Joint Clinical Assessments (JCA) can support getting new innovative cancer medicines to patients faster, if we find the right balance in evidence generation and assessment to facilitate national decisions on access for these innovations. This is the main conclusion from a new report developed by Evidera, and commissioned by the EFPIA Oncology Platform.

Our aspiration is to get the latest advances in cancer treatment to patients as quickly as possible. With less than one year to go before the new system is applied to oncology medicines and advanced therapy medicinal products (ATMPs), health technology developers (HTDs) are urgently seeking transparent, pragmatic and evidence-based guidance on how the changes will impact the development, assessment and subsequent introduction of new medicines in national healthcare systems.

EU-level JCAs are a central feature of a new Regulation on Health Technology Assessment (HTA) that will assess the clinical effectiveness of new technologies versus current treatments. JCA reports should support access and reimbursement decisions at national level. The new rules aim to improve and accelerate the availability of innovative therapies for patients, ensure efficient use of resources, and strengthen the quality of HTA by harmonising the approach to clinical assessments across the EU and reducing multiplicative efforts across individual EU HTA bodies.

Details on how this approach will work are urgently needed so that companies developing new medicines can plan accordingly. At EU level, the HTA Coordination Group (HTACG) is finalising how JCAs will operate. The Coordination Group will rely also on proposed methods of how to conduct the assessments prepared in the last years by EUnetHTA21, a consortium of 13 European HTA bodies contracted by the European Commission, as initial input.

The Evidera report simulated a JCA report by applying the EUnetHTA 21 proposed methods guidelines to three cancer medicines that were approved in recent years to understand the implications of the proposed methods on future JCAs in oncology. The analysis resulted in a very wide assessment scope (i.e. a very large number of population and comparator combinations (or PICOs), ranging from 15 to 57) which is expected to happen frequently when utilising the additive proposed scoping approach in the highly dynamic oncology landscape. To address this wide anticipated scope, all three technologies would have required the use of complex analysis methods or real-world data, even in the cases where ‘gold standard’ randomised controlled trials were available. In addition, JCA should also consider all oncology-relevant endpoints (ORE) not just overall survival (OS), which the methods guidelines do not prioritize. A variety of important endpoints, beyond overall survival, are routinely collected and should be considered as they capture different impacts of a treatment on patients lives, as well as downstream relevance for patient-clinician decision-making. The EUnetHTA21 guidelines do not consider the specificities of medicines for cancer and novel therapies that use genes, tissues and cells. If applied from 2025, this approach would hamper access to some of the most cutting-edge products in the research pipeline.

Treatment of cancer is one of the most dynamic areas of medicine with ground-breaking therapies currently being developed. It requires a suitably flexible approach to how therapies are evaluated and how new treatments are compared to existing products.

“We have seen enormous progress in oncology and, considering the rapidly advancing science, there are more breakthroughs on the horizon. We want to match that innovation in how we utilise the new HTA Regulation to create faster access for patients across Europe. Collectively, we need a clear, workable and predictable framework for all stakeholders to be fully functional by the time the first products are assessed,” said Nathalie Moll, EFPIA Director-General.

“EFPIA looks forward to working with the Coordination Group, the European Commission and the HTA Stakeholder Network to help define optimal implementing arrangements and guidelines that will enable the future system to deliver on its objective of bringing medicines to patients faster,” she added.

Recommendations

The EFPIA Oncology Platform has proposed concrete measures to address the shortcomings identified in the new report, including:

• Ensure meaningful and timely involvement of health technology developers, clinical experts and patients in defining how new therapies will be assessed under JCAs.
• Utilise an optimised evidence-based scoping process
• Provide comprehensive and flexible advice, critical to accommodate for the dynamic oncology treatment landscape.
• Leverage state-of-the-art methodology and all available evidence, including real-world data and the latest data synthesis techniques.
• Consider the totality of oncology-relevant endpoints, going beyond survival alone to include cancer-free survival, quality of life and patient-relevant outcomes.

Read the report in full.