New Innovative Licensing and Access Pathway aims to accelerate access to transformative medicines in the UK 

Three investigational therapies for rare and serious conditions have become the first to enter the UK’s renewed Innovative Licensing and Access Pathway (ILAP), a collaborative initiative designed to accelerate development and patient access through coordinated support from regulators, the NHS, and health technology assessment (HTA) bodies. 

These therapies, targeting neonatal-onset ornithine transcarbamylase (OTC) deficiency, Duchenne muscular dystrophy, and a rare neurodegenerative disorder, have received the first ‘Innovation Passports’ under the UK’s renewed ILAP. The scheme, jointly launched by the Medicines and Healthcare products Regulatory Agency (MHRA), National Health Service (NHS), and UK health technology assessment (HTA) bodies NICE, Scottish Medicines Consortium (SMC), and the All Wales Therapeutics and Toxicology Centre (AWTTC), aims to help promising medicines reach NHS patients faster. 

The Innovation Passport provides developers with coordinated, multi-agency support from the early stages of clinical development, including access to trial design advice and NHS engagement. This support focuses on areas of high unmet medical need, where treatment options are limited or non-existent. 

From 16 initial applications, three rare disease candidates were selected for this first round. These include potential therapies for neonatal-onset OTC deficiency, a metabolic disorder that prevents the body from breaking down ammonia and can be fatal in infancy without liver transplantation; Duchenne muscular dystrophy, a progressive muscle-wasting disease affecting one in 3500–5000 boys with no current cure; and a rare neurodegenerative disease affecting children for which no treatment is available. 

Rare diseases collectively affect around three million people in the UK, though each condition impacts fewer than 0.1% of the population. Small patient numbers and complex trial requirements often make drug development challenging, highlighting the value of early, coordinated engagement across agencies. 

The selection of these products aligns with national priorities set out in England’s Rare Diseases Action Plan, the 10-Year Health Plan for England, and the Life Sciences Sector Plan, which together seek to accelerate diagnosis, treatment, and innovation in rare and underserved conditions. Joe Truitt, CEO of iECURE, developer of the OTC deficiency candidate ECUR-506, commented:  

“This unique initiative is designed to accelerate patient access to transformative new medicines, and we’re proud to be part of that effort.” 

According to the MHRA, ILAP is the only end-to-end pathway globally that brings together the regulator, the NHS, and HTA bodies to provide aligned guidance on safety, effectiveness, and value from the start of development. By doing so, it aims to shorten the time between early research and patient access. 

The refreshed pathway builds on the version that was first launched in 2021, incorporating lessons learned and focusing more selectively on transformative products that address unmet clinical needs. NHS partners are now embedded as core members, enabling faster, system-wide adoption. Key improvements include bespoke support, predictable timelines, early patient and NHS engagement, and a single point of contact for each product. 

Lawrence Tallon, Chief Executive of the MHRA, described the updated ILAP as “collaborative regulation at its best – patients, healthcare partners, industry, and regulators working together to deliver meaningful benefits for public health.” He added that 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.” 

Partner organizations across the UK have welcomed the new phase of ILAP. Dr Sam Roberts, Chief Executive of NICE, said:  

“This is a welcome and significant milestone for the ILAP. As an organization 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 organizations, 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, noted that the SMC’s participation will help “get innovative medicines faster to people who need them,” supporting Scotland’s commitments under its rare diseases action plan. Professor James Coulson, Clinical Director of AWTTC, added that the inclusion of these innovative products “is an important development in the assessment of rare disease medicines, helping to ensure timely and equitable access across the UK.”