profit industry, yet little is known about the nature of these relationships. Objective: To describe the nature of industry funding and partnerships between PAOs and for-profit companies in the United States. Design, Setting, and Participants: A survey was conducted from September 1, 2013, to June 30, 2014, of a nationally representative random sample of 439 PAO leaders, representing 5.6% of 7865 PAOs identified in the United States. Survey questions addressed the nature of their activities, their financial relationships with industry, and the perceived effectiveness of their conflict of interest policies. Main Outcomes and Measures: Amount and sources of revenue as well as organizational experiences with and policies regarding financial conflict of interest. Results: Of the 439 surveys mailed to PAO leaders, 289 (65.8%) were returned with at least 80% of the questions answered. The PAOs varied widely in terms of size, funding, activities, and disease focus. The median total revenue among responding organizations was $299 140 (interquartile range, $70 000-$1 200 000). A total of 165 of 245 PAOs (67.3%) reported receiving industry funding, with 19 of 160 PAOs (11.9%) receiving more than half of their funding from industry. Among the subset of PAOs that received industry funding, the median amount was $50 000 (interquartile range, $15 000-$200 000); the median proportion of industry support derived from the pharmaceutical, device, and/or biotechnology sectors was 45% (interquartile range, 0%-100%). A total of 220 of 269 respondents (81.8%) indicated that conflicts of interest are very or moderately relevant to PAOs, and 94 of 171 (55.0%) believed that their organizations' conflict of interest policies were very good. A total of 22 of 285 PAO leaders (7.7%) perceived pressure to conform their positions to the interests of corporate donors. Conclusions and Relevance: Patient advocacy organizations engage in wide-ranging health activities. Although most PAOs receive modest funding from industry, a minority receive substantial industry support, raising added concerns about independence. Many respondents report a need to improve their conflict of interest policies to help maintain public trust.

Clinical Sequencing Exploratory Research Consortium: Accelerating Evidence-Based Practice of Genomic Medicine.

Despite rapid technical progress and demonstrable effectiveness for some types of diagnosis and therapy, much remains to be learned about clinical genome and exome sequencing (CGES) and its role within the practice of medicine. The Clinical Sequencing Exploratory Research (CSER) consortium includes 18 extramural research projects, one National Human Genome Research Institute (NHGRI) intramural project, and a coordinating center funded by the NHGRI and National Cancer Institute. The consortium is exploring analytic and clinical validity and utility, as well as the ethical, legal, and social implications of sequencing via multidisciplinary approaches; it has thus far recruited 5,577 participants across a spectrum of symptomatic and healthy children and adults by utilizing both germline and cancer sequencing. The CSER consortium is analyzing data and creating publically available procedures and tools related to participant preferences and consent, variant classification, disclosure and management of primary and secondary findings, health outcomes, and integration with electronic health records. Future research directions will refine measures of clinical utility of CGES in both germline and somatic testing, evaluate the use of CGES for screening in healthy individuals, explore the penetrance of pathogenic variants through extensive phenotyping, reduce discordances in public databases of genes and variants, examine social and ethnic disparities in the provision of genomics services, explore regulatory issues, and estimate the value and downstream costs of sequencing. The CSER consortium has established a shared community of research sites by using diverse approaches to pursue the evidence-based development of best practices in genomic medicine.

An Ethical Framework for Allocating Scarce Life-Saving Chemotherapy and Supportive Care Drugs for Childhood Cancer.

Shortages of life-saving chemotherapy and supportive care agents for children with cancer are frequent. These shortages directly affect patients' lives, compromise both standard of care therapies and clinical research, and create substantial ethical challenges. Efforts to prevent drug shortages have yet to gain traction, and existing prioritization frameworks lack concrete guidance clinicians need when faced with difficult prioritization decisions among equally deserving children with cancer. The ethical framework proposed in this Commentary is based upon multidisciplinary expert opinion, further strengthened by an independent panel of peer consultants. The two-step allocation process includes strategies to mitigate existing shortages by minimizing waste and addresses actual prioritization across and within diseases according to a modified utilitarian model that maximizes total benefit while respecting limited constraints on differential treatment of individuals. The framework provides reasoning for explicit decision-making in the face of an actual drug shortage. Moreover, it minimizes bias that might occur when individual clinicians or institutions are forced to make bedside rationing and prioritization decisions and addresses the challenge that individual clinicians face when confronted with bedside decisions regarding allocation. Whenever possible, allocation decisions should be supported by evidence-based recommendations. "Curability," prognosis, and the incremental importance of a particular drug to a given patient's outcome are the critical factors to consider when deciding how to allocate scarce life-saving cancer drugs.

Physicians and Insider Trading.

