Rare diseases affect over 300 million people worldwide.1 Whilst individually rare, collectively, these diseases carry a significant human and economic burden. Advanced therapies, including gene therapies, have played a significant role in providing treatment to these rare diseases.
The Sydney Children’s Hospitals Network (SCHN) has been heavily involved in delivering advanced therapies, including gene therapy, as part of clinical trials and clinical care. SCHN was the first site to deliver adeno-associated virus (AAV) gene therapies approved in Australia and was the first lead global recruitment site for clinical trials investigating an AAV gene therapy intervention. SCHN is now a recognised leader in advanced therapies, locally and internationally.2-3 It is important for SCHN to build the capacity and capability of healthcare professionals as more gene therapies are expected to be delivered soon.
Growing knowledge and confidence in delivering AAV gene therapy
The AAV Gene Therapy Education Program is a multidisciplinary education program comprised of a series of online modules and workshops. It is designed to grow the knowledge and confidence of healthcare professionals who may be involved in delivering AAV gene therapy and/or caring for patients who receive AAV gene therapy. It is also available for any healthcare staff who have a general interest in learning about AAV gene therapy. The goals of this project including the following.
- To deliver a structured multidisciplinary education program to health professionals involved in the administration of gene therapy (including nursing staff, medical professionals, allied health and pharmacy) within SCHN.
- To demonstrate an increase in knowledge regarding AAV gene therapy in staff involved in the direct administration of gene replacement therapy within SCHN.
- To create a scalable education program that can be expanded and replicated in other health care settings.
- To represent, understand and address the needs and patient perspectives of families affected by rare genetic diseases in the educational material provided.
Learning objectives include clinical care and ethical considerations
We conducted a needs assessment to identify knowledge gaps amongst healthcare staff who may deliver gene therapy or care for patients receiving gene therapy, including neurology medical officers and nursing staff, junior medical staff, and intensive care physicians. Following the needs assessment, we created an education program consisting of online modules and workshops to address the complexity of AAV gene therapy. We divided the learning objectives into two themes:
- clinical care and safety
- ethical considerations and responsibility.
The clinical care and safety theme focused on the procedures and clinical frameworks needed to ensure safe and effective patient care, including longer term monitoring for adverse effects. The second theme of ethical considerations and responsibility focused on systems readiness, patient selection, consent and communication. The learning objectives for the program are:
- describe the relevance for AAV gene therapy in treating rare diseases
- identify serious adverse events associated with AAV gene therapy
- identify the practical and ethical challenges in the implementation of AAV gene therapy.
Alongside the online modules, we targeted educational workshops to certain departments and medical staff to ensure that we were presenting the training materials to staff who are critical in providing gene therapy treatment and care to our patients. This included neurology medical and nursing staff, junior medical staff, and clinical research staff at our clinical research centres. We directed participants directed to the online modules for further learning and resources. The online modules of the education program has been made available to any healthcare professional and can be accessed here.
Improvements in confidence when providing AAV gene technology to patients
We conducted pre and post-education surveys as part of our evaluation program. A total of 58 staff attended the five face-to-face/hybrid workshops, and at the time of evaluation, there were 40 learners enrolled in the online modules. The evaluation results highlighted that there was a positive change in confidence and knowledge levels regarding AAV gene technology after attending the workshop or engaging in the online modules. Before receiving education, 68% of respondents indicated that they were not at all confident in providing AAV gene technology to patients. Following receiving education, this decreased to 14%.
We evaluated basic knowledge on AAV technology via three quiz questions. Results showed that there was an increase in basic knowledge on AAV technology, with an increase in the number of correct answers for all three questions asked. This project was funded by Pzifer through an independent grant. Pfizer were not involved in any aspect of content creation or delivery.
References
- The Lancet Global Health (2024) ‘The landscape for rare diseases in 2024’, The Lancet Global Health, 12(3). doi:10.1016/s2214-109x(24)00056-1.
- Kariyawasam DS, D’Silva AM, Sampaio H, Briggs N, Herbert K, Wiley V, et al. Newborn screening for spinal muscular atrophy in Australia: A non-randomised cohort study. The Lancet Child & Adolescent Health. 2023;7(3):159–70.
- World-first treatment changes outlook for SMA babies (2022) Kids Research. Available at: http://www.kidsresearch.org.au/news/world-first-treatment-changes-outlook-sma-babies (Accessed: 9 July 2024).
