This study examines patient access to orphan medicinal products (OMPs) in Central and Eastern Europe (CEE) over the past five years, focusing on seven countries: Bulgaria, Czechia, Hungary, Poland, Romania, Slovakia, and Slovenia. While these jurisdictions have undergone rapid healthcare transformations, significant disparities in OMP access persist compared to Western Europe. This study aimed to address this gap by identifying barriers and enablers to optimize patient access to OMPs in a sustainable and equitable manner. A mixed-methodology approach was utilized, combining systematic literature reviews, in-depth interviews, and advisory board insights. Perspectives were gathered from a wide range of stakeholders, including policymakers, payers, academia, industry associations, and patient advocacy groups. Additionally, the study incorporated data from CEE-specific initiatives to triangulate findings and evaluate barriers, enablers, and best practices in OMP access. The analysis identified sub-optimal OMP access across most CEE countries, marked by prolonged delays and lower reimbursement rates compared to Western Europe, with Slovenia and Czechia as notable exceptions. Key barriers include limited awareness, inadequate health technology assessment (HTA) frameworks, insufficient financing mechanisms, underutilization of novel access schemes, and fragmented patient engagement. Conversely, enablers include the presence of rare disease policies, OMP-specific HTA frameworks, and patient-inclusive decision-making processes.

Common Position of Scientific Societies: Polish Society of Dermatology (PTD), Polish Society for Fundamental and Clinical Immunology (PTiDiK), Polish Society of Paediatric Oncology and Haematology (PTOHD) Background and Objectives This paper aims to analyse, assess and appraise appropriate use of Normal Polyvalent Immunoglobulins (IgGs) across multiple indications, wherever these therapies are known to be used and have been proven to be an effective therapeutic modality. This assessment is based on a framework proposed by a group of international experts in the recent Green Paper „Appropriate Use of Immunoglobulins in Europe”.1 Based on a systematic application of the framework’s criteria to all IgG conditions and diseases, this paper presents a short-list of indications, where IgG use is appropriate, together with a guidance on optimal therapeutic paradigms. As such, this paper is aimed to assist the clinical community and policy makers in navigating the extremely complex clinical landscape of rare and ultra-rare diseases and conditions, which are either immune-mediated or stem from deficient, dysregulated or dysfunctional immune system. Some of the indications considered in this study are currently not registered for IgG use in Poland or, if registered, not fully covered with reimbursement. In cases where IgG use in such conditions or diseases is found to be appropriate, this study may be also considered by the relevant regulatory and/or HTA bodies as a contribution towards reassessment or reappraisal.

Dysimmune inflammatory neuropathies (DINs), including Guillain-Barré syndrome (GBS), chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), and multifocal motor neuropathy (MMN), are rare immune-mediated disorders of the peripheral nervous system associated with substantial diagnostic, therapeutic, functional, psychosocial, and policy-related unmet needs. This narrative review synthesises published evidence, clinical guidance, and patient-perspective inputs to characterise the European DIN patient pathway and identify priorities for improved care. The review highlights persistent challenges in early recognition, differential diagnosis, timely treatment initiation, long-term immunoglobulin use optimisation, rehabilitation, outcome measurement, and patient engagement. It also identifies important inequities in access to immunoglobulin therapy and emerging treatments, driven by plasma supply constraints, heterogeneous reimbursement criteria, affordability barriers, and inconsistent implementation of evidence-based care. Addressing these gaps will require harmonised DIN care pathways, systematic use of patient-reported outcomes, integrated rehabilitation, strengthened plasma supply resilience, real-world evidence generation, and structured patient involvement in clinical, regulatory, reimbursement, and policy processes. A coordinated European approach could improve outcomes for patients with DINs and provide a model for patient-centred policy reform in rare neurological diseases.