Patient Advocacy Organizations Research Articles

The Development and Impact of AYA Can—Canadian Cancer Advocacy: A Peer-Led Advocacy Organization for Adolescent and Young Adult Cancer in Canada

Adolescents and young adults (AYAs; 15–39 years) diagnosed with cancer face disparities in outcomes and survival. Patient advocacy organizations can play a pivotal role in advancing outcomes for underserved health conditions, such as AYA cancer. In 2018 a group of AYA patient advocates founded AYA Canada (later renamed to “AYA Can—Canadian Cancer Advocacy”), a peer-led national organization aimed at improving the experiences and outcomes of Canadian AYAs affected by cancer. The aim of this article is to describe the development and impact of AYA Can. AYA Can was incorporated as a not-for-profit organization in 2021 and became a registered charity in 2023. Since 2018, AYA Can has established a thriving community of practice comprising nearly 300 patients, healthcare providers, researchers, and charitable organizations with an interest in advocacy for AYA cancer. Other activities have included advocacy at academic conferences and on scientific committees, collaboration with scientists to advance AYA cancer research, training the next generation of AYA patient advocates through a “patient ambassador program,” and developing a national resource hub to centralize knowledge and information on AYA cancer. Through its work to foster collaboration and amplify patient priorities on a national scale, AYA Can has become a leading voice for AYA cancer advocacy in Canada.

Abstract PO2-09-10: Addressing Breast Cancer Disparities: Co-Creating Digital Interventions with Patients, Navigators & the Community to Address Social Determinants of Health in the Memphis Statistical Area

Abstract Background: The Memphis Statistical Area, which covers counties in Arkansas, Mississippi, and Tennessee, leads in disparities in mortality rates between Black breast cancer patients compared to White counterparts1. Urban Health Education & Support Services (UHESS), in partnership with RabbleHealth, and West Cancer Center & Research Institute (WCCRI), initiated a solutions-oriented, community-based scalable initiative in January 2023 to measurably address social determinants of health (SDOH), which often drive cancer inequities that disproportionately impact individuals of marginalized communities. By leveraging a community-driven, co-creation sustainable approach, the team sought to measurably improve breast cancer equality utilizing digital enablement and the knowledge of local breast cancer survivors and leading healthcare professionals, including navigators. Methods: A four-phase approach was leveraged to 1. improve access to locally available breast cancer services in communities that are traditionally underserved and 2. measure the public health impact against root causes of breast inequities (i.e., care pathway metrics), and 3. create a repeatable, scalable model to expand to other underserved communities. In Phase 1, UHESS leveraged the Mid-South Regional Breast Cancer Coalition, which convenes ten local breast cancer patient advocacy organizations, to recruit focus groups for co-creation throughout all phases of the initiative. The working group consisted of eight representatives from a variety of Memphis-based cancer organizations, faith-based organizations, navigator programs, and WCCRI. This phase centered around establishing trust with the local patient advocacy community. In Phase 2, an initial patient survey was circulated to identify key resources that reduce health inequities and barriers to access. Human-centered design facilitators supported a series of Listen & Learn workshops with patients to convey root causes of gaps in access to relevant services and with navigators to understand professional needs of navigators and scale the ability improve patient access to resources. To co-create a solution, facilitated patient and navigator workshops were held in Phase 3 to gather patient-centric user experience and design enhancements for a digital patient engagement app. These requirements as well as navigator-defined metrics needs were digitized and organized by SDoH. Phase 4, the Deploy & Measure phase, will begin in September of 2023 and the community-based deployment model focuses on three drivers including: (a) embedding within navigator workflow, (b) activating faith-based organizations, and (c) leveraging societies. Health equity performance data will be analyzed weekly to rapidly target those ZIP codes with the greatest need. Results: Co-design efforts included over 50 members of the Greater Memphis community and final initiative results will be presented at the meeting. Planning for further research regarding community impact and model scalability is already underway. Conclusion: By leveraging a community-driven, co-creation sustainable approach, this model enables a better way to measurably improve breast cancer equality utilizing digital enablement and the knowledge of local breast cancer survivors and leading healthcare professionals, including navigators. Initiative Funding Acknowledgements: Gilead Life Sciences & Blue Cross Blue Shield of Tennessee Foundation 1. Vidal G, Bursac Z, Miranda-Carboni G, White-Means S, Starlard-Davenport A. Racial disparities in survival outcomes by breast tumor subtype among African American women in Memphis, Tennessee. Cancer Med. 2017 Jul;6(7):1776-1786. doi: 10.1002/cam4.1117. Epub 2017 Jun 14. PMID: 28612435; PMCID: PMC5504313. Citation Format: Gregory Vidal, Stacey Tinianov, Aubrey Kelly, Andrea Curry, Carla Baker. Addressing Breast Cancer Disparities: Co-Creating Digital Interventions with Patients, Navigators & the Community to Address Social Determinants of Health in the Memphis Statistical Area [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO2-09-10.

