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The USA spent $99 billion on orally administered and clinician-administered anticancer therapies (excluding supportive care) in 2023 and spending is projected to increase to $180 billion by 2028. This increased spending on anticancer therapies largely reflects the high launch prices of novel therapeutics and increases in the prices of existing products, even in the absence of new evidence of clinical benefit or changes in use. Consequently, high prices have impeded Americans' access to and affordability of necessary anticancer therapies and thus increased their risk of cost-related non-adherence, cancer recurrence and mortality. To address the rising prices and concerns regarding Americans' spending on anticancer therapies, state and federal governments have, over the past decade, enacted legislation that caps out-of-pocket spending, expands subsidies and requires drug price negotiations. In this Perspective, we summarize US policies aimed to lower the costs of anticancer therapies, discuss the implications of such reforms and propose additional solutions needed to reduce costs and increase value., Competing Interests: Competing interests S.B.D. is a member of the Institute for Clinical and Economic Review’s (ICER) Midwestern Comparative Effectiveness Advisory Council and a member of the Medicare Payment Advisory Commission (MedPAC). This work does not necessarily represent the official position of ICER or MedPAC. The other authors declare no competing interests., (© 2024. Springer Nature Limited.)

Immune checkpoint blockade (ICB) therapies are one of the greatest advances in the history of cancer care and are now commonly used in the management of many different malignancies. However, much remains unknown about the factors that affect the efficacy and side effect profile of these agents. This review delves into the published literature that evaluates the intricate interplay between race, age, gender, and social determinants in shaping outcomes following ICB across solid tumors and hematologic malignancies. We examine the pivotal phase 2 and 3 trials to evaluate the demographics of participants and outcomes based on these variables, if reported. Most, but not all, trials reported some basic demographic information like age, sex, race, ethnicity, and/or geographic area for enrollment. Clinically relevant biological markers that could affect ICB outcomes such as obesity or markers of social determinants of health were largely not reported. Trials were generally representative for men and women based on expected prevalence for a given malignancy, but often under-represented non-white participants and rarely enrolled patients from the global south. Subgroup analyses were conducted in many ICB trials for solid malignancies, but rarely conducted for hematologic malignancies. These analyses largely showed similar qualitative benefit across subgroups, but adverse events were rarely reported by subgroup. This review adds to our understanding of the populations that these clinical trials have studied and highlight the urgent need to redouble our efforts at increasing the diversity of the population in future ICB trials., Competing Interests: Competing interests: No, there are no competing interests., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Mantle cell lymphoma is a challenging subtype of B-cell non-Hodgkin lymphoma treat characterized by its aggressive nature and propensity for relapse or refractory (R/R) disease for many patients. The introduction of Bruton's tyrosine kinase inhibitors has significantly improved the outcomes for patients with R/R MCL, but a considerable proportion of patients eventually experience disease progression or develop resistance to these agents. In recent years, immunotherapeutic approaches have emerged as promising treatment options. The treatment landscape is quickly progressing with the FDA approval of CAR-T cell therapy as well as several promising bispecific antibody therapies and antibody-drug conjugates in clinical development. This review article aims to provide a comprehensive overview of the current state of immunotherapeutic options available for patients with R/R MCL., (Copyright © 2023. Published by Elsevier Inc.)

Purpose of Review: Cardiovascular medications improve health and prevent early death. However, high drug prices reduce the use of these medications and strain the health system. The Inflation Reduction Act (IRA) of 2022 allows Medicare to negotiate drug prices with manufacturers and reduces out-of-pocket drug costs for Medicare beneficiaries. This article explores the potential impact that the IRA will have on the treatment of cardiovascular disease., Recent Findings: Cardiovascular disease medications are likely to be selected for price negotiations under the IRA, leading to savings for patients and for Medicare. Recent work suggests that the IRA's reforms to the Medicare Part D drug benefit will meaningfully reduce out-of-pocket costs for important cardiovascular medications. The IRA is expected to impact cardiovascular disease treatments via price negotiations and through the broader access to medications afforded by improvements to Part D coverage design., (© 2023. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.)

