Three potential new therapies are the first to enter the Innovative Licensing and Access Pathway (ILAP).
The scheme is designed to help promising new medicines reach NHS patients faster.
The three therapies are recipients of ‘Innovation Passports’, which give developers coordinated support from the earliest stages of development, with priority access to services such as clinical trials support and NHS engagement.
ILAP 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.
The three therapies will work pm neonatal onset ornithine transcarbamylase (OTC) deficiency, Duchenne muscular dystrophy and a rare neurodegenerative condition affecting children.
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.”