Abstract PO5-26-07: Surgery for Invasive Lobular Carcinoma: A Patient Experience Survey from the Lobular Breast Cancer Alliance

Abstract The Lobular Breast Cancer Alliance (LBCA) is a nonprofit, patient advocacy organization committed to raising awareness and promoting research into invasive lobular carcinoma (ILC). While ILC accounts for 15% of breast cancers, knowledge about ILC remains limited. ILC’s hallmark is lack of E-cadherin, which results in non-cohesive formation and impacts accuracy of imaging tests and staging. Consequently, patients with ILC have higher rates of positive surgical margins, mastectomies, and axillary lymph node (LN) dissections. To understand the surgery experience of individuals with ILC, LBCA conducted an anonymous online survey from 6/21-7/2/23, distributed via the LBCA newsletter, social media, and partner organizations. Questions addressed pre-operative imaging, type of surgery, surgical margins, LN status, decision making about and patient experience with surgery. Analyses were limited to those respondents who had surgery. Study limitations include potential recall and selection bias. Results 1,482 individuals with a diagnosis of ILC completed the survey and 1,426 had undergone surgery. 76% were from North America, 11% from England, and 4% from Australia. Average age at diagnosis was 56 (range 31-86). At diagnosis, 38% were clinically stage 1; 37% stage 2; 17% stage 3; and 4% de novo metastatic. 75% had a mammogram that indicated dense breasts. Of 1,426 surgery respondents (SR), 95% reported that ILC was identified in only one breast at diagnosis. 80% had a pre-surgical MRI. MRI revealed larger tumors than seen on other imaging for 43% and revealed previously unseen contralateral ILC for 3%. 375 (26%) of SRs were diagnosed with ipsilateral disease and had a pre-surgical MRI that did not reveal new information about their ILC. Of these, post-surgical pathology reports indicated 16% had additional ipsilateral foci, 2% contralateral disease, and 40% had larger tumors than seen on pre-surgical imaging. 614 (43%) of SRs had one or more lumpectomy (L), 560 (39%) of SRs had a double mastectomy (DM), and 451 (32%) of SRs had a single mastectomy (SM). 123 (20%) of SRs who had one or more Ls, had a subsequent SM or DM. For those undergoing L (614), reasons for this choice included: recommendation from their care team (83%), wanted to keep their breast (25%), thought it was better for their overall survival (OS) (14%), and 13% indicated they wanted breasts but did not want reconstruction. Of the 560 women who had a DM, 60% indicated that they thought a DM would improve their OS, 50% were concerned about the risk of local recurrence, 47% doubted that imaging would detect future ILC tumors, and 45% noted concern that ILC is more likely to be bilateral. Of note, only 9% had ILC or ILC and another invasive breast cancer in both breasts at initial diagnosis and 31% who had a pre-surgical MRI changed their initial surgical treatment plan. Post surgery, pathology for DM group revealed larger tumors than seen on imaging (57%), and 6% had tumors found in a second breast that were not seen on imaging. For those 451 who had an SM, reasons for this choice included: 75% said it was what their care team recommended, 35% thought it would improve OS, 20% feared risk for local recurrence, and 15% doubted imaging would detect future ILC. Of 614 respondents who had L, 33% had positive margins at first surgery. Of those, 17% reported multiple Ls and M; and 24% reported clear margins were never achieved. Of the 1011 SM or DMs reported, 10% did not achieve clear margins after these surgeries. Conclusion The survey demonstrates the experience of a large cohort of women with ILC, reporting high rates of uncertainty about accuracy of imaging, high rates of repeat surgery, bilateral mastectomies, and positive margins. Patients are factoring concerns about potential extent of disease and future recurrence in their surgical decision making. These results support the need for improved pre-operative staging and imaging modalities specific to ILC. Citation Format: Laurie Hutcheson, Janice Axelrod, Tracy Cushing, Colleen Fitzwater, Rinath Jeselsohn, Gitte Joergensen, Maria Karsten, Miranda Kimm, Megan Kruse, Theresa Langdon, Julia Levine, Mason Mitchell-Daniels, Rita Mukhtar, Barbara Neilsen, Shani Paluch-Shimon, Therese Pross. Surgery for Invasive Lobular Carcinoma: A Patient Experience Survey from the Lobular Breast Cancer Alliance [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO5-26-07.

Health Care Lobbying and Oncology.

The health care industry spends more on lobbying than any other industry, with more than $700 million spent in 2022. However, health care lobbying related to cancer has not been characterized. In this study, we sought to describe overall health sector lobbying spending and oncology-related lobbying spending across patient and clinician organizations. We obtained lobbying data from OpenSecrets.org and the Federal Election Commission. Overall health sector lobbying spending was categorized by OpenSecrets into 4 groups: pharmaceuticals/health products, health services/health maintenance organizations (HMOs), hospitals/nursing homes, and health professionals. We then identified and categorized 4 oncology-related lobbying groups: oncology physician professional organizations (OPPOs), prospective payment system (PPS)-exempt cancer hospitals, patient advocacy organizations, and provider networks (eg, US Oncology Network). We described temporal trends in lobbying spending from 2014 to 2022, in both overall dollar value (inflation-adjusted 2023 dollars) and in per-physician spending (using American Association of Medical Colleges [AAMC] data for number of hematologists/oncologists) using a Mann-Kendall trend test. Among the overall health sector lobbying, pharmaceuticals/health products had the greatest increase in lobbying spending, with an increase from $294 million in 2014 to >$376 million in 2022 (P=.0006). In contrast, lobbying spending by health professionals did not change, remaining at $96 million (P=.35). Regarding oncology-related lobbying, OPPOs and PPS-exempt cancer hospitals had a significant increase of 170% (P=.016) and 62% (P=.009), respectively. Per-physician spending also demonstrated an increase from $60 to $134 for OPPOs and from $168 to $226 for PPS-exempt cancer hospitals. Overall, OPPO lobbying increased as a percentage of overall physician lobbying from 1.16% in 2014 to 3.76% in 2022. Although overall health sector lobbying has increased, physician/health professional lobbying has remained relatively stable in recent years, spending for lobbying by OPPOs has increased. Continued efforts to understand the utility and value of lobbying in health care and across oncology are needed as the costs of care continue to increase.

