Late-stage clinical trials rarely deliver neat storylines. But across Europe’s trial networks in 2025, a few readouts pointed in the same direction: toward treatments built to modify disease biology and paired with better ways to measure whether patients actually feel and function better. Naturally, not every program hit its mark, and several headline results are still awaiting full peer-reviewed publication. Even so, the year offered a clearer view of where many European investigators are placing their bets — and which questions 2026 is poised to answer.

A recurring theme was precision: drugs designed to act on a single target, with studies large enough to test whether mechanistic promise translates into meaningful benefit.

Cardiology: Targeted Therapy Reshapes Hypertrophic Cardiomyopathy (HCM) Care

In cardiology, that theme played out in obstructive HCM, where many patients still start with beta-blockers. In the MAPLE-HCM trial, aficamten — a cardiac myosin inhibitor designed to reduce the hypercontractility that drives left ventricular outflow tract obstruction — was tested head-to-head against metoprolol as a monotherapy. Reported in 2025, aficamten improved peak oxygen uptake and hemodynamics and reduced symptoms more than metoprolol in symptomatic patients.

The findings are enough to raise a practical question for European clinicians: if a mechanism-directed drug outperforms a guideline mainstay in a rigorous trial, where should it sit in the treatment sequence?

“If authorized, aficamten could broaden therapeutic options for patients with symptomatic obstructive hypertrophic cardiomyopathy in Europe,” trialist Pablo Garcia-Pavia, MD, PhD, head of the Heart Failure and Inherited Cardiac Diseases Unit at the Department of Cardiology at Hospital Universitario Puerta de Hierro Majadahonda, Madrid, Spain, told Medscape News Europe. He stressed that uptake will hinge on regulatory approval, labeling, cost-effectiveness assessments, and real-world safety data.

For 2026, the watch list is less about whether myosin inhibition can work and more about durability and implementation: which patients benefit most, how broadly it can be adopted across different health systems, and how safety looks once use expands beyond trial populations.

Oncology: Raising the Bar on Endocrine Therapy

In oncology, endocrine therapy in early hormone receptor-positive, HER2-negative breast cancer is effective but imperfect; late recurrences remain common. That backdrop helps explain interest in giredestrant, an investigational oral selective estrogen receptor degrader. At the 2025 San Antonio Breast Cancer Symposium, an interim analysis from the phase 3 lidERA trial reported a statistically significant improvement in invasive disease-free survival vs standard endocrine therapy. Trial details, including its broad international footprint with many European sites, are posted on ClinicalTrials.gov.

The key task for 2026 will be to scrutinize the full dataset once it is published: the absolute benefit, tolerability across clinically important subgroups, and how giredestrant fits alongside — or instead of — other intensification strategies used in higher risk disease.

Inflammation and Skin: Measuring Outcomes Patients Feel

In inflammatory disease, European researchers are also pushing beyond clinician-reported scores toward outcomes that patients recognize in daily life. Lebrikizumab (brand name, Ebglyss) is an injectable monoclonal antibody that targets interleukin 13, a key cytokine in atopic dermatitis. With the drug already authorized in the EU, the multicenter European ADTrust study protocol is testing how treatment affects well-being and long-term control in routine practice — including by using the World Health Organization-Five Well-Being Index, a tool not commonly incorporated into atopic dermatitis studies.

“Creating patient well-being and supporting patients to return to a normal life is our primary goal,” said Matthias Augustin, MD, who is recruiting patients in Germany. Augustin is a lecturer at the University Medical Center Hamburg-Eppendorf and director of the UKE Institute for Health Services Research in Dermatology and Nursing, Hamburg, Germany.

In ADTrust, the wager is that systematically measuring well-being — alongside skin clearance and itch — can produce a more patient-centered definition of success that is portable across European health systems.

The road to disease modification looks harder in neurodegeneration, and 2025 brought that tension into focus. Prasinezumab is an investigational monoclonal antibody that targets aggregated alpha-synuclein, a protein implicated in Parkinson’s disease. Exploratory analyses from the phase 2 PASADENA study suggested a possible signal in faster-progressing subgroups, but the program has also underscored how difficult it is to prove slowed progression. The phase 2b PADOVA trial continues to follow patients, and Roche has said it plans to advance prasinezumab into phase 3 development.

That makes 2026 a pivotal year for the field: not because anyone expects a single antibody to settle Parkinson’s biology but because study design itself is evolving — toward longer follow-up, better phenotyping of progression rates, and endpoints more likely to capture meaningful change. Europe’s role, with large multicountry enrollment and standardized movement-disorder expertise, is central to that effort. 

Not all late-stage research is in classic disease areas. In dermatology, two phase 3 studies — SCALP-1 and SCALP-2 — evaluated clascoterone 5% topical solution for androgenetic alopecia. Clascoterone is a topical androgen-receptor inhibitor, intended to blunt dihydrotestosterone signaling in the scalp without systemic antiandrogen exposure. In December 2025, the sponsor reported topline efficacy results from trials enrolling patients in the US and Europe.

As with other end-of-year headlines, the clinical significance will be clearer once full results and safety data are published. For European clinicians, the broader question is whether topical androgen blockade can offer a meaningful option for patients who do not tolerate — or do not respond to — existing therapies.

Digital Health: Europe’s Parallel Bet on Infrastructure

Alongside drug development, Europe’s health policy agenda is increasingly treating digital infrastructure as a clinical tool in its own right. In 2025, the EU-funded EDITH project published a roadmap for a “Virtual Human Twin” — a framework for building computational models of an individual (or patient group) that pull together multiple streams of health data (such as clinical records, imaging, lab results, and other measurements) into a dynamic “digital representation” of a person’s health. The aim is not a virtual avatar but a decision-support concept: A model that could, in principle, help clinicians and health systems simulate risk, anticipate disease trajectories, and compare likely outcomes of different interventions to support prevention, diagnosis, and treatment decisions across EU settings. The European Commission has positioned the initiative within its broader digital strategy for health and care.

Liesbet Geris, PhD, professor of biomechanics and computational tissue engineering at the University of Liège, Liège, and KU Leuven, Leuven, both in Belgium, who coordinates EDITH, described the promise in practical terms, while warning against treating artificial intelligence (AI) as a universal fix. She explained it could help clinicians standardize complex decisions and reduce unnecessary workload. But “AI won’t solve everything,” she stressed.

In 2026, the success of “digital twin” approaches may depend less on algorithms than on governance: interoperable data, transparent validation, and workflows that clinicians can realistically use.

Taken together, the year’s trials and technology programs share a sober throughline. Europe is investing in interventions that are more targeted, more measurable, and — at least in intent — more personalized. The next 12 months will test whether that precision can survive the messy realities of clinical practice: heterogeneous patients, constrained budgets, and health systems under strain.

Garcia-Pavia reported receiving speaking fees from BMS and consulting fees from Alexion, BioMarin Pharmaceutical, BMS, Cytokinetics, Edgewise Therapeutics, Rocket Pharmaceuticals, and Lexeo Therapeutics. Augustin and Geris reported having no relevant financial relationships.

Sophie Cousins, MIPH, is a global health journalist who has reported from more than 20 countries.