How regulation can help to unlock the potential of preventative medicines

Sarah Montagne, Bayer Senior Regulatory & Policy Advisor (UKI), ABPI Regulatory Science Board Sponsored Group, Dr Dan O’Connor, ABPI Director of Regulatory Policy and Early Access and Andy Collier, Regulatory Policy Manager, share reflections from an ABPI assembled expert roundtable held on Tuesday 28 May 2024.

If the health of the nation is to improve and pressure on the health service is to ease, preventative strategies must play a pivotal role. In all quarters, from industry and academia to patients and policymakers, there is a growing consensus that prevention must become a more significant part of health policy and pharmaceutical development programmes. Doing this will require a sustained commitment and targeted work to develop a proactive approach to poor health outcomes and rebalance the delivery of healthcare.

Any successful plan will require better patient access to preventative medicines that can either halt or suppress the onset of a disease or its symptoms. It will include products such as vaccines and new active substances, as well as repurposed older medicines.

ABPI members are increasingly active in this innovative area, developing products for some of the biggest national and international health challenges, including HIV/AIDs, Alzheimer’s, Cancer and Diabetes.

Roundtable on preventative medicines

In order to help advance the opportunities for developing preventative medicines, the ABPI held a regulatory-focused roundtable event with multiple stakeholders on 28 May. The subjects discussed included:

• The specific challenges to resolve when successfully developing medicines in the preventative space.
• How the regulatory system could evolve to support and optimise the field.
• Are there activities, incentives and policies that could help
• How to balance evidence generation from formal clinical trials with real-world data collection.

Current approval pathways and associated regulatory requirements are mainly positioned for treatment in an acute care setting, where clinical efficacy and safety is determined in patients with frank symptoms. Attendees of the roundtable agreed that the regulatory framework could evolve to manage the additional challenges of earlier intervention better. These changes include the need to efficiently and timely validate often novel surrogate markers as clinical trial endpoints, recognise and mitigate the uncertainty in the approach to ensure optimised dosing aspects (when to intervene and for how long), and help development methods to determine the unique benefit/risk balance. Finally, future changes should also recognise the difficulties in collecting long-term data outside of clinical trials in the health system for post-authorisation regulatory commitments.

Key points of the discussion highlighted:

• A need for streamlined regulatory approaches for developing surrogate biomarkers as endpoints in clinical studies and in the real world.
• Subject to regulatory acceptance, breakthrough technologies such as artificial intelligence (AI), in silico, and novel non-clinical models could help generate data to reduce the uncertainties in the development programme.
• Opportunities for data collection are often fragmented and sourced from varied and disparate systems, making the landscape difficult for companies to navigate. A clear data strategy from the MHRA and health system could help align expectations and opportunities.
• The regulatory framework must consider the critical role of diagnostics and the interplay between the two regulatory frameworks, reflecting the partnership working that is needed to improve outcomes in this space.
• Novel approaches to collecting patient-reported outcomes could be helpful in determining the long-term safety and effectiveness of preventative medicines delivered through digital health solutions and wearable technologies.
• There needs to be an alignment of evidential standards for medicines regulatory approval and HTA decisions.

Summary

Preventative medicines offer the potential to improve health outcomes and deliver broader economic benefits. The regulatory system should be optimised to enable better development and licencing of preventative medicines. Enabling the rollout of preventative medicines at scale could support progress towards UK public health goals, while offering a chance of global leadership in a critical area of innovative medicines.

A visible and accommodating regulatory and access pathway that recognises the unique challenges is part of the solution. A future ABPI roundtable will examine the issues raised with the goal of developing a whitepaper for wider policy consideration and implementation.