Patient Advocacy Organizations Research Articles

A patient-driven registry on Behçet's disease: the AIDA for patients pilot project.

This paper describes the creation and preliminary results of a patient-driven registry for the collection of patient-reported outcomes (PROs) and patient-reported experiences (PREs) in Behçet's disease (BD). The project was coordinated by the University of Siena and the Italian patient advocacy organization SIMBA (Associazione Italiana Sindrome e Malattia di Behçet), in the context of the AIDA (AutoInflammatory Diseases Alliance) Network programme. Quality of life, fatigue, socioeconomic impact of the disease and therapeutic adherence were selected as core domains to include in the registry. Respondents were reached via SIMBA communication channels in 167 cases (83.5%) and the AIDA Network affiliated clinical centers in 33 cases (16.5%). The median value of the Behçet's Disease Quality of Life (BDQoL) score was 14 (IQR 11, range 0-30), indicating a medium quality of life, and the median Global Fatigue Index (GFI) was 38.7 (IQR 10.9, range 1-50), expressing a significant level of fatigue. The mean Beliefs about Medicines Questionnaire (BMQ) necessity-concern differential was 0.9 ± 1.1 (range - 1.8-4), showing that the registry participants prioritized necessity belief over concerns to a limited extent. As for the socioeconomic impact of BD, in 104 out of 187 cases (55.6%), patients had to pay from their own pocket for medical exams required to reach the diagnosis. The low family socioeconomic status (p < 0.001), the presence of any major organ involvement (p < 0.031), the presence of gastro-intestinal (p < 0.001), neurological (p = 0.012) and musculoskeletal (p = 0.022) symptoms, recurrent fever (p = 0.002), and headache (p < 0.001) were associated to a higher number of accesses to the healthcare system. Multiple linear regression showed that the BDQoL score could significantly predict the global socioeconomic impact of BD (F = 14.519, OR 1.162 [CI 0.557-1.766], p < 0.001). Preliminary results from the AIDA for Patients BD registry were consistent with data available in the literature, confirming that PROs and PREs could be easily provided by the patient remotely to integrate physician-driven registries with complementary and reliable information.

Data-driven strategies for increasing patient diversity in Bristol Myers Squibb-sponsored US oncology clinical trials.

Determining whether clinical trial findings are applicable to diverse, real-world patient populations can be challenging when the full demographic characteristics of enrolled patients are not consistently reported. Here, we present the results of a descriptive analysis of racial and ethnic demographic information for patients in Bristol Myers Squibb (BMS)-sponsored oncology trials in the United States (US) and describe factors associated with increased patient diversity. BMS-sponsored oncology trials conducted at US sites with study enrollment dates between 1 January 2013 and 31 May 2021 were analyzed. Patient race/ethnicity information was self-reported in case report forms. As principal investigators (PIs) did not report their own race/ethnicity, a deep-learning algorithm (ethnicolr) was used to predict PI race/ethnicity. Trial sites were linked to counties to understand the role of county-level demographics. The impact of working with patient advocacy and community-based organizations to increase diversity in prostate cancer trials was analyzed. The magnitude of associations between patient diversity and PI diversity, US county demographics, and recruitment interventions in prostate cancer trials were assessed by bootstrapping. A total of 108 trials for solid tumors were analyzed, including 15,763 patients with race/ethnicity information and 834 unique PIs. Of the 15,763 patients, 13,968 (89%) self-reported as White, 956 (6%) Black, 466 (3%) Asian, and 373 (2%) Hispanic. Among 834 PIs, 607 (73%) were predicted to be White, 17 (2%) Black, 161 (19%) Asian, and 49 (6%) Hispanic. A positive concordance was observed between Hispanic patients and PIs (mean = 5.9%; 95% confidence interval (CI) = 2.4, 8.9), a less positive concordance between Black patients and PIs (mean = 1.0%; 95% CI = -2.7, 5.5), and no concordance between Asian patients and PIs. Geographic analyses showed that more non-White patients enrolled in study sites in counties with higher proportions of non-White residents (e.g. a county population that was 5%-30% Black had 7%-14% more Black patients enrolled in study sites). Following purposeful recruitment efforts in prostate cancer trials, 11% (95% CI = 7.7, 15.3) more Black men enrolled in prostate cancer trials. Most patients in these clinical trials were White. PI diversity, geographic diversity, and recruitment efforts were related to greater patient diversity. This report constitutes an essential step in benchmarking patient diversity in BMS US oncology trials and enables BMS to understand which initiatives may increase patient diversity. While complete reporting of patient characteristics such as race/ethnicity is critical, identifying diversity improvement tactics with the highest impact is essential. Strategies with the greatest concordance to clinical trial patient diversity should be implemented to make meaningful improvements to the diversity of clinical trial populations.

