Registration is free to attend!
Talks from John Scott (Deputy Director of the Division of Biostatistics in the FDA's Center for Biologics Evaluation and Research) and Lisa Hampson (Lecturer in Statistics at Lancaster University)
U.S. regulatory considerations and case studies for rare diseases
In this talk, I will present an overview of the U.S. Food and Drug Administration’s policies and practices for encouraging development of products for rare diseases and of evaluating clinical evidence for the safety and effectiveness of such products. I’ll discuss study designs that may be particularly appropriate for rare disease product development, and address some of their statistical implications. Finally, I’ll present case studies of products that were approved for rare diseases using unusual or innovative study designs and/or regulatory pathways.
John Scott is Deputy Director of the Division of Biostatistics in the FDA's Center for Biologics Evaluation and Research, where he has also served as a statistical reviewer for blood products and for cellular, tissue and gene therapies. Prior to joining the FDA in 2008, he worked in psychiatric clinical trials at the University of Pittsburgh Medical Center and did neuroimaging research with the Neurostatistics Laboratory at McClean Hospital, Harvard Medical School. He has authored or co-authored numerous articles in areas including Bayesian and adaptive clinical trial design and analysis, drug and vaccine safety, data and text mining, and benefit-risk assessment. He holds a Ph.D. in Biostatistics from the University of Pittsburgh and an M.A. in Mathematics from Washington University in St. Louis, and is an associate editor of the journal, Pharmaceutical Statistics.
Bayesian methods for the design and interpretation of clinical trials in rare diseases
For studies in rare diseases, the sample size needed to meet a conventional frequentist power requirement can be daunting, even if patients are to be recruited over several years. Rather, the expectation of any such trial has to be limited to the generation of an improved understanding of treatment options. We propose Bayesian approaches for the conduct of rare disease trials comparing an experimental treatment with a control when the primary endpoint is binary or normally distributed. We describe processes which can be used to systematically elicit from clinicians opinions on treatment efficacy in order to establish Bayesian priors for unknown model parameters. The proposed approaches are illustrated by describing applications to two Bayesian randomised controlled trials, namely a study in childhood polyarteritis nodosa and a study in chronic recurrent multifocal osteomyelitis. Once prior distributions have been established, consideration of the extent to which opinion can be changed, even by the best feasible design, can help to determine whether a small trial is worthwhile.
Lisa Hampson is a Lecturer in Statistics at Lancaster University. Her research interests are in clinical trials, including group sequential tests and Bayesian methods for trials in rare diseases and dose-escalation. Her recent research has focused on developing methods for clinical trials of new medicines for children. She holds a PhD in Statistics from the University of Bath.
An opportunity to meet statisticians from across the pharmaceutical industry in a relaxed and informal setting. An exciting program of events and a chance to work in small groups on a data analysis challenge. Lunch provided.
A Non-PSI Event - Protecting confidentiality and privacy in clinical trial and medical data sets
We are increasingly living in a data driven world. Data are collected in many different ways for a variety of purposes. As such, concerns around protecting the privacy of individuals have increased in recent times.
A PSI Training Course - Practical Approaches to Designing Adaptive Clinical Trials
This hands-on course will provide a deep dive into 4 software packages used to design adaptive clinical trials.
The course will start by providing a general overview of adaptive designs, explaining the different type of adaptations possible and the benefits of each design. Following this, participants will be given the opportunity to have a go at designing trials in R (using RPACTS), EAST, FACTS, and nQuery.
PSI Training Course - Bayesian Practical Course using R and SAS
This practical training course will give a deep dive into performing Bayesian analyses in R and SAS. It is aimed at statisticians who need to be able to conduct Bayesian analyses as part of their day to day work. By the end of the course participants will be able to conduct their own analyses.
This webinar will address operational issues of paramount importance within the healthcare industry with a view to using statistics for the benefit of patients. In attending this webinar, you'll hear more about work being conducted to address some operational issues we face in the health care industrys e.g. patient rectuitment, drug supply and meeting NHS 18 week targets.
PSI Toxicology SIG workshop – 16th and 17th March 2020
The Toxicology SIG provides a forum for statisticians working in regulatory/investigative toxicology, as well as most other pre-clinical areas, to discuss issues and interact with one another.
This 1.5-day workshop will involve approximately 20 statisticians, focusing on discussions around “best practice” in the statistical analysis of various data types.
The afternoon of Day 1 will include a 4.5 hour Bayesian training course focused towards applications in toxicology/pre-clinical, provided by Prof. Dr. Katja Ickstadt and is included in the workshop fee.
The cost will be £270 including VAT per delegate, inclusive of food and one night’s accommodation (and the training course). The workshop is being held at the Crowne Plaza Hotel, Heathrow.
The agenda and topics that will be discussed are yet to be finalised, but please get in touch with email@example.com if you have suggestions. Full details will be circulated in the coming weeks.
This course is aimed at Statisticians and Programmers experienced in SAS, but little or no experience with R.
An Introduction to R studio and the R language, statistical graphics, programming statistical models, simulations and more…
Non-proportional hazards and applications in immuno-oncology
Designs of clinical trials with time to event primary endpoints usually rely on hazards being constant over time. A major challenge in immuno-oncology is the delayed onset of benefit with such therapies and the presence of non-proportional hazards. The impact of this needs to be accounted for in sample size calculations, analysis methodology and reporting. At this meeting, we will examine possible strategies to handle such features, which may not be fully known when the trial is initiated.
The ITIT course will take 25 delegates new to the industry on a complete drug development experience from discovery to marketing. They will visit 6 companies from October 2020 to July 2021 to learn about 6 topics from experts in their field. The ITIT course will have 6 sessions in continental Europe and 3 - 4 sessions in the UK. It promises to be a truly memorable course.