EU-5 has become EU-4! What happened to the UK?

Focus on the EU5: that is the geographical scope we have become used to identifying market opportunities and preparing the ground for orphan drug launches. After all, these 5 countries (France, Germany, Italy, Spain and UK) represent approximately 80% of potential healthcare spend in rare diseases in Europe and are duly prioritised by pharma companies.

Now we are hearing requests for a different geographical scope when pharma is planning launches for orphan drugs and ATMPs: “Focus on the EU5” or more recently, “Focus on the EU4”. So what has happened to the UK?

This webinar will set out two opinions: the first for including the UK as a priority market when developing launch strategies for orphan drugs and ATMPs; the second against including the UK as a strategic priority. By effectively exploring different arguments, the listener will get an objective perspective as to the value of UK prioritisation in developing orphan drugs and ATMPs launch strategies.

On 1st Jan 2021, the UK officially left the European Union. In advance of this, UK politicians and agencies were preparing to ensure the UK remained a country for investment by the pharmaceutical industry. The Life Sciences Industrial Strategy was set up as a collaborative program to address potential gaps and work towards a ‘more efficient and effective health system’.

In addition to this, the UK regulator, MHRA, set up an Innovative Licensing and Access Pathway (ILAP) in Jan 2021. Just one month later, the MHRA was keen to announce the first ILAP given to a rare cancer drug. NICE is also looking to ensure it remains cutting edge in the world of HTAs (and is currently undergoing a new review of its appraisal methodology) and is currently consulting various stakeholders with a revised process due later this year. The appraisal review is part of NICE’s 5-year ambitious strategy from 2021-2026.

A recent study showed that less than half of orphan drugs are routinely funded by the English National Health Service (NHS) -1. The funding is ‘helped’ to a certain extent by Specialised Commissioning and the Cancer Drugs Fund. The same study showed access to orphan drugs is the lowest in the EU5: compare Germany at 93% and France at 81%. Another study found the UK is ~1.5 times lower than Germany, France or Italy when considering the per capita Euro spend on prescription drugs – 2. Add Brexit into the mix and perhaps it is not surprising that over 80% of our last requests for orphan drug launch support have excluded the UK as a strategic priority.

REFERENCE 1 https://ojrd.biomedcentral.com/articles/10.1186/s13023-019-1078-5   2 Patient Access to Medicines for Rare Diseases in European Countries – ScienceDirect