9937 ISTH Gene Therapy, Change Guide

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 1: Change Area

1. Select the Change Areas your or your HTC care team will focus on

Patient eligibility
Referral to centers offering treatment
Patient preparation
Treatment delivery
Post-administration management
Providing patient support
Care coordination
Assessing laboratory measurements of coagulation when using novel agents

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

Based on inclusion/exclusion criteria from clinical trials, which patients will be eligible for GTH? (Work from list of inclusion/exclusion criteria for therapies currently in phase 3 trials) *This question is required.

Based on the current status of patient (disease severity, frequency of bleeds, frequency of infusions, impact on quality of life, etc), is GTH appropriate? *This question is required.

Do patients have neutralizing antibodies for the AAV serotype being considered for therapy?

Referral to centers offering treatment:
Referring Patients to Treatment Centers Where GTH is Available (If Treatment Will Not be Available in Local HTC)

What information will be needed for making a referral? *This question is required.

How will information be communicated (EHR)?
*This question is required.

Patient Preparation:
Preparing Patients for Gene Therapy

What is considered optimal patient care in the days/weeks/months leading up GTH infusion? *This question is required.

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.

What specific monitoring (if any) is required during and immediately following administration of Gene Therapy for hemophilia *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.

If yes, what emergency measures are in place?

Post-administration management:
Post-administration Care

Short-term monitoring

What is the duration of short-term monitoring immediately following the gene therapy infusion (inpatient or outpatient?) What needs to be monitored? *This question is required.

Long-term monitoring (safety and efficacy)

What is the recommended frequency for long-term monitoring? *This question is required.

What assessments need to be performed? *This question is required.

What are the specific requirements for the laboratory assessments associated with GTH? *This question is required.

What is the treatment plan for acute bleeds with Gene Therapy for hemophilia? *This question is required.

Providing Patient Support

What additional support will patients need before, during, and after treatment? *This question is required.

Main concerns
Effects on identification as a PWH

What education will patients require in preparation for gene therapy?

Treatment options
Comparison with current treatment
Selection criteria
Known and unknown risks and benefits
Post-treatment expectations
Importance of long-term monitoring

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.

If not, how will it need to be modified?

Will EHR forms need to be updated and/or modified for Gene Therapy? *This question is required.

If yes, how so?

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.

If so, how will this be accomplished and by whom?

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?

If yes, whose responsibility is it?

Step 3: Determine necessary changes to integrate new therapy

2. Determine necessary changes to integrate gene therapy for hemophilia

Step 4: Outline concrete steps needed to implement changes

3. Outline concrete steps needed to implement changes

Step 5: Identify existing systems and resources to leverage to successfully make changes

4. 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 those barriers

5. Determine potential barriers to change and how the team might address those barriers

6. Please enter your email, a copy of your Change Guide will be sent to this address: *This question is required. This question requires a valid email address.