Hemophilia A

New England CEPAC
The Institute for Clinical and Economic Review (ICER) announced today that it plans to assess the comparative clinical effectiveness and value of valoctocogene roxaparvovec (BioMarin Pharmaceutical, Inc.) and emicizumab (Hemlibra®, Genentech) for the treatment of hemophilia A. Valoctocogene roxaparvovec is an investigational gene therapy, and a decision regarding FDA approval is expected by mid-2020. Emicizumab was approved in October 2018.

For questions or additional information, please contact Catherine Koola, Program Manager, at ckoola@icer-review.org

ASSOCIATED MEETING & MATERIALS

New England CEPAC
August 3, 2020 10am-4pm ET

TBA

The New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for hemophilia.

Key Dates

Associated Materials

01/03/2020 – 01/22/2020

01/24/2020

01/24/2020 – 02/13/2020
Public Comments

01/24/2020

02/25/2020
Revised Scoping Document

03/30/2020
Research Protocol

04/16/2020
Model Analysis Plan

06/03/2020
Draft Evidence Report

06/03/2020
Draft Voting Questions

06/03/2020 – 06/30/2020
Public Comments

07/23/2020
Revised Voting Questions

07/23/2020
Evidence Report

07/23/2020
Response to Public Comments

08/03/2020
Meeting Agenda

08/03/2020
Evidence Presentation

08/27/2020
Final Evidence Report and Meeting Summary

08/27/2020
Report-at-a-Glance

08/27/2020
Final Policy Recommendations


ASSOCIATED MEETING & MATERIALS

New England CEPAC
March 29, 2018 10am-4pm ET

Boston Marriott Cambridge
50 Broadway
Cambridge, MA, 02142

The New England CEPAC will convene to review the comparative clinical effectiveness and value of emicizumab for treatment of hemophilia A.

Key Dates

Associated Materials

08/11/2017

08/11/2017 – 09/06/2017

09/11/2017

09/11/2017 – 09/29/2017

10/10/2017

11/21/2017

11/21/2017

01/26/2018

01/26/2018

Open to public comment until February 23, 2018 at 5pm ET. For more information on submitting a public comment, visit our engagement page.