Financial conflicts of interest among presenters, panellists and moderators at haematology and oncology FDA workshops.

OBJECTIVE: To assess the characteristics and financial conflicts of interest of presenters, panellists and moderators at haematology and oncology workshops held jointly with or hosted by the US FDA. SETTING: We included information on all publicly available haematology or oncology FDA workshop agendas held between 1 January 2018 and 31 December 2022. EXPOSURE: General and research payments reported on Open Payments, industry funding to patient advocacy organizations reported on their webpages or 990 tax forms and employment in both pharmaceutical and regulatory settings. RESULTS: Among physicians eligible for payments, 78% received at least one payment from the industry between 2017 and 2021. The mean general payment amount was $82,170 for all years ($16,434 per year) and the median was $14,906 for all years ($2981 per year). Sixty-nine per cent of patient advocacy speakers were representing organizations that received financial support from the pharmaceutical industry. Among those representing regulatory agencies or pharmaceutical companies, 16% had worked in both settings during their careers. CONCLUSIONS AND RELEVANCE: Our findings in this cross-sectional study show a majority of US-based physician presenters at haematology and oncology workshops held jointly with members of the US FDA have some financial conflict of interest with the pharmaceutical industry. These findings support the need for clear disclosures and suggest that a more balanced selection of presenters with fewer conflicts may help to limit bias in discussions between multiple stakeholders.

Impact of the Hemostasis and Thrombosis Research Society trainee workshop in recruiting the next generation of nonmalignant hematologists.

The United States is facing a shortage of physicians dedicated to nonmalignant hematology to meet future needs. The Hemostasis and Thrombosis Research Society (HTRS) developed a medical education program for trainees, "HTRS Trainee Workshops: Building a Career in Hemostasis and Thrombosis" in 2016. The aim of this study is to evaluate the impact of the workshop in recruiting the next generation of nonmalignant hematologists. Two surveys (post-workshop survey and alumni survey) were conducted. The post-workshop survey occurred within 30 days of each workshop and was completed by 81.9% (n = 185) of participants. Majority of respondents reported that the workshop had a positive impact to their practice and/or research (93.0%, n = 174) and career development (87.7%, n = 164). For the alumni survey which was conducted in 2018, 73 participants responded to the survey (38.2% response rate). Of the 38 respondents who had graduated from fellowship at the time of the survey, almost all chose a career in academic medicine. 41.7% (n = 15) reported their specialty as adult nonmalignant hematology and 25.0% (n = 9) as pediatric hematology/oncology with a nonmalignant hematology focus. 41.1% (n = 30) developed collaborative professional relationships, and 78.1% (n = 57) reported that the workshop had a positive influence in their choice to pursue nonmalignant hematology as a career. 67.1% (n = 49) were actively involved in research in nonmalignant hematology, with the most common being clinical research. This survey suggests that the HTRS Trainee Workshop is meeting its goals to recruit, train, and mentor the next generation of nonmalignant hematologists.

The role of hematologists in a changing United States health care system.

Major and ongoing changes in health care financing and delivery in the United States have altered opportunities and incentives for new physicians to specialize in nonmalignant hematology. At the same time, effective clinical tools and strategies continue to rapidly emerge. Consequently, there is an imperative to foster workforce innovation to ensure sustainable professional roles for hematologists, reliable patient access to optimal hematology expertise, and optimal patient outcomes. The American Society of Hematology is building a collection of case studies to guide the creation of institutionally supported systems-based clinical hematologist positions that predominantly focus on nonmalignant hematology. These roles offer a mix of guidance regarding patient management and the appropriate use and stewardship of clinical resources, as well as development of new testing procedures and protocols.

Planning for the future workforce in hematology research.

The medical research and training enterprise in the United States is complex in both its scope and implementation. Accordingly, adaptations to the associated workforce needs present particular challenges. This is particularly true for maintaining or expanding national needs for physician-scientists where training resource requirements and competitive transitional milestones are substantial. For the individual, these phenomena can produce financial burden, prolong the career trajectory, and significantly influence career pathways. Hence, when national data suggest that future medical research needs in a scientific area may be met in a less than optimal manner, strategies to expand research and training capacity must follow. This article defines such an exigency for research and training in nonneoplastic hematology and presents potential strategies for addressing these critical workforce needs. The considerations presented herein reflect a summary of the discussions presented at 2 workshops cosponsored by the National Heart, Lung, and Blood Institute and the American Society of Hematology.

