With the goal of addressing these challenges and enabling the development of a joint perspective on clinical aspects of medicines, in vitro diagnostics and high-risk medical devices, the European Health Technology Assessment Regulation (EU HTAR) was adopted in 2021.
By increasing transparency and reducing the duplication of assessment efforts, the EU HTAR’s fundamental aim is to accelerate patient access to treatments throughout Europe.
To promote increased patient and clinician involvement and ensure their individual needs are addressed, there will be formal and regular collaboration among stakeholders.
Smaller countries in the EU with less established HTAR bodies will uniquely benefit from congruity of efforts. An important note made in the EFPIA Patient W.A.I.T Indicator 2022 Study is that there is wide variation in terms of access to new medicines … and the HTAR can help to speed this up.1
Although this regulation has numerous benefits for stakeholders, they also face uncertainty regarding the mandatory shift.
Sources of concern include the lack of stakeholder input during the drafting process and cohesion among different components of the regulation (such as joint scientific consultations [JSCs] and joint clinical assessments [JCAs]), the increased complexity of JCA documentation and the accelerated timeline for comprehensive submission development.
Addressing HTAR implementation challenges
Leading up to the 2025 implementation deadline for the HTAR, it will be crucial for the industry to understand these complexities and how to address them. Below are a few examples of areas that stakeholders should be aware of and how they can shape their compliance approach.
Limited stakeholder involvement
The HTAR was formulated with inclusivity in mind, involving all 27 member states in a comprehensive collaborative process that examined methodologies and sought input from patients and clinicians.
The European Community (EC) has initiated training for patient and clinical experts in terms of contributing crucial information for the validation of Population, Intervention, Comparator(s), Outcomes (PICOs) and draft JCA reports.
However, limited resources pose challenges for effective input collection among MS, patients and clinical experts.
Notably, consultation with sponsors during the JCA phase is limited and pharmaceutical companies’ participation in scoping is necessary owing to differing PICO specifications — even though it deviates from the European Network of Health Technology Assessment 21 (EUnetHTA 21) Joint Action 3 (JA3).
Increased involvement will ensure factual accuracy and facilitate the draft report review meeting with relevant stakeholders and the assessment team.
Encouraging proactive stakeholder engagement: Staying up to date with EUnetHTA 21 guidance and the European Commission’s implementation acts will be imperative for the industry.
Additionally, proactive engagement with the co-ordination group (CG), HTAR bodies and national decision makers will be necessary.
Although the 2025 deadline is approaching, there still remains time for stakeholders to offer their input and vital feedback before the implementation acts are ultimately settled. This valuable input may be submitted individually or collectively through industry associations.
The HTAR in its current form introduces a limited timeframe to generate comprehensive evidence, which adds complexity to the analytical process.
Upon submitting a drug, the European Medicines Agency (EMA) informs the HTA co-ordination group secretariat, providing information about the drug’s regulatory aspects and claimed indications, thereby initiating the JCA process.
However, the process as currently outlined takes 4 months before sponsors receive the final PICO, leaving them with just 3 months to finalise the dossier. Given that typical timelines require at least 4–5 months for completion, these constrained schedules place additional pressure on sponsors.
Adapting existing processes to meet deadline challenges: Pharmaceutical companies must model their strategy around future processes and governance within the EU JCA framework to prepare for HTAR submission processes.
To meet the dossier timeline and requirements, literature reviews should be comprehensive, addressing variations in MS and incorporating EU epidemiological data from the past 5 years to assess clinical management and internal validity.
Following the example set by EMA and the US Food and Drug Administration (FDA), sponsors should integrate early dialogues, such as JSC, into their procedures.
This necessitates fostering close collaboration among various departments, including health economics and outcomes research (HEOR), market access, biostatistics, regulatory and clinical development.
Complex analytics process
The completion of regulatory filing triggers the JCA process, beginning with the CG’s survey to collect MS-determined PICO information. However, the current HTAR is a combination of country specific PICOs, rather than a scoping process that streamlines evidence requirements.
As a result, a company launching a new cancer drug could be required to develop evidence to support more than 10 different PICOs.
Anticipating PICOs to tackle complexities: JCA requirements can only be met with detailed comparative evidence. By planning in advance, this need can be more easily met.
Prior to starting the JCA, sponsors will benefit from having a clear view of what PICOs to expect.
To do so effectively, there needs to be an understanding of payer-relevant populations and comparators in each market, which can be facilitated with input from local affiliates, clinicians and patients.
If comparative evidence generation is not feasible, careful evaluation of available alternatives through assessment of indirect treatment comparison (ITC) will become a necessary option.
The future of industry after HTAR
The implementation of the HTAR marks a significant transformation in industry processes with far-reaching implications.
The initial adoption of this large-scale shift poses inherent challenges, but it’s expected that the EU HTAR will continue to evolve until the 2025 deadline, driven by the resolution of stakeholder concerns throughout the implementation journey.
To best prepare for the impending changes, the industry can take proactive measures such as engaging with HTAR bodies and other stakeholders, anticipating PICOs based on available information and adapting internal processes to facilitate easier implementation of future adjustments.
Once the requirements and processes are well-established, the long-term benefits will profoundly shape the landscape of therapeutic development and innovation.
Reducing the duplication of efforts will save valuable time and resources, ultimately leading to increased accessibility of treatments across the EU. This enhanced accessibility, in turn, will contribute to more equitable and widespread access to treatments for various patient communities.
Key term reference guide
- JSCs allow industry to consult with the European Medicine Agency (EMA) and multiple EU HTA bodies to obtain guidance on the information, data, analyses and other evidence that are likely to be required from clinical studies.
- JCAs are health technology assessments that cover the health problem and current use of the technology, description of the technical characteristics of technology, safety and clinical effectiveness.
- Population, Intervention, Comparator(s), Outcomes (PICO) is the framework used to define the scope of the JCA. Within the framework, a research question to be addressed is defined by patients or population(s) of interest, the intervention being addressed, the relevant comparator(s), the intervention under assessment it should be compared with and the outcomes of interest.
- European Network of Health Technology Assessment 21 (EUnetHTA 21) was the consortium of 13 HTAR bodies tasked with supporting the implementation of the HTA regulation between September 2021 and September 2023.
- Joint Action 3 (JA3) was an objective within the EUnetHTA 21 to increase the use, quality and efficiency of joint HTA work at European level to efficiently support policy and decision making in the health systems.
- The Co-ordination Group (CG) comprises a number of representatives from HTA agencies of EU MS, health ministries, EMA and the EC who oversee the implementation of JCAs and other joint work related to the HTAR.