Introduzione
European Statistical Forum 2026 – Design and Analysis of Trials in Rare Diseases and Gene Therapies
The development of therapies for rare diseases and gene therapies presents a distinct set of scientific, statistical, and operational challenges.
Small and heterogeneous patient populations, limited natural history data, and ethical constraints on randomisation require trial designs that go beyond traditional approaches. These challenges directly affect study powering, endpoint selection, control strategies, and the robustness of evidence generation.
Gene therapies, in particular, aim for one-time, disease-modifying interventions rather than chronic symptom management introducing additional complexity in trial design, manufacturing, delivery, and long-term safety and efficacy assessment. Ensuring regulatory acceptance under these conditions requires innovative statistical methodologies, transparent decision frameworks, and careful integration of external data sources, where applicable.
This conference will explore how adaptive and Bayesian designs, natural history data, real-world evidence, single-arm trials, and master protocols can be applied to leverage limited data while maintaining scientific rigor, ethical integrity, and regulatory acceptability.
Attention will also be given to operational practicalities, including global patient identification, long-term follow-up, and early regulatory engagement.
The 16th edition of the European Statistical Forum (ESForum) focuses on the design, analysis, and regulatory evaluation of clinical trials in rare diseases and gene therapies, bringing together statistical innovation and real-world application.
Key Conference Topics
- Innovative trial designs and case studies in rare disease development
- Statistical strategies for small sample sizes and high variability
- Advanced analytical methods supporting gene therapy programs
- Regulatory perspectives and evidentiary expectations for emerging therapies
In addition to the scientific sessions, this conference offers ample opportunities for networking and collaboration.
Scientific Board
Jens-Otto Andreas, BSO Lead Data & Innovation Analyst at UCB Biosciences GmbH
Lisa Comarella, Senior Director Biostatistics at Alira Health Biometrics
Fabiola La Gamba, Associate Director Biostatistic at Novartis
Who Should Attend ESForum 2026?
This conference is designed for professionals involved in the design, analysis, and regulatory evaluation of clinical trials, including statisticians, pharmacometricians, clinical development scientists, data scientists, and regulatory affairs specialists from pharmaceutical and biotechnology companies, CROs, academic institutions, hospitals, and research centers.
Whether you're designing your first gene therapy trial or refining statistical strategies for rare disease development, ESForum 2026 provides the expertise you need to navigate complex clinical trial design challenges.
ESForum – Building Opportunities
The European Statistical Forum is a reference conference for professionals involved in the statistical design, analysis, and regulatory evaluation of clinical trials. ESF 2026 offers a focused platform to translate advanced statistical concepts into practical solutions for rare disease and gene therapy development.
Organized by LS Academy, a trusted provider of life sciences training and conferences since 2010, ESForum brings together biostatisticians, clinical development scientists, and regulatory affairs specialists to translate statistical innovation into practical solutions.
Quote di iscrizione
€ 645,00 Early Bird fee until 15 October 2026
€ 765,00 Ordinary fee
€ 460,00 Freelance, Individual, Academy, Public Administration
€ 65,00 Networking Dinner
Fee includes: seat at the conference, pdf copy of presentations of Speakers who allow the distribution, networking lunch, coffee breaks, organisational office assistance, certificate of attendance.
Informazioni utili
The conference will take place at:
NH Budapest City
Vígszínház u.3 – 1137 Budapest
The NH Budapest City hotel is in the Újlipótváros business district on the Pest side of the river. Some of Budapest’s star attractions are right on the doorstep, and the nearby metro station whisks you to more tourist hotspots.
From the airport
Taxi: it´s a 40 minutes trip.
Bus: you can take the bus line 200 from the Airport Budapest, station “Repüloter, P+R” to get to the station “Kobanya-Kispest”. From there take the metro line M3 (blue line) for the station “Nyugati Palyaudvar”. You can reach the hotel in a walking distance from about 5 minutes.
From the train station
From Nyugati Station: exit the station and turn right for the street “Szent Istvan Krt”. From there please turn right again for the street “Hegedüs Gyula”. Afterwards please turn left for the street “Vigszinhaz”. The hotel is located on the right.
Closest metro station: Nyugati Palyaudvar (M3-blue line).
An informal networking dinner will also be organized, offering a refined setting to continue the conversation, exchange insights, and build valuable connections with industry peers.
More details coming soon.
Edizioni Passate
Registrati
Versione Stampabile
Sponsors
Media Partner
Patrocinio
NH Budapest City
Vígszínház u.3 – 1137 Budapest