The European Hematology Association Roadmap for European Hematology Research: a consensus document.

The European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better funded, more focused European hematology research. Initiated by the EHA, around 300 experts contributed to the consensus document, which will help European policy makers, research funders, research organizations, researchers, and patient groups make better informed decisions on hematology research. It also aims to raise public awareness of the burden of blood disorders on European society, which purely in economic terms is estimated at €23 billion per year, a level of cost that is not matched in current European hematology research funding. In recent decades, hematology research has improved our fundamental understanding of the biology of blood disorders, and has improved diagnostics and treatments, sometimes in revolutionary ways. This progress highlights the potential of focused basic research programs such as this EHA Roadmap.The EHA Roadmap identifies nine 'sections' in hematology: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation. These sections span 60 smaller groups of diseases or disorders.The EHA Roadmap identifies priorities and needs across the field of hematology, including those to develop targeted therapies based on genomic profiling and chemical biology, to eradicate minimal residual malignant disease, and to develop cellular immunotherapies, combination treatments, gene therapies, hematopoietic stem cell treatments, and treatments that are better tolerated by elderly patients.

Premix versus On-Demand Workflow Models in a Veterans Affairs Hematology/Oncology Pharmacy: Impact on Estimated Waste Costs and Patient Wait Time.

Background: The balance between reducing patient wait time and mitigating waste of parenteral products has not been well described in literature. Objective: Evaluate the patient wait times and cost-effectiveness of employing a premix versus an on-demand workflow model for compounding parenteral admixtures in a hematology/oncology infusion setting. Methods: This single center, retrospective cost analysis compiled manually documented monthly waste reports and estimated drug pricing for the institution to calculate the cost of waste during both premix and on-demand compounding workflows. Time to administration was audited for one week with both models. Results: Over a period of 28.5 months following the premix model, 564 products were documented as wasted ($1,196,014.01 in estimated drug purchasing cost). Over a period of 3 months following the on-demand model, 12 products were wasted ($34,823.98 in estimated drug purchasing cost). Switching models reduced the monthly average number of wasted products from 20 to 4 per month; the average cost of waste was reduced from $41,965.40 to $11,607.99 per month (P < .0001). Overall patient wait time from clearance until administration, excluding any recommended wait times after premedication administration (if applicable), was similar in both models: an average of 38.26 minutes in the premix model and 40.97 minutes in the on-demand model. Conclusion: Premixing parenteral admixtures was not cost effective at our institution. After resuming an on-demand compounding model, the monthly cost of waste (based on drug pricing alone) was reduced by over 70%. The wait time from clearance to treatment administration was similar in both models.

Evaluation of non-research and research industry payments to pediatric hematologist/oncologists in the United States between 2013 and 2021.

Financial interactions between healthcare industry and pediatric hematologist/oncologists (PHOs) could be conflicts of interest. Nevertheless, little is known about financial relationships between healthcare industry and PHOs. This cross-sectional analysis of the Open Payments Database examined general and research payments to PHOs from healthcare industry in the United States between 2013 and 2021. Payments to the PHOs were analyzed descriptively. Trends in payments were assessed using generalized estimating equation models. Of 2784 PHOs, 2142 (76.9%) PHOs received payments totaling $187.3 million from the healthcare industry between 2013 and 2021. Approximately, $46.3 million (24.8%) were general payments and $137.7 million (73.5%) were funding for research where PHOs served as principal investigators (associated research funding). Both general payments and associated research funding considerably increased between 2014 and 2019. The number of PHOs receiving general payments and associated research funding annually increased by 2.2% (95% CI: 1.2-3.3%, p < .001) and 5.0% (95% CI: 3.3-6.8%, p < .001) between 2014 and 2019, respectively.

