It was just before 4 p.m. when Hemophilia Federation of America (HFA) President and CEO Sharon Meyers, EdD, CFRE, turned on her webcam and tested her microphone in the organization’s office in Washington, DC. More than 1,500 miles away in her home state of Colorado, HFA Vice President of Policy and Advocacy Sonji Wilkes did the same. The two were comparing notes in the presenter studio of Zoom Webinar and were excited to kick off Dateline Live: Navigating Approved Products and Emerging Therapies. With the push of a button, HFA begin what would be 11 hours of webinars focused on educating patients about treatment options for people with a bleeding disorder.
Each year HFA compiles a comprehensive list of therapies approved by the U.S. Food and Drug Administration as well as all investigational therapeutic products undergoing clinical trial and publishes that list in a special issue of its quarterly magazine, Dateline Federation. Dateline Live took the success and value of the print issue and brought it to life over three days of online education programming. More than 200 community members registered for the event, which took place June 23-25, 2021.Â
While the event was a new concept that the HFA team thought of in early 2021, there was unanimous consent among staff on the decision to act quickly to host it. “As patients, we have the right to know our options. But it is our responsibility to educate and empower ourselves about our disorder,” Wilkes said. “From our community’s history we know that patients and their families look to advocacy organizations such as HFA to provide the facts and information needed. HFA remains committed to being a trusted source of information for you. We are steadfast in our commitment to ensure a safe blood supply and transparency around treatment options and their efficacy.”
Eric Iglewski, LMSW, moderated a session titled “Get Counted: The Importance of Registries,” in which a panel of community leaders discussed how registries are a critical way for researchers to monitor safety signals and other trends, learn from the patient experience and predict future needs or concerns. The panel was comprised of HFA Director of Public Health and Outcomes Projects Mabel Crescioni, DrPH, JD, LLM; National Hemophilia Foundation’s head of research, Michelle Witkop, DNP; and Plasma Protein Therapeutics Association’s senior vice president of public affairs, Julie Birkofer.
Iglewski also moderated “Shared Decision-Making: Making the Most of the Patient-Provider Relationship.” Longtime advocates and active members of the community Will Hubbert and Cazandra MacDonald joined the panel to provide patient perspectives. The provider perspective was brought by Chris Guelcher, a hematology/oncology clinic nurse at Children’s National in Washington, DC, and Robert Sidonio, MD, associate professor of pediatrics at Emory University School of Medicine, clinical director of the hemophilia treatment center (HTC) and associate director of hemostasis and thrombosis, Department of Pediatrics, Children’s Healthcare of Atlanta.
“What to Expect When Participating in Clinical Trials” was presented by Julie Smith, CCRP, who was able to provide the perspective of an HTC clinical research manager. Smith co-manages a team of six clinical research coordinators at the University of Colorado Denver Hemophilia and Thrombosis Center.
David Clark, a retired chemical engineer with more than 35 years of experience in the biotechnology, plasma and tissue industries, hosted the “Emerging Therapies: The Latest in Clinical Trials” session. Patients learned about the ever-changing landscape of bleeding disorders treatment, basics of clinical trial progression and items of discussion for patients/caregivers to consider with their health care provider when thinking about participation in a trial.
Acronyms abound in the bleeding disorders world-how do you make sense of FVIII, FIX, vWF, QoL, HCV, MASAC, ISTH, EHC, AAV and the plethora of others? To help community members learn what these letters mean and represent, HFA’s director of policy and principal legal counsel, Miriam Goldstein, JD, along with Wilkes, hosted an interactive session called “Bleeding Disorders Alphabet Soup: What Do These Letters Mean?”
In addition to the educational webinars presented by HFA, representatives from four pharmaceutical manufacturers-CSL Behring, Genentech, HEMA Biologics and Sanofi Genzyme-hosted sessions that provided community members with updates and information about available treatment options. In keeping with their mission of connecting patients, HFA also hosted rap sessions, led by an HTC nurse, that were open to community members and caregivers as an opportunity to meet with other community members and discuss their experiences. Rap sessions were grouped by diagnosis of hemophilia A or B, von Willebrand disease, inhibitor or other rare bleeding disorder.
When asked why this event was made a priority for the HFA team, Wilkes said, “It was 17 years ago when I was choosing a treatment option for my newborn son with severe hemophilia A. The overwhelming feeling remains vivid. It was a confusing and scary process, despite having wonderful support from the staff at our local hemophilia treatment center. I was a mom, unsure of how to even spell hemophilia at that point, just wanting to make the right call for my baby’s health. But, I didn’t know what questions to ask, what specifics to consider or even what our options were.”
Recordings of each session and biographies about each speaker are available on HFA’s website at www.hemophiliafed.org/dateline-live. Subscriptions to the quarterly print magazine are free; sign up today at www.hemophiliafed.org. To request a copy of the 2021 Product Guide & Emerging Therapies Special Issue, please send your name and address to firstname.lastname@example.org.