Informed Consent among Clinical Trial Participants with Different Cancer Diagnoses.

IMPORTANCE: Informed consent is essential to ethical, rigorous research and is important to recruitment and retention in cancer trials. OBJECTIVE: To examine cancer clinical trial (CCT) participants' perceptions of informed consent processes and variations in perceptions by cancer type. DESIGN AND SETTING AND PARTICIPANTS: Cross-sectional survey from mixed-methods study at National Cancer Institute-designated Northeast comprehensive cancer center. Open-ended and forced-choice items addressed: (1) enrollment and informed consent experiences and (2) decision-making processes, including risk-benefit assessment. Eligibility: CCT participant with gastro-intestinal or genitourinary, hematologic-lymphatic malignancies, lung cancer, and breast or gynecological cancer (N = 334). MAIN OUTCOME MEASURES: Percentages satisfied with consent process and information provided; and assessing participation's perceptions of risks/benefits. Multivariable logistic or ordinal regression examined differences by cancer type. RESULTS: Most patient-participants felt well informed by the consent process (more than 90% overall and by cancer type) and. most (87.4%) reported that the consent form provided all the information they wanted, although nearly half (44.8%) reported that they read the form somewhat carefully or less. More than half (57.9%) said that talking to research staff (i.e., the consent process) had a greater impact on participation decisions than reading the consent form (2.1%). A third (31.1%) were very sure of joining in research studies before the informed consent process (almost half of lung cancer patients did-47.1%). Most patients personally assessed the risks and benefits before consenting. However, trust in physicians played an important role in the decision to enroll in CCT. CONCLUSIONS AND RELEVANCE: Cancer patients rely less on written features of the informed consent process than on information obtained from the research staff and their own physicians. Research should focus on information and communication strategies that support informed consent from referring physicians, researchers, and others to improve patient risk-benefit assessment and decision-making.

Association of Perceived Benefit or Burden of Research Participation With Participants' Withdrawal From Cancer Clinical Trials.

Importance: Attrition in cancer clinical trials (CCTs) can lead to systematic bias, underpowered analyses, and a loss of scientific knowledge to improve treatments. Little attention has focused on retention, especially the role of perceived benefits and burdens, after participants have experienced the trial. Objectives: To examine the association between patients' perceived benefits and burdens of research participation and CCT retention. Design, Setting, and Participants: This survey study was conducted at a National Cancer Institute-designated comprehensive cancer center in the Northeast region of the US. The sample included adult patients with a cancer diagnosis participating in cancer therapeutic trials. Data were collected from September 2015 to June 2019. Analysis of study data was ongoing since November 2019 through October 2022. Exposures: Self-reported validated survey instrument with a list of 22 benefits and 23 burdens of research participation that can be rated by patients with a 5-point Likert scale ranging from 1 (strongly disagree) to 5 (strongly agree). Main Outcomes and Measures: A primary outcome was actual withdrawal from the CCT, and a composite outcome was composite withdrawal that included both actual withdrawal and thoughts of withdrawing. Bivariate and multivariable logistic regressions were used. Results: Among the 334 participants in the sample, the mean (SD) age was 61.9 (11.5) years and 174 women (52.1%) were included. Top-cited benefits included both aspirational and action-oriented goals, including helping others (94.2%), contributing to society (90.3%), being treated respectfully (86.2%), and hoping for a cure (86.0%). Worry over receiving a placebo (61.3%), rearranging one's life (41.9%), and experiencing bothersome adverse effects (41.6%) were notable burdens. An increased burden score was associated with a higher probability of actual withdrawal (adjusted odds ratio [OR], 1.86; 95% CI, 1.1-3.17; P = .02) or composite withdrawal (adjusted OR, 3.44; 95% CI, 2.09-5.67; P < .001). An increased benefit score was associated with lower composite withdrawal (adjusted OR, 0.40; 95% CI, 0.24-0.66; P < .001). For participants who reported the benefits as being equal to or greater than the burdens, 13.4% withdrew. For those who perceived the benefits as being less than the burdens, 33.3% withdrew (adjusted OR, 3.38; 95% CI, 1.13-10.14; P = .03). The risk of withdrawal was even higher for the composite outcome (adjusted OR, 7.70; 95% CI, 2.76-21.48; P < .001). Conclusions and Relevance: This survey study found that patients perceived important benefits from CCT participation, and this perception was associated with trial retention, even among those who also perceived substantial burdens. A broader dialogue among stakeholders can inform an ethical and patient-centric focus on benefits throughout the course of a CCT to increase retention.

