The use of real-world data (RWD) and real-world evidence (RWE) to support pharmaceutical decision making has been a dominant trend over the last decade (ranking as the #1 ISPOR HEOR trend for the last 3 years!¹). RWE is now routinely used to support some regulatory, access and pricing decisions however, scepticism remains, and there is still a feeling that RWE is still underutilised.

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.

Welcome to the final article in the Decisive Dialogue series on artificial intelligence (AI).
2024 was an impactful year for manufacturers employing AI in their evidence generation or developing AI as a medical device (AIaMDs) in the UK.
This article will summarise key events of 2024 from the UK AI market access space and highlight interesting milestones for 2025.

Decisive Consulting and uMed announce partnership to drive data-driven market access success in Cardiometabolic and Parkinson's Disease research 

New collaboration launches at ISPOR 2024, offering pharmaceutical and biotech companies rapid access to unique, customisable data and expert guidance to bridge HTA evidence gaps. 

PRESS RELEASE: London, UK, 18 November 2024

The Spanish Registry of Clinical Studies (REec), managed by the Spanish Agency of Medicines and Medical Devices (AEMPS), has reached a significant milestone by publishing its 10,000th clinical trial.

The MHRA’s 2024/25 business plan offers exciting opportunities for pharmaceutical companies looking to bring innovative medicines and devices to market. Key priorities include maintaining public trust, improving regulatory excellence through strategic partnerships, and enhancing service delivery with new IT systems like SafetyConnect and RegulatoryConnect. The plan also supports UK life sciences growth, pandemic preparedness and sustainability efforts in healthcare product regulation.

We are now less than three months away from the introduction of the Joint Clinical Assessment (JCA) for ATMPs and oncology products – representing a significant landscape shift for the European Pharmaceutical sector.  

Faced with the many new treatments in development for Heart Failure and the prospect of assessing value relative to effective and largely genericised standard of care – should we start to think differently about how we classify and measure effectiveness in heart failure?

Personalised medicine is altering the healthcare landscape by providing individualised therapeutic methods based on unique patient characteristics. This shift presents a complex array of opportunities and challenges for manufacturers developing precision therapies, particularly in navigating the evolving health technology assessment (HTA) processes.

Australia publishes recommendations for a new HTA roadmap to improve market access and faster patient uptake of new medicines by the end of 2024

There is consensus among patients, clinicians, industry and the government that the time to access new health technologies in Australia is too long and that HTA methods must be sustainable for future innovations in medical technology.

The Innovative Medicines Fund was launched in June 2022 to ensure patient access to promising non-cancer medicines when further data are necessary to resolve outstanding uncertainty. Until recently, only four products had received interim funding through the IMF and there were no formal NICE recommendations for managed access under this agreement.
On 24th July 2024, NICE published the final draft guidance for Hemgenix (etranacogene dezaparvovec), which was ‘recommended with managed access as an option for treating moderately severe or severe haemophilia B (congenital factor IX [FIX] deficiency) in adults without anti‑FIX antibodies’.

On 3rd June 2024, a new collaboration, Joint Nordic HTA Bodies (JNHB), was announced, aiming to streamline health technology assessments (HTAs) in the Nordics through joint HTA assessment of medicinal products.

This second article on the current and fascinating developments in artificial intelligence (AI) will dive deeper into the evaluation of synthetic control arms (SCAs). If you are new to synthetic data or missed the first article, make sure to catch up here. As a reminder, synthetic data are created by generative AI models trained to learn the patterns and statistical properties of a sample real-world data set.

Spain has become a global leader in clinical trials due to the strength of its healthcare system and professionals, pioneering regulatory work, public-private collaboration, and the generosity of patients. Farmaindustria highlights these factors, especially on International Clinical Trials Day, while also pointing out new challenges to maintain this leadership.

In recent weeks, Germany's Federal Council (Bundesrat) voiced their disapproval of the proposal for confidential discounting following a read-out of the Medical Research Act (MFG), echoing the same recommendation to reject the proposal reached by the Federal Health Committee.

Earlier this year the UK Department of Health and Social Care (DHSC) concluded a consultation on its revised proposals to update the 5-year Statutory Scheme for cost-containment of NHS spending on branded medicines, with additional updates phased in throughout the year, here we take a look at the most recent changes

The Italian Medicines Agency (AIFA) has restructured its evaluation process by merging the Technical-Scientific Advisory Commission (CTS) and the Pricing and Reimbursement Committee (CPR) into a single entity, known as the Commissione Scientifica ed Economica del farmaco (CSE).

This new guideline provides recommendations for setting criteria and procedures that govern drug pricing decisions and market entry or withdrawal of new pharmaceuticals, offering a framework for their subsequent re-evaluation.

At Decisive Consulting, we are exploring the complex considerations surrounding access to innovative gene therapies for sickle cell disease, a rare disease with high unmet need.

In celebration of International Women’s Day 2024, Decisive Consulting are proud to explore and provide insight for our community on the importance of advocating for inclusion in healthcare and academia.