Healthy Sleep Practices for Consumers of Home Total Parenteral Nutrition: A Mixed-Methods Community-Based Participatory Study

BackgroundConsumers of overnight home parenteral nutrition (HPN) often experience sleep disruption; however, existing healthy sleep recommendations are widely inapplicable to consumers. ObjectivesThe aim of this mixed-methods, community-based participatory research study was to develop tailored recommendations on healthy sleep practices for HPN consumers. MethodsThe multipart study involved the following: 1) an initial draft of sleep recommendations based on the evaluation of existing general sleep hygiene guidelines by an expert panel of clinicians and consumers with lived experience; 2) semi-structured focus groups with consumers and clinicians; 3) pre- and post-knowledge tests completed by consumers, and 4) final approval of the recommendations by the expert panel. ResultsThe literature synthesis resulted in 51 recommendations evaluated for relevance for HPN consumers. Focus groups with 20 HPN consumers and clinicians contributed additional recommendations based on lived experience. Ultimately, the final resource included recommendations spanning 4 sections: getting ready for bed, preparing the bedroom for sleep, daytime behaviors, and overall strategies for better sleep. Of the 36 recommendations, 58% were derived from existing general sleep hygiene guidelines, and the remaining 42% addressed sleep challenges experienced uniquely by consumers, including nocturnal polyuria, noise/light from medical equipment, and infusion schedules. Knowledge tests completed by 10 additional consumers indicated a modest increase in sleep health knowledge. ConclusionsThe curated healthy sleep resource tailored for HPN consumers was facilitated by a multidisciplinary expert panel, a strategic collaboration with members of the HPN community and their clinicians, and in partnership with patient advocacy and support organizations. The wide distribution of these resources may improve the overall well-being of HPN consumers.

The book is just being written: The enduring journey of parents of children with emerging- ultrarare disorders.

Ultra rare disorders are being diagnosed at an unprecedented rate, due to genomic sequencing. These diagnoses are often a new gene association, for which little is known, and few share the diagnosis. For these diagnoses, we use the term emerging-ultrarare disorder (E-URD), defined as <100 diagnosed individuals. We contacted 20 parents of children diagnosed with an E-URD through the Duke University Research Sequencing Clinic. Seventeen completed semi-structured interviews exploring parental perspectives (7/17 had children in publications describing the phenotype; 4/17 had children in the first publication establishing a new disorder). Data were analyzed using a directed content approach informed by an empowerment framework. Parents reported a range of responses, including benefits of a diagnosis and challenges of facing the unknown, some described feeling lost and confused, while others expressed empowerment. Empowerment characteristics were hope for the future, positive emotions, engagement, and confidence/self-efficacy to connect with similar others, partner with healthcare providers, and seek new knowledge. We identified a subset of parents who proactively engaged researchers, supported research and publications, and created patient advocacy and support organizations to connect with and bolster similarly diagnosed families. Other parents reported challenges of low social support, low tolerance for uncertainty, limited knowledge about their child's disorder, as well as difficulty partnering with HCPs and connecting to an E-URD community. An overarching classification was developed to describe parental actions taken after an E-URD diagnosis: adjusting, managing, and pioneering. These classifications may help genetic counselors identify and facilitate positive steps with parents of a child with an E-URD.

Abstract 808: iCCaRE engagement of faith-based organizations to co-create and co-disseminate infographics addressing disparities in prostate cancer literacy and clinical and biospecimen studies