[Diagnosis-related groups as an instrument to develop suitable case-based lump sums in hematology and oncology]. / Diagnosis-Related Groups als Steuerungssystem zur Entwicklung leistungsgerechter Fallpauschalen in der Hämatologie und Onkologie.

In 2003 a new reimbursement system was established for German hospitals. The approximately 17 million inpatient cases per year are now reimbursed based on a per-case payment regarding diagnoses and procedures, which was developed from an internationally approved system. The aim was a better conformity of costs and efforts in in-patient cases. In the first 2 years after implementation, the German diagnosis-related group (DRG) system was not able to adequately represent the complex structures of treatment in hematological and oncological in-patients. By creating new diagnoses and procedures (International Classification of Diseases 10 (ICD-10) and Surgical Operations and Procedures Classification System (OPS) catalogues), generating new DRGs and better splitting of existing ones, the hematology and oncology field could be much better described in the following years. The implementation of about 70 'co-payment structures' for new and expensive drugs and procedures in oncology was also crucial. To reimburse innovations, an additional system of co-payments for innovations was established to bridge the time until innovations are represented within the DRG system itself. In summary, hematological and oncological in-patients, including cases with extraordinary costs, are meanwhile well mapped in the German reimbursement system. Any tendencies to rationing could thereby be avoided, as most of the established procedures and costly drugs are adequately represented in the DRG system.

[Rationalization, rationing, prioritization: terminology and ethical approaches to the allocation of limited resources in hematology/oncology]. / Rationalisierung, Rationierung, Priorisierung: Terminologie und ethische Begründungsansätze zur Allokation bei begrenzten Ressourcen in der Hämatologie/Onkologie.

The field of oncology with its numerous high-priced innovations contributes considerably to the fact that medical progress is expensive. Additionally, due to the demographic changes and the increasing life expectancy, a growing number of cancer patients want to profit from this progress. Since resources are limited also in the health system, the fair distribution of the available resources urgently needs to be addressed. Dealing with scarcity is a typical problem in the domain of justice theory; therefore, this article first discusses different strategies to manage limited resources: rationalization, rationing, and prioritization. It then presents substantive as well as procedural criteria that assist in the just distribution of effective health benefits. There are various strategies to reduce the utilization of limited resources: Rationalization means that efficiency reserves are being exhausted; by means of rationing, effective health benefits are withheld due to cost considerations. Rationing can occur implicitly and thus covertly, e.g. through budgeting or the implementation of waiting periods, or explicitly, through transparent rules or policies about healthcare coverage. Ranking medical treatments according to their importance (prioritization) is often a prerequisite for rationing decisions. In terms of requirements of justice, both procedural and substantive criteria (e.g. equality, urgency, benefit) are relevant for the acceptance and quality of a decision to limit access to effective health benefits.

[Benefit-risk evaluation of new drugs as a basis for decisions on prioritization in hematology/oncology: methodical challenges and problem-solving strategies]. / Nutzen-Risiko-Bewertung neuer Wirkstoffe als Grundlage für Priorisierungsentscheidungen in der Hämatologie/Onkologie: Methodische Herausforderungen und Lösungsansätze.

Innovations in the drug treatment of cancer patients pose several medical and ethical challenges. The increasing incidence and prevalence of cancer, in combination with the availability of new and usually highly expensive anticancer drugs, are associated with a significant increase in the general costs for the treatment of cancer patients. Therefore, the development of scientific strategies for judgments on benefits is indispensable. In this paper, the authors analyze the benefit assessment and the benefit-risk assessment as a foundation for decisions on prioritization in hematology and oncology. In a first step, regulatory aspects and shortcomings regarding the design of clinical trials in oncology before and after approval of anticancer drugs are identified as factors that contribute to difficulties in establishing the effectiveness of new drugs entering the health care market. The authors will conclude with suggestions for the improvement of benefit-risk analyses and the generation of scientific data necessary for such analyses.

[France, an attractive country for international clinical research: 2010 survey assessed by Leem (French association of pharmaceutical companies)]. / Place de la France dans la recherche clinique internationale : enquête 2010 du Leem France et recherche clinique internationale.