Executive Summary of the Ovarian Cancer Evidence Review Conference.

The Centers for Disease Control and Prevention awarded funding to the American College of Obstetricians and Gynecologists to develop educational materials for clinicians on gynecologic cancers. The American College of Obstetricians and Gynecologists convened a panel of experts in evidence review from the Society for Academic Specialists in General Obstetrics and Gynecology and content experts from the Society of Gynecologic Oncology to review relevant literature, best practices, and existing practice guidelines as a first step toward developing evidence-based educational materials for women's health care clinicians about ovarian cancer. Panel members conducted structured literature reviews, which were then reviewed by other panel members and discussed at a virtual meeting of stakeholder professional and patient advocacy organizations in February 2022. This article is the executive summary of the relevant literature and existing recommendations to guide clinicians in the prevention, early diagnosis, and special considerations of ovarian cancer. Substantive knowledge gaps are noted and summarized to provide guidance for future research.

Patient and Healthcare Professional Perspectives from ESMO 2022 on Bladder and Kidney Cancer: A Podcast.

In this plain language podcast, highlights from the European Society for Medical Oncology (ESMO) Congress are discussed for a second year in a row, from the perspective of both a patient advocate and a healthcare professional. The patient advocacy track at the congress included two patient-focused sessions each day on a variety of topics. Here, the authors discuss the importance of involving patients in the design of clinical trials, as well as strategies to improve dialogue and connections between clinicians, researchers and patients. Patient advocacy organisations provide essential services to patients with cancer and their caregivers, and patient advocates play a critical role in helping to inform patients and caregivers in making clinical decisions. Congresses such as ESMO provide an important platform for patient advocates to connect with each other and with physicians and researchers to ensure that patients are placed at the centre of the conversation and are up to date on the latest findings that affect them. The authors also discuss the latest research on genitourinary cancers, focusing on bladder and kidney cancer. Promising results are emerging for combination antibody-drug conjugates and immunotherapy for patients with hard-to-treat, locally advanced or metastatic bladder cancer who are ineligible for platinum-based chemotherapy. In the management of kidney cancer, we may be reaching an end for immune checkpoint inhibitors on their own; the path ahead will be to find new targets and combinations. Podcast Audio (MP4 169766 KB).

National Coalition for Cancer Survivorship State of Survivorship Survey.

e18684 Background: The National Coalition for Cancer Survivorship (NCCS) conducts an annual State of Survivorship Survey to delve into the cancer survivor journey. The survey is needed to identify health disparities, gaps in quality care and assessment of the health care landscape that needs policy change at the national level. The survey objective is to better understand the differences in cancer survivor attitudes, experiences, and needs at every stage of the cancer journey. The survey identifies who the under-served audiences in the cancer community are, what are their specific challenges, and how the system can better meet their needs. Methods: Phase one was 15 virtual interviews of young adults; Hispanic adults; men. Phase two was a nationwide survey of 2,078 patients and survivors. Surveys were recruited through an online non-probability sample with quotas set to ensure demographically representative audiences. Phase three was a survey invitation to all NCCS email contacts, patient advocacy organizations, followers on social media to take the survey. The NCCS connected group was 670 patients and survivors. Results: We segmented the patients into experiential categories: those who have had positive experiences during their journey, mixed, and negative. Nearly three-quarters of respondents reported they were very satisfied with their treatment and care, due to a high degree of trust in and the relationship with their health care team. However, large disparities exist for those in the Negative Experience group. 68% of respondents overall said they always felt their health care provider listened to and respected their concerns, while only 47% of Hispanic respondents and 47% of individuals with metastatic cancer agreed. Similarly, 68% overall said they could always talk to their health care provider about their concerns, while 46% of Hispanic respondents and 50% of individuals with metastatic cancer agreed. Further, levels of trust were high, but groups with lower levels of trust included young adults, Blacks, Hispanics, people living with metastatic cancer. The Negative Experience group gave providers lower satisfaction scores and used terms like “bullied,” “ignored,” “test subject,” and “rushed” to describe their care. While majorities of patients said their health care team was a critical support network during treatment, followed by family/friends and faith, the Negative Experience and Mixed Experience audiences were significantly less likely to report support in any areas of their life during treatment. Conclusions: The survey is sharing the lived experience of cancer care today and brings to light the health disparities. The results show that part of this disconnect is demographic — people of color, younger, and lower socioeconomic status patients are more likely to be in the Mixed and Negative Experience segments. And part of it is experiential — these patients struggle through the their treatment and care.

