Breakthrough Agreement On EU HTA Regulation “Falls Short”
Executive Summary
The European Parliament and EU Council have reached an agreement on taking forward proposals for greater cooperation on health technology assessments.
Following negotiations with the Council of the European Union on proposed regulations on health technology assessments, the European Parliament has conceded its position that joint clinical assessments should be mandatory for member states carrying out HTAs on medicines and medical devices. However, industry representatives doubt that safeguards secured by the parliament to prevent a bigger burden on companies go far enough.
“While the European Commission's original legislative proposal was certainly a step into the right direction, last night’s agreement falls short of addressing the main issue of duplication of clinical assessments, EUCOPE, the European Confederation of Pharmaceutical Entrepreneurs, told the Pink Sheet on 22 June.
The Background To The Regulations
The commission published its proposal for a Regulation on health technology assessment (HTA) and amending Directive 2011/24/EU on patients' rights in cross-border healthcare in January 2018. (Also see "Industry Welcomes Mandatory EU Joint Clinical Assessments For HTA" - Pink Sheet, 1 Feb, 2018.)
The proposal recognized that numerous HTA procedures across the EU lead to delays in access to medicines for patients, duplication of work and inefficient use of resources for the numerous national and regional HTA bodies, as well as a lack of predictability and higher costs for industry. The commission’s aim was to improve the situation by ensuring greater cooperation across Europe on joint clinical assessments, joint scientific consultations, joint horizon scanning and voluntary cooperation in other areas.
The European Parliament adopted its position on the proposals in 2018.
(Also see "EU-Wide Joint Clinical Assessments Edge Closer" - Pink Sheet, 3 Oct, 2018.) It took years for the Council of the European Union to adopt its position and one sticking point was the plan to oblige member states to use the joint clinical assessments in their own HTAs.
However, Portuguese health minister, Marta Temido, hailed the agreement, describing it as a “decisive breakthrough” on a new law that would benefit patients, manufacturers and member states' health systems. “We will all stand to gain when innovative, safe and effective health technologies can reach the market more quickly. EU-level cooperation is the way forward to make this happen,” she said.
Meanwhile, parliament rapporteur Tiemo Wölken from the parliament’s center-left S&D group said the regulation would “dramatically reduce duplications of clinical assessments, ensure sustainable cooperation on HTA and provide us with a framework to better face health issues, such as rare diseases, personal medicine and orphan medicines.” He added that parliament had “fought for stronger provisions on the uptake of joint clinical assessments and that it secured safeguards to ensure that joint assessments cannot be ignored.”
Red Lines
Negotiations on the legislation took place on 21 June. They took six hours, ending close to 1am, a source close to the council told the Pink Sheet. The council represents EU member state health ministers.The source said that both parties were aware of each other’s “red lines” and came to talks prepared to “wrap up” an agreement before the Portuguese presidency of the council ends on 30 June.
One of the most contentious issues was whether the joint clinical assessments (JCAs) should be binding. The commission originally intended that member states would be bound by conclusions of the JCAs and that they would not be permitted to carry out their own clinical assessments. The parliament supported this when it adopted its position in 2018.
However, some member states, including Germany and France, were opposed to such measures and claimed that they were “a clear encroachment on the competences of member states.” (Also see "Germany and France Round On European Commission HTA Cooperation Proposals " - Pink Sheet, 19 Apr, 2018.). The council finally agreed its position earlier this year and its version of the proposals determined that member states should “give due consideration” to the JCA reports. (Also see "EU Council Agrees Compromise Position On Joint Clinical Assessments" - Pink Sheet, 26 Mar, 2021.)
“It was a priority for the council to stick to its position” during this week’s negotiations, said the source. He added that the final text would likely retain the language on “due consideration” though he said finalizing the text could take some weeks.
Meanwhile, a source close to the parliament said it was “unfortunate” that member states are not obliged to use the conclusions of joint clinical assessments “This was the wish from the European Parliament, but it did not survive the negotiations,” she said. Nevertheless, parliament secured some safeguards to ensure that the assessments are properly considered, she said. Parliament had argued that a voluntary cooperation between member states on HTA (EUnetHTA) was already in place and that the point of the regulation would be to take this further by making it mandatory. (Also see "HTA Cooperation: European Parliament Gets Ready To Placate Council" - Pink Sheet, 29 Jun, 2018.)
These safeguards include provisions obliging member states to annex joint clinical assessments to their own national health technology assessments. In addition, member states have to report to the commission on how the JCAs were used and to justify any decision not to use them.
Burden
The new legislative text will try to reduce administrative burden on companies, particularly smaller entities. For example, developers will only have to submit information, data or other evidence for the purposes of a joint clinical assessment once at EU-level. However, member states can ask for further information if they do further assessments beyond the JCA.
Nevertheless, EUCOPE says that the decision to make the assessments non-binding will lead to a bigger workload. “This will inevitably result in duplication of work, posing continued burdens on companies and, in particular for small to mid-size companies that lack the resources to face both the uncertainty and the additional workload that such a process might bring,” said Alexander Natz, EUCOPE’s secretary general.
EUCOPE also warns that in the agreed text, the joint clinical assessments will be phased in over a period of years depending on the type of product. Oncology products will undergo a JCA first, followed by advanced therapies. “As of today, no assessment methodology has been commonly agreed for these very specific products. We call, again, for regulatory flexibility in that important matter,” Natz said.
The organization has been calling for more flexibility for some products, including orphan drugs and advanced therapies so that the framework “can manage evidential uncertainty in specific cases where appropriate.”
Other Issues
The agreed text also introduces a stakeholder network to help ensure inclusiveness and transparency. It will support the work of the Coordination Group, which is made up of HTA representatives from member states and which oversees the joint clinical assessments and joint scientific consultations. “The Commission will be in charge of setting it up, through an open call for applications – addressed to patient associations, consumer organizations, non-governmental organizations in the field of health, health technology developers and health professionals – details and eligibility criteria to be set up,” said the source close to the parliament.
The two parties also agreed that there would be two types of voting modalities if the coordination group does not reach a consensus. For scientific matters, decisions will be made by a simple majority vote, but in political matters, decisions will be made through a qualified majority vote where some member state votes have greater weighting.
Next Steps
Member states will now need to validate the agreement on the proposed legislation so that it can be adopted at a council meeting. Parliament must then adopt it at a plenary meeting, after which it will be published in the Official Journal. It will become applicable