With the European Union’s new Regulation (EU) 2021/2282 on health technology assessment, the evaluation of certain new therapies will now be performed jointly at the European level for the first time. The aims are to speed up access to new therapies, reduce duplication of work and harmonize the methodologies used for the clinical aspects of the evaluation. The new European HTA procedure is to be carried out in parallel with the European approval process. The EU HTA Regulation, which came into force in January 2022, creates the legal and organisational framework for cooperation between member states in joint scientific consultations and joint clinical evaluations of new medicines.
Now, the framework needs to be substantiated: over the next three years, the processes, methods and requirements for EU HTA will be further prepared for the Commission by the EUnetHTA21 consortium, represented by the member states’ HTA bodies, taking into consideration the input of stakeholders.
The issues at hand
When the EU HTA Regulation was first conceived, the European Commission highlighted a number of issues that the new EU HTA procedure was meant to tackle :
- The issue of divergent HTA processes and methodologies in the Member States which impede and distort market access and reduce business predictability for health technology developers, with increased costs for market access and a negative impact on innovation.
- The issue of duplication of work for national HTA bodies and developers caused by multiple parallel assessments and low uptake of EUnetHTA joint reports, which lead to delays and inequalities in the availability of innovative health technologies for European patients.
- Finally, the ad-hoc and project-based cooperation was deemed unsustainable in the long run due to the inefficient and uncertain allocation of resources.
Regrettably, after heavy backlash from certain Member States, the position was softened, proposing that Member States shall “give due consideration” to the joint clinical assessments. The result is that joint clinical assessments will not replace the national procedures for clinical assessment, but rather a joint clinical report will be considered as part of the national proceedings. The Regulation will be introduced in a staged approach over several years, starting with oncology treatments and Advanced Therapy Medicinal Products (ATMPs) in 2025, Orphan Medicinal Products (OMPs) in 2028, and finally expanded to all other therapies in 2030.
While a few formal steps still remain before the EU HTA Regulation becomes official in 2025, it is absolutely paramount that the methodological and procedural rules do not create added layers of complexity and additional barriers.
What does this mean? At EUCOPE, we believe the EU HTA procedure must lead to sufficient harmonisation of existing methodologies to allow wide uptake of joint EU HTA reports by member states to avoid the risk of additional clinical assessments being demanded at Member State level, with increasing burdens for developers and delays in patients’ access to innovative treatments.
EUCOPE’s Five #EUHTAPriorities
In order to prevent the duplication of work, reduce burdens and increase the predictability for all stakeholders, EUCOPE has the following five (5) priorities for the EU HTA Procedure:
- Joint scientific consultation must be offered to all developers
- A flexible methodology is needed that reflects the specificities of OMPs and ATMPs
- There must be procedures for resolving the issue of multiple and competing comparator requests from the Assessors
- The procedure must ensure the broad involvement of relevant stakeholders
- A transparent and balanced selection of experts for rare disease and specialised technologies is necessary
Let’s examine these priorities a bit deeper:
Priority 1: Joint scientific consultation must be offered to all developers
Scientific advice is a crucial step in the evaluation of a medicinal product in order to align the relevant methodologies for the assessment, and the evidence to include in the dossier. All developers must therefore be offered the opportunity of early dialogue with the HTA bodies through joint scientific consultation.
A robust submission is in the best interest of all stakeholders involved, including HTA bodies, payers, and patients. In order to meet the high demand for joint scientific consultation, adequate EU funding must be allocated. Alternatively, a fee-paying mechanism could be introduced. If developers are not able to receive scientific advice through the EU procedure, a complementary procedure should be explored jointly by national HTA bodies.
Priority 2: A flexible methodology is needed that reflects the specificities of OMPs and ATMPs
To prevent duplication, alignment must be strived for between the choice of comparator used by the EMA Committee for Orphan Medicinal Products (OMP) and that used by the relevant EU HTA subgroup and its appointed assessor and co-assessor. The recognition of the benefit of the product as confirmed by the European Commission through the Orphan Drug Designation (ODD) must be respected.
To improve patients’ access, evidence generated outside of the strict randomised controlled trial (RCT) design must be accepted such as real-world evidence, while postlaunch evidence generation should be routinely implemented. It must be noted that OMPs and ATMPs in particular, due to smaller patient populations, rely on less conventional methodological approaches in order to improve their intepretability.
Priority 3: There must be procedures for resolving the issue of multiple and competing comparator requests from the Assessors
Above all, the choice of comparator must be based on available clinical evidence. When the comparator is a pharmaceutical compound, it must have a marketing authorisation for that indication and line of treatment. Conversely, national policies aimed at cost considerations, such as economic off-label use, should not form the basis of choice of the comparator.
Since the choice of the comparator is often based on political considerations, a list of possible agreed comparators should be drawn up at the scoping stage through discussions with the developer. The comparator used in the investigative study should be included in this list. In order to make benefit from the “one-stop-shop” established with the EU HTA Regulation, the maximum choice of listed pharmaceutical comparators should not exceed three (3) for any given assessment.
Priority 4: The procedure must ensure the broad involvement of relevant stakeholders
Patients have important insights into their respective disease areas and it is positive that they will be involved in a meaningful way in joint clinical assessments A representative selection of patient associations must be included in the Stakeholder Network.
Health technology developers are key stakeholders and should be included in the scoping meetings to inform the selection of the appropriate evidence and comparators. Developers must be given the opportunity to respond with technical clarifications as needed and provide a fact check of the joint clinical assessment report. Otherwise, technical mistakes might lead to poor outcomes to the disadvantage of patients.
Priority 5: A transparent and balanced selection of experts for rare disease and specialised technologies is necessary
When selecting experts for rare disease and specialised technologies, conflicts should be evaluated on an individual basis. Decisions about whether to include or exclude an expert must be transparent and alternative methods of input should be provided, such as structured questionnaires.
Recruitment of experts should be done via a general open call when seeking broader feedback, while the Stakeholder Network should be utilised to help identify individual experts for one on one and group discussions.
Putting the EU on the Right Path
Our expectation is that the finalised methodological and procedural rules for the EU HTA Procedure will secure an efficient and robust assessment process and a predictable environment for industry. An EU HTA Procedure that prevents the duplication of work, reduces burdens and increases predictability for all stakeholders, including patients, will put the EU on the right path.
For more information about our work on EU HTA, please visit our Resources page.
 Eur-lex.europa.eu (2018): “Commission staff working document Impact assessment Strengthening of the EU Cooperation on Health Technology Assessment (HTA). https://eur-lex.europa.eu/legal-content/FR/ALL/?uri=CELEX%3A52018SC0041
April 27, 2022