A novel patient-centric longitudinal data registry platform to generate insights into real-world cholangiocarcinoma (CCA) clinical practice.

4084 Background: Treatment of advanced CCA has changed dramatically over the past five years with the advent of molecularly targeted therapy, now approved in the 2nd line and beyond. However, real world utilization of molecular profiling and targeted therapy is still unknown and these data are critical to empower treatment decision-making. Methods: In 2019, the Cholangiocarcinoma Foundation (CCF) and Ciitizen (a wholly owned subsidiary of Invitae Corporation) collaboratively launched a registry platform that directly consents patients and collects comprehensive medical records. De-identified data including clinical characteristics, molecular testing, interventions, and outcomes are extracted and standardized for research use. The data is longitudinal with regularly planned updates; registry participants can be re-engaged to obtain additional data and communicate tailored insights. Results: We quantified the rate of molecular testing, the presence of targetable biomarkers, and the utilization and outcomes on matched targeted therapies for 372 individuals with CCA. 328 (88.2%) individuals had molecular testing, with the identified targetable mutations and matched therapies reported. Of 328 individuals who had molecular testing, 111 (33.8%) individuals had one or more targetable mutations (116 mutations total). Only 46% (51) of individuals with targetable mutations received targeted therapy. Of the 54% (60) individuals with targetable mutations that did not receive any matched therapy, the majority (61.7%, 37) were in early disease or first line of treatment and therefore not yet eligible for targeted therapy. These individuals will be informed that based on their molecular profile, a targeted therapy may be an option, and suggest they discuss with their treating physician. Conclusions: A novel prospectively-maintained database registry for CCA has been formed in collaboration between a patient advocacy organization (CCF) and industry (Invitae). Prevalence of actionable biomarkers was higher than historically expected and may reflect patient utilization bias of the registry platform. Molecular profiling and access to targeted therapeutics remains suboptimal at this time in CCA. Future directions for the registry include identifying and targeting disparities in care and supporting biopharmaceutical development and regulatory decisions. [Table: see text]

Black voices on gynecologic cancer: Understanding experiences.

