How Much Evidence Is Enough? Research Sponsor Experiences Seeking Regulatory Acceptance of Digital Health Technology-Derived Endpoints.

Introduction: Digital health technologies (DHTs) provide opportunities for real-time data collection and assessment of patient function. However, use of DHT-derived endpoints in clinical trials to support medical product labelling claims is limited. Methods: From November 2020 through March 2021, the Clinical Trials Transformation Initiative (CTTI) conducted a qualitative descriptive study using semi-structured interviews with sponsors of clinical trials that used DHT-derived endpoints. We aimed to learn about their experiences, including their interactions with regulators and the challenges they encountered. Using applied thematic analysis, we identified barriers to and recommendations for using DHT-derived endpoints in pivotal trials. Results: Sponsors identified five key challenges to incorporating DHT-derived endpoints in clinical trials. These included (1) a need for additional regulatory clarity specific to DHT-derived endpoints, (2) the official clinical outcome assessment qualification process being impractical for the biopharmaceutical industry, (3) a lack of comparator clinical endpoints, (4) a lack of validated DHTs and algorithms for concepts of interest, and (5) a lack of operational support from DHT vendors. Discussion/Conclusion: CTTI shared the interview findings with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and during a multi-stakeholder expert meeting. Based on these discussions, we provide several new and revised tools to aid sponsors in using DHT-derived endpoints in pivotal trials to support labelling claims.

Transforming patient engagement in clinical trials: Moving from a transactional relationship to human-centered care.

Engagement with patients during the clinical trial process encompasses all of the interactions that those running clinical trials have with the participants. The essence of patient engagement is to ensure patients feel supported, connected and empowered before, during and after the clinical trial process. There has been significant progress in the past few years and, while there is still a long way to go, 92% of clinical trial executives believe that clinical trials are now more patient-centric than ever.

From Individuals to Systems and Contributions to Creations: Novel Framework for Mapping the Efforts of Individuals by Convening The Center of Health and Health Care.

BACKGROUND: People with lived health care experiences (often referred to as "patients") are increasingly contributing to health care and are most effective when they are involved as partners who can contribute complementary knowledge alongside other stakeholders in health care. OBJECTIVE: Convening The Center aimed to bring together "people known as patients"-the center of health care-to address priorities as they defined them. METHODS: According to the original project design, an in-person gathering was to be conducted; however, as a result of the COVID-19 pandemic, the in-person gathering was transformed into a series of digital gatherings, including an in-depth interview phase, small-group gatherings, and a collective convening of 25 participants (22 women and 3 men from the United States, India, Costa Rica, Sweden, and Pakistan). Each participant was interviewed on Zoom (Zoom Video Communications Inc), and the interview data were thematically analyzed to design a subsequent small group and then full cohort Zoom sessions. Visual note-taking was used to reinforce a shared understanding of each individual- and group-level conversation. RESULTS: The interviews and gatherings for Convening The Center offered unique perspectives on patient activities in research, health innovation, and problem-solving. This project further developed a novel, two-spectrum framework for assessing different experiences that patients may have or seek to gain, based on what patients actually do, and different levels of patients' involvement, ranging from individual to community to systemic involvement. CONCLUSIONS: The descriptors of patients in academic literature typically focus on what health care providers think patients "are" rather than on what patients "do." The primary result of this project is a framework for mapping what patients "do" and "where" they do their work along two spectra: from creating their own projects to contributing to work initiated by others and from working at levels ranging from individual to community to systems. A better understanding of these spectra may enable researchers to more effectively engage and leverage patient expertise in health care research and innovation.

A History of #BCSM and Insights for Patient-Centered Online Interaction and Engagement.

PURPOSE: Participation in cancer support groups can provide a sense of community and may better prepare patients for interactions with their health care team. Online interactions may overcome some barriers to in-person support group participation. #BCSM (breast cancer social media), the first cancer support community established on Twitter, was founded in 2011 by two breast cancer survivors. The aims of this study are to describe the growth and changes in this community and to discuss future directions and lessons that may apply to other online support communities. METHODS: Symplur Signals was used to obtain all #BCSM Twitter data from January 1, 2011, to January 1, 2020 (00:00:00 Coordinated Universal Time for both). Hashtag use by selected stakeholder groups, user locations, weekly tweet chat activity, and topics were determined. RESULTS: From year 1 (2011) to year 9 (2019), tweets using the #BCSM hashtag increased by 424%. Tweets by patient advocates increased by 226%, with a peak in 2016. Impressions, a measure of potential tweet views, by patient advocates increased by 517%. Tweets by doctors and nonphysician health care professionals increased by 693%. Weekly #BCSM tweet chat activity peaked in 2013, increasing by 58.1% from 2011. Chat topics have included survivorship, metastatic breast cancer, death and dying, advocacy, and highlights from national breast cancer meetings. CONCLUSIONS: #BCSM has experienced tremendous growth since 2011, although there are challenges to community sustainability. The weekly chats, as well as discussions utilizing the hashtag but occurring outside of scheduled chat times, serve as an important resource for patients and offer physicians an opportunity to both support and learn from patients.

