The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has announced that a treatment for a rare cancer-causing genetic disorder is the first to be awarded the Innovation Passport under the MHRA’s Innovative Licensing and Access Pathway (ILAP). The ILAP was launched in January 2021 to speed up development and access to innovative medicines in the UK post-Brexit.
The first Innovation Passport has been awarded to belzutifan, a treatment developed by Merck, Sharp & Dohme (UK) Limited (MSD UK Limited) for adults with von Hippel Lindau disease, a rare genetic disease caused by mutations in the VHL tumour suppressor gene. The treatment has also been granted orphan designation in the EU, as well as orphan designation and breakthrough therapy designation by the US Food and Drug Administration (FDA).
The Innovation Passport, a new medicine designation, acts as the gateway to enter the ILAP. The approval process for belzutifan will now be accelerated through the ILAP, which means that patients could benefit much sooner from the treatment than they otherwise would.
The decision to award the Innovation Passport to MSD UK Limited was taken jointly by the MHRA, the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC). The ILAP aims to foster a collaborative approach between the MHRA, its partners and the medicine developer, with the common goal of getting the best products to the people who need them as safely and quickly as possible.
The ILAP is open to both commercial and non-commercial developers of a wide range of medicines, including medicines for rare diseases, repurposed medicines and advanced therapy medicinal products (ATMPs). It is aimed towards a variety of products, from very early entries based on non-clinical data to products with mid-development ‘global’ dossiers.
The ILAP has multiple entry points, depending on:
Products towards the end of their development are generally not suitable for the ILAP and applicants are encouraged to apply early in the development of their products in order to maximise the benefits under the pathway.
The ILAP does not replace the promising innovative medicine (PIM) designation of the early access to medicines scheme (EAMS) and applicants can apply for both. EAMS remains an important scheme for earlier patient access towards the end of the development programme in areas of unmet medical need and where major advantage over existing therapies can be demonstrated. The ILAP is broader in its scope and is open to all innovative products.
Since its launch in January, the MHRA has stated that it has so far received ten applications for the ILAP. The fee to apply for an Innovation Passport is £3,624 and the full criteria for ILAP eligibility can be found here.
The Innovation Passport is the first step in the ILAP. Once candidates have received the Innovation Passport, they will work together with the MHRA and its partners to create a product-specific target development profile (TDP) for the new medicine.
The TDP is a living document that will create a product-specific roadmap with the goal of delivering early patient access to the treatment. The TDP offers access to specialist toolkits to design an efficient and ‘regulation and access ready’ development programme. The initial TDP fee is £4,451.
Patient engagement plays a key part in the ILAP and patient views on the benefits and risks of medicines are to be integrated at every stage of the pathway.
Under the ILAP, data can be submitted for review on a rolling basis as it becomes available, similarly to the approach taken for the approval of the Pfizer/BioNTech Covid-19 vaccines. This will speed up the approval process in comparison to the conventional approach that requires all data from clinical trials to be completed before a drug can be cleared for use.
The ILAP scheme demonstrates the innovative steps being taken by the UK in providing access to cutting edge medicine following Brexit and is a positive move towards ensuring the UK remains an attractive location for those in the life sciences sector.
This article has been co-written with Nilly Tabatabai, a trainee solicitor in the commercial, IP and IT team.