Does the EU HTAR really put patients at the center?

As Antonella Cardone, CEO of Cancer Patients Europe (CPE) has noted, broadening access is a priority since innovation with medicines has little value if patients can’t access them2.

By centralizing the health technology assessment of clinical evidence of new products, the JCA process has the potential to address issues such as the access delay that is caused by differing evidence requirements across the EU. As of January 2025, the JCA process will become mandatory for oncology drugs and advanced therapy medicinal products (ATMPs).

In a recent interview with Cencora, Cardone said CPE welcomes the spirit of the HTAR in aiming to accelerate “patient access to treatments across Europe by increasing transparency and reducing duplication of assessment efforts.”

However, she added that the organization is concerned with how this will be achieved and that it does not lead to delay and duplication of work at the country level.

Currently, access to medicinal products differs widely across the EU. This affects both the speed at which products get to patients in different markets, as well as the restrictions placed on labels. 

From a speed perspective, the gap from marketing authorization to gaining access to the reimbursement list is vast, on average just 37 days in Germany compared with 887 days in Romania³. 

When it comes to label restrictions, in Germany, 87% of oncology products approved by the European Medicines Agency (EMA) have full, unrestricted availability, while in Poland that unrestricted figure is as low as 8%, according to data from the European Federation of Pharmaceutical Industries³. 

Concerns over the assessment process
The PICO (Population, Intervention, Comparator, Outcomes) framework will be used to define the key questions that need to be answered during the clinical assessment, such as the relative effectiveness and safety compared with other treatment options⁴. The national health technology assessment (HTA) bodies are expected to align on particular comparators and endpoints within the JCA process. 

The expectation is that by harmonizing the HTA process, the speed and access barriers for many patients in the EU will be addressed. 

At the same time, questions about the assessment process remain. Cardone noted in her interview with Cencora that, while in the past, cancers such as breast or lung cancer were traditionally considered to be one disease,  innovative diagnostics and the identification of specific biomarkers have characterized these as many diseases treated with targeted therapies and addressing a smaller population.

“We fear that innovation in diagnostics and targeted and precision oncology are not reflected in the new EU HTA Regulation,” she said, adding that these cannot be assessed with the same methods as traditional health technologies. 

“The evidence of the clinical added benefit to patients and societal impact cannot only be assessed through randomized clinical trials or large clinical trials.”

“Treatment comparison and confirmation of lifetime durability, such as patient-free survival (PFS), do not respond to the same traditional criteria. Applying traditional criteria would significantly delay the process and delay access to some of the most innovative treatments in some of the hardest to treat conditions. A similar situation would happen for rare cancers later in the JCA implementation process for rare diseases.”

Rather, Cardone noted that a pragmatic approach to setting the scope is needed. She stressed that the ideal would be to identify a common comparator and common endpoints to avoid duplication and thereby realize the spirit of accelerating access to new treatments.

Progressing toward harmonization

As the deadline for implementation draws nearer, there has been steady progress in developing the JCA process. The EUNetHTA 21 consortium has developed a draft framework, which will be discussed and signed off by the Member State Coordination Group on HTA, and officially implemented in January 2025.

While the framework can largely be viewed as well considered, it remains to be seen how the patient perspective is incorporated.

“One of our main concerns is about the stakeholders’ involvement and, specifically, patient involvement, as it would be ideal to standardize when and how in the JCA process patient input is expected and how it will be considered,” Cardone said.

She emphasized the importance of relying on primary endpoints and patient-reported endpoints during the JCA assessment process. Evidence should include patient-preference studies, surveys run by patient organizations, and real-world data on the technology or the history of the disease and real-world outcomes.

Elevating the patient voice

As patient groups have noted, without clear commitment in the framework to embedding patient insights from the outset, there is a danger that patient involvement will simply be a tick-the-box exercise. 

Cardone has emphasized the importance of patient-centeredness throughout the HTA process, and of considering patients as partners in that process. Specifically, she has highlighted the need to ensure patients’ perspectives and preferences are considered in the evaluation and adoption of new medical treatments².

In her recent interview with Cencora, she offered concrete examples of how the patient perspective can and should be considered.

“We recommend considering the best practices already existing to standardize and facilitate patients’ input in the JCA, such as the PREFER project’s outcomes, and the Summary of Information for Patients (SIP) template,”

PREFER refers to the Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle project, which seeks to strengthen patient-centric decision-making across the lifecycle of medicinal treatments⁵. 

Cardone explained that the SIP template is a plain-language summary describing a treatment under appraisal and is designed to support patient and carer expert input to HTA. It is typically completed by the industry as part of their HTA submissions, reviewed, and provided to patient and carer experts by the HTA body, she added. 

The SIP approach was developed by the Scottish Medicines Consortium and adapted for international use . It is currently being piloted by NICE in the UK and the Pharmaceutical Benefits Advisory Committee (PBAC) in Australia.  

“The intention is that implementation of the SIP will lead to greater quality patient input into an HTA,” Cardone shared.
She added that clear guidelines should be established for real-world evidence generation and for direct and indirect comparisons. “How they will be used in the JCA process should be determined to avoid the risk of inconclusive recommendations that will translate into a replication of the assessment, delaying rather than accelerating the access to innovative treatments,” she said.

“Anticipating the JCA scoping process would also help accelerate access. We can learn from the European Medicines Agency and start the process much sooner.”

