During the 2023 American Society of Clinical Oncology (ASCO) Genitourinary Cancers Symposium, a panel of experts identified some of the key challenges to achieving health equity in bladder cancer care and discussed what can be done to improve access to clinical trials, mitigate financial toxicity, and promote value-based care.
Expanding Eligibility Criteria for Clinical Trials
Elad Sharon, MD, MPH, Co-Director, Cancer Therapy Evaluation Program, National Cancer Institute, began the session by discussing the importance of expanding clinical trial eligibility criteria to accrue more diverse patient populations.
“Expanding eligibility criteria on clinical trials is an essential means of improving health equity,” he said.
Dr Sharon explained that although eligibility criteria are necessary to define the patient population being studied, isolate the potential effect of an experimental agent, and ensure that clinical trials are conducted safely, exclusions can lead to treatment dilemmas and uncertainty.
Specifically, overly stringent eligibility criteria can restrict patient access to investigational drugs and limit the ability to generalize the results to the broader population of patients who will ultimately use those drugs.
Over the past several years, encouraging progress has been made to ensure that more patients are eligible for clinical trials. In 2016, ASCO and Friends of Cancer Research (Friends) initiated a joint project to develop and advance strategies to change the exclusionary nature of eligibility criteria in clinical trials.1 This led to the development of key recommendations that catalyzed efforts to improve the applicability and accessibility of clinical trials to patients with brain metastases, human immunodeficiency virus infection, younger age, organ dysfunction, and prior/concurrent malignancies. These guidelines were implemented by the National Cancer Institute to modernize the Cancer Therapy Evaluation Program protocol and were used to inform FDA guidance eligibility criteria for oncology clinical trials.
ASCO/Friends continue to work with stakeholder experts, including ASCO’s Cancer Research and Health Equity Committees, to develop evidence-based, consensus recommendations that focus on further expanding eligibility criteria to make trial populations more reflective of the general cancer population. In the ASCO/Friends May 2021 clinical trial eligibility criteria update, key recommendations were made pertaining to laboratory reference ranges and testing intervals.2 This publication included the following recommendations specific to laboratory references:
- Laboratory test results should be used only as exclusion criteria when scientifically justified and when abnormal test results confer safety concerns
- Laboratory reference values should account for potential normal variations due to race, ethnicity, age, sex, and gender identity
- Routine assessment of laboratory test–based exclusion criteria should be conducted during the course of clinical research and drug development as investigational agents progress from earlier- to later-phase clinical trials.
Addressing Financial Toxicity
Addressing challenges associated with financial toxicity was the focus of a presentation by Vidit Sharma, MD, MS, Assistant Professor, Urology, Mayo Clinic, Rochester, MN, who cited data from a SEER-Medicare analysis, which projected that costs associated with bladder cancer treatment will increase to $11.6 billion by 2030.3 He emphasized that the estimated costs in this analysis do not include additional expenses of therapeutic agents that have not yet been approved by the FDA.
Dr Sharma went on to highlight results from a review exploring the economic burden of cancer in the United States,4 which showed that patients must pay not only the direct out-of-pocket costs of medical care, but the indirect costs related to lodging, travel, and fees paid to entities other than healthcare providers. From a societal perspective, a bladder cancer diagnosis can be even more costly, he noted, since total expenses include direct costs to payers and patients as well as indirect costs associated with reduced and unpaid leave for patients and caregivers.
“The costs that we can measure—and these are the direct costs that we usually measure—are often the tip of the iceberg of the costs that our patients usually face,” Dr Sharma said.
He also discussed data from a survey of 138 patients with bladder cancer, indicating that nearly 1 in 4 respondents experienced financial toxicity, which was defined as “having to pay more for medical care than you can afford.”5 Financial toxicity was more frequently reported in younger patients, Black patients, and those without a college degree, and correlated with lower quality-of-life scores. Patients who experienced financial toxicity were also more likely to delay care than those who did not have financial toxicity.
Dr Sharma asserted that approximately 30% of direct Medicare costs associated with bladder cancer are related to complications. Thus, reducing the rate of these complications can reduce costs. He stated that the first step in mitigating financial toxicity is to identify patients at risk and then connect them to resources (ie, social workers and financial navigators) before the start of treatment. It is also important to include financial concerns in the shared decision-making process with patients, he said.
Promoting Value-Based Care
Cary Stimson, MD, JD, Assistant Professor, Urology, and Senior Vice President of Value Transformation, Office of Population Health, Vanderbilt University Medical Center, Nashville, TN, discussed the role of value-based care in addressing financial toxicity associated with bladder cancer care.
When considering value-based care, paying for “healthcare that works” is critical because it includes both how clinicians are compensated for what they do and the care that they provide to their patients, Dr Stimson said, adding that value is determined by dividing outcomes by the amount spent. Clinicians provide value-based care when they are willing to accept financial risk for the costs of care.
Dr Stimson went on to cite several examples demonstrating how the principles of value-based care can be applied to diagnostic, treatment, and surveillance strategies in the management of bladder cancer.
Regarding diagnosis, he said that photodynamic transurethral resection of bladder tumor did not reduce recurrence, and was therefore not cost-effective when compared with white light at 3 years. Regarding treatment, Dr Stimson said that alvimopan (Entereg) can shorten the time to gastrointestinal recovery after radical cystectomy, thereby reducing hospital stays and lowering costs, making this a high-value drug. In contrast, atezolizumab (Tecentriq) is not cost-effective compared with cytotoxic chemotherapy as second-line therapy for metastatic bladder cancer. Regarding surveillance, he said that CellDetect staining has been shown to have higher sensitivity and negative predictive value compared with cytology and UroVysion fluorescence in situ hybridization.
Dr Stimson concluded by stating that there are many “value opportunities” in the management of bladder cancer and that providers should continue to seek out these opportunities and be prepared to implement them.
- American Society of Clinical Oncology. Broadening eligibility criteria to make clinical trials more representative. https://old-prod.asco.org/research-data/clinical-trials/clinical-trial-eligibility-criteria. Accessed March 22, 2023.
- Spira AI, Stewart MD, Jones S, et al. Modernizing clinical trial eligibility criteria: recommendations of the ASCO-Friends of Cancer Research laboratory reference ranges and testing intervals work group. Clin Cancer Res. 2021;27:2416-2423.
- Mariotto AB, Enewold L, Zhao J, et al. Medical care costs associated with cancer survivorship in the United States. Cancer Epidemiol Biomarkers Prev. 2020;29:1304-1312.
- Yabroff KR, Lund J, Kepka D, Mariotto A. Economic burden of cancer in the United States: estimates, projections, and future research. Cancer Epidemiol Biomarkers Prev. 2011;20:2006-2014.
- Castilla-Lennon MM, Choi SK, Deal AM, et al. Financial toxicity among patients with bladder cancer: reasons for delay in care and effect on quality of life. J Urol. 2018;199:1166-1173.