At the ISPOR Conference in Washington DC, USA on May 2022,
Dr. Ariel Beresniak represented Data Mining International in the Expert Panel alongside :

Dr. Alicia Granados of Sanofi, Michael Drummond Professor of Health Economics at York University, Sheela Upadhyaya Expert in Rare Diseases and ISPOR Chair Elect, Durhane Wong-Rieger President of the Canadian Organization for Rare Disorders, and Karen Facey who chaired the panel.

Thank you to panellists of ISPOR2022 in Washington for their insights shared in this Issue Panel Report, including :

▶️ Alicia Granados, MD, PhD PH says small, heterogeneous populations showing a lack of clear optimal outcomes make evidence generation based on QALYs extremely challenging

▶️ Ariel Beresniak MD, MPH, PhD reminds us that the four underlying assumptions of the QALY are not validated, and cannot be used in rare diseases, whilst suggesting alternative/complementary approaches

▶️ Michael Drummond indicates that the QALY leaves out aspects of value for in rare diseases, such as severity of disease, insurance value, real option and equity,…

▶️ Sheela Upadhyaya says that further flexibility is needed to account for uncertainty in the rare diseases and for other types of evidence that the QALY does not capture

▶️ Durhane Wong-Rieger insists that the QALY is not designed for rare diseases and prevents access to treatments for patients, and that a new approach is needed

ISPOR 2022 Issue Panel Report – QALYs and RDs

#ISPOR #heor #rarediseases #qaly