Utilizing a Delphi Panel to develop a gene treatment algorithm

Overview

Facing tight deadlines and complex logistics, Woven Health Collective facilitated development of a treatment algorithm for gene therapy in Duchenne Muscular Dystrophy. Utilizing a Delphi Panel framework, the team navigated a four-month timeline, ensuring expert consensus and seamless collaboration. The project met critical launch dates and resulted in significant scientific contributions, including presentations at major medical conferences.

the challenge

In July 2022, a biotechnology company approached Woven to assist with developing a treatment algorithm focused on addressing adverse events related to gene therapy in patients with Duchenne Muscular Dystrophy.

The company was considering a systematic literature review, but time constraints may have put that approach at higher risk. An established content vendor needed until October 2022 to complete their portion of the project, while the company required a completed algorithm by January 2023 to meet launch deadlines.

An already compressed timeline would be further impacted by multiple fourth quarter holidays, restricting HCP availability and complicating meeting logistics.

the solution

A truly integrated effort would be needed to deliver this algorithm in only four months. Woven recommended the Delphi Panel framework (see below), a specialized methodology that leverages multiple rounds of expert consensus through surveys and questionnaires.

Given the limited literature available, this method allowed the company to gather expert consensus on the administration of, and safety monitoring for gene therapy in DMD patients.

• Seasoned medical publications staff coordinated the scientific and content aspects of the Delphi Panel and organized several rounds of expert surveys.
• Skilled logistics managers planned and produced an effective live meeting.
• A central account director liaised between internal and external teams to ensure seamless collaboration and maintain alignment throughout the process.
  
  

the timeline

the Delphi Panel process

A Delphi Panel is an expert consultation method designed to reach consensus over several rounds of data collection and refinement. This project used three rounds:

Round 1:

A phone survey identified key concepts for administration and safety monitoring of gene therapy in DMD patients.

Round 2:

A second phone survey used to gather proposed monitoring parameters and safety considerations.

Round 3:

Then, an in-person meeting was held with the aim of reaching consensus on proposed guidance statements.

the consensus

Monitoring: The prescribing physician should perform physical assessments, with a high likelihood of patient hospitalization.

Laboratory Studies: Baseline labs to be repeated within a week, including specific evaluations like ANA, CRP, and myoglobin.

Diagnostic Studies: Recommendations included EKGs, echo, and neuromuscular strength assessments.

Treatment: In the case of inadequate response, escalate steroid therapy and consider advanced treatments like plasmapheresis or immunosuppressant therapy.

the results

• Seamless execution allowed for live Delphi Panel meeting January 23, 2023.
• Abstract submitted to American Society of Gene & Cell Therapy (ASGCT) February 3, 2023, resulting in a poster presentation in May of 2023.
• Second abstract submitted to Muscular Dystrophy Association (MDA) February 15, 2023 resulting in a poster presentation in March of 2023.
• Peer-reviewed manuscript developed with a target submission in June of 2023

the takeaways

Evaluating the merits of multiple development approaches (ie, literature review vs Delphi Panel) allowed for the selection of the best process given the specific parameters of the program.

The integration of Woven’s scientific, editorial, and logistics
capabilities enables the delivery of high-quality consensus-based publications, even when there are significant time constraints.