Community Voices in Research (CVR): A lived experience expert-centred approach to advance the future of inheritable bleeding disorders

Established in 1975, the Nurse’s Health Study stands among the largest prospective investigations into the risk factors for major chronic diseases in women, with over 275,000 participants ^[22]. Motivated by this extensive longitudinal study, NBDF recognised a lost opportunity within the inheritable BD community to better understand LEE experiences, including those during the HIV and hepatitis crisis of the 1970s and 1980s, when at least 50% of LEEs with haemophilia contracted HIV ^[23] and 80% contracted Hepatitis C ^[24]. Consequently, the idea to establish a patient-reported, web-based registry to gather the experiences of PIBD and their families as LEEs was born, enabling timely data collection and reporting of their experiences and concerns.

MyBDC was NBDF’s first effort to comprehensively describe the LEE perspective longitudinally through electronic surveys and place it at the center of research. Initial goals were to transform the lived experience into evidence, identify trends and patterns, and develop LEE-inspired research priorities. This would include input from LEE subgroups who had not previously participated in data collection efforts, e.g. those receiving care outside of HTCs.

The development of MyBDC, which evolved into CVR, included input from key stakeholders and diverse LEEs. Initial stakeholders were identified through NBDF chapters and other advocacy organisations, and then invited to focus groups held during large national meetings. Later, stakeholder groups were built around identifying and meeting certain diversity metrics in order to ensure a wide range of perspectives. These metrics include race, ethnicity, gender, sexual orientation, age, and roles within the community (e.g., LEEs with various diagnoses, HTC clinicians, researchers, NBDF chapter and Hemophilia Federation of America representatives, government and industry partners, payors, and other research entities, such as ATHN).

Since the launch of CVR, enrolment has continued to increase (Figure 5). Data on topics collected (Figure 3) provide information to stimulate ideas for research and answer descriptive or correlational research questions.

Figure 5.

Cumulative enrolment in CVR since 2019

Starting in early 2020 and in partnership with NBDF, external researchers have been able to develop and launch surveys via CVR with specific research questions based on the literature and pre-existing data. Examples include the validation of a Pain PROM and a discrete choice experiment. Table 2 shows publications to date. Surveys can be tailored and sent to participants conforming to specific inclusion and exclusion criteria. Results are provided to external researchers in a deidentified and aggregated manner.

Through the PD, participants continue to have access to pending and completed surveys. They may also view aggregated survey responses in chart format, with the option to custom filter (e.g., by age and diagnosis) to further understand how their answers compare to others like them. NBDF staff regularly develop and curate new content aimed at continuing engagement.

CVR’s ability to swiftly query participants on relevant topics and gauge the community's stance in real time enables a “rapid research cycle”. A five-question Vaping Survey, aiming to understand the impact of lung injury associated with the use of e-cigarettes and launched in August 2019, was the first test of this capability. Upon survey completion, respondents were directed to the CDC website and offered additional vetted education ^[37]. Another example is the ‘COVID-19 Questionnaire’, launched in April 2020, aimed at understanding how the pandemic was initially affecting participants. Based on the results, NBDF developed and launched COVID-19-specific resources to support LEE needs.

As CVR evolved and expanded, it became necessary to change platform vendors to improve its capabilities for both LEEs and researchers. A transition and re-development period in 2023 resulted in a more attractive and intuitive tool, offering enhanced functionality and safety measures.

CVR surveys are voluntary and, generally, uncompensated. Periodically, however, incentives are offered to encourage enrolment and participation in additional research surveys. During the COVID-19 pandemic, recruitment through social media resulted in rapid enrolment, raising concerns about potential bot activity and the creation of false accounts. A complex algorithm based on multiple criteria (e.g., mismatches between enrolment and baseline demographic information, survey completion time impossibly short, patterns of consecutive surveys with systematic numerical sequences, etc.) was developed to identify potential invalid entries. If more than two criteria were met, the statistician reviewed the individual responses, and a clinical expert validated the final determination for inclusion or exclusion from analyses. Entries that did not meet these criteria were permanently deleted. To mitigate the chances of this reoccurring, the CVR team implemented a stricter enrolment process, including the Completely Automated Public Turing Test to Tell Computers and Humans Apart (CAPTCHA), email addresses verification and use of pre-established clinical and demographic criteria to flag or deny enrolment. The team also monitors enrolment trends, addressing suspicious activity quickly, and financial incentives are used judiciously.

CVR is a nimble, versatile, and powerful community tool with the primary aim of incorporating the LEE firsthand experience and perspective into research (Figure 6).

Figure 6.

The power of CVR

During the inception of CVR, NBDF identified an unexplored opportunity within the inheritable BD community to gain deeper insights into the LEE experience. With nearly five years of implementation, CVR is making substantial strides in comprehensively gathering the experiences directly from those most impacted by inheritable BDs and their family members, devoid of filters from clinicians or researchers. The current interaction of CVR facilitates swift responses to societal events that may impact the lives of LEEs by furnishing timely and pertinent data for research purposes.

CVR has enriched researchers' understanding of the nuanced needs of LEEs and their families directly from them, enabling their unique viewpoints to be incorporated into research, education, and advocacy. It ensures that the information collected is relevant and accurately represents participants’ views and experiences. As CVR’s enrolment and vision expansion outpaced the initial platform's capabilities, a new vendor was engaged in 2023 to accommodate growth and analytical needs, paving the way for ongoing adaptation to the evolving research landscape. Collaboration with state NBDF chapters, other non-profits, government entities, and ATHN remains integral to the research process, fostering regular interactions both virtually and in person.

The CVR PD serves as a bridge between clinicians, researchers, and LEEs, facilitating effective information exchange. It provides education and research findings and enables LEEs to link with additional research opportunities. CVR also furnishes a robust cohort for research purposes and integrates participants' input into pre-clinical trial designs, as well as the development of educational and promotional materials. Its infrastructure facilitates rapid research cycles, monitors longitudinal changes, and identifies forthcoming research inquiries pertinent to all stakeholders.

CVR represents a pivotal starting point for researchers to actively engage with LEEs through the VAPs/PAPs programmes. Advisory boards now include LEEs in the discussion of future research proposals, enabling a unique, timely, and personal perspective to research questions and relevant findings. Including LEEs early in the research process as members of the team and utilising CVR data helps identify and pursue community-prioritised research gaps.

CVR has accelerated the movement to engage LEEs at regional and national levels, in rural communities, and outside the HTC network. Strategies to augment LEE engagement with CVR include the development of the personal ‘health summary’ (summarising participants’ health history and current health concerns), and an updated family baseline survey (including self-reported caregiver burden items), and a direct linkage to additional research opportunities beyond CVR surveys and clinicaltrials.gov.

CVR data has enabled assessment of several LEEcentred gaps in research, including pain issues ^[27,31,34], gene therapy preferences ^[28–30,37], perceived social support and mental health ^[31,34], racial and ethnic disparities among PIBD ^[33], and the disability paradox when comparing persons with haemophilia to the general population ^[36]. It has also increased awareness of how timely issues affect LEEs participating in CVR and has resulted in educational initiatives focused on vaping and COVID-19. As more participants enrol and the data matures, the opportunities to identify additional gaps in research will increase further.

The inclusion of LEEs to enrich research has been effectively highlighted through the implementation of CVR. This approach not only offers a mechanism for engagement, but also allows for a deeper insight into their perspectives. It is imperative that LEEs become active partners in every phase of research. Such involvement presents LEEs and researchers with the opportunity to develop more relevant studies and educational initiatives in a timely manner.