New Framework for Innovative Drugs and Biosimilars in the UK

Starting on January 1, 2021, the Medicines and Healthcare Products Regulatory Agency (MHRA) will become a freestanding regulator, taking over full responsibility for evaluating and approving all types of medicinal products in the UK. It is seizing the opportunity to amend the existing regulatory framework by making the approval process faster and less burdensome for pharmaceutical companies.

To this effect, the MHRA has announced two major changes to the medicines regulatory landscape: a new licensing pathway for innovative drugs and a reduction of the clinical trial data requirements for the approval of biosimilars.

New licensing pathway for innovative drugs

The new pathway, available January 1, will offer a new dynamic and interactive approach to licensing based on regulatory flexibilities to enable an “adaptive authorization” of promising drugs. This will introduce an ongoing benefit-risk assessment throughout the life cycle of a medicinal product. To realize this new approach to licensing and assessment under this new adaptive authorization, MHRA is planning on relying on real-world evidence (RWE) and novel clinical trial designs.

Drugs eligible for this pathway will be granted “innovative medicine designation,” which will lead to a product-specific roadmap for the development of the product called “target development profile.” This will exist in parallel with other schemes aimed at accelerated assessment, such as the “early access to medicines scheme” operated by MHRA. At the core of this scheme will be multi-stakeholder collaboration, allowing for the collection of input from healthcare professionals, patients, the National Health Service and the National Institute for Health and Care Excellence, the health technology assessment body in the UK.

Reduced biosimilar data requirements

The MHRA has also issued a draft guideline that aims to reduce the clinical trial data requirements for biosimilar submissions by removing the need for a comparative efficacy trial in the majority of cases. MHRA has proposed that a comparative efficacy trial would not generally be considered necessary when sufficient comparative analytical and functional data are available and that therefore the need for clinical efficacy/safety trials of biosimilars would apply only to exceptional cases.

This is in line with the MHRA’s position highlighted in a recently published paper that comparative biosimilar efficacy trials are "unnecessary to demonstrate that drug candidates are equivalent to reference products" and that extensive analytical testing and a pharmacokinetic study are "usually sufficient."

The MHRA has always strived to facilitate innovation and create a regulatory system within which the development of life-saving therapies can thrive. Alongside driving the thinking on how best to utilize the potential of RWE in generating meaningful data, MHRA has also been at the forefront of harnessing the potential of digital technology and artificial intelligence to advance healthcare. The rapid pace of regulatory adaptations necessitated by the COVID-19 pandemic has set the example for swift regulatory transformation. The MHRA has publicly expressed its intention to stay harmonized with international standards and seek a closer alignment with other regulatory agencies, such as the U.S. Food and Drug Administration, to ensure that post-Brexit the UK continues to be an attractive and welcoming place for innovation and investment in the healthcare sector. The proposed changes to the licensing pathway for innovative new drugs and reduced biosimilar data requirements are part of this broad effort.