e18680 Background: Structural racism and bias affects care of Black gynecologic cancer patients. Factors associated with disparities in Black cancer death rates have been associated with structural racism which has systematically limited the standard of living (wealth, housing, education, healthy food and healthcare) for Black people. Recent studies point to implicit bias, and lack of cultural sensitivity playing a significant role in the health outcomes of Black cancer patients. For instance, despite NCCN guidelines, oncologists underprescribe chemotherapy for older Black women with ovarian cancer and underdose chemotherapy for Black women with cervical cancer. Moreover, Black women dying from advanced uterine cancer are given pain medication at lower rates &amp; there are disparities in palliative care utilization amongst Black patients with metastatic gynecologic cancer. The voices of Black patient advocates are frequently missing from research studies. By centering the voices of Black patient advocates, we can identify new means of addressing &amp; positively impacting disparities within the clinical care spectrum (from diagnosis to survivorship). Our study group, Black Voices on Gynecologic Cancer: Understanding Experiences, aimed to identify common and unique scenarios of racism and bias in the experiences of Black patients with gynecologic cancer seeking medical attention. Methods: Focus groups were advertised using social media and partner patient advocacy organizations. We conducted five focus groups of 24 Black gynecologic cancer survivors using a Zoom platform. Key themes were identified through individual and group review of transcripts. Results: The themes that emerged from the focus groups included: (1) lack of communication and/or clarity in patient/provider communication - “… my first appointment with my gyn I did…share that…I'm having these hot flashes, night sweats…so he gave me some medicine, but… they´d never really addressed and had a conversation about like this is what your body will be experiencing…So, I really have been dependent on other women that have gone through this.”, (2) insufficient mental health support throughout and after treatment, (3) experiences with misdiagnosis (4) provider’s inadequate management of patient pain, (5) failure to mention clinical trials and (6) difficulty navigating insurance. Conclusions: Our study identified gaps in the care of Black gynecologic cancer patients. The second phase of BVOGUE will reduce structural barriers that prohibit authentic patient and community engagement around gynecologic cancer care and participation in academic research for Black cancer patients. We will pilot modules for both cancer education and research advocacy. This model will serve as a roadmap for all cancer centers creating action plans for engagement, inclusion, and participation.

A Milestone in the Treatment of Ataxias: Approval of Omaveloxolone for Friedreich Ataxia.

The exciting news about the US FDA approval of omaveloxolone as the first-ever drug to be approved for an inherited ataxia is welcome news for patients and families that deal with this devastating disease as well as for health care providers and investigators with an interest in this and other rare diseases. This event is the culmination of long and fruitful collaboration between patients, their families, clinicians, laboratory researchers, patient advocacy organizations, industry, and regulatory agencies. The process has generated intense discussion about outcome measures, biomarkers, trial design, and the nature of approval process for such diseases. It also has brought hope and enthusiasm for increasingly better therapies for genetic diseases in general.

The Importance of Patient-Focused Drug Development in Pemphigus and Pemphigoid

The Importance of Patient-Focused Drug Development in Pemphigus and Pemphigoid

Recommendations for the Pharmacological Treatment of Osteoporosis – Update 2023 of the German Osteoporosis Guideline

Abstract Aim The German osteoporosis guideline was updated on the basis of a systematic literature review and through a structured consensus process by an interdisciplinary, international (Germany, Austria, Switzerland) expert group representing the 20 member societies of the Joint Osteological Association (DVO) and patient advocacy organizations (BfO). This manuscript summarizes the changes in the German osteoporosis guideline with regard to pharmacological interventions and offers pragmatic pathways for implementation. Method Systematic literature search and structured consensus building according to AWMF regulations. Results The sclerostin antibody romosozumab was adopted into the guideline. In addition, the effectiveness of teriparatide was re-evaluated. For both bone anabolic agents evidence from randomized trials underlined a superiority over oral bisphosphonates with regard to fracture risk reduction. Between the antiresorptive treatments, differences regarding superiority/inferiority could only be shown with respect to surrogate parameters, not with regard to fractures. Data about the duration of treatment of all available drugs is limited. All non-bisphosphonate treatments require follow-up treatment. Conclusion An individualized treatment concept should be developed in a shared decision process, including a bone anabolic-first strategy in high-risk patients. Recommendations on treatment duration, drug holidays and sequential strategies should be considered.