Experiences of Patients After Withdrawal From Cancer Clinical Trials.

Importance: Cancer clinical trials (CCTs) provide patients an opportunity to receive experimental drugs, tests, and/or procedures that can lead to remission. For some, a CCT may seem like their only option. Little is known about experiences of patient-participants who withdraw or are withdrawn from CCTs. Objective: To examine patient-participants' experiences during withdrawal from CCTs. Design, Setting, and Participants: This qualitative, descriptive study used a semistructured interview designed specifically for it, with open-ended and probing questions. The study took place at a National Cancer Institute-designated comprehensive cancer center affiliated with the University of Pennsylvania. The need for a sample of 20 interviewees was determined by code and meaning saturation (ie, no new themes revealed and identified themes fully elaborated). Interviews were transcribed verbatim and analyzed with a qualitative software program. Data coded with the software were refined into categories reflecting broad themes. A criterion-based sampling approach was used to select a subset of adult patients with cancer who were former CCT participants and who agreed on exit from those CCTs to a later interview about withdrawal experiences. They were contacted one by one by telephone from September 2015 through June 2019 until 20 agreed. Data analysis was completed in October 2020. Main Outcomes and Measures: Themes characterizing patient-participants' perceptions of their withdrawal experiences. Results: Respondents' mean (SD) age was 64.42 (8.49) years; 12 (63.2%) were men. Most respondents were White (18 respondents [94.7%]) and college educated (11 respondents [55.0%]). Cancer stage data were available for 17 participants, 11 of whom (64.7%) had stage IV cancer at CCT enrollment. Thirteen respondents reported withdrawal as a result of disease progression, and 5 withdrew because of adverse effects. Other reasons for withdrawal included acute illness and participant uncertainty about the reason. Analysis of interview data yielded 5 themes: posttrial prognostic awareness, goals of care discussions, emotional coping, burden of adverse effects, and professional trust and support. Subthemes included regrets or hindsight, urgency to start next treatment, and weighing benefits and burdens of treatment. Limited discussions about patient-participants' immediate posttrial care needs left many feeling that there was no clear path forward. Conclusions and Relevance: Patient-participants transitioning from a CCT described feeling intense symptoms and emotions and awareness that their life span was short and options seemed to be limited. Communication that includes attention to posttrial needs is needed throughout the CCT to help patient-participants navigate posttrial steps. Research should focus on components of responsible and ethical CCT transitions, including types and timing of discussions and who should begin these discussions with patient-participants and their families.

Meeting the Transitional Care Needs of Older Adults with COVID-19.

Older adults with COVID-19 who survive hospitalizations and return to their homes confront substantial health challenges and an unpredictable future. While understanding of the unique needs of COVID-19 survivors is developing, components of the evidence-based Transitional Care Model provide a framework for taking a more immediate, holistic response to caring for these individuals as they moved back into the community. These components include: increasing screening, building trusting relationships, improving patient engagement, promoting collaboration across care teams, undertaking symptom management, increasing family caregiver care/education, coordinating health and social services, and improving care continuity. Evidence generated from rigorous testing of these components reveal the need for federal and state policy solutions to support the following: employment/redeployment of nurses, social workers, and community health workers; training and reimbursement of family caregivers; widespread access to research-based transitional care tools; and coordinated local efforts to address structural barriers to effective transitions. Immediate action on these policy options is necessary to more effectively address the complex issues facing these older adults and their family caregivers who are counting on our care system for essential support.

An Assessment of State-Led Reform of Long-Term Services and Supports.

Health care in the United States is fragmented, inefficient, and rife with quality concerns. These shortcomings have particularly serious implications for adults with disabilities and functionally impaired older adults in need of long-term services and supports (LTSS). Three strategies have been commonly pursued by state governments to improve LTSS: expanding noninstitutional care, integrating payment and care delivery, and realigning incentives through market-based reforms. These strategies were analyzed using an evaluation framework consisting of the following dimensions: ease of access; choice of setting/provider; quality of care/life; support for family caregivers; effective transitions among multiple providers and across settings; reductions in racial/ethnic disparities; cost-effectiveness; political feasibility; and implementability. Although the analysis highlights potential benefits and drawbacks associated with each strategy, the limited breadth of the evidentiary base precludes an assessment of impact across all nine dimensions. More importantly, the analysis exposes the interdependent, complex system of care within which LTSS is situated, suggesting that policy makers will require a holistic and long-term perspective to achieve needed changes. Addressing the nation's LTSS needs will require a multipronged strategy incorporating a range of health and social services to meet the complex care needs of a diverse population in a variety of settings.