Abstract Black men hold the highest prostate cancer burden among all ethnicities; thus, there is an urgent need for research to inform the development and implementation of a culturally tailored intervention to reduce disparities and improve outcomes. In response, the Inclusive Cancer Care Research Equity (iCCaRE) Consortium was created to advance health equity and reduce disparities in prostate cancer. Addressing the unique needs of Black men require a genuine bidirectional relationship between scientist and the community. In the Black community, faith-based organizations are respected institutions advocating for health equity and justice. Given their vital role as community health gatekeepers, the iCCaRE Consortium partners with faith-based organizations. Rooted in community engaged research (CER), we conducted an environmental scan through stakeholder meetings to discuss the prevailing challenges and opportunities to meet the needs of the Black community along the prostate cancer care continuum. iCCaRE investigators including Partnership Engagement Services (PES) core partnered with faith-based organizations to ensure and advance community responsive cancer prevention. Together, with our faith-based leaders (N=18), we identified gaps including issues associated with the care continuum from screening to diagnosis and from treatment to survivorship; disparities according to health coverage and social determinants of health, and best practices to assist patients including care coordination, patient navigation, digital innovations, and programs offered by patient advocacy organizations. In response to the needs of the community and the strengths of faith-based organization, we prioritized our initial focus on increasing community prostate cancer literacy and activation using co-design educational infographics for both traditional and technology-based communication platforms. The infographics provided evidence-based facts about prostate cancer burden in the Black community; and using a call to action approach, we emphasized the importance of taking action for life saving screening, prevention and healthy lifestyle. Taking action in prostate cancer symptom recognition, survivorship care, and quality care. Also, co-designed infographics were created for clinical studies and biospecimen awareness and participation and the importance of advocacy and equitable care. Another component incorporated face to face trainings and presentations to enhance their knowledge of cancer screening tools, epidemiology of prostate cancer, and pathways to care. This presentation highlighted the first step towards community based intervention development, implementation and dissemination towards equity and justice for prostate cancer prevention and patient/survivor improvements for Black men. Citation Format: James C. Morrison, Kimlin T. Ashing, Gaole Song, Timethia J. Bonner, Che Ngufor, Getachew Dagne, Arnold Merriweather, John McCall, Ewan Cobran, Cassandra N. Moore, Fornati Bedell, Rotimi Oladapo, Floyd B. Willis, Runcie Chidebe Chidebe, Noreen A. Stephenson, JoAnne S. Oliver, Vinessa Gordon, Folakemi Odedina. iCCaRE engagement of faith-based organizations to co-create and co-disseminate infographics addressing disparities in prostate cancer literacy and clinical and biospecimen studies [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 1 (Regular Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(6_Suppl):Abstract nr 808.

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

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. We included information on all publicly available haematology or oncology FDA workshop agendas held between 1 January 2018 and 31 December 2022. 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. 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. 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.

Prioritizing Patient Experiences in the Management of Diabetes and Its Complications: An Endocrine Society Position Statement.

Diabetes can be an arduous journey both for people with diabetes (PWD) and their caregivers. While the journey of every person with diabetes is unique, common themes emerge in managing this disease. To date, the experiences of PWD have not been fully considered to successfully implement the recommended standards of diabetes care in practice. It is critical for health-care providers (HCPs) to recognize perspectives of PWD to achieve optimal health outcomes. Further, existing tools are available to facilitate patient-centered care but are often underused. This statement summarizes findings from multistakeholder expert roundtable discussions hosted by the Endocrine Society that aimed to identify existing gaps in the management of diabetes and its complications and to identify tools needed to empower HCPs and PWD to address their many challenges. The roundtables included delegates from professional societies, governmental organizations, patient advocacy organizations, and social enterprises committed to making life better for PWD. Each section begins with a clinical scenario that serves as a framework to achieve desired health outcomes and includes a discussion of resources for HCPs to deliver patient-centered care in clinical practice. As diabetes management evolves, achieving this goal will also require the development of new tools to help guide HCPs in supporting PWD, as well as concrete strategies for the efficient uptake of these tools in clinical practice to minimize provider burden. Importantly, coordination among various stakeholders including PWD, HCPs, caregivers, policymakers, and payers is critical at all stages of the patient journey.

Comparison of financial toxicity among patients with non-metastatic versus metastatic renal cell carcinoma.

408 Background: Little is known about the financial impact of renal cell carcinoma (RCC). We sought to ascertain real world data on financial implications and how financial toxicity (FT) impacts quality of life in patients with localized RCC versus patients with metastatic RCC. Methods: An online survey was conducted by the Kidney Cancer Research Alliance (KCCure), a non-profit patient advocacy organization, from 7/22 to 9/22. The survey included questions about costs and financial concerns and the COST questionnaire. Pearson’s correlation (r) and Kendal’s tau test were used to analyze the COST questionnaire, financial burden and hardship. Results: Out of 1062 responders 623 had localized RCC. Out of these patients 395 did not recur and 204 were willing to answer questions related to cost and financial hardship. 289 responders had metastatic disease and were on systemic therapy and 177 pts answered the COST questionnaire. In localized disease 28% reported that their diagnosis has not reduced their income, in 26% the diagnosis had reduced their income very much.31% experienced insurance denials for imaging, 30% faced delays in care due to pre-approval requirements, 45% faced high out-of-pocket costs for medical care. 24% of patients reported taking a hardship withdrawal from a retirement account, 28% stopped funding or lowered contributions to an existing retirement account, 20% borrowed from friends or family. In metastatic patients 14% reported difficulties to pay their premiums. 28% reported that their medical situation has not reduced their income and 26% claimed that their disease reduced their income very much. 36% reported high out of pocket costs are a barrier to care. 44% have received financial support through a manufacturer or a foundation. Median COST score in non-metastatic RCC was 32 (range 19-44)) and was significantly correlated to age, NCCN distress score, risk of recurrence and supplement intake (p &lt; 0.05). Metastatic patients had a significantly higher median COST score of 22 (range 4-36)) that was also correlated to age, NCCN distress score, time since diagnosis and supplement intake (p &lt; 0.05). Conclusions: RCC imposes financial hardship on patients. Younger patients with a higher NCCN distress score and a shorter time since diagnosis are more likely to suffer financial hardships. Patients are willing to pay for supplements. Financial counseling should be considered in these patient subgroups regardless of stage. [Table: see text]

A summit on amplifying voices of patients, caregivers, and people with disabilities in Inflation Reduction Act drug price negotiations.