Allogeneic hematopoietic cell transplant (allo-HCT) for eligible patients with acute myeloid leukemia (AML) in first complete remission is a central treatment paradigm to achieve durable remission. However, disease relapse after allo-HCT remains a significant concern and generally portends a poor prognosis. There is significant interest regarding the role for maintenance therapy after allo-HCT for patients with high risk of relapse, regardless of the presence of measurable residual disease. While there are currently no therapies approved for maintenance therapy for AML after allo-HCT, there are a number of ongoing investigations examining the role of maintenance therapies that include targeted agents against FLT3-ITD or IDH mutations, hypomethylating agents, immunomodulatory therapies and cellular therapies. In this review, we examine the current landscape and future strategies for maintenance therapy for AML after allo-HCT., Competing Interests: YBC has served as a consultant for Novartis, Incyte, Jasper, Celularity, Equilium, Actinium, Daiichi and Abbvie. The remaining author declares that the research was conducted in the absence of any commercial or financial relationships that could be constructed as a potential conflict of interest., (Copyright © 2022 Nayak and Chen.)

Financial toxicity describes the financial burden and distress that can arise for patients, and their family members, as a result of cancer treatment. It includes direct out-of-pocket costs for treatment and indirect costs such as travel, time, and changes to employment that can increase the burden of cancer. While high costs of cancer care have threatened the sustainability of access to care for decades, it is only in the past 10 years that the term "financial toxicity" has been popularized to recognize that the financial burdens of care can be just as important as the physical toxicities traditionally associated with cancer therapy. The past decade has seen a rapid growth in research identifying the prevalence and impact of financial toxicity. Research is now beginning to focus on innovations in screening and care delivery that can mitigate this risk. There is a need to determine the optimal strategy for clinicians and cancer centers to address costs of care in order to minimize financial toxicity, promote access to high value care, and reduce health disparities. We review the evolution of concerns over costs of cancer care, the impact of financial burdens on patients, methods to screen for financial toxicity, proposed solutions, and priorities for future research to identify and address costs that threaten the health and quality of life for many patients with cancer., (© Society of Behavioral Medicine 2021. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.)

Objectives: Hepatic encephalopathy (HE) is associated with hospital readmissions and mortality. We sought to determine whether cognitive testing and stool frequency at discharge predicted 30-day readmission or death in cirrhotic patients admitted with overt HE., Methods: We approached consecutive inpatients with cirrhosis and overt HE when they were within 48 hours of discharge. Patients underwent cognitive tests, including Psychometric Hepatic Encephalopathy Score (PHES), and stool frequency was documented. Chart review identified Model for End-Stage Liver Disease-sodium (MELD-Na) and the presence of non-HE extrahepatic organ failures. Cox proportional hazards models were used to evaluate predictors of time to the primary composite outcome of hospital readmission for HE or death within 30 days, censoring for liver transplantation., Results: Of 51 patients consented and enrolled, 14 patients met the primary composite outcome. In unadjusted Cox models, 4 variables predicted HE readmission or death: MELD-Na (hazard ratio [HR] 1.10 [1.01-1.20], P = 0.03), respiratory failure (HR 4.26 [1.47-12.35], P = 0.008), total number of HE extrahepatic organ failures (HR 1.79 [1.12-2.88], P = 0.02), and score on a PHES subtest, Number Connection Test A (per 30 seconds; HR 1.25 [1.06-1.47], P = 0.01). PHES and 24-hour stool frequency did not predict the primary outcome. When controlling for MELD-Na, respiratory failure predicted the primary outcome (HR 3.67 [1.24-10.86], P = 0.02)., Conclusion: Cognitive testing and stool frequency at discharge did not predict poor outcomes in patients admitted with HE, while respiratory failure appeared to be a strong predictor.

