Description: Early phase trials are recognised to have a major impact on later phase confirmatory trials. This course presents state of the art methodology for Phase I dose-escalation studies that are delivered a mixture of lectures and practical sessions. The topics include model-based dose-escalation in-patients studies (such as Continual Reassessment Method [CRM], Bayesian logistic regression model [BLRM], Escalation with Overdose Control [EWOC]), designs incorporating multiple endpoints, and implementation of these designs in a software (specifically, R).

Please note: We have up to 10 NIHR-funded places available for statisticians employed by clinical trial units (CTU) in the UK. These are funded by the NIHR as part of Dr Mozgunov’s Advanced Research Fellowship, and the course fee is waived for the eligible attendees. The places will be assigned prioritizing personnel from different CTUs. If multiple attendees apply from the same CTU, the places will be allocated on a first-come-first-served basis. To register, please contact Dr Mozgunov at pavel.mozgunov@mrc-bsu.cam.ac.uk using your CTU email address and mentioning which CTU you are working on.

Location: In-person

Duration: Tuesday 26^th September 2023 (full day)

Course Tutors: Dr Pavel Mozgunov (MRC Biostatistics Unit, University of Cambridge) & Prof Thomas Jaki (MRC Biostatistics Unit, University of Cambridge)

Important Information: Attendees are asked to bring their own laptops with R installed for the practical sessions.

Registration: Short Course on Single-Agent Phase I Dose-Escalation Studies, September 2023 | University of Cambridge

Description: Therapies using a combination of drugs have become the mainstream approach in many therapeutic areas. They, however, require more advanced designs to establish the best dose for further study compared to single-agent trials. This course presents novel dose-finding designs for combinations and dose-schedule trials that are delivered a mixture of lectures, practical sessions, and discussion. The topics will include:

• Model-based and model-free Phase I dual-agent and dose-schedules designs;
• Phase I/II designs for molecularly targeted agents;
• Randomised dose-escalations trials;
• Inclusion of covariates into Phase I dose-escalation;

Throughout the course, the examples of implemented in practice designs will be used to demonstrate the methodology. The workshop will involve practical sessions in R on these designs implementations and discussions on the practical aspects. By the end of the course, you will design your own combination or dose-schedule trial.

Location: In-person

Duration: Wednesday 27th and Thursday 28th September 2023 (two full days)

Course Tutors: Dr Pavel Mozgunov (MRC Biostatistics Unit, University of Cambridge) & Prof Thomas Jaki (MRC Biostatistics Unit, University of Cambridge)

Important Information: Attendees are asked to bring their own laptops with R installed for the practical sessions.

Registration: Short Course on Dose-finding Designs for Combination Treatments, October 2022 | University of Cambridge

Many confirmatory clinical trials turn out to be negative and do not lead to the registration of a new drug or the introduction of a new treatment. Consequently, there is growing interest amongst clinical researchers in the use of efficient methods for conducting early and late phase trials to identify the most promising compounds, and in using interim analyses to allow trials to be stopped as soon as there is sufficient evidence to reach a convincing conclusion.

Adaptive designs allow mid-trial modifications to be made to the trial so that the accumulating data guides investigators to the most appropriate form of evaluation. While offering great flexibility during the trial, they require specialised approaches to be designed and analysed.

In this course, we are going to introduce the definition of adaptive designs and consider its several most common types that are delivered through a mixture of lectures, practical sessions, and discussions.

Specifically, we are going to explore:

1.  Group Sequential Designs

2. Multi-Arm Multi-Stage Designs

3. Sample Size Re-estimation

4. Response Adaptive Randomisation

5. Master protocols

Throughout the course, the examples of implemented in practice designs that the MRC BSU has contributed to recently will be used to demonstrate the methodology. The workshop will involve practical sessions in R on these designs implementations and discussions on the practical aspects. By the end of the course, you will design your own adaptive trial.

Upcoming course dates: 30th October – 1st November (3 Full Days)

Course Tutors:

Prof Thomas Jaki (MRC Biostatistics Unit, University of Cambridge)

Dr Sofia Villar (MRC Biostatistics Unit, University of Cambridge)

Dr David Robertson (MRC Biostatistics Unit, University of Cambridge)

Dr Pavel Mozgunov (MRC Biostatistics Unit, University of Cambridge)

Dr Martin Law (MRC Biostatistics Unit, University of Cambridge)

Important Information: Attendees are asked to bring their own laptops with R installed for the practical sessions.

