Module 1 2021

30/06/2021

HTA challenges (1/2)

• Pressure for early access • Application requirements HTA ≠ HA • Small trials  limited statistical power beyond primary endpoints • Endpoints sufficient validated? • Limited QoL • Lack of natural history and other comparative data, study duration limited • Challenge to develop Health Economic analysis and to demonstrate value for money

The Organisation for Professionals in Regulatory Affairs

31

HTA challenges (2/2)

Already only in EU do all MS have their own reimbursement system – while reimbursement is prerequisite to return for investment • Smaller subpopulation than regulatory approval • Expected initial period of compassionate use

The Organisation for Professionals in Regulatory Affairs

32

16