Best practice in modelling and simulation: initiatives from PSI and EFPIA
Michael O’Kelly (Quintiles)
At an EMA-IFPIA workshop on modelling and simulation in 2011, regulators called for a Best Practice document for projects involving modelling and simulation. Finally, in the last few months, two different working groups have come up with two tools aimed at helping statisticians and their colleagues to promote and use best practice. The European Federation of Pharmaceutical Industries & Associations (EFPIA) working group on Model Informed Drug Discovery and Development (MID3) has just published a wide-ranging 90-page paper on the whys and hows of Best Practice. The paper includes as a supplement a table of 103 examples of the use of modelling and simulation in pharmaceutical science. At the same time, the Board of PSI in December adopted a Best Practice document proposed by the PSI Modelling and Simulation Special Interest Group (SIG). The two initiatives are consistent in their recommendations, with the SIG providing a template that could be used to put the recommendations for best practice into action. This presentation will identify the elements necessary for best practice in simulation of clinical trials, and survey current practice in this area.
Writing Clinical Trial Simulators: there’s more to it than the stats analysis.
Tom Parke (Berry Consultants)
When writing ones first trial simulator it’s inevitable that one’s focus (as a stats programmer) is on the novel aspect of the trial design that has required writing a simulator in the first place. However there are other aspects of trial simulation that will end up as being every bit as important. By understanding these other aspects in advance you’ll be able to better plan to accommodate them and do a better job of writing and using a trial simulator.
Assessment of various continual reassessment method models for dose-escalation phase 1 oncology clinical trials: Clinical trial data and simulation studies
Gareth James (Phastar)
Background: The continual reassessment method (CRM) is considered more efficient and ethical than standard methods for dose-escalation trials in oncology, but requires an underlying estimate of the dose-toxicity relationship (“prior skeleton”) and there is limited guidance of what this should be when little is known about this association.
Aim: To compare the CRM with different prior skeleton approaches and the 3+3 method in their ability to determine the true maximum tolerated dose (MTD) of various “true” dose-toxicity relationships.
Simulating Dose Finding Designs using Bayesian Decisions, with examples for Severe Asthma, Ulcerative Colitis and Alzheimer’s Disease.
Alun Bedding (AZ)
Choosing the correct dose for Phase III is a pivotal part of drug development. Model based approaches are the most appropriate way of doing this and Bayesian decisions are more informative. Simulation is needed in order to understand the operating characteristics of designs given different assumptions. Examples, including those for severe asthma, ulcerative colitis and Alzheimer’s disease will be used for illustration.
In clinical trial design we often use simulations to illustrate operational characteristics where analytical solutions are intractable. In this talk we will look at some case studies where these simulations have aided decision making and so increased understanding within the project team. We will look at an example of complex go/no go criteria and how these criteria were refined based on the results of the simulation. We will look at an example of how simulations were used to explore the impact of partial data on the operational characteristics of a futility analysis.
Simulating a sequences of clinical trials - a whole drug development program, in order to optimize the design, planning and resource allocation
Tom Parke (Berry Consultants)
It’s hard to judge how big a phase 2 should be, what the trade off in power and type error should be, and what the significance of other post phase 2 errors (such dose selection, population selection, estimate of effect size) might be, without considering the whole picture of time to registration, overall probability of technical success and overall value / clinical usefulness should the treatment be successfully registered.
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.