One of the main challenges of the European Joint Clinical Assessment (JCA) does not lie in preparing the dossier itself. It lies in ensuring that the available evidence can address the questions raised by the different Member States.
A comparator relevant to a healthcare system that was not included in the pivotal trial. A subpopulation for which no specific analyses are available. Or clinical outcomes that have not been measured consistently across the available studies.
These are situations that require going beyond the evidence generated through clinical development and building an evidence strategy capable of addressing the requirements of the European assessment with methodological rigour.
We are currently involved in 4 of the 19 Joint Clinical Assessment procedures underway in Europe, spanning oncology, medical devices and advanced therapies. This experience gives us first-hand insight into the challenges of a system that is still in the early stages of implementation, where the ability to anticipate evidence needs and synthesise the available evidence is critical.
One hundred days that involve much more than drafting
Once the final assessment scope has been communicated, the standard timeframe for submitting the dossier is 100 days, or 60 days for certain procedures.
The challenge is not simply the timeframe. It is the complexity of addressing multiple PICOs, covering populations, interventions, comparators and clinical outcomes, that reflect the needs of different healthcare systems across Europe.
Each PICO requires the relevant evidence to be identified, its quality to be assessed, and a determination to be made as to whether it supports a direct comparison or whether alternative evidence synthesis methods are required.
The difference between being prepared and starting from scratch may lie in the work completed before the final scope is known.
Anticipating PICOs, preparing the responses
Although the final scope is not known in advance, many of the questions that may arise can be anticipated.
Analysing clinical practice across countries, their relevant comparators and previous assessments makes it possible to develop potential PICO scenarios and assess them against the available evidence.
This is where the methodological work begins in earnest: conducting systematic literature reviews (SLRs), identifying evidence gaps, assessing the feasibility of indirect comparisons and preparing additional analyses where the data allow.
The aim is not to generate evidence where none exists, nor to force comparisons that cannot be methodologically justified. It is about making the best use of the available evidence, understanding its limitations and developing the most robust possible response for each PICO.
Our involvement in these early procedures is demonstrating the extent to which this exercise requires a combination of knowledge of national HTA systems, expertise in evidence synthesis and the ability to adapt to a new and demanding methodological framework.
A common assessment, multiple evidence needs
The new European framework aims to reduce duplication through a shared clinical assessment. However, a common assessment does not mean that every country has the same evidence needs.
Differences in clinical practice, therapeutic alternatives and relevant populations remain reflected in the definition of the PICOs. And once the JCA has been completed, each Member State retains responsibility for assessing added clinical benefit and making its own pricing and reimbursement decisions.
For this reason, the evidence strategy needs to address the requirements of a European assessment while remaining aligned with the specific needs of national healthcare systems.
Where the difference lies
With orphan medicinal products coming within scope in 2028, followed by all other medicinal products covered by the Regulation in 2030, an increasing number of companies will need to address this challenge.
Experience from the first procedures shows that preparing for a JCA goes far beyond completing a dossier. It requires anticipating potential scenarios, systematically reviewing the evidence, selecting appropriate comparative methods and managing uncertainty transparently.
In a JCA, having evidence is not enough. The difference lies in knowing how to use it to address the questions that will actually be assessed.
If your medicinal product is approaching a Joint Clinical Assessment, anticipating potential PICOs and establishing a robust evidence strategy can make a meaningful difference. Let’s talk.