The development of therapies for rare diseases and gene therapies presents a distinct set of scientific, statistical, and operational challenges.<\/p>\n
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.<\/p>\n
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.<\/p>\n
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.<\/p>\n
Attention will also be given to operational practicalities, including global patient identification, long-term follow-up, and early regulatory engagement.<\/p>\n
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<\/strong>, bringing together statistical innovation and real-world application.<\/p>\n In addition to the scientific sessions, this conference offers ample opportunities for networking and collaboration.<\/p>\n Jens-Otto Andreas, <\/strong>BSO Lead Data & Innovation Analyst at UCB Biosciences GmbH<\/em> This conference is designed for professionals involved in the design, analysis, and regulatory evaluation of clinical trials<\/strong>, including statisticians, pharmacometricians, clinical development scientists, data scientists, and regulatory affairs specialists from pharmaceutical and biotechnology companies, CROs, academic institutions, hospitals, and research centers.<\/p>\n 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.<\/p>\n 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.<\/p>\n Organized by LS Academy<\/a>, 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.<\/p>\n","protected":false},"excerpt":{"rendered":" European Statistical Forum 2026 \u2013 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 … Continued<\/a><\/p>\n","protected":false},"featured_media":16188,"template":"","meta":[],"product_brand":[],"product_cat":[47],"product_tag":[],"class_list":["post-19398","product","type-product","status-publish","has-post-thumbnail","conference_topics-statistica-e-data-management","productgroup-european-statistical-forum","product_cat-conferenze","first","instock","taxable","shipping-taxable","purchasable","product-type-variable"],"_links":{"self":[{"href":"https:\/\/lsacademy.com\/it\/wp-json\/wp\/v2\/product\/19398","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lsacademy.com\/it\/wp-json\/wp\/v2\/product"}],"about":[{"href":"https:\/\/lsacademy.com\/it\/wp-json\/wp\/v2\/types\/product"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/lsacademy.com\/it\/wp-json\/wp\/v2\/media\/16188"}],"wp:attachment":[{"href":"https:\/\/lsacademy.com\/it\/wp-json\/wp\/v2\/media?parent=19398"}],"wp:term":[{"taxonomy":"product_brand","embeddable":true,"href":"https:\/\/lsacademy.com\/it\/wp-json\/wp\/v2\/product_brand?post=19398"},{"taxonomy":"product_cat","embeddable":true,"href":"https:\/\/lsacademy.com\/it\/wp-json\/wp\/v2\/product_cat?post=19398"},{"taxonomy":"product_tag","embeddable":true,"href":"https:\/\/lsacademy.com\/it\/wp-json\/wp\/v2\/product_tag?post=19398"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}Key Conference Topics<\/h3>\n
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Scientific Board<\/h3>\n
\nLisa Comarella, <\/strong>Senior Director Biostatistics at Alira Health Biometrics<\/em>
\nFabiola La Gamba, <\/strong>Associate Director Biostatistic at Novartis<\/em><\/p>\n
\nWho Should Attend ESForum 2026?<\/h3>\n
\nESForum \u2013 Building Opportunities<\/h3>\n