How to expedite patient access to medicines through the Innovative Licensing and Access Pathway (ILAP) in the UK

One word you don’t hear too often in current political discourse relating to Brexit is ‘streamlining’; but that’s exactly what the UK’s regulator hoped its Innovation Passport, which forms part of the Innovation Licensing and Access Pathway, would do for getting new drugs on the market more effectively when it was launched in early 2021.

It’s been two years since Belzutifan, a treatment developed by MSD UK Limited for von Hippel Lindau disease, was awarded the UK’s first Innovation Passport by the The Medicines and Healthcare products Regulatory Agency (MHRA). This provided access to enhanced regulatory and other stakeholder input such as experts from the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) and supported the efficient and timely development of the product leading to regulatory approval in May 2022. This collaborative approach is a key feature of the ILAP and is designed to foster close cooperation between the regulatory agencies, the medicine developer, and other important stakeholders such as health technology assessment (HTA) bodies.

The ILAP aims to provide a faster and more streamlined route to market for innovative medicines, while ensuring that patient safety and clinical effectiveness remain the top priorities. By working together, the MHRA, NICE, and the SMC in Scotland and the All Wales Therapeutics and Toxicology Centre (AWTTC) in Wales can share knowledge, expertise, and data, and make informed decisions that benefit patients. The ILAP also provides opportunities for early dialogue between the medicine developer, the regulatory and HTA agencies, which can help to identify and address potential issues earlier in the development process. This can help to speed up the approval process and ensure that patients can access innovative treatments as quickly as possible.

The ILAP offers an integrated approach to drug development through the involvement of key stakeholders jointly considering a wide range of issues affecting product development, regulatory approval, and market access. ILAP allow weak spots in the product development to be identified and provide the tools to resolve these ahead of the product launch and have the evidence package meeting the needs of both regulators and HTA bodies. It also gives developers the tools for enhanced patient engagement actively incorporating the patient voice in product development.

The pathway allows entry from a very early stage, based on pre-clinical data through to the mid-development. Drug companies can take part in ILAP whether they are a developer of new chemical entities, or biological medicines or work on new indications of existing medicines.

The entry point to ILAP is this much-lauded Innovation Passport application. It can take approximately 8-10 weeks between ILAP submission and decision on the eligibility and awarding the Innovation Passport. Products must fulfil a set of eligibility criteria to get Innovative Passport designation. The criteria fall into three distinct areas:

1: Details of the condition, patient or public health area (product fulfils more than one of a specific area)

• The condition is life-threatening or seriously debilitating.
• The condition is life-threatening or seriously debilitating.
• There is a significant patient or public health need.

2: The medicinal product fulfils one or more of a specific area

• Innovative medicine (e.g., advanced therapy medicinal product [ATMP], or a new mechanism of action, or novel drug-device combination)
• Medicines being developed in a clinically significant new indication
• Medicines for rare disease and/or other special populations such as neonates and children, elderly, and pregnant women
• Development aligning with the objectives for UK public health priorities

3: The medicinal product has the potential to offer benefits to patients

• Improved efficacy or safety, patient care or QoL compared to alternative therapeutic options

After Innovation Passport designation, the drug developer will work together with key stakeholders to define the Target Development Profile (TDP) providing a roadmap of key regulatory and development features, key areas for future engagement, and evidence generation to facilitate not only regulatory assessment, but also the HTA assessments. The TDP will be reviewed and refined on a rolling basis as the product advances through development. During the ILAP, developers will have a broad range of TDP tools available encompassing tools for regulatory as well as HTA interaction, tools to support patient recruitment and enhance patient engagement.

So where do we find ourselves in 2023? As of this January, the MHRA has received a total of 164 applications since the ILAP was implemented, of which 108 were awarded the Innovation Passport Criteria, with the highest proportion of those applications being in the oncology area.

About a quarter of applications were made for drugs in the early stage of development (in which human studies were not yet initiated) and half of the drug applications were at the mid-stage (pre-phase 3 not recruiting).

Overall, the scheme has proven popular in the first two years since its inception. It shows the innovative steps being taken by the UK to allow the best new medicines to get to those patients who need them, and is likely to futureproof the UK’s ambitious vision for life sciences in a post-Brexit landscape.