As people with bleeding disorders know, access to quality healthcare is essential to living well. But there hasn’t been a widely accepted scientific resource to guide how that care should be delivered—until now. The National Hemophilia Foundation (NHF), in partnership with McMaster University in Hamilton, Ontario, Canada, has published a landmark evidence-based clinical practice guideline on care models for hemophilia management. Based on rigorous and innovative study methods, the guideline concludes that an integrated care model is ideal for people with hemophilia and other bleeding disorders.
Integrated care means all the services needed to effectively manage a health condition are provided by a multidisciplinary team like the staff at hemophilia treatment centers (HTCs). This may seem obvious to people with bleeding disorders who already rely on the comprehensive care provided at their HTCs. However, spelling out the conclusion in a clinical practice guideline is essential to supporting the goal for consistent, quality care for every person with a bleeding disorder.
“Patients should be going to facilities that deliver integrated care, and specialists who are delivering care should be providing it in an integrated fashion,” says Mark Skinner, JD, a consultant who served on the panel to create the guideline. He has severe hemophilia A. “Having care delivered through an integrated care model makes a difference to patients. This data support that.”
What does it mean for you?
The guideline is significant for a few reasons. First, it helps establish best practices. Second, it provides consumers solid backing when they go to bat for themselves or family members. This is particularly important when obtaining care outside an HTC. Common scenarios include emergencies and being cared for by community physicians between visits to an HTC. The guideline is a credible resource for care providers who don’t treat bleeding disorders every day.
“The guideline is a tool for patients to use with insurers, employers and policy makers when advocating for access to specialized, high-quality care,” says Ellen Riker, senior vice president at CRD Associates in Washington, DC, and a federal policy adviser to NHF. She served on the guideline panel for NHF. “We influence the environment that we live in. Evidence is often the best way to do it.”
From perception to evidence
NHF has a long history of advocating for the needs of people with bleeding disorders. However, spearheading the development of an evidence-based clinical guideline was a new frontier. That’s why the foundation partnered with McMaster University, which is known for its work in hematology and evidence-based medicine. Further, NHF provided the funding to support the creation of a multidisciplinary panel to do the work over a three-year period. Although many topics for the first guideline were discussed, the panel decided that care delivery was the place to start.
For many patients and medical professionals, it’s common sense that integrated care provides better outcomes. Indeed, when everyone on the healthcare team works together coordinating care and sharing results, there’s more consistency and attention to detail. Before creating the guideline, the data to back up this perception were sorely lacking.
One primary reason for this previous lack of evidence is the rarity of bleeding disorders. There simply aren’t enough people to participate in the kind of large-scale studies usually conducted to make evidence-based recommendations. “Traditionally, you take a look at all of the published evidence and then you synthesize it. So we had to get creative here,” says Menaka Pai, BSc, MSc, MD, FRCPC, one of the panelists. She is an associate professor in the Department of Medicine and an associate member of the Department of Pathology and Molecular Medicine at McMaster University. She is also the transfusion medicine quality lead and consultant laboratory hematologist at the Hamilton Regional Laboratory Medicine Program.
Identifying the ways in which delivering care for hemophilia is similar to delivering care for other chronic diseases was one creative solution to gathering evidence. For example, ample data show that integrated care improves outcomes for people with diabetes. From that, the panel drew some indirect conclusions about how the care delivery model would apply to people with hemophilia.
A further innovative approach was the use of structured interviews with patients, caregivers and healthcare providers, asking about their experiences with care. In addition, the panel contacted experts in the field, seeking unpublished data from smaller studies or personal observations. This method helped the panel tap into what Pai calls “hidden pockets of knowledge” in the community.
Uncommon approach yields unique results
Another notable aspect of the panel was that it was made up of a cross section of the bleeding disorders community. “The fact that patients were sitting with the researchers and the clinicians, the payers and methodologists—all evaluating the evidence on equal footing—means there was a consensus among everyone involved, particularly patients,” Skinner says.
In addition, this guideline wasn’t led by a medical society, which is the case with most guidelines. “This is the first evidence-based guideline of its kind developed by an advocacy organization,” says Marla Feinstein, NHF public policy analyst. She respresented NHF on the committee. “It really encompasses what we do as an organization, helping the community and all stakeholders understand access to care.”
Grounded in rigorous research
To make sure the document would be widely adopted in practice, the panel followed accepted principles for developing transparent, evidence-based guidelines. These principles are promoted by the Institute of Medicine, the National Guideline Clearinghouse and the Grading of Recommendations Assessment, Development and Evaluation (GRADE) working group. This last group sets parameters on how guidelines should be created. “The result is a guideline that is extremely rigorous and that the bleeding disorders community can really put their faith into,” Pai says.
The use of exacting methodology is one reason the guideline was endorsed by three highly respected organizations: the American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH) and the World Federation of Hemophilia (WFH). “ASH agreed that the final guideline is both methodologically rigorous and aligns with our values as an organization,” says Nathan Connell, MD, MPH, a member of the Guideline Oversight Subcommittee and the Committee on Quality at ASH. He is a hematologist at Brigham and Women’s Hospital in Boston, and a Harvard Medical School faculty member.
What’s more, the guideline validates the care model HTCs already use. “ASH members were excited to have the opportunity to participate in the guideline development process—to review and ultimately endorse it—because we feel there is value and benefit for the patient,” Connell says. “Ultimately, what we’re here to do is improve the care for the patients.”
Actionable improvements
Although HTCs already use an integrated care model, the way it’s applied is inconsistent. “There are practice pattern variations around the country,” Skinner says.
The panel carefully analyzed the evidence to come up with recommendations, including suggestions on how to implement them, with a goal of “harmonizing” care delivery across the US, says Pai. The ultimate objective was to create guidelines that improve treatment practices and patients’ health, she adds.
“Some guidelines are scientifically sound but don’t move the needle of care,” Pai says. “This partnership, where you have an academic institution coming together with a highly active patient organization like NHF, helps us focus on that end goal of making a difference in the lives of people affected by the disease.”
What’s next
This first guideline is only the beginning. NHF plans to conduct ongoing reviews to identify ways to improve the evidence base and make the guideline even more actionable for the bleeding disorders community. Additionally, the guideline will hopefully inspire HTCs and specialists to unify their data collection efforts, adds Pai. This may fuel additional research into the treatment of bleeding disorders.
“NHF is the standard-bearer in the rare disease community by saying, ‘Yes, this is a rare disease, but we can still produce good guidelines and put out strong calls for research,’” Pai says. “Just because hemophilia and other bleeding disorders are considered rare does not mean that patients must settle for anything less than the best care.’”