TABLE 2.

Three types of community‐prioritised inherited BD research themes

Category	Prioritised themes
Themes applicable across inherited BDs	Research that is feasible, prioritised for impact on patient outcomes Research to benefit future generations addressing the most pressing needs of the inherited BD community Impact of inherited BD symptoms/manifestations across the lifespan Therapies for non‐haemophilia inherited BDs that are safe/effective/easy to use/affordable/covered by insurance; and for RBDs, disease‐specific; POC management Pain management in PWIBD Mental health in PWIBD Health disparities across under‐represented populations (ethnic, racial, gender [including LGBTQ], and geographic) Future workforce development and evolution of the HTC model to ensure/optimise future care of PWIBD
Priorities specific to individual inherited BDs	Rare and ultra‐rare inherited BDs: Disease‐specific diagnostics and therapeutics, including recombinant replacement concentrates Impact of inherited BD symptoms/manifestations across the lifespan Women with inherited BDs: Novel therapeutics for VWD Impact of inherited BD symptoms/manifestations across the lifespan Joint disease in women Haemophilia carriers Haemophilia: Continuing therapeutic advance (efficacy/safety/ease of administration) toward normal QoL Defining ‘cure’ expectations with gene therapy Moderate/mild haemophilia research (non‐severe)
Infrastructure and capacity opportunities	Elements of national research infrastructure/capacitation/acculturation facilitating and optimising work on a prioritised research blueprint through: National patient‐centred data collection Hypothesis‐driven and feasible basic research/observational studies/clinical and implementation trials Future workforce nourishing an expansive national research enterprise through: Inclusion of an entire, well‐trained inherited BD provider and patient community Regional, national, and international collaborations Multidisciplinary team science drawing expertise from within and outside the inherited BD scientific community Strategic partnerships leveraging existing infrastructure and common goals NHF role in realising the National Blueprint for Inherited Bleeding Disorders Research

Abbreviations: BD, bleeding disorder; HTC, haemophilia treatment centre; LGBTQ, lesbian, gay, bisexual, transgender, queer; NHF, National Hemophilia Foundation; POC, point of care; PWIBD, people with an inherited bleeding disorder; QoL, quality of life; RBD, rare bleeding disorder; VWD, von Willebrand disease.