On September 18, 2023, Cancer Support Community convened patient and caregiver advocates, health care providers, policy experts, and health care innovators and thought leaders for a roundtable discussion on the need to ensure that patients, people with disabilities, and caregivers have a voice in defining "clinical benefit" for the purpose of Medicare Part D drug price negotiations and future health care policies that impact patients. The meeting featured presentations from Lara Strawbridge, Deputy Director for Policy at the Medicare Drug Rebate and Negotiations Group in the Center for Medicare, regulatory expert, Dr Monique Nolan, Counsel at Arnold and Porter, LLP, and 3 panel discussions: IRA Implementation-What Matters to Patients, a discussion of policies expected to impact patients and caregivers who are likely to rely heavily on high-cost drugs or biologics to treat cancer or other chronic illnesses, as well as the future development of novel therapies; The Science of Measuring Patient Experience, a discussion of current science of measuring patient experience and how it should be incorporated into the definition of clinical benefit; and Developing an Infrastructure for External Feedback, a discussion of actions and goals for patient engagement, advocacy opportunities, and how to best coordinate such efforts. This article represents an analysis of relevant resources as well as highlights from these sessions and subsequent discussions. It also outlines principles for engaging patient and provider advocacy organizations, whether in policy, media, or online discussions, surrounding the implementation of the Medicare Drug Price Negotiation Program.

Colorectal Cancer Alliance: Promoting prevention and early detection with screening navigation and a personalized recommendation via a digital screening quiz tool for colorectal cancer.

71 Background: Evaluating the effectiveness of a Colorectal Cancer screening navigation model delivered in a digital platform offered by a patient advocacy organization. Designed as a digital screening quiz tool and live navigation to promote CRC screening, the Colorectal Cancer Alliance’s Screening Quiz is a risk stratification tool that connects respondents to live certified cancer care navigators delivering evidence-based interventions to increase CRC screening, education and patient and family support. The framework utilizes small and mass media, one-on-one education and patient navigation services to help address and reduce barriers to care. Critical interventions which research shows that on-time screening for CRC leads to better outcomes. Methods: The Screening Quiz has been promoted through social sharing, community outreach, media, including influencer campaigns to increase outreach to at-risk communities. Questions assess personal and family history, hereditary syndromes, and demographics to provide a CRC screening recommendation based on current USPSTF guidelines. Respondents overdue for screening who opt in, are triaged to the Alliance’s patient navigation team for follow-up. This interaction includes phone calls and emails for symptomatic individuals or at high risk for CRC, telephonic and email for average-risk uninsured, and emails for average-risk insured. At-risk individuals are connected to resources, such as financial assistance, free at-home screening tests, and colonoscopy scheduling services. Results: July 2022 to August 2023, the Screening Quiz provided over 30,000 screening recommendations. The median age of respondents was 48; 33% of the respondents were between the ages of 45 to 64. Of the respondents, 29% were male and 71% were female. Most were White (74.3%), but nearly half of those who opted into navigation identified as BIPOC, including 14.4% Black/AA, 17.8% Hispanic or Latino, 7.7% Asian, and 2.5% AI/AN. Most respondents were symptomatic (65.5%) but 7.3% were asymptomatic high-risk. These two groups represented 88% of 1,851 respondents triaged to patient navigation. Conclusions: This digital screening tool and live navigation framework may serve as a key solution in driving CRC screening compared to other interventions. Providing access to live navigation, educational resources, and support services for CRC screening and diagnosis.The Alliance is uniquely positioned with its national network of volunteers, partners, and community presence to effectively reach at-risk communities. Aligning with the science of evidence-based interventions, the Alliance will continue to leverage the Screening Quiz in partnership with healthcare and community stakeholders to close Colorectal Cancer screening gaps.

Meaningful Endpoints for Idiopathic Pulmonary Fibrosis (IPF) Clinical Trials: Emphasis on 'Feels, Functions, Survives'. Report of a Collaborative Discussion in a Symposium with Direct Engagement from Representatives of Patients, Investigators, the National Institutes of Health, a Patient Advocacy Organization, and a Regulatory Agency.

