Amid a wave of healthcare regulatory changes in the EU, an update on health technology assessment (HTA) regulation is set to speed up drug access times. However, some industry players remain wary of logistical issues anticipated to accompany this change.
On 12 January, the European Medicines Agency (EMA) launched its long-awaited, revamped Health Technology Assessment regulation (HTAR). “The new regulation will enable our collaboration across decision-makers, reinforcing efforts to improve access to medicines for all patients in the EU,” said Emer Cooke, the EMA’s executive director, in a press release.
An HTA is a review process that evaluates medical, ethical, social, and economic factors to assess the value of new health technologies. The new regulation involves the implementation of joint clinical assessments (JCAs) by the HTA Coordination Group to assess new therapies’ clinical effectiveness and safety in comparison to existing technologies. The newly established group will also work with the EMA to advise developers scientifically, and to produce data that meets the demands of regulators and HTA bodies. Furthermore, the EMA has promised to share information on new applications and health technologies for “planning purposes and horizon scanning”.
This initial launch only impacts HTAs for cancer technologies and advanced therapy medicinal products. However, orphan medicinal products will also be targeted starting January 2028, followed by all medicinal products in 2030.
The HTAR is one of several healthcare regulatory initiatives the European Commission and EMA have announced in recent years, like the EU Competitiveness Fund, the EU pharmaceutical legislation, and new clinical trial regulations.
The difference between the HTAR and these other changes is that this legislation is addressed to policymakers rather than the industry itself, says Dr. Gunnar Sachs, a partner at the international law firm Clifford Chance, in Germany.
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By GlobalDataThe new harmonised assessments could be a major boon for the EU pharmaceutical industry due to faster timelines that set it apart from other geographies, says Sachs. However, he highlights lingering confusion surrounding compliance with the new HTAR.
The HTA makeover
The aim with the new HTA directive is to drive efficiency because it allows for a single HTA process, where previously each individual Member State would do it separately, says Stephen Reese, co-head of the Healthcare and Life Sciences sector at the London, UK office of Clifford Chance. “If it works the way we hope, it should deliver some cost savings, particularly for the pharmaceutical industry,” says Reese.
Before the HTA revamp, HTA decision-making processes varied broadly between different EU Member States. For example, in France and Germany, HTA bodies are public regulatory organisations that work separately from the government, whereas Sweden’s Dental and Pharmaceutical Benefits Agency (TLV) is a government agency. Furthermore, countries like Germany assess therapeutic benefits separately from reimbursement, whereas the two factors are assessed in unison in other Member States. Given the many differences, the European Commission is providing funding support to Member State HTA bodies to allow the smooth implementation of the new framework.
The new change may incentivise some pharma companies to enter into smaller European markets, which may have typically been neglected, due to the quicker, more centralised process, says Sachs.
A wave of regulatory change
As the past few years have brought a tide of EU healthcare regulatory change, responses to the changing systems have ranged from industry approval to confusion. “There are some positives [related to general EU regulatory change] in terms of more openness and transparency from a regulatory standpoint,” says Jacopo Andreose, the CEO of Angelini Pharma, an Italian biotech based in Rome. He praised the new European regulatory sandboxes, which allow small companies and biotechs to have early interactions with the EMA to understand whether a company is on track in terms of development efforts. In recent years, European regulators have created sandboxes for several industries and topics such as pharma, artificial intelligence, and blockchain to improve communication with industry voices and boost development in certain sectors.
One of the first major European regulatory updates was a switch to a centralised clinical trial regulation (CTR) system that involved the implementation of a single EU entry point for clinical trial applications. Florent Gros, CEO at the Dublin, Ireland-headquartered biotech Priothera, says his company was among the first to submit its documents under the new system and has been adapting to the clinical trial centralisation process for the past few years. The new process only took a few weeks, adds Gros.
Patrik Stromberg, the CEO at the Stockholm, Sweden-based biotech AnaCardio, which was also an early adopter of the new CTR, says that with regulatory experts on board, the company was able to consult specialists on the shift. However, this process would be much more difficult for smaller companies that lack such resources, says Stromberg.
Anticipating similar challenges for the HTAR, a lack of consultation and regulatory guidance ahead of the launch has been a point of contention for some in the industry. In a December 4, 2024 press release, the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) released a statement raising concerns about the lack of advice being offered to pharmaceutical companies surrounding the HTAR. “We are concerned that only a fraction of companies submitting for a JCA will be able to discuss their clinical trial plans at the critical planning stage,” the organization said in the statement. Until that point, the HTA Coordination Group had only scheduled five to seven joint scientific consultations for medicinal products.
“If the JCA procedure, which ultimately is the measure of success of joint EU HTA, does not provide transparency, predictability, and workability for companies, joint EU HTA will fail to deliver on its aim of ensuring better access for patients to innovative health technologies,” said EUCOPE, in the same statement.
The European Federation of Pharmaceutical Industries and Associations (EFPIA), also raised concerns about JCAs following the release of a December guidance. The agency praised the inclusion of patient-reported outcomes and the EMA’s “flexibility regarding how [clinical trial] outcomes should be measured and analysed, allowing indirect comparisons across different trials”. However, the trade body criticised a restriction on the number of clinical trial outcomes that Member States could request from health technology developers.
Moreover, the new HTAs outline nine specific domains that developers must follow in preparing for HTAs, including some clinical and non-clinical domains, explains Sachs. There is some concern surrounding the “soft requirements” that companies must comply with non-clinical domains like those related to reimbursement standards for the different Member States, he adds. There is currently very little guidance on this and it is hard to predict what this could look like in the future, says Sachs.
“Right now, the industry has to prepare for the HTAs proactively without actually knowing what they will be facing from the side of the decision makers,” said Reese.
“The new regulation is a kind of compromise that means even though we have a centralised approach with widely harmonised requirements, the final decisions still need to be taken by the EU Member States. The big question is, will this compromise approach actually work? Will the Member States actually follow?” says Sachs.
