TY - JOUR
T1 - Survey of Health Care Provider Understanding of Gene Therapy Research for Inherited Metabolic Disorders
AU - Hansen, Joyanna
AU - Ramachandran, Radha
AU - Vockley, Jerry
N1 - Publisher Copyright:
© 2022 The Authors
PY - 2022/8
Y1 - 2022/8
N2 - Purpose: The treatment of inherited metabolic disorders (IMDs) has traditionally relied on dietary interventions that are difficult to maintain, expensive, and socially isolating. The development of gene therapy for IMDs aims to provide sufficient gene activity to address the underlying causes of these conditions. This study surveyed health care providers (HCPs) to characterize their familiarity with gene therapy technologies and to identify educational needs across roles in a multidisciplinary care team. Methods: The link to a Web-based, 26-question survey was distributed to HCPs in North America and Europe who were involved in IMD patient care. Results were analyzed using descriptive statistics. Findings: Of the 590 survey link recipients, 64 completed the survey. Of these, 35 (55%) respondents were physicians, 23 (36%) were dietitians, 3 (5%) were nurse practitioners, 2 (3%) were genetic counselors, and 1 (2%) was a nurse. Most survey respondents (88% [n = 56 of 64]) reported the belief that gene therapy for IMDs would be available within 5 years of study conduct. Although nearly all physicians (97% [n = 34 of 35]) expressed awareness of gene therapy, rates of reported familiarity were lower among dietitians (57% [n = 13 of 23]); confidence in conversations with colleagues and patients/caregivers was also discordant. Nearly all HCPs wanted education on gene therapy advancements, and the most preferred informational sources were published literature and congress presentations. Implications: There is an urgent need for education on topics related to gene therapy modalities. Professional education on gene therapies is desired across all specialties and will be important for unified treatment practices in IMD care.
AB - Purpose: The treatment of inherited metabolic disorders (IMDs) has traditionally relied on dietary interventions that are difficult to maintain, expensive, and socially isolating. The development of gene therapy for IMDs aims to provide sufficient gene activity to address the underlying causes of these conditions. This study surveyed health care providers (HCPs) to characterize their familiarity with gene therapy technologies and to identify educational needs across roles in a multidisciplinary care team. Methods: The link to a Web-based, 26-question survey was distributed to HCPs in North America and Europe who were involved in IMD patient care. Results were analyzed using descriptive statistics. Findings: Of the 590 survey link recipients, 64 completed the survey. Of these, 35 (55%) respondents were physicians, 23 (36%) were dietitians, 3 (5%) were nurse practitioners, 2 (3%) were genetic counselors, and 1 (2%) was a nurse. Most survey respondents (88% [n = 56 of 64]) reported the belief that gene therapy for IMDs would be available within 5 years of study conduct. Although nearly all physicians (97% [n = 34 of 35]) expressed awareness of gene therapy, rates of reported familiarity were lower among dietitians (57% [n = 13 of 23]); confidence in conversations with colleagues and patients/caregivers was also discordant. Nearly all HCPs wanted education on gene therapy advancements, and the most preferred informational sources were published literature and congress presentations. Implications: There is an urgent need for education on topics related to gene therapy modalities. Professional education on gene therapies is desired across all specialties and will be important for unified treatment practices in IMD care.
KW - gene therapy
KW - gene transfer
KW - inborn errors of metabolism
KW - inherited metabolic disorders
KW - phenylketonuria
KW - survey
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U2 - 10.1016/j.clinthera.2022.07.002
DO - 10.1016/j.clinthera.2022.07.002
M3 - Article
C2 - 35927093
AN - SCOPUS:85135347773
SN - 0149-2918
VL - 44
SP - 1045
EP - 1056
JO - Clinical therapeutics
JF - Clinical therapeutics
IS - 8
ER -