Background: Idiopathic pulmonary fibrosis (IPF) carries significant mortality and unpredictable progression, with limited therapeutic options. Designing trials with patient-meaningful endpoints, enhancing the reliability and interpretability of results, and streamlining the regulatory approval process are of critical importance to advancing clinical care in IPF. Methods: A landmark in-person symposium in June 2023 assembled 43 participants from the US and internationally, including patients with IPF, investigators, and regulatory representatives, to discuss the immediate future of IPF clinical trial endpoints. Patient advocates were central to discussions, which evaluated endpoints according to regulatory standards and the FDA's 'feels, functions, survives' criteria. Results: Three themes emerged: 1) consensus on endpoints mirroring the lived experiences of patients with IPF; 2) consideration of replacing forced vital capacity (FVC) as the primary endpoint, potentially by composite endpoints that include 'feels, functions, survives' measures or FVC as components; 3) support for simplified, user-friendly patient-reported outcomes (PROs) as either components of primary composite endpoints or key secondary endpoints, supplemented by functional tests as secondary endpoints and novel biomarkers as supportive measures (FDA Guidance for Industry (Multiple Endpoints in Clinical Trials) available at: https://www.fda.gov/media/162416/download). Conclusions: This report, detailing the proceedings of this pivotal symposium, suggests a potential turning point in designing future IPF clinical trials more attuned to outcomes meaningful to patients, and documents the collective agreement across multidisciplinary stakeholders on the importance of anchoring IPF trial endpoints on real patient experiences-namely, how they feel, function, and survive. There is considerable optimism that clinical care in IPF will progress through trials focused on patient-centric insights, ultimately guiding transformative treatment strategies to enhance patients' quality of life and survival.

The Policy Alliance Between Hospitals and Debt Collection Agencies: Content Analysis of Public Comments on Regulations on Billing and Collections.

Significant debate persists about the obligations of nonprofit hospitals toward low-income patients. Many issues pertaining to this subject were discussed during the rulemaking process following the passage of the Affordable Care Act of 2010, which set forth rules for hospital billing and collection. In public comments, hospitals, debt collectors, and patient advocates debated what constituted "reasonable efforts" to determine whether a patient qualified for hospital financial assistance before resorting to extraordinary collection actions including lawsuits, wage garnishments, and adverse credit reporting. This study analyzes public comments to the proposed Internal Revenue Service rule on section 501(r)(6). After an initial review of the data, 5 commonly mentioned issues were identified. Respondents were organized into commenter types, and the opinion of each respondent to each issue was coded by 2 separate reviewers. Discrepancies between reviewer determinations were resolved by consensus during follow-up discussions. This analysis revealed a set of common concerns: whether reporting delinquent medical debt to credit bureaus and selling debt to third party buyers should be considered extraordinary collection actions; whether hospitals should be able to use presumptive eligibility to rule patients either eligible or ineligible for financial assistance; and whether hospitals should be held legally liable for the actions of third-party debt collectors. Hospitals and debt collection agencies were allied on most issues, particularly in their shared belief that reporting debt to credit bureaus and selling debt to third parties should not be tightly regulated. Patient advocacy organizations and hospitals had divergent opinions on most issues. The alliance of hospitals and debt collectors in advocating for fewer regulations around collections is part of a history of hospital lobbying to maintain tax-exemption with fewer charity care mandates. This alignment helps explain why third-party debt collection agencies, and aggressive collection tactics, have become commonplace in hospital billing.

Cross-cultural assessment of the influence of the COVID-19 pandemic on the perceived mental health and medical experiences of persons with inherited bleeding disorders and their parents/guardians

ABSTRACT Background Objectives were to 1) assess COVID-19-associated medical and psychological challenges facing persons with inherited bleeding disorders (PIBD) and their parents/guardians (PG) in Germany, the US, and the UK; 2) describe similarities and differences among these countries; 3) identify needs and opportunities for intervention by patient advocacy organizations (PAGs). Research Design & Methods A cross-sectional, international survey was conducted in three countries using validated psychometric instruments and investigator-developed items. Results Five hundred and four surveys were included. Significant differences between countries were found including experiences with medical care, specific thoughts, and concerns about COVID-19, anxiety, and other mental health measures, as well as resources used to cope with stress. Age, education, income, race, IBD diagnosis, PIBD vs. BD group, and gender had moderating effects on resources used. Communication with friends/relatives and use of PAG and HTCs as resources for information/coping decreased in all countries during the pandemic. Conclusions There were similarities and differences between respondents across the country in the perceived impact of the pandemic, mental health scores, and strategies used to cope with stress. Recommendations: strategies to increase PAG access for PIBD and their PG during pandemics and natural disasters, ongoing assessment and adaptation to provide supportive resources to specific patient subgroups.

The role of patient advocacy organisations – nothing about us without us

The role of patient advocacy organisations – nothing about us without us

The Value of Collaboration with Patient Advocacy Organizations to Enhance Initiatives Focused on Patient-Centered Care and Improve Outcomes in Chronic Lymphocytic Leukemia

