Three potential therapies – including one that could be a lifeline for babies born with a fatal metabolic disorder and another for boys with the muscle-wasting condition Duchenne muscular dystrophy – have become the first to enter a new UK scheme designed to help promising new medicines reach NHS patients faster.
The candidates are recipients of ‘Innovation Passports’ under the new Innovative Licensing and Access Pathway (ILAP), announced today by the ILAP Partners: the Medicines and Healthcare products Regulatory Agency (MHRA), National Health Service (NHS), and the health technology assessment (HTA) bodies – the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC), and the All Wales Therapeutics and Toxicology Centre (AWTTC).
The Innovation Passport gives developers coordinated support from the earliest stages of development, with priority access to services such as clinical trials support and NHS engagement, to help speed up access for patients where current treatment options are limited or non-existent.
A world-first approach
What makes ILAP unique is that it is the only end-to-end pathway in the world where healthcare developers, the regulator, the UK-wide national health system, and the health technology assessment (HTA) bodies work together from the outset. By giving developers early, coordinated guidance on safety, effectiveness and value requirements across the system, the scheme is designed to reduce the time it takes for promising new treatments to move through development, licensing and, if approved, to NHS patients.
The new ILAP builds on the original pathway launched in 2021. Building on its successes and feedback, the new version focuses more selectively on transformative products that address unmet clinical needs, with NHS partners now embedded as core members to ensure faster system-wide adoption. Key improvements include enhanced bespoke support, predictable timelines, early patient and NHS engagement, and a single point of contact for each product.
Accelerating development for rare diseases
There are between 5,000 and 8,000 rare diseases. Each affects less than 0.1% of the UK population, but together they affect the lives of 3 million people. For many of these conditions, effective treatments are lacking, leaving patients and families facing limited options.
Developing medicines for rare diseases is particularly challenging, as small patient populations make clinical trials complex and expensive. The new ILAP offers focused support that is particularly valuable in this area, helping to overcome these barriers and accelerate the development of promising new treatments.
From 16 applications in this first round, three investigational products with rare disease indications were awarded an Innovation Passport, the entry point into ILAP. While still in clinical development, they offer hope for conditions including:
Excerpt from the official page. The original is authoritative. Continue reading at UK MHRA ↗