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NHLBI Working Group: Can Global Assays Address Current and Future Challenges in Therapeutic Monitoring of Hemophilia A?
The National Heart, Lung, and Blood Institute held a working group meeting on June 22 & 23, 2011 to address the question posed in the working group title, “Can Global Assays Address Current and Future Challenges in Therapeutic Monitoring of Hemophilia A?” This research question stems from the concern that current available methods to monitor hemophilia A therapy do not always reflect clinical endpoints, cannot effectively monitor therapy with bypassing agents and will not likely be applicable to the new long acting products in development. The goal of the working group was to evaluate the current capabilities of available hemostatic global assays and define the scientific and translational research needed to move the field toward routine clinical application. The working group consisted of clinical and research hemophilia experts from the United States, Canada and Europe; scientists with expertise in global assays and representatives from the Center for Biologics Evaluation & Research, FDA.
Presentations & Discussions:
Dr. Susan Shurin, NHLBI Acting Director, welcomed all participants and Drs. Keith Hoots, Donna Di Michele and Ken Mann outlined the goals of the meeting and charge to the working group members. During the open session working group members and interested parties from academia, government and industry listened to presentations and participated in discussions on current methods used to monitor hemophilia treatment, future needs for monitoring therapy and the challenges to global assay implementation. The first day program was divided into three Sessions with presentations in the following areas:
Dr. Ken Mann and Session Chairs, Drs. Steve Pipe, Diane Nugent & Nigel Key provided their summary of the first day discussions to all participants on the morning of the second day. The meeting identified the need to define the bleeding phenotype and clinical response to therapeutic intervention, address the ways that assays are vetted and standardized and determine how to implement the assays for clinical studies. They provided the following list of characteristics needed in an assay to meet future needs:
They recommended a framework to move the TGA and TEG assays forward that would consist of three working sub-committees (Panels) and an Executive Chair to continue the work begun by this Global Assay Working Group meeting. Because this is of international interest the 1) Clinical Outcome Endpoints, 2) Assay/Technical and 3) Clinical Trial Implementation panels would each have one US and one EU co-chair. The panel co-chairs would select additional members, determine goals, prepare a budget and set timelines for the work over the ensuing year. Dr. Nigel Key agreed to be the Executive Chair and oversee the three panels. Drs. Guy Young and Flora Peyvandi agreed to be the co-chairs for Clinical Outcome Endpoints; Drs. Kathleen Brummel-Ziedins and Benny Sorensen will be the co-chairs for Assay/Technical and Drs. Diane Nugent and Claude Negrier will be the co-chairs for the Clinical Trial Implementation. The industry representatives present agreed to provide support for the Panel activities through a nonprofit organization. A meeting of all industry-stakeholders, Drs. Mann and Key, and the Panel Co-chairs will be held in conjunction with the upcoming International Society on Thrombosis & Haemostasis (ISTH) meeting in Japan to discuss the details of the arrangement.
During the closed session the working group members determined the focus should include both hemophilia A and B, but not be immediately expanded to other hemostatic or thrombotic disorders. They refined the goals for the three panels:
The Panel co-chairs agreed to plan initial meetings at the July 2011 ISTH meeting, constitute the membership of their respective panels and interact with other individuals or groups working on these issues. A writing committee was formed from among the panel co-chairs with the goal of drafting a report summarizing the outcomes of the meeting for publication in a peer-reviewed journal. The paper will explain the reasons for creating the panels and their working goals.
Working Group Members:
Kenneth G. Mann Ph.D., Working Group Chair, University of Vermont
Katheleen Brummel-Ziedins, Ph.D., University of Vermont
NHLBI Staff Members:
Donna Di Michele, M.D., Deputy Director, DBDR; Acting Chief, Thrombosis and Hemostasis Branch
Last Updated September 2011