Introduction: Collaborative efforts in medical education that include patient organizations have been shown to be instrumental in enhancing clinical outcomes and exposing professional and patient learners to practice-changing evidence. Here we describe the educational and practical impact of a strategic partnership between PeerView Institute for Medical Education (PeerView) and CLL Society that has led to successful outcomes across six educational initiatives on management of patients with chronic lymphocytic leukemia (CLL) developed for a variety of professional audiences. Methods: Six educational initiatives targeted healthcare professionals, including hematologist-oncologists, oncology nurses, pharmacists, and other relevant professionals, involved in the management of patients with CLL; patients with CLL also engaged in these activities, which included in-person and virtual live satellite symposia, in-person and virtual live regional workshops, and online self-study activities. Influenced by the partner's participation and accessibility of its patient outreach resources, the instructional design of the interventions was chosen for its ability to deliver skills-based education and comprised case-based content, concise scientific lectures, and expert guidance on using CLL Society tools/resources to engage with patients and encourage them to participate in their own care. Designed to meet the needs of the target audience, the learning objectives addressed knowledge of the science supporting the use of targeted therapies, such as BTKi and BCL2i platforms, in the CLL setting, as well as ability to manage safety concerns, personalize treatment plans, and counsel patients. Furthermore, each initiative emphasized the patient voice through the integration of patient testimonials developed in collaboration with CLL Society or sharing of existing partner tools and resources (eg, Expert Access™ program, Patient Education ToolKIT, Test Before Treat™ biomarker educational program). These resources were made available for download to aid in retention and facilitate implementation of the education into practice, helping providers improve their ability to work in a collaborative, patient-centric manner. Outcomes measurements included pre- and post-assessment questions designed to quantify the impact of the educational content on learners' knowledge, skills, and clinical practice intentions. Results of these assessments were compared with baseline audience findings to determine the effect of the education. Results: To date, these activities have actively engaged 16,855 learners.* Pre- to post-participation score changes across all initiatives demonstrated gains of up to 69% in knowledge and 68% in skills and strategies over baseline. These substantial gains were consistent across all initiatives, highlighting the effectiveness of the educational content. Notable improvements in knowledge and skills were seen in treatment selection, identification and management of safety considerations, and ability to counsel patients with CLL. Additionally, these data demonstrated an increase in the participants' ability to incorporate evidence-based protocols and creation of personalized treatment plans into practice. Hundreds of learners, including patients with CLL, have downloaded the provided tools and resources. *Outcomes data as of 7/26/2023. Conclusions: As shown with these outcomes, the initiatives led to significant improvements in healthcare professionals' knowledge, skills, and intentions regarding CLL patient care and enabled participants to achieve their specific learning objectives and make positive behavior changes that can lead to improved patient outcomes. Furthermore, the outcomes from this collaboration between PeerView and CLL Society demonstrated that incorporation of patient perspectives and tactics proven to enhance gains in knowledge and skills into medical education increases the ability of participants to effectively manage patients with CLL. Overall, these data suggest that collaborative educational initiatives with patient support groups enhance healthcare professionals' ability to deliver effective, compassionate, and modern CLL care, resulting in a positive impact on the lives of patients.

Unmet Needs of Immune Thrombotic Thrombocytopenic Purpura (iTTP)-Specific Pregnancy Counseling, Care and Outcomes in Women with Ittp

Unmet needs of iTTP-specific pregnancy counseling, care and outcomes in women with iTTP INTRODUCTION: Pregnancy is a known trigger for an initial episode or relapse of immune thrombotic thrombocytopenic purpura (iTTP) and outcomes of pregnancy in women with iTTP have historically been suboptimal with high rates of recurrent iTTP, fetal loss, preeclampsia and HELLP syndrome. More recent data suggest that prophylactic immunosuppression to normalize ADAMTS13 levels before conception, and ADAMTS13 monitoring and prophylactic steroids or plasma exchange during pregnancy can improve maternal and fetal outcomes. However, it is not clear whether women with iTTP are routinely counseled regarding these options. We conducted this study to understand the experiences of women with iTTP with reproductive healthcare, how iTTP has impacted their reproductive decisions, and outcomes of pregnancy. METHODS: We developed a web-based (RedCap) survey focused on the reproductive healthcare experiences of women with iTTP with in three major sections: (1) counseling and education around pregnancy that was provided to them, (2) the impact of iTTP on their reproductive decisions, and (3) outcomes of any pregnancies they had experienced. The survey was distributed through social media and email mailing lists of a patient advocacy organization and was kept open for one month (January 2023). Survey data were summarized using counts and percentages. The focus of the analysis was to identify gaps in patient education and care where efforts could be focused on educating clinicians or providing information to patients though patient advocacy groups. RESULTS: Seventy-five females with iTTP responded to the survey. The median age of the respondents was 44 years (range 23 - 65). Sixty-five (74.7%) identified as White, 13 (14.9%) as Black, 6 (6.9%) as Asian and 3 (3.4%) identified as other/prefer not to say. Nearly all (94.6%) were followed by a hematologist or hematologist-oncologist and 82.9% reported being monitored regularly for iTTP. Only 26.7% (20 of 75) reported that their doctor talked to them about measures that may help achieve a successful pregnancy. These included ADAMTS13 monitoring (n=15), plasma exchange treatments (n=10), rituximab (n=10), and aspirin (n=3) (options are not mutually exclusive so total is &amp;gt; 20). Moreover, 30.0% (21 of 70) reported that they were advised by their doctor to not get pregnant due to their iTTP. Some patients (36.0%) avoided pregnancy out of fear of the consequences of iTTP and 28.4% (21 of 74) responded that their iTTP diagnosis has affected their ability to get into a relationship, which impacts their quality of life (Figure 1). Fifty-three patients reported that they had had at least one pregnancy. Of these, 35 had pregnancies before iTTP diagnosis, 10 with the initial presentation of iTTP, and 8 women had a total of 11 pregnancies following the diagnosis of iTTP. Consistent with prior reports, there was a high rate of maternal and fetal complications in pregnancies that occurred in the setting of a first iTTP episode complicating pregnancy, or pregnancies after an iTTP diagnosis (Table 1). iTTP relapse occurred in 27.3% (3 of 11) pregnancies in women with prior iTTP. Fetal loss occurred in 30% of pregnancies with first TTP diagnosis and 9% of pregnancies after iTTP. In both groups, approximately 20% were small for gestational age. Preeclampsia or HELLP syndrome occurred in 50% and 18.2% of pregnancies complicated by initial TTP diagnosis or after iTTP diagnosis, respectively. CONCLUSION: Though pregnancy in women with iTTP is high risk, close monitoring and prophylactic interventions can improve outcomes. While many women with iTTP are counseled regarding the risks of pregnancy, few receive counseling regarding their options to potentially improve pregnancy outcomes. This limits their reproductive choices and impacts their relationships. Educating clinicians and providing information to patients though patient advocacy groups may help address these healthcare gaps.