In order to evaluate the attractiveness of France for conducting international clinical trials, a survey is performed every two years among pharmaceutical companies that are based in France or have affiliates in France. Twenty-nine companies (72% of the French market) including 10 newly participants, have participated in the current survey which included 328 international phase II and III clinical studies, 79 countries, 24,337 centres and 249,704 patients (included in 2008/2009). France (291 patients/million inhabitants) ranked among the best European recruiters in second position behind Scandinavia. Since 2008, protocols are still to be given the go-ahead by French Authorities (Afssaps and CPP) within 60 days, in accordance with European directive. The continuation of reduction in deadlines for hospital contracts is encouraging and highlights the positive effect of CeNGEPS. French state-of-art is well recognized in Oncology/Hematology and Orphan disease which could be an example to improve the competitiveness of France in other key therapeutic areas such as Alzheimer's disease and Immuno-Inflammation.

Investment analysts and the American Society of Hematology.

Investment analysts are a growing presence at the Annual Meeting of the American Society of Hematology (ASH), and financial professionals frequently contact ASH members for information and perspective on drugs, devices, and scientific developments. Recent incidents have raised concerns about consulting relationships between physicians and the investment industry; the appropriate role of medical societies in influencing these relationships is unclear. In this essay, I summarize the current situation, discuss potential risks and benefits from interactions between physicians and investment analysts, and outline issues that all individuals involved in investment industry consulting should consider. I also propose changes in ASH policy that may help safeguard public trust as well as preserve the access of clinicians and scientists to clinically relevant data presented at the Annual Meeting.

The unit costs of inpatient hospital days, outpatient visits, and daycare treatments in the fields of oncology and hematology.

OBJECTIVES: Many economic evaluations are conducted in the fields of oncology and hematology, partially owing to the introduction of new expensive drugs in this field. Even though inpatient days, outpatient visits, and daycare treatments are frequently the main drivers of total treatment costs, their unit costs often lack generalizability. Therefore, we aimed to determine the unit costs of inpatient hospital days, outpatient visits, and daycare treatments specifically for oncological and hematological diseases in The Netherlands from the hospital's perspective. METHODS: Unit costs were collected from 30 oncological and hematological departments of 6 university and 24 general hospitals. Costs included direct labor and indirect labor, hotel and nutrition, overheads and capital. Ordinary least squares regression models were constructed to examine the degree of association between unit costs and hospital and hospital department characteristics. All costs were based on Euro 2007 cost data. RESULTS: At university hospitals, the unit costs per inpatient day were determined at €633 in oncological and €680 in hematological departments. At general hospitals, the mean costs per inpatient day were €400. Unit costs for inpatient hospital days, outpatient visits. and daycare treatments equalled the relative ratio 100:21:44. Direct labor costs were the major cost driver and the type of hospital (university, yes/no) was a strong predictor of unit costs. CONCLUSIONS: The present study provided unit costs for inpatient hospital days, outpatient visits, and daycare treatments in the fields of oncology and hematology. The results may be used as Dutch reference unit prices in economic evaluations assessing oncological and hematological diseases.

Navigating Ethical Practices in the Era of High Cost Hematology.

PURPOSE OF REVIEW: In this review article, we will highlight ethical issues faced by hematologists due to a growing constellation of expensive diagnostics and therapeutics in hematology. We outline the important issues surrounding this topic including stakeholders, cost considerations, and various ethical challenges surrounding access to care, communication about costs, and individual vs. societal responsibilities. We review available tools to navigate these ethical themes and offer potential solutions. RECENT FINDINGS: We identified several gaps in the literature on the topic of ethical issues in hematology treatment and supplement by non-hematological cancer and general medical literature. We propose proactive solutions to address these problems to include cost transparency, utilization of evidence-based decision making tools, application of the four quadrant approach to ethical care, and advanced systems-based practice curriculum for physician trainees.

Preparing Trainees to Deliver High-Value and Cost-Conscious Care in Hematology.

