Community-developed priority areas for future research implementation and facilitation leading up to the Research SOS summit
| Number . | Priority area . | Output of the WG . | Citation . |
|---|---|---|---|
| 1 | Hemophilia A and B | Therapeutics and drug delivery Goals for gene therapy Evolution of PwH-informed care Sex/gender biology pain/comorbidities Enhanced life span data collection Aging Redefining nonsevere phenotypes | 15 |
| 2 | von Willebrand disease, platelet dysfunctions, and other mucocutaneous BDs | Targeted/accessible diagnostics and therapy All disorders All phenotypes Gene therapy Biology of mucocutaneous bleeding Enhanced life span data collection Aging Patient reported outcome measures | 14 |
| 3 | Ultrarare BDs | Targeted/accessible diagnostics and therapy All disorders All phenotypes Gene therapy Biology of rare factor deficiencies Enhanced life span data collection Aging Patient reported outcome measures | 12 |
| 4 | The unique challenges of people with BDs who are women, girls, or have the potential to menstruate | Reproductive bleeding Gene therapy risks in women and girls Noninvasive prenatal diagnosis Sex/gender biology across inherited BDs and phenotypes Hemarthrosis/comorbidities women and girls Inclusion in data collection and clinical trials | 10 |
| 5 | HEDI, health services, and implementation science research | DEI/Access to care Health services research Implementation science Public health Digital health HC delivery networks Access/research in mental health/pain Research/health communications Patient reported outcome measures | 11 |
| 6 | Research facilitation through infrastructure, workforce, and resource development | Research infrastructure optimization Resources, funding procurement, and development Strategies for future workforce development | 13 |
| Number . | Priority area . | Output of the WG . | Citation . |
|---|---|---|---|
| 1 | Hemophilia A and B | Therapeutics and drug delivery Goals for gene therapy Evolution of PwH-informed care Sex/gender biology pain/comorbidities Enhanced life span data collection Aging Redefining nonsevere phenotypes | 15 |
| 2 | von Willebrand disease, platelet dysfunctions, and other mucocutaneous BDs | Targeted/accessible diagnostics and therapy All disorders All phenotypes Gene therapy Biology of mucocutaneous bleeding Enhanced life span data collection Aging Patient reported outcome measures | 14 |
| 3 | Ultrarare BDs | Targeted/accessible diagnostics and therapy All disorders All phenotypes Gene therapy Biology of rare factor deficiencies Enhanced life span data collection Aging Patient reported outcome measures | 12 |
| 4 | The unique challenges of people with BDs who are women, girls, or have the potential to menstruate | Reproductive bleeding Gene therapy risks in women and girls Noninvasive prenatal diagnosis Sex/gender biology across inherited BDs and phenotypes Hemarthrosis/comorbidities women and girls Inclusion in data collection and clinical trials | 10 |
| 5 | HEDI, health services, and implementation science research | DEI/Access to care Health services research Implementation science Public health Digital health HC delivery networks Access/research in mental health/pain Research/health communications Patient reported outcome measures | 11 |
| 6 | Research facilitation through infrastructure, workforce, and resource development | Research infrastructure optimization Resources, funding procurement, and development Strategies for future workforce development | 13 |
DEI, diversity, equity, and inclusion; HC, healthcare; PwH, people with hemophilia.