Tom Lazenby/LinkedIn

May 26, 2026, 01:15

Tom Lazenby: Accelerating Rare Disease Drug Development Through MHRA’s Investigational Marketing Authorisation

Tom Lazenby, Founder and CEO at Mayet, shared on LinkedIn:

”The MHRA wants to compress rare disease drug development by ten to twelve years.

The mechanism is a single authorisation that combines trial approval with a progressive route to marketing authorisation.

It is called an Investigational Marketing Authorisation.

Announced 21 May 2026. Consultation open until 30 July.

What the framework actually proposes:

Rolling data submissions and modular assessments rather than a single dossier
Adaptive trial designs as a default option, including basket and umbrella studies
Acceptance of surrogate endpoints where conventional endpoints are not feasible
Technology-agnostic across gene therapies, ATMPs, and repurposed medicines

The line in the announcement that matters most for sponsors and CROs running rare disease trials is not the speed.

It is this:
‘Structured post-authorisation evidence generation for safety, quality and efficacy.’

A faster pathway in does not mean a lighter regulatory expectation out.

The framework pairs the streamlined approval with continuous, structured evidence generation that runs through to post-authorisation.

This is the same principle that lives inside ICH E6(R3) for trial conduct.

Sponsor accountability does not end at submission.

It continues through the whole lifecycle, and the evidence has to be available, not assembled.

If you are running rare disease trials in the UK, three things to think about this week:

Whether your study evidence trail can support modular assessment rather than dossier submission
Whether your vendor oversight programme can demonstrate ongoing performance, not point-in-time qualification
Whether your post-trial evidence generation is a continuous process or a closing activity

The consultation is open.

The MHRA wants input by 30 July.”

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