News - May 10, 2022

PharmaBoardroom Opinion: New EU HTA Procedure – Will it Reduce the Complexity and Burdens for Manufacturers?

This article originally featured in PharmaBoardroom. You can find the original article here. 

EUCOPE’s Secretary-General Dr. Alexander Natz sees both opportunities and pitfalls with the EU HTA procedure, saying that the next three years will decide the crucial steps and whether the new procedure leads to reduced burdens for developers and faster patient access to innovative therapies.

With the entry into force of the Regulation on health technology assessment (EU) this January, the European Commission and EUnetHTA21, a consortium of Member States HTA bodies, are racing to develop the procedural rules and the methodology for EU Health Technology Assessment (HTA). This is before the first medicinal products will be subject to the new procedure starting 12 January 2025.

The new EU HTA procedure will significantly change the requirements before placing innovative medicinal products on the market, with certain new products having to go through a joint European assessment as a first step. Based on this, it is worth assessing how and why we got here, and if this new procedure will replace the fragmented patchwork of national frameworks that currently exist, or if it will only represent yet another step in the process.

The methodologies and procedural rules need to prevent duplication and decrease the burdens on developers to ensure a well-functioning EU HTA procedure. When the Regulation was first conceived, the European Commission highlighted a number of issues the new EU HTA procedure was meant to tackle[1], the most important being divergent HTA processes and methodologies in Member States and the duplication of work for national HTA bodies and developers.

Together, these issues impede and distort market access and reduce business predictability for health technology developers, ultimately leading to increased costs, delays and inequalities in patient access and an overall negative impact on innovation.

A harmonised approach is needed

Currently, national HTA bodies reach different decisions regarding the clinical added value of new technologies. These different outcomes from the same evidence would have been largely prevented with the original proposal, since Member States were to “apply the joint clinical reports” and not carry out their own clinical assessments.

Unfortunately, the final text agreed between Member States does not allow for a full binding effect of Joint Clinical Assessment reports. This means that national HTA bodies are free to draw their own value judgements and conclusions on the overall clinical benefit.[1]

This poses the risk of the duplication of work and continued burdens on companies, since Member States can still ask developers for clarifications and request additional evidence in order to conduct complementary clinical analyses, after a joint clinical assessment. These extra burdens will be especially significant for small to mid-sized companies, who typically lack the resources to face such uncertainty and the additional workload.

To prevent this, the EU HTA procedure must be based on a sound common methodology for evidence assessment and have mechanisms that limit divergent HTA outcomes at Member State level. This would help to re-establish at least the idea of a “one-stop-shop” from the original text.

A successful EU HTA Regulation requires a harmonisation of the current multiple and various national requirements regarding the timing of the procedures, data requirements and the assessment scope. The next three years have several challenges that need to be addressed. Among them are the disagreements between national HTA bodies over the choice of comparators, endpoints and the use of data not generated in randomised clinical trials (RCTs).

The Power of Predictability

Scientific advice remains a crucial step in the evaluation of a medicinal product, both in the authorisation and the HTA proceedings. It serves to align on the relevant methodologies for the assessment, and the evidence to include in the dossier at an early stage of clinical development. It also reduces the chance that an assessment will be discontinued because of different views regarding the scope of the assessment and the evidence to be included.

At the moment, there are a limited number of available slots for Joint Scientific Consultations with the appointed Assessors, that will be allocated on a yearly basis. This is a major point of concern. It means that not all developers that are subject to a joint EU HTA will receive scientific advice prior to doing so.

A robust submission is in the best interest of all stakeholders involved, including developers, HTA bodies, payers and patients. To ensure this mutually beneficial result, all health technology developers should be offered the opportunity of early dialogue with the HTA bodies through Joint Scientific Consultation. Early engagement between developers and HTA bodies should support the uptake of innovation in healthcare systems.

RARE-ING to go

The EU HTA procedure would be a major step forward with the European Commission’s approach, especially for patients with rare diseases or companies bringing Orphan Medicinal Products (OMPs) to the market. Certain Member States currently have distinct HTA processes in place for OMPs and specialised therapies such as Advanced Therapy Medicinal Products (ATMPs), to ensure highly innovative products can reach patients in a timely manner.

As an organisation that represents small to mid-sized health technology companies focused on rare diseases, it is quite positive that the EU HTA Regulation spells out the need for methods that recognise the specificities of newer health technologies, including for OMPs and ATMPs. A flexible methodology is needed to improve patients’ access, and involves acceptance of non-randomised clinical trial designs, the use of Real World Evidence (RWE) and post-launch evidence generation.

Will the EU HTA regulation improve access and reduce burdens?

While the EU HTA Regulation fell short of directly addressing access delays caused by different national processes and outcomes, it established the framework for continued collaboration that could lead to the increased harmonisation of national procedures.

EUCOPE’s Five EU HTA Priorities outlines the steps needed to make the procedure a success by: offering joint scientific consultation to all developers, guaranteeing a flexible methodology for the specificities of OMPs and ATMPs, implementing procedures for resolving the issue of multiple comparator requests, involving all relevant stakeholders and ensuring a transparent and balanced selection of experts.

With a proper EU HTA procedure, health experts, producers and patients will have a basis to help address and facilitate access to innovative medicines. Our role is to ensure the final EU HTA Regulation reflects a flexible, predictable and uncomplicated framework that increases European competitiveness, improves the lives of patients and ushers in a new era for European cooperation on health.

[1] (2018): “Commission staff working document Impact assessment Strengthening of the EU Cooperation on Health Technology Assessment (HTA). [Date of Access 29 March 2022]
[2] (2021): “Eucope’s Statement on EU HTA – Portuguese Presidency compromise” [Date of Access 29 March 2022]