PhD Optimizing rare disease registry, statistical design and trial tools
Research in the Amsterdam Leukodystrophy Center aims at improving insight into leukodystrophies and developing treatment. The disease “vanishing white matter” (VWM) is a major research focus since long. VWM mostly affects children and causes increasing neurological handicap and early death.
We defined this disease 30 years ago and identified the related genes in 2001. In 2004, we started a patient registry for VWM, containing now data from approximately 500 patients worldwide with information on the disease course for up to 30 years. With growing insight into disease mechanisms, several therapeutic interventions have been developed and tested with promising results. VWM is ultra-rare, hampering trial design and analysis.
As PhD student, you will work on the analysis of the VWM registry. Disease courses change over the years and an important question is from when on is the disease course of untreated patients in the registry comparable with the disease course of current patients? From when on can registry data be regarded as relevant real life data, which can be used for comparison in clinical trials?
You will also work on emerging and recently completed trials in VWM and design and optimize tools for statistical analysis of small data sets.
- Familiarize yourself with VWM and clinical trial methodology.
- Analyze the disease course of patients with VWM, using data from the VWM registry, and determine from when on the disease course is similar to the current disease course.
- Write a narrative review of the different existing statistical approaches for single-arm and two-arm drug trials for ultra-rare diseases, their suitability in specific settings, and their limitations.
- Develop an optimal design of a single arm phase 2 trial in children <6 years with external control data from a historical cohort from the VWM registry.
- Develop an optimal randomization schedule of a randomized two arm phase 2/3 trial in patients ≥6 years.
- Develop an optimal design of a randomized two-arm delayed start phase 2/3 trial in patients ≥6 years.
We are looking for an enthusiastic PhD student with:
- Master degree in statistics, epidemiology, econometrics or related field.
- Proven interest in the (bio)medical field.
- Good programming skills, preferably in R or Python.
- Fluent in English.
- Motivated and able to work independently and in teams.
- Excellent communication skills.
- A flying start to your career in scientific research.
- Plenty of room for your drive to shape tomorrow's healthcare.
- Working on large-scale and in-house research, with motivated colleagues from all over the world.
- A contract for 12 months (with the intention to extend).
- Salary scale OIO: in the first year € 3.017 till € 3.824 in the fourth year based on full-time employment and a year-end bonus of 8.3%. Calculate your net salary here.
- Holiday hours: 186,4 per year for fulltime and a possibility to save additional hours.
- Pension accrual with BeFrank, a modern, comprehensible and fairly priced pension.
- For >7 km each way, 100% reimbursement for public transport travel costs and, for private transport, €0.18 per km up to a maximum of 40 km each way.
- Do you prefer walking or cycling? Take advantage of our good bike scheme. Moreover, you will receive a reimbursement of €0.18 per km.
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We offer an excellent research environment in two collaborating teams. One is a multidisciplinary team focusing on many different aspects of leukodystrophies. The leukodystrophy team consists of 6 research PI’s, one post-doc, 2 trial coordinators, 4 child neurologists involved in trial execution, 6 technicians, 12 PhD students and numerous interns. Some work on clinical aspects of the disease using several patient registries. Some work in the lab and execute studies on disease mechanisms, with the aim to better understand the disease and find openings for treatment. Some work on clinical trials in patients. Some of these trials are based on our own results and have been initiated by our team.
The second team concerns Epidemiology and Data Science, which consists of 7 research teams and aims to improve clinical research and healthcare through novel methodological contributions. The department consists of biostatisticians, epidemiologists, quantitative psychologists, health economists, and bioinformaticians and about 40 PhD students. This project will be carried out in the Decision Modeling Center, a multidisciplinary team of 3 research PI’s, 5 assistant professors, 2 postdocs and 9 PhD students. The team works closely with other statistical and epidemiological teams in the department such as the big stats team and meets at monthly departmental research meetings.
Working in those two teams means that you will gain insight into many different aspects of leukodystrophies and clinical trial development.
During the publication period, applications will be handled continuously. If the vacancy is filled, it will be closed prematurely.
If you have any questions about this position, please feel free to contact Prof. Dr. Marjo van der Knaap, pediatric neurologist, via ms.vanderknaap@amsterdamumc.nl, or prof. Dr. Hans Berkhof, statistician, via h.berkhof@amsterdamumc.nl.
For more information about the application procedure, please contact Rhiannon Sandfort, Recruitment advisor, via r.e.sandfort@amsterdamumc.nl.
A reference check, screening and hiring test may be part of the procedure. Read here whether that applies to you. If you join us, we ask you for a VOG (Certificate of Good Conduct).
Internal candidates will be given priority over external candidates in case of equal suitability.
Acquisition in response to this vacancy is not appreciated.