First in Human Clinical Trials in the United Kingdom – Past, Present and Future?

抄録

In my talk, I focus on three main topics:

1) An overview of clinical trials conducted in the UK, particularly advanced medicines

2)The initiatives to make drug development more efficient, focussed and thereby faster

3)How early-phase clinical trial units need to adapt to stay relevant

I started working in clinical pharmacology 30 years ago. Since then, I only had success with five medicines progressing from FTIM to market authorization — two of which are gene silencers. I will be discussing the SWOT of small molecule versus gene-based medicines and comparing FDA/EMA market authorizations until now.

I will discuss ex-vivo and in-vivo gene editing, and newer in-vivo therapies such as NTLA-2001 or VT-1001 but will use gene silencing using RNAi as an example to dive deeper into some principles pertaining to the development of these medicines.

Shortening times from FIH to MA from 5 to 10 years to only 3 to 5 will have effects on how we work. For biotech firms, a very important aspect of fundraising is the time until revenue can be generated. With RNAi even in prevalent indications, these shorter timelines to MA have already been seen. Regulators in all regions help by offering fast-tracking approvals and I will discuss ILAP in the UK.

Advanced study designs and integrated protocols play a prominent role in this context and the inclusion of patients in FTIM studies may be the only option to start human studies but there is the option to use the same strategies in many other programs where healthy participants could be – but don’t have to be – included.