Continued from cover
Kidding aside, members of the bleeding disorders community have plenty of thoughts about and hopes for the future. The session provided a forum for panelists and audience members to share ideas of how to achieve those goals.
Valentino said an NHF community survey revealed three key goals for the future: sustainability, leveraging technology and incorporating the voice of the community. Sustainability involves treatment centers, hematologists and being able to sustain the community into the future. Leveraging technology includes not only telehealth, but also digital therapeutics and diagnostics. And incorporating the voice of the community includes diversity, equity and inclusion for both community members and health care professionals.
Panelist Matt Delaney, who has von Willebrand disease and a rare platelet disease, joined NHF’s public policy team as a government relations specialist two months ago. As a member of the under-30 population, Delaney said it’s important to focus on adolescents and children.
“We’ve made so many medical advancements within the last few years that have changed so many lives for the better,” Delaney said.
“But as a result, a lot of young people in particular haven’t had the same experience as older members of the community. They don’t have the same perspective or history.”
Delaney said that lack of historical literacy can make it difficult to engage younger members of the community. “We’re truly standing on the shoulders of giants,” he said, “and it’s important for younger people to be reminded of that.”
Maria Santaella, RN-DC, MSN, CPHON, worked as a nurse in HTC clinics for 17 years and now serves as NHF’s director of research.
“At NHF, I can look at everything from the macro level,” she said, “and I’m starting to see how fragile the HTC model that we currently have is.” To sustain HTCs into the future, Santaella believes people both inside and outside the clinics need to be involved.
Michael Recht, MD, PhD, chief science officer for the American Thrombosis and Hemostasis Network, said researchers know that in order to make advances in the bleeding disorders community, they have to keep answering questions.
“But the right questions are not always the questions that are most interesting to me as a researcher,” he said. To discover the right questions, researchers need to ask community members questions about what will impact them and their families, he said.
Recht also noted that funding for research into bleeding disorders needs to be diversified. “Advances in hemophilia research over the past 10 years have been amazing. Now, we’ve got to do that for all of the bleeding disorders,” he said.
Heather Dean, vice president and U.S. hematology head at Takeda, outlined three key priorities for the future.
The first is paying very close attention to understanding the journey of underrepresented populations. That involves real-world data from underrepresented communities, including involving them in clinical trials. “We’re changing the way we recruit and communicate availability to all patients for trials,” she said.
Another hot topic is women who have bleeding disorders. “There’s so much more awareness about acknowledging this really important group of patients,” Dean said.
And the third priority is access to care. “Patients have such unique and nuance-specific needs, and it’s incumbent on all of us to make sure they have access to those therapies,” she said.
Peter Chen, PhD, head of U.S. medical for Sanofi, also discussed clinical trials involving underserved populations, and emphasized the importance of researchers listening to patients.
“What I hope patients realize is how much they can influence clinical trial design and therapies,” he said. “Surveys still show a strong dissatisfaction and unmet needs with their disease state. We’re trying to bring patients to as near-normal life as possible, and that wouldn’t be the case if it weren’t for the patient voice.”
Mary-Lacey Reuther, head of U.S. healthcare policy and external affairs at CSL Behring, said the health care financing environment will be tougher in 2030 for the rare disease community, which has pretty much been inoculated from that type of pressure until now.
“There are a lot of voices in this space, and the community is such advocates,” she said “Continuing to do that, and doing even more, will be more important in the future.”
Reuther said it will also be important for the bleeding disorders community to continue collaborating with a cross section of other patient organizations. ■