A national assessment of standards of care for inherited bleeding disorders in Canada.

AIM: The Canadian Integrated and Comprehensive Care Standards for Inherited Bleeding Disorders were adopted in June 2020. In early 2023, a self-assessment of each of the 26 Canadian inherited bleeding disorder treatment centre's (IBDTCs) capacity to meet the Standards was conducted. The goal was to validate the standards by assessing appropriateness and adherence. As a result, centres can compare their own practices and capacity against those of all centres, identify barriers to adherence, identify opportunities for remedial actions and use the results locally as evidence for needed resources. METHODS: Healthcare providers (HCPs) in the 26 IBDTCs were provided with a checklist to assess adherence to each of the 66 standards of care. Centre participation was voluntary but strongly encouraged by the healthcare provider and patient associations. RESULTS: All 26 centres completed the self-assessments. Collectively, centres reported meeting 88.8% of the standards. Adherence to each standard ranged from 40% to 100%. Forty-one (41) of the standards were adhered to by 90% or more of the centres, 12 by 80%-89% of the centres and 13 by fewer than 80% of the centres. A report consolidating all the assessments was sent to the 26 centres. CONCLUSION: None of the comments received in the self-assessment reports indicated that a given standard was irrelevant, unrealistic or unnecessary. These data are strong indicators that the standards, as written, are appropriate. The self-assessments, however, reveal alarming deficiencies in staffing levels, notably in physiotherapy, psychosocial support and data entry and data management. These constitute a barrier to comprehensive care for many centres. The findings echo similar conclusions from a previous assessment conducted in 2015.

Achieving the unimaginable: Health equity in haemophilia.

Historically, treatment based on the availability of clotting factor replacement has resulted in an arcane guideline for the correction of factor deficiencies in people with haemophilia (PwH). While all other disease entities seek to restore function to a normal level, PwH are restricted to factor nadirs still equivalent to mild or moderate disease, resulting in continued risk of bleeding. A new treatment paradigm is needed based on the defined needs of PwH. A treatment model was developed by a panel of haemophilia providers, patient advocates and health economists to establish specific treatment milestones and targeted outcomes. The panel defined a series of treatment milestones to characterize the activity and outcomes linked to level of factor deficiency correction. All agreed that the ultimate goal should be 'functional cure' and 'health equity'. Seven levels to achieving a functional cure were identified, (a) Sustain life; (b) Minimal joint impairment; (c) Freedom from any spontaneous bleeds; (d) Attainment of 'normal' mobility; (e) Able to sustain minor trauma without additional intervention; (f) Ability to sustain major surgery or trauma; and (g) Normal haemostasis. A parallel set of patient-reported outcomes to achieve health equity was identified. These guidelines are now comparable with other disorders where the goal is to replace missing proteins to attain normal activity levels. As we are no longer limited by plasma supply due to the manufacture of recombinant factors, mimetics, and the early success of gene therapy, health equity is now achievable.