Better Bleeding Disorders Care Begins with You

When it comes to chronic and life-changing medical conditions, better care starts with better research. Until a few years ago, clinical investigators drove the direction of bleeding disorders research. The wants and needs of those most affected by inherited bleeding disorders were never strategically harnessed to determine research goals.

That’s about to change with the National Hemophilia Foundation’s (NHF) National Research Blueprint. Since 2020, this initiative has aimed to shape the future of bleeding disorders research, putting people with inherited bleeding disorders, as well as their families and caregivers, front and center.

NHF’s efforts to prioritize the voices of those most affected by bleeding disorders within a formal research planning framework began in 2018, when NHF members attended a National Heart, Lung, and Blood Institute workshop on inhibitor research. One of the main recommendations from the event was patient inclusion in prioritizing research.

To improve the lives of people with inherited bleeding disorders, NHF and its nationwide chapters have long relied on community input. Spurred by the leadership of President and CEO Leonard A. Valentino, M.D., NHF realized it had built-in relationships and know-how to take the recommendation from the NHLBI workshop a step further.

“Imagine the impact we can create by focusing our collective expertise, resources, and energy toward areas that can create the most dynamic impact for people with bleeding disorders and their families. We could change lives for the better and create a lasting generational impact,” Valentino says.

He adds: “No one individual or organization can own this challenge. As the largest national organization dedicated to improving lives for people with inherited bleeding disorders, NHF is embracing our role, and responsibility, to champion this effort and serve as the conduit through which the research and patient community can come together to create a national blueprint of patient-centered research with quantifiable outcomes.”

Spearheading what the future of research should look like was a logical and important step for NHF, says Michelle Witkop, DNP, FNP-BC, who served as NHF’s vice president of research strategy until she retired in 2022. Her successor, Maria Santaella, has had a leadership role in the National Research Blueprint (NRB) since the beginning and now oversees the initiative.

Listening to what patients want not only makes for better community relations, it also makes for better research, Witkop says. “Patient-centered, coordinated research has fewer Institutional Review Board amendments and has better recruitment. It also finishes the research phases and is assimilated into practice more quickly,” she says.

The Start of Something Different

From the beginning, NHF prioritized listening to all community voices. Since summer 2020, NHF has conducted community working groups and listening sessions to capture patient, caregiver, and family input.

In early 2021, NHF launched a cross-community survey, developed by and administered through NHF’s chapters and Hemophilia Federation of America member organizations. As part of the survey, 335 participants — including 125 people with bleeding disorders and 112 health care professionals — identified three major research priorities. At the top of the list was continuing the hemophilia treatment center model of care. Survey participants also prioritized more research on new therapies across bleeding disorders and research on improving access to care, including differences in care in various communities. NHF also gathered input from participants in Community Voices in Research, a community-powered registry supported by NHF.

Input from the listening initiatives formed the basis for NHF’s inaugural State of the Science (SOS) Research Summit, held in September 2021 to help design and implement the NRB framework.

The four-day virtual summit was organized by SOS steering and advisory committees. Donna M. DiMichele, M.D., the former deputy director of the Division of Blood Diseases and Resources at NHLBI, spearheaded the 2018 NHLBI workshop and has served as a consultant on the NRB’s leadership team since its launch in 2020.

Through a contract with the Center for Information and Study on Clinical Research Participation, NHF convened several months of virtual listening sessions with diverse working groups representing adults with bleeding disorders, caregivers, patient organizations, chapter and member organization directors, health care providers, and industry representatives to develop research recommendations.

In line with NHF’s commitment to the principles of health equity, diversity, and inclusion (HEDI), the virtual summit allowed for greater participation nationwide. Of the 887 attendees, nearly 40 were in remote areas, representing underserved and minority populations.

The central question posed to them: Where can we make the greatest impact? Topics included research priorities for hemophilia A and B and other, ultra-rare inherited bleeding disorders, as well as science around the health of women, girls, and those with the potential to menstruate. Participants also highlighted the need to prioritize patient-centricity and HEDI principles in health services research.

The community input initiatives pinpointed several areas that warranted further research, including mental health, joint disease, treating the aging population, and pain management.

Making Your Voices Heard

Sammie Valadez, of Peru, Illinois, was one of 10 patients recommended by their local NHF chapters to serve as subject matter experts during the virtual working group sessions. Valadez brings eight years of personal and family experience with bleeding disorders to the discussion. She and her two daughters have von Willebrand disease. Her oldest daughter also has factor VII deficiency.

A name change for the patient subject matter expert working group was the first order of business. “We were getting confused with researchers, since they are subject matter experts in their field,” Valadez says. The group decided that “lived experience experts,” or LEEs, better reflects who they are, says Valadez, who serves as the LEE working group co-chair. The term now refers to people with inherited bleeding disorders, their family members, and
their caregivers.

Before she started serving on the NRB group, Valadez says, she didn’t think community members’ voices were heard when it came to research. For the past three years, LEEs have met twice a month to discuss community input and impact on developing the NRB.

The development of the NRB offers hope and empowerment to people directly affected by bleeding disorders, Valadez says. “Most patients have no idea how they can be part of the research and what comes from research, so bringing us into the research from the beginning is wonderful,” she says.

The NRB doesn’t just benefit people with bleeding disorders and their families. “The focus of LEE-centered research means clinical researchers have a new partner with unique insight into what are the most impactful questions we should be answering,” says NHF Chief Medical and Scientific Officer Michael Recht, M.D., Ph.D., co-chair of the NRB Steering Committee alongside Valentino.

“The innovative aspect of the NRB process is including and valuing the LEE voice. It’s the first time where research questions and clinical trials will be centered around the issues brought to the forefront by LEEs,” Recht says.

He and other clinical researchers say the patient-centered approach to research is long overdue. “As I reflect back, I have come to realize the questions I was asking and attempting to answer were the questions interesting to me,” Recht says. “I had never enlisted my patients or families to help me formulate the questions I was trying to answer.”

For LEEs like Valadez, having a seat at the decision-making table is a welcome change, but not everyone embraced the idea initially. “At first, they were hesitant about their place, afraid of speaking up,” Witkop says. But with Santaella’s guidance and encouragement, Witkop says, many more LEEs now embrace their role in determining research goals.

“The goals of the NRB and the pivotal role being played by LEEs in realizing these goals are helping to bring unique solutions to overcoming the knowledge gaps and experiential barriers to active LEE participation in advancing their own standard of care through research engagement, regardless of type of bleeding disorder,” DiMichele says.

An important aspect of the NRB is that it is grounded in the principles of equitable and inclusive access to care and research. “As the LEEs now say, ‘Nothing about us without us!’ And that is as it should have been and should always be, now and in the future,” DiMichele says. “Although this has been a perennial goal for this community, we have never been so close to achieving it in such a strategic and inclusive way.”

For the NRB to succeed, community members must continue to make their voices heard, Witkop says. “They know where the gaps are and what needs to happen to make things move forward,” she says. “By being involved, making themselves knowledgeable, and becoming part of the team, they create change and energy. With the knowledge of the researchers, that synergy creates the spark of innovation. We can all be part of it, but not without each other.”

Stay Up to Date: Find more details about the National Research Blueprint here.