Although insider trading is illegal, recent high-profile cases have involved physicians and scientists who are part of corporate governance or who have access to information about clinical trials of investigational products. Insider trading occurs when a person in possession of information that might affect the share price of a company's stock uses that information to buy or sell securities--or supplies that information to others who buy or sell--when the person is expected to keep such information confidential. The input that physicians and scientists provide to business leaders can serve legitimate social functions, but insider trading threatens to undermine any positive outcomes of these relationships. We review insider-trading rules and consider approaches to securities fraud in the health care field. Given the magnitude of the potential financial rewards, the ease of concealing illegal conduct, and the absence of identifiable victims, the temptation for physicians and scientists to engage in insider trading will always be present. Minimizing the occurrence of insider trading will require robust education, strictly enforced contractual provisions, and selective prohibitions against high-risk conduct, such as participation in expert consulting networks and online physician forums, by those individuals with access to valuable inside information.

Clinical research: Should patients pay to play?

Permitting patients to pay for participation in clinical research threatens the principles of social value and fair subject selection as well as robust clinical trial design.

National Survey of Hematopoietic Cell Transplantation Center Personnel, Infrastructure, and Models of Care Delivery.

Hematopoietic cell transplantation (HCT) is a complex procedure that requires availability of adequate infrastructure, personnel, and resources at transplantation centers. We conducted a national survey of transplantation centers in the United States to obtain data on their personnel, infrastructure, and care delivery models. A 42-item web-based survey was administered to medical directors of transplantation centers in the United States that reported any allogeneic HCT to the Center for International Blood and Marrow Transplant Research in 2011. The response rate for the survey was 79% for adult programs (85 of 108 centers) and 82% for pediatric programs (54 of 66 centers). For describing results, we categorized centers into groups with similar volumes based on 2010 total HCT activity (adult centers, 9 categories; pediatric centers, 6 categories). We observed considerable variation in available resources, infrastructure, personnel, and care delivery models among adult and pediatric transplantation centers. Characteristics varied substantially among centers with comparable transplantation volumes. Transplantation centers may find these data helpful in assessing their present capacity and use them to evaluate potential resource needs for personnel, infrastructure, and care delivery and in planning for growth.

Generalizability of trial results to elderly Medicare patients with advanced solid tumors (Alliance 70802).

BACKGROUND: In the United States, patients who enroll in chemotherapy trials seldom reflect the attributes of the general population with cancer, as they are often younger, more functional, and have less comorbidity. We compared survival following three chemotherapy regimens according to the setting in which care was delivered (ie, clinical trial vs usual care) to determine the generalizability of clinical trial results to unselected elderly Medicare patients. METHODS: Using SEER-Medicare data, we estimated survival for elderly patients (ie, age 65 years or older, n = 14097) with advanced pancreatic or lung cancer following receipt of one of three guideline-recommended first-line chemotherapy regimens. We compared their survival to that of similarly treated clinical trial enrollees, without age restrictions, with the same diagnosis and stage (n = 937). All statistical tests were two-sided. RESULTS: Trial patients were 9.5 years younger than elderly Medicare patients. Medicare patients were more often white and tended to live in areas of greater educational attainment than trial enrollees. For each tumor type, Medicare patients who were 75 years or older had median survivals that were six to eight weeks shorter than those of trial patients (4.3 vs 5.8 months following treatment with single agent gemcitabine for advanced pancreatic cancer, P = .03; 7.3 vs 8.9 months following treatment with carboplatin and paclitaxel for stage IV non-small cell lung cancer, P = .91; 8.2 vs 10.2 months following treatment with CDDP/ VP16 for extensive stage small cell lung cancer, P ≤ .01), whereas younger Medicare patients had survival times that were similar to those of trial patients. CONCLUSIONS: Results of clinical trials for advanced pancreatic cancer and lung cancers tended to correctly estimate survival for Medicare patients aged 65 to 74 years, but to overestimate survival for older Medicare patients by six to eight weeks. These estimates of Medicare patients' survival may aid subsequent patients and their oncologists in treatment decision-making.

Are hybrid umbilical cord blood banks really the best of both worlds?

Since the first use of umbilical cord blood (UCB) as a medical therapy, the number of UCB banks worldwide has grown. Public UCB banks offer the option of altruistic donation, whereas private banks allow a product to be stored for the exclusive use of the paying client. With many more UCB products banked privately than publicly in countries such as the USA, hybrid models blending aspects of public and private banking have been proposed. One such bank is in operation in the UK. In this paper we review the hybrid UCB model and conclude that it offers limited benefit to the general public. Furthermore, compared with private banking, this model provides few advantages and potential disadvantages to private clients.