EN SV HOME THERAPEUTIC AREAS RESEARCH & DEVELOPMENT INVESTIGATOR SPONSORED STUDIES Investigator sponsored studies An Investigator-Sponsored Study (ISS) is an unsolicited preclinical, clinical, outcomes or disease-state study. In an ISS, an investigator independently generates a research proposal, and if approved Sobi provides support for the proposal. Support can include, but is not limited to, drug material, expert advice, funding to pay for expenses directly related to the research based on fair market value, or a combination of these items. The investigator, or the institution the investigator is affiliated with, serves as the study sponsor. As such, the sponsor assumes responsibility for initiating and conducting the study, directing the administration of study drug (if applicable), ensuring compliance with all local laws and regulatory requirements, and analysing and communicating all study results. Sobi recognises the important role that ISSs can play in expanding the knowledge related to Sobi's products and their associated disease areas. This research can advance science and contribute to the development of better medicines for patients consistent with the company's overall research and global development strategies. Areas of interest Haematology Haemophilia The following areas of research are currently seen as supporting novel, independent research that advances general knowledge for Sobi's products within haemophilia. Clinical studies Studies on intensification of prophylaxis, to aim for normalisation of FVIII levels Biomarkers and novel assessments of microbleeding, bleed resolution, inflammation and joint damage Studies on immunogenicity and induction of immune tolerance Joint health and dysfunction Outcomes related to comorbidities of ageing haemophilia population e.g., cardiovascular, diabetes mellitus, cancer Patient, caregiver and health-economic studies Such as Quality of Life, psychosocial function, pain, work productivity, pharmacoeconomics, and health resource utilisation Immune Thrombocytopenia (ITP) Differentiation of the thrombopoietin receptor class preclinically Patient reported outcomes and measures of convenience Bleeding outcomes in ITP Burden of disease (outcomes and financial cost) Sequencing of medications used to treat ITP (switching between therapies) Cost effectiveness in selected populations or cost-settings Adherence studies Real World Evidence generation Chronic Liver Disease (CLD) Bleeding outcomes associated with thrombocytopenia in chronic liver disease planned for an invasive procedure Immunology The following areas of research are currently seen as supporting novel, independent research that advances general knowledge for Sobi's product such as anakinra. Clinical studies Clinical outcomes in IL-1 autoinflammatory diseases Clinical outcomes and predictors of response (e.g., biomarkers, patient characteristics) to anakinra therapy in rheumatoid arthritis Proof of concept and efficacy studies for the role of anakinra to affect clinical outcomes in IL-1 driven diseases. These diseases must be well-characterized and have strong evidence for the role of IL-1 in their pathophysiology. Given the current situation with the COVID-19 pandemic, the review of prospective studies with anakinra is temporarily halted, having in mind to focus on this disease and the current patients with rare disease treated with anakinra. Review process Submission requirements SOBI ISS PORTAL SOBI ISS PORTAL SUPPORT Sobi ISS Portal support 754 KB About us At a glance Rare diseases Therapeutic areas Haematology Immunology Specialty Care portfolio Investors Reports & presentations Share information Calendar Career Open positions Meet our employees Media Press releases Gallery Sobiplay Sitemap Cookie Policy Privacy Policy Terms of use Cookies Settings © Swedish Orphan Biovitrum AB (publ) Reg. no. 556038-9321