ISSUE

: There has been an explosion in the number of new cell and gene therapies and treatments for rare diseases in development. This will result in a heavy demand for health technology assessments. Ideally HTA might look at each treatment individually but demand in the system requires greater efficiency. Many biopharmaceutical companies developing drugs individually invest in similar and sometimes overlapping work to develop evidence and economic models. Small-sized companies may not have the capabilities or finances to invest in HTA at an early stage. The panel will debate the trade-off between what is efficient and what is optimal to ensure fair HTA appraisals and patient access and how patient-led initiatives might help.

OVERVIEW

: The panel will debate the pros and cons of developing greater efficiencies in HTA for innovative therapies and explore approaches to balancing optimal HTA methods with greater efficiency. Josie Godfrey will moderate the session and describe the challenges of the increase in cell and gene therapies and orphan drugs. She will pose questions including: Will the HTA burden of cell and gene therapies and for rare diseases be too high for current approaches to continue? Small companies may not have the resources to develop evidence for HTA. Should we address this or accept it? Can we identify and agree upon areas where collaboration could help build efficiency in the system? What role is there for patient-led initiates?

The panelists will represent different stakeholder and geographical perspectives. Ron Akehurst will represent a HTA perspective arguing that current approaches will continue to be effective without the need for major changes. Fleur Chandler will present a patient perspective arguing for greater collaboration and efficiencies in approach. Sachin Kamal-Bahl will represent an industry perspective that a pragmatic approach is needed to address the increasing demands on the system and identify efficiencies.