Precision Medicine Clinical Trials

18th November - 19th November 2024, Newcastle upon Tyne, UK

Dates and times

Monday 18th November (9.15am to 5.15pm) and Tuesday 19th November (9.00am to 4.00pm) 2024

Location

Newcastle upon Tyne

Newcastle is easily accessible via train (frequent trains from London and Edinburgh) and air (Newcastle airport has multiple daily flights to London Heathrow, Schiphol, and Frankfurt).

Cost and Registration

Private sector: £640 Public sector: £425 Students: £320

To enrol, please go to the Newcastle University Webstore

Registration includes lunch and refreshments over the two days and a course dinner on the evening of day 1.

Description

Precision medicine is about going beyond assessing whether a new treatment works on average to predicting which subgroups of patients receive benefit and to what extent. When subgroups, often defined by biomarkers, genetic, phenotypic or psychosocial characteristics, are associated with a treatment’s efficacy or toxicity, precision medicine offers substantial advantages to patients, trial sponsors, and the wider healthcare system. However, a barrier to realising the promise of precision medicine is the inappropriate use of traditional clinical trial design and analysis, which rely on estimates of population-averaged effects.

In this course we introduce the concept of precision medicine and cover innovative approaches. This includes novel designs (biomarker-guided, basket, umbrella, adaptive signature, adaptive enrichment designs and Sequential Multiple Assignment Randomized Trials) as well as efficient analysis approaches (e.g. Bayesian hierarchical modelling, identifying patient subgroups using high-dimensional genetic data) to help estimate sub-group specific treatment effects. These approaches have all been developed to improve the power, quality of information, and patient benefit provided by clinical trials. Examples from a wide variety of therapeutic areas will be discussed, with implementation in R software. Perspectives will be given on the future development of design, conduct and analysis of clinical trials in the field.

Tutors

Lectures

  1. Precision medicine and traditional clinical trials

  2. Basket trials and borrowing of information

  3. Umbrella and platform trials

  4. Analysing high-dimensional biomarker data in trials (Part I)

  5. Adaptive enrichment and error rate control

  6. Adaptive interventions and chronic disease approaches (Crossover trials, N-of-1 trials, SMART designs)

  7. Analysing high-dimensional biomarker data in trials (Part II)

  8. Practical considerations for precision medicine trials

FAQs

Q: Is the course running in person or online?

A: Currently we are only planning to run the course in person

Q: I’m not a statistician, will I benefit from the course?

A: The course is predominantly aimed at applied statisticians, but hopefully many parts of the course will be useful to non-statisticians! Some lectures may be more difficult but if you don’t mind some Greek letters and other mathematical symbols, you should be okay.

Quotes from 2023 participants

“The overall course was excellent, extremely knowledgeable presenters and the material was very well presented.”

“Very interesting course. Teachers very attentive to the needs of participants and available for any clarification”

“It was great, very organized, helpful resources, interesting talks.”