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

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 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. 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