The new Innovative Licensing and Access Pathway welcomes first investigational products
Source: gov.uk (2025-10-14)

Developers work with the NHS, the medicines regulator and the UK health technology assessment bodies from the early stages of clinical development, as part of the Innovative Licensing and Access Pathway.

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:
- Neonatal onset ornithine transcarbamylase (OTC) deficiency: a rare metabolic disorder that prevents the body from breaking down ammonia, often proving fatal in infancy without liver transplantation.
- Duchenne muscular dystrophy: a progressive muscle-wasting disease affecting 1 in 3,500 – 5,000 boys, currently with no cure.
- A rare neurodegenerative condition affecting children, with no current cure or treatment available.
Their selection supports wider commitments set out in the government’s Rare Diseases Action Plan (https://www.gov.uk/government/publications/england-rare-diseases-action-plan-2025), which aims to improve diagnosis, treatment and care for people living with rare conditions. It also aligns with the ambitions of the 10 Year Health Plan for England (https://www.gov.uk/government/publications/10-year-health-plan-for-england-fit-for-the-future) and the Life Sciences Sector Plan (https://www.gov.uk/government/publications/life-sciences-sector-plan), which together set out how the UK will harness research and innovation to improve patient outcomes and maintain the UK’s position as a global leader in life sciences.

These products are still in development. Any future availability will depend on the evidence of safety, effectiveness and value.

Developers interested in ILAP can now apply for the next round. Full guidance is available on the MHRA website (https://www.gov.uk/government/publications/innovative-licensing-and-access-pathway-ilap).

Lawrence Tallon, Chief Executive of the MHRA, said:

“The new ILAP is collaborative regulation at its best – patients, healthcare partners, industry, and regulators working together to deliver meaningful benefits for public health. By embedding NHS partners from the start and focusing on transformative medicines, this world-leading, integrated pathway keeps the UK at the forefront of innovative medicine and helps overcome some of the barriers in rare disease development. It ensures work is centred on patient needs and provides hope to families who currently have limited treatment options.”

Dr Sam Roberts, Chief Executive of NICE, said:

“This is a welcome and significant milestone for the ILAP. As an organisation committed to getting the best care to people fast, NICE welcomes any initiative that helps developers get transformative medicines into the NHS. The collaboration between partner organisations, industry and patients has really helped shape the new ILAP offer. We look forward to continuing this exciting collaboration, helping to deliver faster patient access to life-changing medicines.”

Robbie Pearson, Chief Executive of Healthcare Improvement Scotland, said:

“At Healthcare Improvement Scotland our aim is to make health and care better for the people of Scotland. We are delighted that the SMC, part of Healthcare Improvement Scotland, is participating in ILAP to get innovative medicines faster to people who need them. The Scottish Government is committed to improving the lives of people living with a rare condition through the Rare diseases action plan (https://www.gov.scot/publications/progress-report-scotlands-action-plan-rare-diseases/) and we are proud to support the development of innovative therapies that could benefit patients and their families across the UK.”

Professor James Coulson, Clinical Director of AWTTC, said:

“The AWTTC welcomes the award of Innovation Passports for three investigational products as part of the refreshed ILAP. The inclusion of these innovative products into ILAP is an important development in the assessment of rare disease medicines, helping to ensure timely and equitable access across the UK. We look forward to continuing this important collaboration with our partner organisations to deliver safe and effective treatments to all our patients and promote the UK as a world-leader in drug development and life-sciences.”

Joe Truitt, Chief Executive Officer of iECURE, developer of the OTC deficiency candidate, said:

“We are honoured that iECURE’s investigational therapy, ECUR-506, has been awarded an Innovation Passport to enter the ILAP. This unique initiative is designed to accelerate patient access to transformative new medicines, and we’re proud to be part of that effort. We look forward to continuing our collaboration with UK health partners to advance our innovative targeted gene insertion platform and work to bring breakthrough treatments for severe OTC deficiency to patients and their families as efficiently and safely as possible.”

Notes to editors

Entry to ILAP is open to commercial and non-commercial developers worldwide. Applicants must submit medicines or drug-device combinations that have not yet entered their confirmatory trial, which will give more opportunity to benefit from the support offered within the pathway. More details can be found on the MHRA website (https://www.gov.uk/government/publications/innovative-licensing-and-access-pathway-ilap).

The ILAP is a UK-wide initiative, comprising the following partners:
- The All Wales Therapeutics and Toxicology Centre (AWTTC) (http://www.awttc.org/) (on behalf of NHS Health boards and Trusts in Wales)
- The Medicines and Healthcare products Regulatory Agency (MHRA) (https://www.gov.uk/government/organisations/medicines-and-healthcare-products-regulatory-agency)
- National Institute for Health and Care Excellence (NICE) (https://www.nice.org.uk/)
- NHS England (https://www.england.nhs.uk/)
- Scottish Medicines Consortium (SMC) (https://www.scottishmedicines.org.uk/) (on behalf of NHS boards in Scotland)
- Department of Health Northern Ireland
Supporting partners include:
- Department of Health and Social Care (DHSC)
- National Institute for Health Research (NIHR)
- Office for Life Sciences (OLS)
- Scottish Government
- Welsh Government
- Two of the developers selected in this first ILAP round have chosen not to be named publicly at this stage.
- The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines, medical devices and blood components for transfusion in the UK by ensuring they work and are acceptably safe. All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks.
- The MHRA is an executive agency of the Department of Health and Social Care.
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