Registration: Adaptive Methods in Clinical Research 30 October – 1 November 2023

Description: The majority of biologic and small molecule drugs perturb protein targets to exert their effects. With the recent explosion in the availability of large-scale genetic association data, it is increasingly feasible to identify genetic variants that proxy the effect of perturbing a protein drug target. Such leverage of genetic data thus offers an efficient and cost-effective approach for identifying drug targets and studying their effects. While this course is entirely self-contained, it follows on naturally from the Mendelian Randomization course. Participants who are interested in both topics may wish to apply for both courses.

Location: Online

Duration: 1 week, 5 live sessions (x2 courses per year)

Course Tutors: Dr Dipender Gill, (Novo Nordisk/Imperial College London), Dr Ville Karhunen (University of Oulu), Dr David Ryan (University College London), Dr Stephen Burgess (MRC Biostatistics Unit, University of Cambridge)

Upcoming course dates: 27th November – 1st December 2023

Course fee: £300 for all participants

Registration: Genetics in Drug Development | November 2023

More information: Genetics in Drug Development course (mendelianrandomization.com)

Description: Studies based on Mendelian randomization are increasingly being used to distinguish causal relationships from observational associations in epidemiology and to prioritize potential targets for pharmaceutical intervention. This course intends to explain both simple and more complex statistical methods for causal inference in Mendelian randomization studies, and the instrumental variable assumptions on which they are based. The course includes several computing practicals in R.

Location: Online

Duration: 3 weeks, 4 live sessions per week (x2 courses per year)

Course Tutors: Dr Stephen Burgess (MRC Biostatistics Unit, University of Cambridge), Dr Verena Zuber, (Imperial College London), Dr Apostolos Gkatzionis (MRC Unit, University of Bristol), Dr Andrew Grant (University of Sydney), Dr Amy Mason (Cardiovascular Epidemiology Unit, University of Cambridge), Dr Dipender Gill (Novo Nordisk/Imperial College London)

Upcoming course dates: 6th November – 24th November 2023

Course fees: £200 (student registration), £380 (public sector registration), £500 (private sector registration)

Registration: Mendelian Randomization Course | November 2023

More information: Mendelian Randomization course (mendelianrandomization.com)

Description: This short course introduces students to Bayesian statistical methods in biomedical settings, and provides skills for designing, assessing and interpreting Bayesian analyses using the R and JAGS statistical software. The emphasis throughout will be on practical, applied modelling: code to carry out analyses will be provided.

Location: Online

Duration: The course will be delivered via the Moodle online learning platform. The course consists of 7 half-days worth of content, and will take place over 7 half-days across 3 weeks starting on Friday 17^th November 2023.

Course tutors: Dr Anne Presanis (MRC Biostatistics Unit, University of Cambridge), Dr Robert Goudie (MRC Biostatistics Unit, University of Cambridge), Dr Christopher Jackson (MRC Biostatistics Unit, University of Cambridge)

Course dates: Friday 17th November 2023 – Friday 1 December 2023

Course fees:

Students and/or LMIC participants: £200

Academic: £380

Industry: £500

Registration: Open Soon

More information: Bayesian Statistics

Description: Bayesian statistics is an approach to data analysis, wherein prior beliefs about a certain quantity of interest can be formally incorporated and updated when new data become available. There is a big role for Bayesian statistics to play in making clinical trials more efficient. This two-day short course is designed to provide an introduction to both basic aspects and advanced features of Bayesian methods with applications to clinical trials. Day 1 will be a primer in Bayesian design and analysis of clinical trials, including fundamentals of Bayesian statistics, resemblance between Bayesian and classical trial designs, prior elicitation, and practical implementation of Bayesian designs in statistical software. Day 2 will focus on Bayesian adaptive methods for clinical trials, where modifications during the trial are enabled. The course will be a mix of lectures and practical session during which the attendees will apply learned methods in problems motivated by real clinical trials.

Location: In Person –

MRC Biostatistics Unit
East Forvie Building
Forvie Site
Robinson Way
Cambridge Biomedical Campus
Cambridge
CB2 0SR

Duration: 2 days

Course Tutors:

Dr Haiyan Zheng
Dr Pavel Mozgunov
Prof Thomas Jaki

Course fees: £300 (student registration), £460 (public sector registration), £600 (private sector registration)

Programme and Course outline