Organizing Online Health Content: Developing Hashtag Collections for Healthier Internet-Based People and Communities.

Twitter use has increased among patients with cancer, advocates, and oncology professionals. Hashtags, a form of metadata, can be used to share content, organize health information, and create virtual communities of interest. Cancer-specific hashtags modeled on a breast cancer community, #bcsm, led to the development of a structured set of hashtags called the cancer tag ontology. In this article, we review how these hashtags have worked with the aim of describing our experience from 2011 to 2017. We discuss useful guidelines for the development and maintenance of health-oriented communities on Twitter, including possible challenges to community sustainability and opportunities for future improvement and research.

Transforming research: engaging patient advocates at all stages of cancer research.

In recent years there has been a push to include patients and advocates in clinical research, including in clinical trial design and approval processes. Additionally, increased advocate-driven research funding has led to seats for advocates in the funding review process. However, despite advances that include having patients and advocates involved in clinical research projects that may directly, or eventually, affect their care and treatment, we have much further to go to make this a truly inclusionary participation in research. We have even further to go to actively incorporate patients and advocates throughout the research continuum. Here we review and present current programs and projects that have successfully incorporated patients and advocates into translational research, and propose mechanism and strategies to actively engage patients throughout the research continuum. Our goal in translational cancer research is to improve outcomes for cancer patients. To more successfully and quickly meet this objective we should incorporate patient driven research questions earlier in the process, specifically at the level of basic benchtop research.

Patient Participation at Health Care Conferences: Engaged Patients Increase Information Flow, Expand Propagation, and Deepen Engagement in the Conversation of Tweets Compared to Physicians or Researchers.

BACKGROUND: Health care conferences present a unique opportunity to network, spark innovation, and disseminate novel information to a large audience, but the dissemination of information typically stays within very specific networks. Social network analysis can be adopted to understand the flow of information between virtual social communities and the role of patients within the network. OBJECTIVE: The purpose of this study is to examine the impact engaged patients bring to health care conference social media information flow and how they expand dissemination and distribution of tweets compared to other health care conference stakeholders such as physicians and researchers. METHODS: From January 2014 through December 2016, 7,644,549 tweets were analyzed from 1672 health care conferences with at least 1000 tweets who had registered in Symplur's Health Care Hashtag Project from 2014 to 2016. The tweet content was analyzed to create a list of the top 100 influencers by mention from each conference, who were then subsequently categorized by stakeholder group. Multivariate linear regression models were created using stepwise function building to identify factors explaining variability as predictor variables for the model in which conference tweets were taken as the dependent variable. RESULTS: Inclusion of engaged patients in health care conference social media was low compared to that of physicians and has not significantly changed over the last 3 years. When engaged patient voices are included in health care conferences, they greatly increase information flow as measured by total tweet volume (beta=301.6) compared to physicians (beta=137.3, P<.001), expand propagation of information tweeted during a conference as measured by social media impressions created (beta=1,700,000) compared to physicians (beta=270,000, P<.001), and deepen engagement in the tweet conversation as measured by replies to their tweets (beta=24.4) compared to physicians (beta=5.5, P<.001). Social network analysis of hubs and authorities revealed that patients had statistically significant higher hub scores (mean 8.26×10-4, SD 2.96×10-4) compared to other stakeholder groups' Twitter accounts (mean 7.19×10-4, SD 3.81×10-4; t273.84=4.302, P<.001). CONCLUSIONS: Although engaged patients are powerful accelerators of information flow, expanders of tweet propagation, and greatly deepen engagement in conversation of tweets on social media of health care conferences compared to physicians, they represent only 1.4% of the stakeholder mix of the top 100 influencers in the conversation. Health care conferences that fail to engage patients in their proceedings may risk limiting their engagement with the public, disseminating scientific information to a narrow community and slowing flow of information across social media channels.

What Do Patients Prefer? Understanding Patient Perspectives on Receiving a New Breast Cancer Diagnosis.