Background: Current practice and policies maintain that it is very important to disclose to potential research subjects that their treatment will be selected by randomization. In contrast, it typically is not considered important to disclose to patients how doctors select their treatment. Unfortunately, when the available treatment options are similar to one another, this approach has the potential to inadvertently undermine both clinical research and clinical care. Hence, it is important to assess whether, in the context of similar treatment options, individuals support current practice of using very different disclosure practices in research and care., Methods: Respondents were randomly presented with either (1) a "drug" scenario involving two medications for hypertension (called CTD and TRT) whose risk-benefit profiles are very similar, or (2) a "dose timing" scenario involving morning versus nighttime dosing of the same antihypertensive. Respondents were asked whether, in the presented scenario, they agree, using a 7-point scale (1 = strongly disagree, 4 = neutral, 7 = strongly agree), that it is important to disclose to potential research subjects that whether they receive CTD or TRT/AM or PM dosing will be determined by randomization. Respondents were also asked whether they agree, using the same 7-point scale, that it is important to disclose to patients who face the same treatment options how their doctor will decide whether they receive CTD or TRT/AM or PM dosing., Results: The survey was sent to 3330 online GfK KnowledgePanel members and completed by 2130 (response rate, 64.0%). Respondents indicated that it is somewhat important to disclose to potential subjects that whether they receive CTD or TRT/AM or PM dosing will be determined by randomization (mean, 5.10 [95% CI, 5.02 to 5.17]). Respondents also indicated that it is slightly more important to disclose to patients who face the same treatment options how the doctor will decide whether they receive CTD or TRT/AM or PM dosing (mean, 5.29 [95% CI, 5.22 to 5.36]; p < .001). In addition, 66.4% indicated that, in the setting of similar options, it is equally important to disclose how treatment is selected in research and care, 20.5% indicated it is more important to disclose this information in clinical care, and 13.1% indicated it is more important to disclose it in research., Conclusion: When the available options are similar to each other, individuals do not support current practice and policies that maintain that disclosure of how treatments are selected is very important in clinical research but not important in clinical care. Future research will be needed to evaluate the feasibility of developing disclosure practices and policies for this context that are consistent with individuals' views.

Background: Significant debate surrounds the issue of whether written consent is necessary for pragmatic randomized, controlled trials (RCTs) with low risk., Objective: To assess the U.S. public's views on alternatives to written consent for low-risk pragmatic RCTs., Design: National experimental survey (2 × 2 factorial design) examining support for written consent versus general notification or verbal consent in 2 research scenarios., Setting: Web-based survey conducted in December 2014., Participants: 2130 U.S. adults sampled from a nationally representative, probability-based online panel (response rate, 64.0%)., Measurements: Respondent's recommendation to an ethics review board and personal preference as a potential participant on how to obtain consent or notification in the 2 research scenarios., Results: Most respondents in each of the 4 groups (range, 60.3% to 71.5%) recommended written informed consent, and personal preferences were generally in accord with that advice. Most (78.9%) believed that the pragmatic RCTs did not pose additional risks, but 62.5% of these respondents would still recommend written consent. In contrast, a substantial minority in all groups (28.5% to 39.7%) recommended the alternative option (general notification or verbal consent) over written consent., Limitation: Framing effects could have affected respondents' attitudes, and nonrespondents may have differed in levels of trust toward research or health care institutions., Conclusion: Most of the public favored written informed consent over the most widely advocated alternatives for low-risk pragmatic RCTs; however, a substantial minority favored general notification or verbal consent., Primary Funding Source: Time-sharing Experiments for the Social Sciences and Intramural Research Program of the National Institutes of Health Clinical Center.

In an effort to control health costs, payers are increasingly turning to step therapy (or "fail first") policies in pharmacy benefit design. These policies restrict coverage of expensive therapies unless patients have already failed treatment with a lower-cost alternative. More than other utilization management tools such as formulary tiering, step therapy raises important ethical concerns regarding the proper balance between cost control and the ability of patients and clinicians to tailor care to the needs of the individual patient. This article provides eight design criteria to guide the ethical development and evaluation of step therapy policies and describes six clinical scenarios in which step therapy may be appropriate. The ethical criteria and scenarios are intended to provide guidance and transparency for insurers, patients, clinicians, and policy makers in choosing and paying for the appropriate therapies., (Project HOPE—The People-to-People Health Foundation, Inc.)