The Lived Experience of Pediatric Gene Therapy: A Scoping Review.

Little is known about patients' and families' lived experiences of participating in pediatric gene therapy (GT) clinical trials. Currently, pediatric GT research targets a broad range of indications--including rare and ultra-rare diseases--which vary in severity and in the availability of alternative therapies. Pediatric GT differs meaningfully from adult GT because the decision to participate involves a dyad of both the child and parent or caregiver/s. It is critical to understand patients' and caregivers' perceptions and experiences of social, emotional, physical, and logistical burdens or benefits of participating in such trials, and how they weigh and prioritize these factors when deciding whether to participate. We conducted a scoping review of the current literature in this subject area with objectives to (1) provide an overview of existing literature, (2) identify gaps and areas for further research, and (3) better understand the lived impact of pediatric GT research on patients and their parents/caregivers. Four themes emerged, including (1) weighing risks and benefits (2) timing of GT trial participation, (3) value of clear communication, and (4) potential impact on quality of life. Notably, our sample surfaced articles about how patients/parents/caregivers were thinking about GT-their understanding of its safety, efficacy, and risks-rather than accounts of their experiences, which was our initial intention. Nevertheless, our findings offer useful insights to improve the informed consent process and promote a more patient- and family-centered approach. Moreover, our findings can contribute to patient advocacy organizations' efforts to develop educational materials tailored to patients' and families' expressed informational needs and perspectives, and can inform more patient- and family-centered policies from GT clinical trial sponsors.

The impact of metastatic breast cancer (mBC) treatment on patient reported productivity loss among patients with HER2+ mBC in the U.S.

352 Background: Little is known about the impact of treatments and other factors on productivity loss among HER2+ mBC patients. This study aimed to assess the work productivity and activity impairment among employed women with HER2+ mBC. Methods: A cross-sectional survey was completed by the members of two patient advocacy organizations in the U.S. Those who were employed for paid work (≥20 hours/week) and receiving systemic treatments for HER2+ mBC for the past 3-months were recruited. Participants self-reported their sociodemographic, clinical and treatment history, time burden related to disease management, and completed the work productivity and activity impairment (WPAI) and Functional Assessment of Cancer Therapy Breast (FACT-B) quality of life (QoL) questionnaires. Patients were grouped based on their current regimen: (1) Oral only, (2) one parenteral drug (1 IV), (3) 2 IVs, (4) 3+ IVs. Association between WPAI scores and sociodemographic and clinical characteristics was assessed using simple linear regression. Results: A total of 118 HER2+ mBC patients participated [mean (SD) age 47.2 (9.5) years, 69.5% were employed full-time and 30.5% part-time, 89% were White, 13.6% resided in rural area]. Among 118 patients, 38 (32.2%) were on 1 IV, 38 (32.2%) on 2 IVs, 31 (26.6%) on 3+ IVs and 11 (9.3%) on oral only regimen. The median travel time (4 hrs vs. 6 hrs) and time spent at healthcare visits (7.3 hrs vs.13.4 hrs) was numerically higher among those on 3+ IVs than those on 1 IV. The median percent work time missed (7.7% vs. 5.8%), overall work impairment (38.2% vs.31.3%) and activity impairment (40.0% vs. 30.0%) in the past 7 days was numerically higher in patients on 3+ IVs than on 1 IV. Working part-time, residing in rural area, having low household income (&lt;25K vs. &gt;75K) and non-commercial insurance was associated with greater overall work impairment (all p-values &lt;0.05). Longer time since mBC diagnosis, longer time on treatment and higher QoL score were associated with lower overall work impairment (all p-values &lt;0.05). Working part-time, low household income, non-commercial insurance, longer time on treatment and higher QoL score were similarly associated with activity impairment (all p-values &lt;005). Conclusions: Socioeconomic and geographical factors were important determinants of work productivity loss among HER2+ mBC patients. IV treatment is the mainstay of therapy for HER2+ mBC patients and being on a simpler regimen for a longer time is likely to reduce work productivity loss and activity impairment. Improving access to novel monotherapy infusion treatment in rural community oncology practices may offer an opportunity to reduce patient time burden and work productivity loss among HER2+ mBC patients and improve health-related quality of life. Additional research from a larger diverse sample is warranted to further understand health disparities in HER2+ mBC patients.