Adaptive Pathways and conditional approval

Thursday, 16.00

Francois Houyez

Valid point
differences in compassionate usage programs between countries effectively create disparity in access across Europe, even between countries with similarly efficient healthcare systems. Francois mentioned 4 years- but need to verify in which setting.


Advantages

Disadvantages

Tomas Salmonson

Comment-
conditional earlier approval can make it difficult to recruit for the registration trials. And I wonder why that is!!! Guess the trial design is not attractive for patients? 

Susan Forda

comparison between US and EU approvals of innovative therapies-

for indications that let to special pathways for both sides.

US has more fast access designations than the EU

US takes less time to approval than EU

BUT less post-marketing studies in the EU than in the US. 


And- faster approval does not lead to more withdrawls
So far, no withdrawl after accelerated approval/ conditional approval. In the US, none on break-through and one withdrawl for a priority review!



Niklas Hedberg, TLV

used a great analogy of a stream to describe how new therapies come down after a damm (the regulators) and how the downstream tries to cope.


Hans-Georg Eichler

Use of real-world data in the Adaptive Pathways pilots

and again the very valid point that IF registries, they need to have the right quality

Discussion
interesting enough, this is an adaptive pathway discussion and the first comment goes directly to drug prices. Niklas made the point that there is the worry that adaptive licensing effectively out-sources part of the development cost to society (instead of the company paying for it). 
Very valid point by Tomas Salmonson- what is the point if we approve drugs for a wide indication (like HepC) but then only give them to a few patients because of price? That makes no sense from a public health perspective!

Niklas, TLV, suggestion to discuss conditions with a consortium of different Pharma working in that space to discuss the specific challenges and needs way before a single products hits the market.