BACKGROUND AND OBJECTIVE: There is variability in physician practice regarding delivery method and timeliness of test results to cancer patients. Our aim was to survey patients to determine if there was a difference between actual and preferred care for disclosure of test results. METHODS: A de-identified survey was distributed to online cancer support groups to query patients about their experience regarding communication of cancer testing and timeliness. Analyses of the differences between actual and preferred communication and wait times were performed. RESULTS: Overall, 1000 patients completed the survey. The analysis herein was restricted to 784 breast cancer survivors. Survey responders were predominately White (non-Hispanic; 89 %), college educated (78 %), and media 'savvy' (online medical media usage; 97 %). Differences between actual and preferred care were identified for the domains of mode of communication and wait times for initial breast cancer diagnostic biopsies and other tests. A total of 309 (39 %) of 784 patients received face-to-face communication for a new cancer diagnosis, with 394 (50 %) patients preferring this option (p < 0.0001). In addition, 315 (40 %) of 784 patients received their cancer biopsy result within 2 days, with 646 (82 %) patients preferring this option (p < 0.0001). Differences were also identified between actual and preferred care for multiple other test types. CONCLUSIONS: Actual care for timeliness and modes of communication did not reflect patient-desired care. National and local initiatives to improve performance are needed. As a first step, we recommend that each patient be queried about their preference for mode of communication and timeliness, and efforts made to comply.

Twitter Social Media is an Effective Tool for Breast Cancer Patient Education and Support: Patient-Reported Outcomes by Survey.

BACKGROUND: Despite reported benefits, many women do not attend breast cancer support groups. Abundant online resources for support exist, but information regarding the effectiveness of participation is lacking. We report the results of a Twitter breast cancer support community participant survey. OBJECTIVE: The aim was to determine the effectiveness of social media as a tool for breast cancer patient education and decreasing anxiety. METHODS: The Breast Cancer Social Media Twitter support community (#BCSM) began in July 2011. Institutional review board approval with a waiver of informed consent was obtained for a deidentified survey that was posted for 2 weeks on Twitter and on the #BCSM blog and Facebook page. RESULTS: There were 206 respondents to the survey. In all, 92.7% (191/206) were female. Respondents reported increased knowledge about breast cancer in the following domains: overall knowledge (80.9%, 153/189), survivorship (85.7%, 162/189), metastatic breast cancer (79.4%, 150/189), cancer types and biology (70.9%, 134/189), clinical trials and research (66.1%, 125/189), treatment options (55.6%, 105/189), breast imaging (56.6%, 107/189), genetic testing and risk assessment (53.9%, 102/189), and radiotherapy (43.4%, 82/189). Participation led 31.2% (59/189) to seek a second opinion or bring additional information to the attention of their treatment team and 71.9% (136/189) reported plans to increase their outreach and advocacy efforts as a result of participation. Levels of reported anxiety before and after participation were analyzed: 29 of 43 (67%) patients who initially reported "high or extreme" anxiety reported "low or no" anxiety after participation (P<.001). Also, no patients initially reporting low or no anxiety before participation reported an increase to high or extreme anxiety after participation. CONCLUSIONS: This study demonstrates that breast cancer patients' perceived knowledge increases and their anxiety decreases by participation in a Twitter social media support group.

Toolbox to Reduce Lumpectomy Reoperations and Improve Cosmetic Outcome in Breast Cancer Patients: The American Society of Breast Surgeons Consensus Conference.

BACKGROUND: Multiple recent reports have documented significant variability of reoperation rates after initial lumpectomy for breast cancer. To address this issue, a multidisciplinary consensus conference was convened during the American Society of Breast Surgeons 2015 annual meeting. METHODS: The conference mission statement was to "reduce the national reoperation rate in patients undergoing breast conserving surgery for cancer, without increasing mastectomy rates or adversely affecting cosmetic outcome, thereby improving value of care." The goal was to develop a toolbox of recommendations to reduce the variability of reoperation rates and improve cosmetic outcomes. Conference participants included providers from multiple disciplines involved with breast cancer care, as well as a patient representative. Updated systematic reviews of the literature and invited presentations were sent to participants in advance. After topic presentations, voting occurred for choice of tools, level of evidence, and strength of recommendation. RESULTS: The following tools were recommended with varied levels of evidence and strength of recommendation: compliance with the SSO-ASTRO Margin Guideline; needle biopsy for diagnosis before surgical excision of breast cancer; full-field digital diagnostic mammography with ultrasound as needed; use of oncoplastic techniques; image-guided lesion localization; specimen imaging for nonpalpable cancers; use of specialized techniques for intraoperative management, including excisional cavity shave biopsies and intraoperative pathology assessment; formal pre- and postoperative planning strategies; and patient-reported outcome measurement. CONCLUSIONS: A practical approach to performance improvement was used by the American Society of Breast Surgeons to create a toolbox of options to reduce lumpectomy reoperations and improve cosmetic outcomes.