Table 2.
Self‐reported ability | Physicians directly involved in care of people with hemophilia (%) | Other respondents (%) |
---|---|---|
Ability to explain the science of AAV‐mediated liver‐directed gene therapy for hemophilia to a colleague (n = 177) | ||
1—I’ve never heard of gene therapy | 3 | 3 |
2—I do not have a clear understanding of how gene therapy works | 6 | 6 |
3—I’ve learned about gene therapy, but still don’t think that I could explain it well to someone else | 26 | 41 |
4—I know enough about gene therapy to feel comfortable educating colleagues, patients, and caregivers | 54 | 40 |
5—I consider myself an expert | 11 | 10 |
Ability to answer patient questions about gene therapy for hemophilia based on clinical trial results to date (n = 143) | ||
1—I would not be able to answer questions about clinical trials of gene therapy for hemophilia | 2 | 7 |
2—I have read about some of the studies, but would not feel very comfortable answering questions | 11 | 24 |
3—I could answer a few basic questions about the studies | 27 | 28 |
4—I feel comfortable answering questions about clinical trial results in gene therapy | 50 | 30 |
5—I consider myself an expert | 10 | 11 |
Ability to describe how gene therapy may impact an individual’s current treatment for hemophilia (n = 143) | ||
1—I am not able to describe how gene therapy may impact an individual’s current treatment | 1 | 9 |
2—I have a limited understanding of how gene therapy may impact current treatment practice for patients | 16 | 22 |
3—I am able to explain how gene therapy may impact current treatment practice for patients | 35 | 21 |
4—I am very comfortable with my ability to explain to my patients and colleagues how gene therapy may impact current treatment practice | 38 | 35 |
5—I consider myself an expert | 10 | 13 |
Abbreviation: AAV, adeno‐associated viral.