Euro Roundup: Council of the EU adopts ‘pharma package’ to advance legislative reform agenda
The Council of the European Union has adopted new rules for the pharmaceutical sector, advancing efforts to reform legislation governing the industry.
Lawmakers voted 26 to 1 in favor of the reforms. Under the legislation, new drugs will have eight years of data exclusivity, plus one year of market protection from off-patent competitors. Companies will have the chance to secure two more years of exclusivity by meeting certain criteria and by fulfilling an unmet need, bringing the total protected period up to 11 years.
A transferable exclusivity voucher can provide an additional year of exclusivity. The EU is implementing the vouchers to incentivize drugmakers to develop priority antibiotics. Companies cannot use vouchers on products with annual gross sales of more than €490 million ($556 million) in the preceding four years.
The next step is for the Parliament to adopt the regulation and directive that make up the pharma package. After that happens, the legislation will be published in the EU’s Official Journal and come into force.
EMA sets 10 priorities for driving change in medicines for women’s health
The European Medicines Agency (EMA) has identified 10 actions it could support to ensure women are better represented in drug development.
EMA’s priority areas emerged from the multistakeholder workshop that the agency held this week. The agency held the workshop in response to evidence that persistent gaps in research, evidence generation, and drug development mean many women’s health needs are insufficiently understood and addressed. EMA linked the evidence gaps to poorer health outcomes and unmet medical needs in women.
At the two-day event, EMA and more than 100 leading voices on women’s health converged on the set of priority actions. The actions will inform a roadmap on medicines for women’s health that EMA plans to publish this year.
In each area, EMA sees an opportunity for its teams to lead, collaborate, or influence the action. The priorities include supporting the inclusion of pregnant and breastfeeding individuals in clinical trials and training the European medicines regulatory network on sex- and gender-sensitive assessment matters.
EMA’s tenth action is to develop a framework of measurable outcomes and key performance indicators and to report regularly on its progress. The data could reveal progress against the “concrete deliverables with planned short-, medium-, and long-term timelines” that EMA plans to include in the roadmap.
The planned response extends beyond EMA. The workshop generated “a clear path to action” that has a role for national bodies, María Lamas, director of the Spanish Agency for Medicines and Health Products, said.
“National regulatory authorities can drive progress across many areas,” Lamas said. “A key priority is to ensure that women are properly represented in clinical trials and that the data generated capture their experiences and needs, providing the evidence required for informed decisions and better care.”
Believing AI has ‘outrun the framework,’ EU policy unit recommends actions
A European Parliament policy unit has warned that AI-enabled biological design technology has “outrun the framework.”
The Policy Department for Transformation, Innovation and Health shared the warning in a briefing report for Parliament committees. The report focused on AI for predicting, designing, or modifying proteins, peptides, and the DNA or RNA that produce them. While the policy unit identified six EU instruments that apply to such tools, no single framework focuses on the intersection of AI and biology.
Officials identified gaps in the patchwork of instruments covering the technology. The gaps have raised concerns, in part because tools that can design a new drug could, in principle, be used to create a more dangerous pathogen.
The policy unit named three operational instruments that would close the main gaps. Mandatory checks of DNA synthesis orders against the nucleic acid’s function, rather than its sequence, could mitigate risks. However, the policy unit cautioned that industry-wide deployment of function-aware detection methods would require coordinated adoption by providers and a public methodology for evaluating compliance.
Another recommendation covers two AI Act pathways. The policy unit recommended capability-based designation for foundation models and an explicit carveout for narrow open-source tools. The third main response is to operationalize the proposed Biotech Act’s Biothreat Radar, aligned with the Biological Weapons Convention.
The policy unit provided advice on “avoiding regulatory-jigsaw,” including by recommending that EMA guidance is coordinated with the AI Office and the Advisory Group on Biosecurity. The coordination workload would mainly fall on EMA. The policy unit also proposed assessing whether notified bodies can absorb AI-specific work or whether funding should be provided to build capacity.
EMA seeks feedback on clinical trials of sickle cell and thalassemia medicines
EMA has released draft guidelines on the clinical requirements for medicines to treat sickle cell disease (SCD) and thalassemia.
SCD and thalassemia are inherited disorders that affect how well red blood cells carry oxygen. There are overlaps in how the conditions are treated. For example, Vertex and CRISPR Therapeutics’ gene-editing therapy Casgevy is approved in SCD and beta-thalassemia. EMA has published separate draft guidelines on the two conditions, but the documents share a structure and some content.
Both guidelines focus on therapeutic confirmatory studies, covering topics including patient selection, efficacy assessment, and safety. EMA has proposed different endpoints for the indications, listing the rate of vaso-occlusive crises as a way to assess SCD drugs and transfusion independence as a measure of the efficacy of curative thalassemia medicines.
The final sections of both documents address studies in pediatric and older patients. EMA largely used the same text in each guideline. Notable differences include a line about the potential need to defer testing SCD gene therapies in children until at least one year of safety and efficacy data are available.
Both guidelines are open for comment until 31 January.
SCD Guideline, Thalassemia Guideline
Lobby group calls for UK to follow Health Canada’s lead to close ‘biosimilar void’
An association for off-patent drugmakers has called for policymakers to act to avoid a “biosimilar void” that could cause the UK to miss out on a “golden age of biologic loss of exclusivity.”
Multiple blockbuster biologics are set to lose patent protection through 2032, creating opportunities for healthcare systems to lower costs by switching to cheaper biosimilar copies. Medicines UK, a trade group for generic and biosimilar companies, forecasts that copies of most top-selling biologics will launch in the UK.
However, the trade group is concerned about 50 biologics with annual UK sales below $50 million. While uptake of the 50 drugs is limited, Medicines UK’s report suggests the low sales may reflect the value for money of the branded biologics. Cheaper biosimilars could “transform niche biologics into high‑volume commercial successes while dramatically reducing NHS costs,” provided the UK strengthens its position.
Medicines UK’s “practical measures” for ensuring that the UK remains a competitive market include “following Health Canada’s approach of presuming confirmatory studies will not be required before a biosimilar is licensed in the UK.” Health Canada published guidance on biosimilar development this year.
Other News:
The European Commission has published an expert opinion on bioabsorbable orthopedic screws. The experts called for the notified body to ask the manufacturer to run certain studies, including a pre-authorization clinical trial comparing the device to the gold standard of fixation.
