This issue features an in-depth look at the National Hemophilia Foundation (NHF)-McMaster Guideline on Care Models in Hemophilia (See “Well-grounded Guideline”), published in the journal Haemophilia and presented at NHF’s 68th Annual Meeting in July in Orlando, Florida.
This guideline is a major achievement for NHF. It furthers its goal to find better treatments and cures for bleeding disorders through education, advocacy and research support. The Guideline on Care Models acknowledges that the healthcare landscape in the US is changing rapidly, as is the natural course of hemophilia. Now that people with hemophilia are living longer and living better, it is essential that evidence-based clinical practice guidelines are created to support patient-centered decision-making. These guidelines help ensure patients with hemophilia receive the right care, delivered in the right way.
The Guideline on Care Models is the first published clinical practice guideline in any field of medicine conceived of and sponsored by a patient organization. It also is unique in a few other respects.
All clinical practice guidelines must be built on a solid foundation of evidence, which can be a challenge in rare diseases like hemophilia, where there are relatively few published scientific papers. So the team of health researchers from McMaster University developed new methods to gather and develop evidence to support the guideline. These new methods, described in supporting papers in Haemophilia, can be used to bolster guideline development in other rare diseases.
The panel that made the final recommendations is also distinctive. Very often, panels consist exclusively of physicians. These physicians may have tremendous experience treating the target disease, but they may not be able to speak to all of the aspects of clinical care that are important to patients. By contrast, NHF and McMaster University aimed for both breadth and depth of expertise in assembling the panel. Panelists included not only physicians but also people with hemophilia, parents of people with hemophilia, individuals with other rare diseases, payers, researchers, nurses, physical therapists and genetic counselors. This group offered a host of different viewpoints, all of which were considered in developing the guideline.
The purpose of the Guideline on Care Models is to identify best practices in hemophilia care, and to discuss the range of care providers and services that are most important for people with hemophilia across the US. The hope of the panel, and the NHF-McMaster development team, is that the guideline will be an important resource for anyone affected by or providing care for this disease.
Menaka Pai, BSc, MSc, MD, FRCPC, is an associate professor in the Department of Medicine and an associate member of the Department of Pathology and Molecular Medicine at McMaster University in Hamilton, Canada. She was a member of the guideline panel.