A socioecological qualitative analysis of barriers to care in colorectal surgery

BackgroundAlthough specific social determinants of health have been associated with disparities in surgical outcomes, there exists a gap in knowledge regarding the mechanisms of these associations. Gaining perspectives from multiple socioecological levels can help elucidate these mechanisms. Our study aims to identify social determinants of health that act as barriers or facilitators to surgical care among colorectal surgery stakeholders. MethodsWe recruited participants representing 5 socioecological levels: patients (individual); caregivers/surgeons (interpersonal); and leaders in hospitals (organizational), communities (community), and government (policy). Patients participated in focus groups, and the remaining participants underwent individual interviews. Semistructured interview guides were used to explore barriers and facilitators to surgical care at each socioecological level. Transcripts were analyzed by 3 coders in an inductive thematic approach with content analyses. The intercoder agreement was 93%. ResultsSix patient focus groups (total n = 18) and 12 key stakeholder interviews were conducted. The mean age of patients was 54.7 years, 66% were Black, and 61% were female. The most common diseases were colorectal cancer (28%), inflammatory bowel disease (28%), and diverticulitis (22%). Key social determinants of health impacting surgical care emerged at each level: individual (clear communication, mental stress), interpersonal (provider communication and trust, COVID-related visitation restrictions), organizational (multiple forms of contact, quality educational materials, scheduling systems, discrimination), community (community and family support and transportation), and policy (charity care, patient advocacy organizations, insurance coverage). ConclusionKey social determinants of health-impacting care among colorectal surgery patients emerged at each socioecological level and may provide targets for interventions to reduce surgical disparities.

Provisional practice recommendation for the management of myopathy in VCP-associated multisystem proteinopathy.

Valosin-containing protein (VCP)-associated multisystem proteinopathy (MSP) is a rare genetic disorder with abnormalities in the autophagy pathway leading to various combinations of myopathy, bone diseases, and neurodegeneration. Ninety percent of patients with VCP-associated MSP have myopathy, but there is no consensus-based guideline. The goal of this working group was to develop a best practice set of provisional recommendations for VCP myopathy which can be easily implemented across the globe. As an initiative by Cure VCP Disease Inc., a patient advocacy organization, an online survey was initially conducted to identify the practice gaps in VCP myopathy. All prior published literature on VCP myopathy was reviewed to better understand the different aspects of management of VCP myopathy, and several working group sessions were conducted involving international experts to develop this provisional recommendation. VCP myopathy has a heterogeneous clinical phenotype and should be considered in patients with limb-girdle muscular dystrophy phenotype, or any myopathy with an autosomal dominant pattern of inheritance. Genetic testing is the only definitive way to diagnose VCP myopathy, and single-variant testing in the case of a known familial VCP variant, or multi-gene panel sequencing in undifferentiated cases can be considered. Muscle biopsy is important in cases of diagnostic uncertainty or lack of a definitive pathogenic genetic variant since rimmed vacuoles (present in ~40% cases) are considered a hallmark of VCP myopathy. Electrodiagnostic studies and magnetic resonance imaging can also help rule out disease mimics. Standardized management of VCP myopathy will optimize patient care and help future research initiatives.

Emotional experiences of family caregivers of children with Dravet syndrome

BackgroundSince the psychosocial implications of Dravet syndrome (DS) are much more serious and far-reaching than in other types of epilepsy, caring for a DS child seriously affects the entire family. This study describes the emotional experiences of family caregivers of DS children and evaluates the way caregiving affects their perceived quality of life. MethodsAn anonymous, self-administered online questionnaire was sent to family caregivers of DS children through the online patient advocacy organization the Association for People with Severe Refractory Epilepsy DRAVET.PL. It focussed on the psychosocial impact of caregiving for DS children, the perceived burden of caregiving, caregivers’ emotional experiences and feelings related to caregiving, and the impact of DS on the perceived quality of life. ResultsCaregivers stressed that caring for a DS child is associated with a significant psychosocial and emotional burden that affects the entire family. Although most caregivers reported that it was the child’s health problems and behavioral and psychological disorders that were the most challenging aspects of caregiving, they were also burdened by the lack of emotional support. As caregivers were profoundly engaged in caregiving, they experienced a variety of distressing emotions, including feelings of helplessness, anxiety and fear, anticipated grief, depression, and impulsivity. Many caregivers also reported that their children’s disease disrupted their relationships with their spouses, family, and healthy children. As caregivers reported experiencing role overload, physical fatigue, and mental exhaustion, they stressed the extent to which caregiving for DS children impaired their quality of life, their social and professional life, and was a source of financial burden. ConclusionsAs this study identified specific burden domains affecting DS caregivers’ well-being family carers often need special attention, support, and help. To alleviate the humanistic burden of DS carers a bio-psychosocial approach focusing on physical, mental, and psychosocial interventions should include both DS children and their caregivers.

