Navigating EU Joint Clinical Assessments & Joint Scientific Consultations from January 2025 Onward
Emma Hawe, MSc
Head, Data Analytics and Design Strategy
RTI Health Solutions
Transcript:
What Do We Need to Know About Joint Clinical Assessments (JCAs)?
Joint clinical assessments centralise the clinical domains of the Health Technology Assessment process on a European level. Non-clinical HTA domains, which mainly involve economic evaluation, remain with the national HTA bodies of the 27 member states. The national decision-makers also retain the final appraisal of a medicine and its recommendation for reimbursement for subsequent pricing negotiations.
The aim of joint clinical assessments is to increase the quality, transparency, and timeliness of clinical HTA so that access to innovative medicines is accelerated for EU patients. Whether the new legislation and centralised process will meet these aims remains to be seen.
It is important to understand that the joint clinical assessment process runs in parallel to the marketing authorisation process of the European Medicines Agency.
The goal is endorsement of the final joint clinical assessment report within 30 days of the European Commission decision to approve the medicine, which puts considerable time pressure on the entire process. It is fair to say that a good part of this pressure is placed squarely on the shoulders of the manufacturer. The process is kicked off with the submission of the regulatory dossier to the EMA.
In practice, this implies that the HTA bodies are now firmly in the driving seat and the manufacturers no longer retain control over when to launch the HTA process. This assumes the submission is subject to the EU HTA Regulation, which applies to all new oncology drugs and cell and gene therapies from January 2025, orphan medicinal products from January 2028, and lastly, all new drugs from 2030 onward.
It also means that the clinical efficacy and safety evidence submitted as part of the EMA regulatory dossier will effectively be mirrored in the joint clinical assessment, placing an even greater emphasis on the importance of including payer-relevant endpoints and comparators in the pivotal, registration-enabling clinical trials.
Additional Time Pressures for Manufacturers
Many of the details surrounding joint clinical assessments remain to be reconfirmed in the final Implementation Act, which completed public consultation in April 2023. We are eagerly awaiting its publication.
From the manufacturer’s perspective, there are several key inflection points in the joint clinical assessment process. The first is the receipt of the request for dossier submission from the HTA Coordination Group. It will include the scope of the assessment, the deadline for submission, the dossier template, and its requirements.
As currently outlined, the deadline for dossier submission is within 90 days from the date of the request. This is where the scope and requirements of the request become crucial. The scope is defined by the PICOs, which stands for Population, Intervention, Comparator, and Outcomes.
Manufacturers will have little opportunity to interact with the PICO scoping process. That means that they will not have enough information up front to adequately prepare for the joint clinical assessment. The time to gather, analyse, and submit the requested information will be extremely limited.
Without the ability to comment on the PICOs, manufacturers are concerned that requests for subpopulations, comparators, and outcomes might be included that cannot possibly be addressed in a scientifically adequate way within the 90-day deadline.
This could especially be the case for complex, less well-known and understood conditions with smaller, heterogeneous populations where the standard of care across the EU might vary considerably.
Joint clinical assessment will require additional data analyses within an extremely compressed timeframe, putting pressure on pharma biostatisticians, epidemiologists, and real-world researchers. The open question is: will this framework and timeline be effectively workable in practice, or will it result in a loss of scientific quality?
There appears to be a very limited amount of time in the joint clinical analysis process for companies to respond to clarification questions and additional information requests. It also leaves little time to perform factual checking of the final report.
How are Companies Responding to Expected Challenges with Joint Clinical Assessments, and What is RTI Health Solutions Doing to Support Them?
We see our clients preparing to navigate joint clinical assessment in several ways. On one hand, companies are adjusting their organisational structure, capacities, and resourcing because joint clinical assessment centralizes the clinical domains of the HTA process that formerly resided within each country.
On the other hand, companies are seeking greater alignment between the preparation of the EMA regulatory dossier and the joint clinical assessment, as both procedures will be running in parallel. This emphasises the need for joint ownership of regulatory affairs and market access functions on an EU level.
Given the timeline and scoping challenges, success will depend on anticipating the PICOs likely to determine the scope of the joint clinical assessment dossier request.
As with our comprehensive support in preparation for global HTAs, RTI Health Solutions works in advance with clients to anticipate likely and appropriate PICOs. We prepare the highest quality evidence in advance, and nimbly adjust to the final scope and label changes.
In addition, we provide a range of strategy, planning, evidence preparation, and support services. Given the 3-month cut-off for the JCA dossier, epidemiology, and indirect treatment comparisons, these services include systematic literature reviews and timely updates.
Continuous stakeholder engagement will be essential, including early dialogues with patients, caregivers, payers, and HTA opinion leaders. Early scientific advice concerning the joint scientific consultations come into play.
Joint Scientific Consultations
Joint scientific consultations are a non-binding scientific advice procedure in parallel consultations with participating HTA bodies and the EMA. They are being introduced together with the joint clinical assessments and are typically provided before the start of pivotal clinical trials.
The aim is to provide common EMA and HTA recommendations for the design of the planned registration-enabling clinical studies. That includes fundamental criteria, such as patient populations, comparator selection, relevant endpoints, study design, and the economic evidence generation plan.
The ultimate success of joint clinical assessments and market access in Europe will increasingly hinge on including key payer-relevant endpoints and comparators in a product’s pivotal clinical trials, with submissions occurring in parallel to the EMA and the JCA.
At RTI Health Solutions, we support clients along the entire scientific advice process, including the preparation of joint scientific consultation briefing books. We can analyse and interpret the scientific feedback clients receive and develop tailored recommendations for the design of clinical trials that specifically address requirements of the EMA and HTA bodies.
