Emergence of the Joint Clinical Assessment in the European Union

Anne Heyes, MBA
Vice President, Head of Value & Access, Europe
RTI Health Solutions

Sweeping Developments in Health Technology Assessment and Their Impact on Manufacturers Throughout the Pharmaceutical and Medtech Landscape

The Inflation Reduction Act (IRA) was implemented in the United States in 2023. Its impacts are already being felt – the first 10 drugs have been selected for the new Medicare Drug Price Negotiation Program.

The European Regulation on Health Technology Assessment (HTAR) is due to be introduced in January 2025 for new oncology medicines, advanced therapies (also known as cell and gene therapies), and medical technologies. Therapies for rare diseases will be introduced from January 2028 and for all new medicines starting January 2030. And yes: line or label extensions, known as type 2 variations at the EMA, also are included in the legislation.

The European HTAR will introduce joint clinical assessments and joint scientific consultations, which will significantly alter the requirements and procedures for HTAs, market access, and pricing across Europe.

In addition, the European Union (EU) has proposed a sweeping revision of its pharmaceutical legislation – the largest change in 20 years. The new law, expected by 2025 or 2026, aims to shorten the period of regulatory protection. However, it incentivizes the development of therapies that address high unmet medical needs as well as the use of comparative clinical trials, among other factors.

How is the Pharmaceutical Industry Responding to These New Practical Realities?

We see companies responding on several levels.

On the portfolio level, because small molecules or multiple-indication orphan drugs are disincentivised by the IRA, companies might consider rebalancing their investments to include more large-molecule drugs or biologicals instead.

On the disease-area level, companies might be thinking of prioritising the larger indication therapies, because the IRA clock starts with the first approval. They might also do so because of the additional workload and the uncertainties around the initial EU JCAs. Companies could find choosing to pursue research for the larger rather than the smaller and rarer populations more justifiable.

For individual drugs in development, we will see changes in strategy, particularly for phase 2b or 3, where pivotal trials are being designed or have already started. Manufacturers are equipping themselves to address potential challenges they may face in drug development as they move toward regulatory approval and market access in the US and EU.

They will need to rethink or even more closely align their strategies for clinical development, regulatory processes, market access, and pricing. Integrated evidence planning and evidence generation will be extremely important. Early preparation to address the specific perspectives and priorities of all the various healthcare stakeholders, particularly those of the payer and HTA bodies, is crucial.

How is RTI Health Solutions Helping Clients Address These Challenges?

We support clients on portfolio and disease-area levels as well as with individual drugs in development. Our experts have first-hand experience with the IRA and an in-depth understanding of the EU HTAR, including the JCAs and joint scientific consultations.

On a portfolio and disease-area level, we partner with our clients' own teams to merge their expertise with ours. We can help develop IRA and EU HTAR impact analyses, anticipate which development programs might be affected and in what ways, and formulate actionable recommendations to inform clients’ decision-making processes.

Supporting clients through the entire life cycle of a new product is a particular strength of RTI Health Solutions. From very early on, we help our clients understand disease burdens, unmet medical needs, and patient preferences. We support clinical trial design by incorporating multistakeholder viewpoints, with a particular focus on payer- and patient-relevant endpoints, such as clinical outcomes assessments and patient-reported outcomes measures.

Pharma manufacturers in the 2 largest pharmaceutical markets globally will face complex and wide-ranging changes. We help clients navigate these challenges throughout product development.