MSCA-DN PhD in regulatory & access pathways for leukodystrophy (Utrecht, NL)
Amsterdam UMC, Netherlands
- Institution
- Amsterdam UMC
- Country
- Netherlands
- Position type
- PhD
- Subject area
- Medicine & Health
- Application deadline
- 5 October 2026
- Funding
- Fully funded position
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About this position
Job description
POLARIS doctoral network
POLARIS is a European Marie Skłodowska-Curie Doctoral Network focused on accelerating therapy development for leukodystrophies: rare genetic disorders affecting the brain's white matter. The network aims to strengthen the full translational pathway, from understanding disease mechanisms and developing disease models to preclinical therapy testing, clinical readiness and patient-informed treatment priorities.
To achieve this, POLARIS brings together expertise in leukodystrophy biology, advanced disease models, therapeutic innovation, biomarker development, clinical translation, patient engagement, and ethical, regulatory and health-economic aspects of therapy development. The network will train a cohort of doctoral candidates in an international and interdisciplinary environment, combining academic research, industry-based preclinical development, secondments, network-wide training and transferable-skills development.
The PhD
For this position, the recruiting organisation is the Medicines Evaluation Board (MEB) in Utrecht, the Netherlands. You will be employed by the MEB and embedded in the POLARIS consortium. You will be supervised by Dr Marjon Pasmooij and work closely with the Amsterdam Leukodystrophy Center at Amsterdam UMC and the Amsterdam UMC platform Medicijn voor de Maatschappij, a partner of RARE-NL.
You will initiate and conduct several studies on regulatory pathways and sustainable access to innovative therapies for rare diseases. Your research will address:
You will use a combination of regulatory document analysis, expert interviews and case studies. This will allow you to bring together different scientific perspectives and contribute to knowledge that can support the development and accessibility of therapies for rare diseases.
As part of the POLARIS network, you will also undertake secondments. These will provide hands-on experience in regulatory science and orphan-drug analysis at Amsterdam UMC platform Medicijn voor de Maatschappij, as well as experience with cross-border access and reimbursement within the European Reference Network for Rare Neurological Diseases (ERN-RND) at University Hospital Tübingen.
Your PhD will be awarded by the Faculty of Medicine, Vrije Universiteit Amsterdam.
POLARIS is a European Marie Skłodowska-Curie Doctoral Network focused on accelerating therapy development for leukodystrophies: rare genetic disorders affecting the brain's white matter. The network aims to strengthen the full translational pathway, from understanding disease mechanisms and developing disease models to preclinical therapy testing, clinical readiness and patient-informed treatment priorities.
To achieve this, POLARIS brings together expertise in leukodystrophy biology, advanced disease models, therapeutic innovation, biomarker development, clinical translation, patient engagement, and ethical, regulatory and health-economic aspects of therapy development. The network will train a cohort of doctoral candidates in an international and interdisciplinary environment, combining academic research, industry-based preclinical development, secondments, network-wide training and transferable-skills development.
The PhD
For this position, the recruiting organisation is the Medicines Evaluation Board (MEB) in Utrecht, the Netherlands. You will be employed by the MEB and embedded in the POLARIS consortium. You will be supervised by Dr Marjon Pasmooij and work closely with the Amsterdam Leukodystrophy Center at Amsterdam UMC and the Amsterdam UMC platform Medicijn voor de Maatschappij, a partner of RARE-NL.
You will initiate and conduct several studies on regulatory pathways and sustainable access to innovative therapies for rare diseases. Your research will address:
- regulatory, organisational and scientific factors that influence the development of orphan medicines in academic settings;
- requirements for small-scale (“n-of-few”) manufacturing and quality control of advanced therapy medicinal products (ATMPs);
- patient-reported outcomes relevant to leukodystrophy therapies;
- cross-border access and reimbursement pathways for innovative therapies in Europe.
You will use a combination of regulatory document analysis, expert interviews and case studies. This will allow you to bring together different scientific perspectives and contribute to knowledge that can support the development and accessibility of therapies for rare diseases.
As part of the POLARIS network, you will also undertake secondments. These will provide hands-on experience in regulatory science and orphan-drug analysis at Amsterdam UMC platform Medicijn voor de Maatschappij, as well as experience with cross-border access and reimbursement within the European Reference Network for Rare Neurological Diseases (ERN-RND) at University Hospital Tübingen.
Your PhD will be awarded by the Faculty of Medicine, Vrije Universiteit Amsterdam.
Apply directly to Amsterdam UMC
Emerging Scholars Council is not the employer and does not recruit for this position. It is advertised by Amsterdam UMC, and your application goes to them. We help students prepare and strengthen their applications.
Go to the official application page →Applications close 5 October 2026.
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