On 26 February 2021, Belzutifan, a treatment for adults with the rare cancer-causing genetic disorder Von Hippel-Lindau disease, was awarded the first “Innovation Passport” under the recently established Innovative Licensing and Access Pathway (ILAP).

ILAP

At the end of the Brexit transition period on 1 January 2021, the MHRA became the UK’s standalone drug regulator. In seeking to attract life sciences development in the post-Brexit era, a new approval pathway was established – the ILAP.

In a press release at the start of the year, the MHRA clearly stated the ambition for the ILAP is to reduce the time to market for innovative medicines. Guidance states that the pathway involves enhanced input and interactions with the MHRA and partners (including the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC)).

The MHRA further notes that the ILAP can be accessed at multiple entry points depending on:

• the stage of development of the product
• the data available
• the ambition of the applicant to engage with UK stakeholders
• the applicant’s appetite for new innovative ways of working

The “Innovation Passport” acts as the gateway to enter the ILAP and is a new medicine designation. The next step in the pathway is the preparation of a target development profile (TDP) document by the MHRA, NICE and the SMC. The TDP is a living document which sets out the regulatory and development milestones, identifies potential pitfalls and creates a roadmap to achieving early patient access in the UK. The TDP can assist pharmaceutical companies with clinical trial design and evidence generation.

These features mean that the ILAP offers pharmaceutical companies the opportunity to engage in dialogue with regulators, health technology assessment (HTA) agencies and other partners at a pre-clinical stage, rather than after the completion of Phase 3 trials having committed substantial time and expenses towards a specific treatment.  In addition, ILAP will offer companies a rolling review similar to the one that was done with the Pfizer/BioNTech COVID-19 vaccine which will also help speed up the approval process.

It remains to be seen whether drugs with an Innovation Passport will still tend to go through the traditional phases of clinical trials (albeit on an accelerated basis) or whether we will see greater flexibility in this respect. Those entering into licensing deals will need to bear this in mind when agreeing to make payments that are pinned to development or regulatory milestones, as milestones that conventionally feature in such agreements potentially may be bypassed.

In the press release announcing the award of the first Innovation Passport to Belzutifan, the MHRA notes that since its launch at the start of the year they have received 10 applications for Innovation Passports.

We note that the ILAP does not replace the Early Access to Medicines Scheme (EAMS) and applicants can apply for approval via both ILAP and EAMS.

The therapy

Belzutifan, developed by Merck Sharp & Dohme (MSD), is a novel, investigational candidate in MSD’s oncology pipeline and is currently being evaluated in a variety of clinical trials. The drug functions to inhibit the accumulation of proteins which can lead to the formation of tumours. The discovery of these proteins was awarded the Nobel Prize in Physiology or Medicine in 2019. In June 2020 Belzutifan was granted orphan designation in the EU and both orphan designation and breakthrough therapy designation by the US Food and Drug Administration (FDA).