PD08-01 FINANCIAL TOXICITY IN NON-METASTATIC RENAL CELL CARCINOMA PATIENTS

You have accessJournal of UrologyCME1 Apr 2023PD08-01 FINANCIAL TOXICITY IN NON-METASTATIC RENAL CELL CARCINOMA PATIENTS Michael Staehler, Hedyeh Ebrahimi, Severin Rodler, Ulka Vaishampayan, Pavlos Msouel, Mark Ball, Ithaar Derweesh, and Dena Battle Michael StaehlerMichael Staehler More articles by this author , Hedyeh EbrahimiHedyeh Ebrahimi More articles by this author , Severin RodlerSeverin Rodler More articles by this author , Ulka VaishampayanUlka Vaishampayan More articles by this author , Pavlos MsouelPavlos Msouel More articles by this author , Mark BallMark Ball More articles by this author , Ithaar DerweeshIthaar Derweesh More articles by this author , and Dena BattleDena Battle More articles by this author View All Author Informationhttps://doi.org/10.1097/JU.0000000000003239.01AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail Abstract INTRODUCTION AND OBJECTIVE: Little is known about the financial impact of renal cell carcinoma (RCC). We sought to ascertain real world data on financial implications and how FT impacts quality of life in patients following surgery for localized 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 related to the diagnosis of cancer, 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 initially localized RCC. Out of these patients 395 did not recure and 204 were willing to answer questions related to cost and financial hardship. Median age was 54 (range 19-84). Patients identified as white (89%), living in the US (84%) and had a bachelor's degree or beyond (53%). 15%, 59%, and 26% lived in urban, suburban, and rural locations. The approximate annual household income was <50.000US$ in 13%, <100,000US$ in 32% and >200,000US$ in 17%. 65% had private insurance, 13% are on Medicare, 3% on Medicaid and 0.5% were uninsured. 55% of patients work full time, 11% work part time, 4% are unemployed looking for work, 8% unemployed not looking for work, 18% are retired and 4% are on disability. 28% of patients reported that their medical diagnosis has not reduced their income at all and 26% report that their diagnosis has reduced their income very much. 31% of patients reported experiencing insurance denials for imaging scans, 30% faced delays in care due to pre-approval requirements, 45% reported facing high out-of-pocket costs for medical care, 18% reported being unable to go outside of a provider network. 25% reported spending over $100 a month on health-related products not covered by insurance. To cover medical costs 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. Median COST score was 32 (range 19-44)) and was significantly correlated to age, NCCN distress score, risk of recurrence and supplement intake (p<0.05). CONCLUSIONS: Patients diagnosed with localized RCC are impacted by financial hardship and financial toxicity, with more than a quarter of patients reporting that the diagnosis significantly reduced their income. More research is needed to characterize the full extent and root cause of financial implications to this group of patients. Financial counseling should be recommended as part of every cancer diagnosis regardless of stage. Source of Funding: None © 2023 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetails Volume 209Issue Supplement 4April 2023Page: e232 Advertisement Copyright & Permissions© 2023 by American Urological Association Education and Research, Inc.MetricsAuthor Information Michael Staehler More articles by this author Hedyeh Ebrahimi More articles by this author Severin Rodler More articles by this author Ulka Vaishampayan More articles by this author Pavlos Msouel More articles by this author Mark Ball More articles by this author Ithaar Derweesh More articles by this author Dena Battle More articles by this author Expand All Advertisement PDF downloadLoading ...