PURPOSE OF REVIEW: Despite national-level directives to reduce healthcare waste and promote high-value care (HVC), clinical educators struggle to equip trainees with the knowledge and skills needed to practice value-based care. In this review, we analyze ongoing efforts in graduate medical education (GME) to enhance trainee competence in delivery of high-value and cost-conscious care. RECENT FINDINGS: Surveys of residents and program directors have shown that while many training programs want to offer formal training in high-value care delivery, few succeed. Although several studies suggest that trainees model stewardship behaviors after clinical preceptors, there remains a shortage of faculty role models skilled in providing HVC. Preparing future hematologist-oncologists to provide cost-conscious care will require significant cultural change at the institutional and program levels and will depend heavily on the development of skilled clinical role models.

Choosing Wisely® in Hematology: Have We Made a Difference?

PURPOSE OF REVIEW: The Choosing Wisely® initiative, led by the American Board of Internal Medicine Foundation in collaboration with national professional medical societies, aims to help patients choose care that is essential, free from harm, and evidence-based. The American Society of Hematology has advocated practices specific to hematology for physicians and patients to examine carefully. Here, we summarize various barriers to adopting these practices, interventions used to improve adoption, and challenges in measuring the effectiveness of these interventions. RECENT FINDINGS: The Choosing Wisely® campaign has become an international effort with more than 20 countries worldwide having embraced it. Such widespread interest indicates that the campaign initiated an important dialog between patients and physicians about overutilization of resources. Evidence showing the positive impact of interventions on adopting these practices is accumulating, but their effect on improving clinical outcomes is uncertain. Decreasing overuse of resources is a cultural change in perspective for practitioners and patients alike. We believe that healthcare delivery is transitioning from being volume-based to value-based. As we continue to support the Choosing Wisely® campaign, we need to implement strategies to document and measure the influence of our value-based recommendations on physician practices, patient care and attitudes, and healthcare costs.

The Financial Effect of Medicare Coverage Design and Safety Net Options for Cancer Care.

BACKGROUND: National spending on specialty medications accounted for approximately $193 billion in 2016. The coverage design for Medicare Parts B and D has shifted medication costs to patients, which may prohibit patients from starting or maintaining therapy due to affordability. As a result, patients have enrolled in safety net financial options, such as patient assistance and foundation programs. Safety net options may provide savings not otherwise realized by Medicare; however, they may have a negative financial effect on health systems and pharmaceutical manufacturers. OBJECTIVES: To (a) quantify financial savings to Medicare as a result of patient enrollment in patient assistance programs and (b) quantify the financial effect of safety net options for patients, manufacturers, and the academic medical center that participated in this study. METHODS: A single-center, nonrandomized, retrospective pilot study of Medicare beneficiaries was conducted. Patients who were prescribed hematology/oncology specialty medications and enrolled in safety net options between July 2015 and June 2017 were included. Investigators collected data related to fill history, drug cost, and prescription coverage. The primary outcome was the overall cost savings to Medicare as a result of patient enrollment in patient assistance programs. Secondary outcomes included total patient out-of-pocket savings as a result of foundation copayment support, financial effect on manufacturers as a result of patient assistance programs, and health system revenue impact as a result of safety net options. Descriptive statistics were used. RESULTS: This study included 114 patients. Medicare saved $5,083,816.83 over 2 years as a result of patient assistance programs. Eight foundations provided $240,350.04 in patient insurance copayments. Nine manufacturers provided 2,243 free drug doses, valued at $3,379,032.34. The participating medical center missed the opportunity for $6,481,543.55 in revenue due to patient assistance programs. CONCLUSIONS: The participating medical center's efforts to improve access to oncology care took considerable time and resources. These activities, as well as unreimbursed infusion services, were costs to the medical center that may not be recognized by Medicare. Manufacturers also supported patient access through their sponsored patient assistance programs. The use of these services and safety net options resulted in cost savings to Medicare and their beneficiaries. DISCLOSURES: No outside funding supported this study. The authors have nothing to disclose. Findings from this study were part of a podium research presentation at the Great Lakes Pharmacy Residency Conference; April 25, 2018; West Lafayette, IN.