MedVal/PharmaWrite Facilitates Consensus on AE Management Using Delphi Panel

Dec 20

The Challenge

In July 2022, a company approached MedVal/PharmaWrite to develop a treatment algorithm for managing adverse events related to gene therapy for Duchenne Muscular Dystrophy (DMD). The client wanted to submit the results as a congress abstract and a manuscript. Because of the pending abstract deadline for the annual meeting of the American Society of Gene & Cell Therapy (ASGCT), it was important that the process in developing the treatment algorithm was efficient and would lead to insights that would withstand the scrutiny of peer-reviewed publications.

The Solution

MedVal/PharmaWrite recommended a Delphi Panel to gain consensus on the treatment algorithm. A Delphi Panel facilitates expert consensus through multiple rounds of surveys, followed by a live meeting to finalize recommendations. Not only do the surveys have to be clear and precise, but the process needs to be conducted with consideration of the ACcurate COnsensus Reporting Document (ACCORD) guidelines.

The Results

MedVal/PharmaWrite created surveys, engaged with the experts, and managed the live meeting to gather the opinions of the panel and align on consensus. The Delphi Panel was completed on time. Key outcomes included:

The live meeting was conducted in January 2023
An abstract was submitted to ASGCT in February, leading to a poster presentation in May 2023
Another abstract was submitted to the Muscular Dystrophy Association Clinical & Scientific Conference in February, with a poster presented in March 2023
A peer-reviewed manuscript was published in 2024 (Zaidman CM, et al. J Neuromuscul Dis. 2024; 11(3): 687–699)

The Takeaway

Because of the scientific knowledge and thorough expertise of the Delphi Panel process, MedVal/PharmaWrite delivered a high-quality solution that was executed in accordance with professional guidelines and executed a communication plan that allowed for multiple presentations and a publication. This workstream provides important guidance for physicians to improve the management of patients who are treated for DMD with gene therapy.