Interventions to reduce cancer screening inequities: the perspective and role of patients, advocacy groups, and empowerment organizations

BackgroundHealth inequities lead to low rates of cancer screening in certain populations, such as low-income and ethnic minority groups. Different interventions to address this have been developed with mixed results. However, interventions are not always developed in collaboration with the people they target. The aim of our article is to present the viewpoint of patients, survivors, advocates, and lay persons on interventions to increase cancer screening from a health inequity perspective.MethodsWe prepared talking points to guide discussions between coauthors, who included representatives from nine patient and survivor advocacy groups, organizations working for citizen/patient empowerment, and health equity experts. Perspectives and opinions were first collected through video conferencing meetings and a first draft of the paper was prepared. All authors, read through, revised, and discussed the contents to reach an agreement on the final perspectives to be presented.ResultsSeveral themes were identified: it is important to not view screening as a discrete event; barriers underlying an individual’s access and willingness to undergo screening span across a continuum; individually tailored interventions are likely to be more effective than a one-size fits-all approach because they may better accommodate the person’s personal beliefs, knowledge, behaviors, and preferences; targeting people who are unknown to medical services and largely unreachable is a major challenge; including professional patient advocacy groups and relevant lay persons in the cocreation of interventions at all stages of design, implementation, and evaluation is essential along with relevant stakeholders (healthcare professionals, researchers, local government and community organizations etc).ConclusionsInterventions to address cancer screening inequity currently do not adequately solve the issue, especially from the viewpoint of patients, survivors, and lay persons. Several core pathways should be focused on when designing and implementing interventions: advancing individually tailored interventions; digital tools and social media; peer-based approaches; empowerment; addressing policy and system barriers; better design of interventions; and collaboration, including the involvement of patients and patient advocacy organizations.

Burden of Treatments for Respiratory Complications in Extremely Premature Infants: Interviews with Caregivers

Introduction: Extremely premature (EP) infants (<28 weeks gestational age) with respiratory conditions after discharge from the neonatal intensive care unit (NICU) impose a significant burden on caregivers. This study explored caregiver burden post-NICU discharge and perceptions of meaningful change in infant chronic respiratory morbidity. Methods: Adult primary caregivers of EP infants 3–14 months corrected age were recruited through patient advocacy organizations or hospital centers in the USA, Northern Ireland, Germany, and Japan and interviewed by phone. Interviews explored caregiver experiences with infants with respiratory conditions, associated treatment burden, and meaningful change in infant respiratory morbidity as measured by treatment use. Qualitative analysis of interview data was performed using MAXQDA software. Sociodemographic data were summarized using descriptive statistics. Results: Forty-five caregivers (95.6% female) of EP infants were interviewed. Respiratory morbidities post-NICU discharge included coughing (78%), breathing difficulties (76%), wheezing (58%), and bronchopulmonary dysplasia/chronic lung disease of prematurity (56%). Respiratory medications were required by 87% of infants, 80% used home respiratory technology support (e.g., supplemental oxygen), 38% were re-hospitalized, and 33% had emergency department visits. Caregivers considered visits to the emergency department to be the most burdensome treatment requirement they experienced, and reduction in the number of emergency department visits was considered the most meaningful change in treatment use. Conclusion: These findings underscore the significant burden faced by caregivers of EP infants with respiratory morbidities. Development of treatments for respiratory complications should take into consideration the concerns and preferences of caregivers in order to provide a meaningful benefit.

Diversity, equity, and inclusion in clinical trials: A practical guide from the perspective of a trial sponsor

BackgroundConsidering diversity, equity, and inclusion (DEI) in clinical trials ensures that data collected for investigational treatments reflect the populations most likely to benefit from those therapies. Resources and recommendations regarding DEI were assembled by the trial sponsor to assist clinical trial development. MethodsA cross-disciplinary team from the sponsoring organization was assembled to inform trial planning and collate resources that promote DEI throughout the clinical trial life cycle. ResultsRepresentatives from clinical operations, health economic outcomes research, medical affairs, patient advocacy, procurement, and research and development functional groups united together to implement DEI strategies in clinical trials. Planning strategies focus on eligibility, participant/patient engagement, feedback through patient advocacy organizations, and community interactions. Informed site, investigator, and vendor selection at trial startup supports efforts to recruit diverse target trial populations and engage underrepresented businesses; establishing relationships and demographic target-goal tracking should be maintained throughout trial management. Continued communication during trial closeout consolidates learnings and enhances partnerships with trial participants and patient advocacy organizations. The sponsoring organization continuously updates an internal library of resources to facilitate implementation of outlined strategies. ConclusionsThis first iteration of guidance intends to improve the representation of target populations who will ultimately benefit from investigational therapies; to assist sponsor clinical trial teams in developing recruitment and retention plans; and to ensure compliance with federal granting agencies. The sponsoring organization anticipates data from future clinical trials will help characterize the impact of these initiatives to ensure evidence-based practices are used in future clinical trials to enhance DEI.

