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Pharmacometric modeling of composite score outcomes

Lambertville, NJ, USA
March 12 - 13, 2020


The EDSS in multiple sclerosis, the ACR scale for rheumatoid arthritis, or the MDS-UPDRS in Parkinson’s disease; these composite scales are as diverse as the diseases they are designed to measure. Composite scores also arise through patient-reported outcomes (PROs) that aim at measuring symptom status, physical function, mental health, and other measures important to patients. The wide range of novel pharmacometric models and approaches developed during the past years are a testament to the growing importance of this data type. This course will cover recent innovations and provide participants with a rich set of tools for analyzing composite scores in a wide variety of therapeutic areas. The course will illustrate how one can leverage finely grained item-level information but also how summary score data are most efficiently utilized. Throughout the course, questions of both model building and model-use will be discussed and presented in an interactive format including hands-on exercises.

On March 12-13 in Lambertville, NJ, Mats Karlsson and Sebastian Ueckert will give a 2-day course on “Pharmacometric modeling of composite score outcomes”. The course covers data aspects, model building, evaluation and use for composite score outcomes.

Topics covered

  • Modeling data with item-level resolution using item response theory
  • Modeling total score data either as continuous or discrete data under a variety of models
  • Models for responder analysis to handle, e.g., ACR20/50/70 and PASI70/90
  • Pharmacometric modeling of Patient Reported Outcomes (PROs)
  • Model-based optimization of clinical trials with composite score outcomes

Intended course participants

The course is designed for those who have a good working knowledge of pharmacometric analysis with experience in performing NONMEM analyses and/or have attended a NONMEM basic workshop.


Prof. Mats Karlsson
Dr. Sebastian Ueckert

Practical Information

The course will run March 12 - 13 (10:00 am - 5:30 pm on Day 1, 8:30 am - 5:00 pm on day 2) at Lambertville Station Inn & Restaurant, 11 Bridge St, Lambertville, NJ, 08530, USA.

Computer hardware/software - The course will include hands-on training, with the participants working on their own computers.  All programs will run from a USB memory-stick and participants will not be required to install any programs on their computer. All participants must bring their own Windows laptops (for MAC OS and Linux users a virtual windows environment will work).

Course Fee

Regular fee: €1,200
Student fee: €800

Registration fee includes: extensive electronic material including lectures, exercises, programs, additional self-study material including hands-on's and solutions. Morning and afternoon refreshments and lunches are included.

Questions and registration

For questions, please e-mail Sebastian Ueckert.

To register, follow this link: dinkurs.se/CompositeScoresNJ