MSCA-DN PhD in regulatory & access pathways for leukodystrophy (Utrecht, NL)

SIGRA; Amsterdam; On-site; EUR 23,000 - 30,000; Full time

Job summary

Medicines Evaluation Board (MEB) in Utrecht, the Netherlands, hosts a POLARIS doctoral position embedded in the POLARIS consortium. The PhD student will study regulatory pathways and sustainable access to innovative therapies for rare diseases, supervised by Dr Marjon Pasmooij and in collaboration with Amsterdam UMC and other partners.

The project offers secondments across Europe, training opportunities in regulatory science, and a pathway to a PhD awarded by Vrije Universiteit Amsterdam.

Qualifications

• Master's degree in a discipline relevant to the project (medical or pharmaceutical sciences, biomedical sciences, health sciences, epidemiology or related).
• Interest in research at the intersection of rare diseases, regulatory science and access to therapies.
• Ability to work accurately and independently; ability to plan and organize research; good communication skills.

Responsibilities

• Initiate and conduct studies on regulatory pathways and sustainable access to innovative therapies for rare diseases.
• Use regulatory document analysis, expert interviews and case studies to integrate multiple perspectives.
• Undertake secondments to gain hands‑on regulatory science experience at partner institutions.
• Collaborate across Europe within the POLARIS doctoral network and with MEB and university partners.

Skills

Communication skills

English proficiency

Independent work

Research planning

Education

Master's degree in medical/pharmaceutical sciences or related field

Job description

Would you like to contribute to improving access to innovative therapies for people with rare diseases? In this PhD project, you will investigate regulatory pathways, sustainable access and reimbursement for leukodystrophy therapies. You will work across science, policy and patient perspectives.

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 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.

We are looking for a motivated researcher who is interested in rare diseases and the development and accessibility of innovative therapies. You have:

• a Master's degree in a discipline relevant to the project, such as medical or pharmaceutical sciences, biomedical sciences, health sciences, epidemiology or a related field;
• an interest in conducting research at the intersection of rare diseases, regulatory science and access to therapies;
• the ability to work accurately and independently and to plan and organise your research;
• good communication and interpersonal skills and an interest in collaborating with people from different professional and academic backgrounds;
• good academic writing skills and proficiency in English, both written and spoken.

Experience with quantitative and qualitative research methods, relevant professional or research experience, or scientific publications would be an advantage, but are not essential.

We value different backgrounds and perspectives. If you meet the essential requirements and are motivated to develop your expertise in this field, we encourage you to apply.

Eligibility criteria

Important, strict eligibility criteria according to MARIE SKLODOWSKA CURIE programs apply to applicants:

1. You must not already hold a doctoral degree.
2. At the time of the application deadline, you must have a Master's degree and must not have resided or carried out your main activity (work, studies, etc.) in the Netherlands for more than 12 months in the three years prior to the contract start date.
3. You will participate in the network-wide training programme of POLARIS, which includes (international) training activities and planned secondments with POLARIS partner organizations throughout the EU.

• A contract for 12 months, with the intention to extend for a total of 3 years;
• Salary, mobility allowance and benefits according to the applicable MSCA Doctoral Network and host-organisation rules;
• Embedding in the international POLARIS doctoral training network;
• Access to network-wide training activities, secondments and transferable-skills development;
• A challenging and interdisciplinary research project at the interface of rare disease research and regulatory science;
• An open and supportive team culture with opportunities for scientific and professional development.

The Medicines Evaluation Board (MEB) is the Dutch governmental agency responsible for assessing and monitoring the efficacy, safety and quality of human and veterinary medicinal products in the Netherlands. The MEB also contributes to research that supports appropriate treatment with medicines.

You will join the Science Department, which coordinates scientific activities within the MEB. The department is involved in around 25 PhD projects and organises regular PhD and Master's student presentations, alongside learning and social activities. The MEB works closely with the European Medicines Regulatory Network, including the European Medicines Agency.

Dr Marjon Pasmooij is a regulatory scientist and Head of the Science Department at the MEB, as well as Associate Professor of Drug Regulatory Science at Utrecht University. Her work focuses on regulatory science and the translation of innovative therapies, particularly for rare diseases.

You will also be part of the international POLARIS doctoral network. You will work with researchers from different scientific disciplines and collaborate with academic and industry partners across Europe. Through secondments, network-wide training and transferable-skills development, you will build experience beyond your own research project and develop an international research network.

POLARIS is committed to open, transparent and merit-based recruitment. We value diversity and welcome candidates with different backgrounds, perspectives and experiences.

The application deadline is 5 October 2026. We aim to select the final candidate in October/November 2026.

Applicants may, where appropriate and in line with the recruitment procedure, also be considered for other POLARIS doctoral candidate positions that better match their profile and interests. You can highlight up to two additional POLARIS projects of interest in your application letter. This does not replace your application to the primary position, but helps the consortium identify potential alternative matches where relevant.

Would you like to know more about this PhD position? For substantive information about the vacancy, please contact Dr Marjon Pasmooij, via [e-mail].

Acquisition in response to this vacancy will not be considered.