Accelerating Medicines Partnership® Schizophrenia (AMP® SCZ): developing tools to enable early intervention in the psychosis highrisk state.

Accelerating Medicines Partnership® Schizophrenia (AMP® SCZ): developing tools to enable early intervention in the psychosis highrisk state.

Participant FIRST: Improving Communication With and Support For Participants in Trials that End Early

AbstractBackgroundIn 2019, multiple AD/ADRD trials ended earlier than planned. Participants subsequently stepped forward explaining they first learned their trial ended via news coverage, rather than from a site investigator or research staff. As a result, the issue of participant notification—and the emotional impact of being in a trial that stops early—gained attention. This led to calls to identify better ways to communicate with and support participants after trials end early.MethodThe multidisciplinary Participant Follow‐Up Improvement in Research Studies and Trials (Participant FIRST) Work Group met throughout 2021. Members included research participants and study partners, clinical trialists and study personnel, as well as representatives from academia, industry, patient advocacy organizations, and the National Institutes of Health. The work group’s goals were: (1) improving communication with research participants and study partners when trials end early and (2) identifying supports for those affected by early stopping.ResultsThe Participant FIRST Work Group identified 17 key recommendations. Pre‐trial recommendations focus on developing a patient‐centered communication plan and ensuring that participants and study partners understand early stopping (e.g., role of DSMBs, stopping rules) and securities laws that shape companies’ announcements of trial stopping and, thus, how disclosure occurs. Mid‐trial recommendations focus on maintaining up‐to‐date contact information; helping participants and study partners build support networks; and reinforcing education on early stopping. Post‐trial recommendations focus on rapidly communicating with participants via multiple modalities; scheduling a personalized close‐out meeting to provide key information about the trial and discuss next steps; collaborating with patient advocacy organizations to provide support to participants (e.g., via a patient hotline); and ensuring that participants receive top‐line results. The work group calls for funders and sponsors to ensure adequate time and resources are available to communicate with and support participants when trials end early.ConclusionParticipants and study partners invest time, effort, and hope in their AD/ADRD research participation. The research community should hold itself accountable and take intentional steps towards better means of communicating with and supporting participants when clinical trials end early. The Participant FIRST recommendations are a practical guide for researchers and sponsors embarking on that journey.

Public Health Need, Molecular Targets, and Opportunities for the Accelerated Development of Function-Promoting Therapies: Proceedings of a National Institute on Aging Workshop.

People ≥ 65 years are expected to live a substantial portion of their remaining lives with a limiting physical condition and the numbers of affected individuals will increase substantially due to the growth of the population of older adults worldwide. The age-related loss of muscle mass, strength, and function is associated with an increased risk of physical disabilities, falls, loss of independence, metabolic disorders, and mortality. The development of function-promoting therapies to prevent and treat age-related skeletal muscle functional limitations is a pressing public health problem. On March 20-22, 2022, the National Institute on Aging (NIA) held a workshop entitled "Development of Function-Promoting Therapies: Public Health Need, Molecular Targets, and Drug Development." The workshop covered a variety of topics including advances in muscle biology, novel candidate molecules, findings from randomized trials, and challenges in the design of clinical trials and regulatory approval of function-promoting therapies. Leading academic investigators, representatives from the National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA), professional societies, pharmaceutical industry, and patient advocacy organizations shared research findings and identified research gaps and strategies to advance the development of function-promoting therapies. A diverse audience of 397 national and international professionals attended the conference. Function-promoting therapies to prevent and treat physical disabilities associated with aging and chronic diseases are a public health imperative. Appropriately powered, well-designed clinical trials and synergistic collaboration among academic experts, patients and stakeholders, the NIH and the FDA, and the pharmaceutical industry are needed to accelerate the development of function-promoting therapies.