2024: a year in review
2024 has marked a transformative period for healthcare policy across the UK and European Union. As we approach year-end, Decisive Consulting examines the substantial policy developments across major European markets and analyses the anticipated changes for 2025, exploring their implications for the healthcare industry. The past 12 months have seen shifts in regulatory frameworks and assessment procedures, particularly in preparation for the European Joint Clinical Assessment (JCA). From the integration of artificial intelligence to advances in personalised medicine, alongside evolving market access pathways and policy reforms, the healthcare landscape continues to present both challenges and opportunities for all stakeholders.
European Joint Clinical Assessment
We are now less than 1 month away from the introduction of the JCA for ATMPs and oncology products, effective 12th January 2025. The JCA aims to streamline health technology evaluations across member states by providing centralised clinical assessments of new treatments and represents a significant landscape shift for the European Pharmaceutical sector.
Following a year of many updates from the HTA Coordination Group (HTACG), Health Technology Developers (HTDs) must prepare for challenges presented by the need to generate evidence that reflects the requirements of all 27 member states in a single JCA submission.
Early prediction of PICOs (population, intervention, comparator, outcome) for pipeline assets likely to be within the JCA process: early preparation to understand the likely PICOs for medicines undergoing JCA, and knowing how to respond to them or provide a strong rationale for why it is not possible to respond is going to be essential to adhere to the short timelines
Ensuring robust indirect treatment comparisons: the need to demonstrate comparative efficacy will be paramount, and indirect treatment comparisons (ITCs) are set to play a pivotal role in this process
A small number of HTDs will experience the JCA process in 2025 with the outputs seen in late 2025 or early 2026. For all other HTDs, 2025 is an opportunity to build JCA knowledge and understanding across their organisations, predict PICOs, enhance evidence plans and adapt workflows.
Evolving EU4 and UK healthcare policy landscape
England
Various reforms in England in 2024 aimed to create a more efficient, patient-centric healthcare system while fostering an environment conducive to medical innovation in 2025 and beyond.
The 2024 Voluntary Scheme for Branded Medicines Pricing, Access, and Growth (VPAG) was introduced, emphasising collaboration between the NHS, NICE, and the pharmaceutical industry to improve patient access to innovative treatments while ensuring cost-effectiveness.
The MHRA’s 2024/25 business plan emphasises enabling access to innovative healthcare products, regulatory agility and global collaboration further building on the 2023 pro-innovation agenda.
Key priorities include maintaining public trust, improving regulatory excellence through strategic partnerships, and enhancing service delivery with new IT systems like SafetyConnect and RegulatoryConnect
2024 saw the first marketing authorisation through the new International Recognition Procedure (IRP), details of which were unveiled 2023. The first opinion was issued in February, for a new formulation of monoclonal antibody XGEVA (denosumab), with the process taking just 30 days
Updates were also provided on the UK's MedTech regulatory roadmap, outlining plans to implement future medical device regulations. This initiative aims to enhance patient safety and promote innovation within the medical technology sector
The Innovative Medicines Fund (IMF) recommended Hemgenix as its first treatment, setting a precedent for funding advanced therapies targeting rare and severe conditions. This milestone underscores the government's commitment to expanding patient access to cutting-edge treatments through specialised funding pathways.
Document A was removed from NICE submission requirements in a significant procedural update, simplifying the evaluation process while maintaining rigorous standards for safety, efficacy, and quality.
Germany
On 4th July 2024, the Ministry of Health and cabinet supported the proposal for confidential rebates in the Medical Research Act (MFG), despite prior concerns from the Bundesrat around transparency. This now allows confidential price negotiations for manufacturers with active research and development departments in Germany. The provision is set to terminate on 30th June 2028, unless extended.
The proposed confidential rebates are expected to apply selectively to a limited number of drugs each year; it is anticipated that companies will seek these rebates only in specific cases due to administrative burdens. The cabinet emphasised negotiation flexibility and established a Federal Ethics Commission to address industry and scientific concerns. By the end of 2026, the Ministry of Health will assess the impact of confidential rebates on market withdrawals, savings, and any unintended outcomes, such as potential increased administrative costs and changes in drug expenditures.
Automatic substitution for biosimilars was also introduced in March 2024 aiming to address cost efficiencies and marking the first of such regulations in Europe.
France
An emphasis on increased access and rewarding innovation were key themes in France this year.
The PLFSS (Social Security Financing Bill) update focussed around four key pillars to increase access: higher pay-back trigger, temporary reimbursement scheme to bridge between early access and reimbursement, clawbacks to reduce drug supply shortages and environmental waste and biosimilar substitution to increase cost-effective patient access.