Unintended consequences of steps to cut readmissions and reform payment may threaten care of vulnerable older adults.

The US health care system is characterized by fragmentation and misaligned incentives, which creates challenges for both providers and recipients. These challenges are magnified for older adults who receive long-term services and supports. The Affordable Care Act attempts to address some of these challenges. We analyzed three provisions of the act: the Hospital Readmissions Reduction Program; the National Pilot Program on Payment Bundling; and the Community-Based Care Transitions Program. These three provisions were designed to enhance care transitions for the broader population of adults coping with chronic illness. We found that these provisions inadequately address the unique needs of vulnerable subgroup members who require long-term services and supports and, in some instances, could produce unintended consequences that would contribute to avoidable poor outcomes. We recommend that policy makers anticipate such unintended consequences and advance payment policies that integrate care. They should also prepare the delivery system to keep up with new requirements under the Affordable Care Act, by supporting providers in implementing evidence-based transitional care practices, recrafting strategic and operational plans, developing educational and other resources for frail older adults and their family caregivers, and integrating measurement and reporting requirements into performance systems.

Design of comprehensive Alzheimer's disease centers to address unmet national needs.

The problem of Alzheimer's disease (AD) exemplifies the challenges of dealing with a broad range of aging-related chronic disorders that require long-term, labor-intensive, and expensive care. As the baby boom generation ages and brain diseases become more prevalent, the need to confront the pending health care crisis is more urgent than ever before. Indeed, there is now a critical need to expand significantly the national effort to solve the problem of AD, with special focus on prevention. The Campaign to Prevent Alzheimer's Disease by 2020 (PAD2020) aims to create a new paradigm for planning and supporting the organization of worldwide cooperative research networks to develop new technologies for early detection and treatments of aging-related memory and motor impairments. PAD 2020 is developing an implementation plan to justify (1) increasing the federal budget for research, (2) developing novel national resources to discover new interventions for memory and motor disorders, and (3) creating innovative and streamlined decision-making processes for selecting and supporting new ideas. Since 1978 the National Institute on Aging or National Institute of Health (NIH) established an extensive national network of AD research facilities at academic institutions including AD Centers (ADCs), Consortium to Establish a Registry for AD, AD Cooperative Study (ADCS), AD Drug Discovery Program, National Alzheimer's Coordinating Center, National Cell Repository for AD, and AD Neuroimaging Initiative. However, despite the success of these programs and their critical contributions, they are no longer adequate to meet the challenges presented by AD. PAD 2020 is designed to address these changes by improving the efficiency and effectiveness of these programs. For example, the ADCs (P30s and P50s) can be enhanced by converting some into Comprehensive Alzheimer's Disease Centers (CADCs) to support not only research, but also by being demonstration projects on care/treatment, clinical trials, and education as well as by seamlessly integrating multisite collaborative studies (ADCS, AD Neuroimaging Initiative, Patient Registries, Clinical Data Banks, etc) into a cohesive structure that further enhances the original mission of the National Institute on Aging ADCs. Regional CADCs offer greater efficiency and cost savings while serving as coordinating hubs of existing ADCs, thereby offering greater economies of scale and programmatic integration. The CADCs also broaden the scope of ADC activities to include research on interventions, diagnosis, imaging, prevention trials, and other longitudinal studies that require long-term support. Thus, CADCs can address the urgent need to identify subjects at high risk of AD for prevention trials and very early in the course of AD for clinical trials of disease modification. The enhanced CADCs will allow more flexibility among ADCs by supporting collaborative linkages with other institutions and drawing on a wider expertise from different locations. This perspective article describes the University of Pennsylvania (Penn) CADC Model as an illustrative example of how an existing ADC can be converted into a CADC by better utilization of Penn academic resources to address the wide range of problems concerning AD. The intent of this position paper is to stimulate thinking and foster the development of other or alternative models for a systematic approach to the study of dementia and movement disorders.