title: Methodology for the development of the NHF-McMaster Guideline on Care Models for Haemophilia Management.
abstract: BACKGROUND
  Rigorous and transparent methods are necessary to develop clinically relevant and evidence-based practice guidelines. We describe the development of the National Hemophilia Foundation-McMaster Guideline on Care Models for Haemophilia Management, which addresses best practices in haemophilia care delivery.
  
  
  METHODS
  We assembled a Panel of persons with haemophilia (PWH), parents of PWH, clinical experts and guideline methodologists. Conflicts of interest were disclosed and managed throughout. Panel members and key stakeholders were surveyed to develop the guideline questions and identify patient-important outcomes. Systematic reviews of the literature were conducted for all factors important in decision-making: benefits and harms; patient values and preferences; resource implications; acceptability; equity; and feasibility. We used the GRADE approach to create evidence profiles to evaluate the evidence and present key results. Evidence to Decision frameworks were created to guide the Panel in making evidence-based recommendations. When evidence was very low quality or not available, evidence from other chronic disease populations was presented to the Panel to inform the recommendations. Additionally, we systematically pooled observations from experts, and conducted qualitative interviews exploring key stakeholder experiences and perspectives. The Panel made recommendations for each guideline question and elaborated on research priorities, implementation considerations, and monitoring. Final recommendations were circulated for public and peer review.
  
  
  CONCLUSIONS
  Despite the paucity of high-quality evidence typical of a rare condition such as haemophilia, we successfully applied a rigorous and transparent methodology based on GRADE to develop an evidence-based clinical practice guideline.
authors.affiliation: Department of Medicine, McMaster University, Hamilton, ON, Canada.|Department of Pathology and Molecular Medicine, McMaster University, Hamilton, ON, Canada.|McMaster Centre for Transfusion Research, McMaster University, Hamilton, ON, Canada.
authors.forename: M
authors.initials: M
authors.lastname: Pai
authors.affiliation: Department of Clinical Epidemiology and Biostatistics, McMaster University, Hamilton, ON, Canada.
authors.forename: N
authors.initials: N
authors.lastname: Santesso
authors.affiliation: Department of Clinical Epidemiology and Biostatistics, McMaster University, Hamilton, ON, Canada.
authors.forename: C H T
authors.initials: CH
authors.lastname: Yeung
authors.affiliation: McMaster Centre for Transfusion Research, McMaster University, Hamilton, ON, Canada.
authors.forename: S J
authors.initials: SJ
authors.lastname: Lane
authors.affiliation: Department of Clinical Epidemiology and Biostatistics, McMaster University, Hamilton, ON, Canada.
authors.forename: H J
authors.initials: HJ
authors.lastname: Schünemann
authors.affiliation: Department of Medicine, McMaster University, Hamilton, ON, Canada.|Department of Clinical Epidemiology and Biostatistics, McMaster University, Hamilton, ON, Canada.
authors.forename: A
authors.initials: A
authors.lastname: Iorio
mesh_terms: D003695:Delivery of Health Care / Q000379:methods* / Q000592:standards; D019468:Disease Management; D019317:Evidence-Based Medicine; D017408:Guidelines as Topic; D006467:Hemophilia A / Q000628:therapy*; D006801:Humans
keywords: care model; delivery of health care; guideline; haemophilia; integrated care; rare disease