A direct access programme was introduced as a 2-year pilot to streamline access to innovative treatments with immediate reimbursement for medicines with major medical benefit (SMR major), full reimbursement for up to 1 year and a clawback clause for price adjustment after negotiations. Hemegenix was the first medicine authorised under the programme at the end of 2023 with a ~ 2-month lead time.
Italy
In April 2024, AIFA introduced the Commissione Scientifica ed Economica del farmaco (CSE), restructuring its evaluation process by merging the Technical-Scientific Advisory Commission (CTS) and the Pricing and Reimbursement Committee (CPR). The CSE aligns closely with European guidelines, incorporating reference to the JCA framework for consistency across the region. This consolidation aims to streamline processes, ensure universal access to innovative and essential medicines, and adopt a pricing policy that rewards therapeutic value.
Spain
A new health technology assessment (HTA) model was introduced in 2024, set to be implemented at the end of the year through a Royal Decree, to enhance the evaluation and decision-making process with a three-level work structure (giving clear separation between evaluation, positioning and decision making).
Integral to the updates is the integration of clinical assessment with cost-utility analyses using Quality-Adjusted Life Years (QALYs). Robust evidence from randomised controlled trials is mandated, and detailed cost assessments, including VAT considerations, are required. Inclusion of a balance of stakeholders in the decision-making process, including patients and healthcare professionals, is also a priority.
Outside of the EU4 and UK, efforts to harmonise regulatory frameworks and an emphasis on collaboration are gaining momentum in a bid to continue expediting patient access to therapies, particularly in areas of high unmet need.
Other areas to keep an eye on in 2025
Advancements in AI and market access
The UK has made significant strides in artificial intelligence (AI) integration within healthcare. The Medicines and Healthcare Products Regulatory Agency (MHRA) responded to the government's pro-innovation white paper by outlining reforms to the medical device classification system, emphasising the need for demonstrable benefits and robust validation of AI applications. Anticipated guidance in 2025 will aim to align regulatory processes with innovation principles further. This push for AI integration highlights the UK's commitment to maintaining its global position as a leader in healthcare technology innovation.
In 2025, NHS England will begin trialling an AI tool that is able to predict patients at risk of type 2 diabetes as many as 13 years before they develop the condition. Early detection with the AI-ECG risk estimation for diabetes mellitus (Aire-DM) could prevent the condition by prompting changes to lifestyle and diet. The trial will take place at Imperial College Healthcare NHS trust and Chelsea and Westminster hospital NHS foundation trust, making NHS England the first health system globally to use such a tool.
Elsewhere, the use of AI to generate real world evidence (RWE) is being increasingly explored both within the framework of the JCA and on a national level. Key developments include:
Growing recognition of AI’s potential to enhance RWE generation in support of EU JCA assessments, in particular in oncology and advanced therapies
In 2025, expanded use of medical data in Germany will open up opportunities for AI companies to develop tools for MedTech, facilitating the integration of AI in generating evidence for market access. Two laws regarding healthcare data were approved in 2024, and both will come into force 1st January 2025: the Law to Accelerate Digitalisation in the Health Sector and the Law to Improve the Use of Health Data
Personalised medicine
An ongoing shift toward personalised medicine is altering the healthcare landscape, presenting a complex array of opportunities and challenges for manufacturers developing precision therapies, particularly in navigating the HTA processes.
The challenges are multifaceted: traditional randomised controlled trials often struggle to demonstrate value in highly stratified patient populations, while the increasing reliance on RWE introduces new complexities in data quality assessment. Additionally, smaller patient subgroups and complex treatment pathways create greater uncertainty in cost-effectiveness estimates, requiring more sophisticated approaches to value demonstration.
As HTA agencies refine their methodologies to accommodate this rapidly advancing field, a deep understanding of the changing regulatory and reimbursement environment will be paramount for successful market access. Effective engagement and collaboration between manufacturers and HTA bodies will be crucial to harness the benefits of personalised medicine while ensuring equitable access.
Looking Ahead
This review highlights the significant change and progress experienced by the healthcare sector in 2024. With the imminent introduction of the JCA on the horizon, increased emphasis on personalised medicine, and the integration of AI, 2025 promises to be another exciting and challenging year. Stakeholders will need to remain proactive, responsive and adaptable to navigate the evolving regulatory and market access environment effectively and ensure the efficiencies and benefits offered by the changes are translated into improved patient outcomes.
Written by Eleanor Butler
Decisive Dialogue 27th December 2024