Currently, involvement of patient advocacy groups differs quite significantly across Europe, with some countries giving these groups a more central role in the HTA assessment process. For example, Germany’s G-BA and Belgium’s     RIZIV/INAMI both intend to ensure that the experience of patients, pharmacists, healthcare providers  and regulators are all carefully considered during the decision-making⁷. 

At the same time, it is important that patient advocacy groups are better educated in the relevance of the HTA process and the role they can play in it. While knowledge of the process is generally fairly well established at the European level, the literacy gap tends to be felt more at the country level. Local patient associations tend to be understaffed and focus their effort on providing immediate support to patients. For these associations, the HTA appears complicated to understand and follow.   

Elevating the role of patient groups at the local level is likely to require overcoming both capability and capacity challenges. From a capability perspective, that will require helping people who are experts in their disease to also become representatives in the HTA discussion. For most patient advocacy groups, capacity remains a daunting challenge and consideration needs to be given to providing these organizations with funding and logistical support to ensure they have the resources for the JCA process. 

Cardone has previously stressed the importance of campaigning for experienced resources to support patient engagement throughout the JCA and Joint Scientific Consultations (JCS)  processes, and to build patient organizations’ capabilities across the entire process². In recent guidance from the EUnetHTA 21 JSC Secretariat, it was noted that the “inclusion of patients and clinical experts in Parallel EMA/EUnetHTA 21 JSCs is expected on a routine  basis”⁸.

The guidance states: “European-level patients and/or clinical experts are involved via interview/written statement and participate in the discussion meeting (if applicable). The involved experts express their personal opinions and do not act as representatives for any stakeholder organization.”

She has also highlighted accessibility challenges facing patient organizations, such as language barriers, unrealistic timelines, and complexity of the processes involved.

Cardone told Cencora that although patients are now able to participate in reviewing and commenting on the scope during the ‘consolidation of PICO’, there is no standardized way to provide the information to the individual patients involved. 

“We are concerned that individual patients may not have the ability to understand the topic and effectively contribute to the JCA,” she said. “It is also more complicated, if possible, to verify the conflict of interest of an individual versus that of an organization. A patient organization can build the internal capacity to be part of the process, stimulating the internal debate among a number of patients directly affected by the disease and organizing surveys and focus groups.”

She commented that it is not clear how patient input will be considered, adding that standardizing the input process and making patient input accessible through a public repository will lead to greater transparency. Another way to optimize the process would be through the use of dashboards and scorecards summarizing patient perspectives for EU policy makers. 

“A pre-submission meeting with all the stakeholders involved to discuss the dossier requirements, anticipated data problems, and EMA label timelines could be beneficial to an efficient JCA process,” she said.

The future with JCA

While more information about the framework around the JCA is being made available, it is important to consider that the JCA is currently in a transition phase and that harmonizing 27 countries in preparation for it becoming mandatory in 2025 will require significant effort. As such, all stakeholders need to be open and willing to learn and adapt. However, it is equally important to remember that patients have no time to wait. The voices of patients and patient advocacy groups need to be heard and included by decision makers. 

 

About the authors:
Herbert Altmann, Ph.D., is head of market access, pricing and reimbursement for PharmaLex (a part of Cencora). Casper Paardekooper is partner at Vintura (a part of Cencora).

The contents of this article may contain opinions of the author and the noted interview subject and do not represent the opinions of Vintura, PharmaLex or their parent Cencora. Vintura, PharmaLex and Cencora strongly encourage readers to review the references provided with this article and all available information related to the topics mentioned herein and to rely on their own experience and expertise in making decisions related thereto.

¹Regulation on Health Technology Assessment, European Commission. https://health.ec.europa.eu/health-technology-assessment/regulation-health-technology-assessment_en
²The European Health Technology Assessment Regulation: The Political Will, Cancer Patients Europe, Feb 2023. https://cancerpatientseurope.org/the-european-health-technology-assessment-regulation-the-political-will/
³EFPIA Patients W.A.I.T. Indicator 2022 Survey , Breakdown of availability (%, 2018-2021), April 2023. https://www.efpia.eu/media/s4qf1eqo/efpia_patient_wait_indicator_final_report.pdf
⁴What is the role of a PICO question for a EUnetHTA assessment? eunetHTA. https://www.eunethta.eu/pico/
⁵Giving Patients’ Preferences a Voice in Medical Treatment Life Cycle: The PREFER Public–Private Project, The Patient – Patient-Centered Outcomes Research, Feb 2017. https://link.springer.com/article/10.1007/s40271-017-0222-3
⁶Guidance to Manufacturers for Completion of Summary Information for Submitting Patient Groups Form, Scottish Medicines Consortium. https://www.scottishmedicines.org.uk/media/2771/guidance-on-summary-information-for-patient-groups.pdf
⁷https://www.riziv.fgov.be/nl/themas/kost-terugbetaling/door-ziekenfonds/geneesmiddel-gezondheidsproduct/Paginas/hervorming-terugbetalingsprocedures-geneesmiddelen.aspx
⁸Guidance on Parallel EMA/EUnetHTA 21 Joint Scientific Consultation, European Medicines Agency and EUnetHTA 21 JCS Secretariat, September 2023. https://www.ema.europa.eu/en/human-regulatory/research-development/scientific-advice-protocol-assistance/parallel-joint-scientific-consultation-regulators-health-technology-assessment-bodies