Currently, the first expected approval for gene therapy for hemophilia (GTH) is not expected for another year. Much time is likely needed prior to approval for Hemophilia Treatment Centers (HTC’s) to implement changes. We encourage HTC teams who are preparing to integrate GTH into patient care to use this change guide to ensure HTC readiness. Included below are key topics and relevant questions that need to be addressed to prepare for and integrate GTH.
Change domains included in this guide are:
1. Patient eligibility
2. Referral to centers offering treatment
3. Patient preparation
4. Treatment delivery
5. Post-administration management
6. Providing patient support
7. Care coordination
8. Assessing laboratory measurements of coagulation when using novel agents
Once you’ve explored the questions and the current status within your HTC, work as a team to develop a specific action plan to implement change.
To facilitate the process of integrating GTH, this guide has sections dedicated to each of these high level topics, with a set of relevant questions that need to be addressed to ensure HTC readiness.
Step 1: Identify a change domain
Step 2: Discuss questions, identifying any additional information your team may need to answer the questions for your HTC and its patients
Step 3: Determine necessary changes to integrate GTH
Step 4: Outline concrete steps and timeline needed to implement changes
Step 5: Identify existing systems and resources that can be leveraged to facilitate successful transition
Step 6: Determine potential barriers to change and how the team might address these barriers
Step 2: Discuss questions, identifying any additional information you or your team may need to answer the questions from your HTC and its patients
Patient eligibility:
Identifying Patients Eligible and Appropriate for New Therapies
Referral to centers offering treatment:
Referring Patients to Treatment Centers Where GTH is Available (If Treatment Will Not be Available in Local HTC)
Patient Preparation:
Preparing Patients for Gene Therapy
Treatment delivery:
Administration of Gene Therapy
Post-administration management:
Post-administration Care
Long-term monitoring (safety and efficacy)
Providing Patient Support
Care coordination:
Coordinating Care Among HTC Care Team Members
Assessing Laboratory Measurements of Coagulation when using Novel Agents
Integrating Research Questions Into Clinical Practice
Step 3: Determine necessary changes to integrate new therapy
Step 4: Outline concrete steps needed to implement changes
Step 5: Identify existing systems and resources to leverage to successfully make changes
Step 6: Determine potential barriers to change and how the team might address those barriers
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