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9085 ISTH Gene Therapy, Change Guide


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 1: Change Area
1. Select the Change Areas your or your HTC care team will focus on
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
Is there any pre-infusion medication that needs to be administered? *This question is required.
Does the GTH interact/interfere with vaccinations (seasonal or otherwise)? *This question is required.
Do patients need to be currently on prophylaxis treatment (and for how long) to be considered for GTH? *This question is required.
Treatment delivery:
Administration of Gene Therapy
Are the storage requirements clearly defined and present/achievable in your HTC? *This question is required.
Is your center currently capable of providing the biosafety level needed for GTH (ie, biosafety level 1, see NIH AAV vector risk group guidance)? *This question is required.
Is appropriate training available for all staff members who will be handling viral infusions? *This question is required.
Is the HTC pharmacy trained and prepared for Gene Therapy for Hemophilia (receipt, handling, storage, etc)? *This question is required.
Are emergency measures in place in case of infusion reaction (physician, crash cart, etc)? *This question is required.
Post-administration management:
Post-administration Care
Short-term monitoring
Long-term monitoring (safety and efficacy)
 Providing Patient Support
What additional support will patients need before, during, and after treatment? *This question is required.
What education will patients require in preparation for gene therapy?
Care coordination:
Coordinating Care Among HTC Care Team Members
Will the current system/process for coordination of patient care work for Gene Therapy for Hemophilia? If not, how will it need to be modified? *This question is required.
 Will EHR forms need to be updated and/or modified for  Gene Therapy? *This question is required.
Assessing Laboratory Measurements of Coagulation when using Novel Agents
Will gene therapy require new methods of assessing coagulation? *This question is required.
Are resources available at your HTC to integrate new testing measures?  *This question is required.
Will samples need to be sent out for testing? If so, how will this be accomplished and by whom? *This question is required.
Integrating Research Questions Into Clinical Practice
Is there an existing list or a list in development of relevant gene therapy research questions? *This question is required.
 Is there a process for integrating research questions into the HTC clinical practice? *This question is required.
Is there a group or person within the HTC responsible for promoting and monitoring this integration activity? *This question is required.
Is there a group or person within the HTC responsible for including patients in a gene therapy for Hemophilia registry?
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
This question requires a valid email address.