EU HTA: If we want faster access to cancer treatment for all Europeans, we need to get it right 

Although more people are being diagnosed with cancer, deaths are slowing down. New therapies, along with advances in prevention, screening and new treatment modalities, are improving and saving lives every day. For example, the number of lung cancer patients that are alive one year after diagnosis is more than 10 times higher than in 1995.

While there is still much work to do if we are to further improve outcomes for all cancers, we see opportunities to usher in new waves of innovation.

How will the new EU Health Technology Assessments (HTA) Regulation facilitate improving cancer outcomes?

Our aspiration is to get these treatments to patients as quickly as possible. However, the imminent implementation of the HTA Regulation and introduction of Joint Clinical Assessments (JCA) in 2025 create uncertainty about the speed at which breakthrough cancer treatments will reach patients in the future.

via EFPIA

EU-level JCAs will be used to inform the value rating of a medicine and ultimately pricing and reimbursement decisions at national level. The new rules aim to improve the availability of innovative medicines for patients, ensure the efficient use of resources and strengthen the quality of HTA by harmonizing the approach to clinical assessments across the EU and reducing multiplicative efforts across individual EU HTA bodies. We share the objectives of HTA reform. However, at the same time we are concerned about how JCAs will work in practice based on the current proposals.

With fewer than seven months until the future EU HTA system becomes operational, details on how this approach will work are urgently needed so that companies developing new medicines can plan accordingly. For companies developing innovation in the oncology and advanced therapy medicinal products, the EU HTA is no longer a theoretical exercise. They are now preparing JCA dossiers for technologies expected to undergo the European Medicine Agency’s review starting in January 2025 and making the necessary changes to internal operating models to ensure readiness. To get it right for patients, they urgently need additional clarity on the procedures, methods and timelines for the new system.

As science evolves, the EU HTA system should adapt

To help inform this urgent conversation, the EFPIA Oncology Platform commissioned Evidera to undertake detailed research on the proposed reforms. The report applied the EUnetHTA 21 proposed methods guidelines to three recently approved cancer medicines to understand the implications of the proposed methods on future JCAs. Findings show that these guidelines do not consider the development context and specificities of innovative therapies. The research also finds that, if applied from 2025, this approach would hamper access to some of the most cutting-edge products in the research pipeline.

A critical part of the future JCAs will be the scope, or the set of data analysis that are requested for each new medicine application. Evidera’s analysis resulted in a very wide assessment scope (i.e. a very large number of population and comparator combinations, ranging from 15 to 57), which is expected to happen frequently when utilizing the additive approach in the highly dynamic oncology landscape. To address this wide anticipated scope, all three technologies would have required complex analysis methods or real-world data, even in the cases where ‘gold standard’ randomized controlled trials were available. Furthermore, an arithmetic proliferation of analyses, based on such a wide scope, which may not even be fully relevant or used at national level for decision-making, would be detrimental to everybody involved.

Getting it right for patients

More flexibility, pragmatism and closer dialogue with the stakeholders impacted by the JCA (patients and clinicians, but also the company developing the innovative technology) in the application of the methodologies is needed.

For example, in many cancers, survival is improving and, for others, is becoming a chronic condition. JCAs should also consider all oncology-relevant endpoints, not just overall survival, which the methods guidelines do not prioritize. How we assess benefit needs to reflect the specificities of modern cancer treatment, and a wide range of measures and endpoints should be considered useful and assessed in a JCA, as they capture different impacts of a treatment on patients’ lives, as well as downstream relevance for patient-clinician decision-making.

The EU HTA Regulation gives the EU an opportunity to develop a world leading HTA system, but it mustn’t become an additional bureaucratic barrier that delays access. Europe should seize this opportunity and apply state-of-the-art methods to assessments and not use the lowest common denominator.

Based on research by Evidera, the EFPIA Oncology Platform has developed the following recommendations designed to address the challenges that may arise from JCAs:

• Ensure meaningful and timely involvement of health technology developers, clinical experts and patients in defining how new therapies will be assessed.
• Utilize an optimized evidence-based scoping process with clear methodology on how to identify and harmonize EU JCA data needs, while achieving the regulation’s efficiency objectives.
• 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 HTA data synthesis techniques.
• Consider the totality of oncology-relevant endpoints in JCAs going beyond survival alone to include cancer-free survival, quality of life and patient-relevant outcomes.

Delivering for patients

The EU HTA Regulation is a fantastic opportunity to speed up access to medicines for patients in Europe. At a time when science in oncology is transforming how we treat the disease, we want to match that innovation with how we utilize 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. EFPIA is ready to work 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.