Active Studies
Ongoing investigator-led research projects
Investigator-Initiated Research
Supporting independent research initiatives in rare diseases
Our IIR programme partners with independent researchers and academic institutions to advance scientific understanding and clinical outcomes in rare disease research. We provide support for innovative studies that align with our commitment to improving patient lives.
Our Work With the Medical Community
At Orphalan, we continuously engage with healthcare professionals and patient advocacy groups in our common goal of improving care and services for patients with rare disease. Together, by taking a patient-centric approach, in partnership with healthcare professionals, our mission is to collaborate to advance knowledge in rare disease and in particular to identify and find innovative solutions for the unmet needs of patients.
Orphalan along with our network of Key Opinion Leaders strive to design meaningful and important studies to improve patient care and outcomes. It is through close collaborations with healthcare professionals and scientists, that Orphalan is able to address these unmet needs of patients with rare disease and potentially transform their lives through discovery and innovation.
What is Investigator-Initiated Research (IIR)?
As part of our commitment to delivering innovative therapies to patients, Orphalan believes and values in the need to support ethically conducted independent clinical research by qualified third-party investigators.
Orphalan views scientific research produced by third party sponsors as complementary to Orphalan-sponsored research. Investigator initiated research (IIR) are studies with scientific and medical merit developed and sponsored by an independent investigator aligned with Orphalan's areas of interest or scientific priorities. These priorities are to help Orphalan to better understand the benefit/risk profile of our therapies, as well as enabling us to explore new opportunities addressing unmet medical needs.
How IIR Works
Researcher Initiates
An independent investigator designs a study addressing a rare disease question
Proposal Submitted
A formal proposal is sent to Orphalan outlining the study objectives and support required
Orphalan Reviews
Our team assesses scientific merit, ethical standards and alignment with our areas of interest
Study Conducted
The investigator leads the research independently; Orphalan provides agreed funding or study drug
Areas of Interest
Orphalan supports research projects related to our therapeutic products, diagnostic tests and disease of interest and that are aligned with our R&D programs.
Wilson's Disease
Research related to copper metabolism disorders and chelation therapy
Infantile Spasms (West's Syndrome)
West's syndrome and epilepsy research
Glioblastoma
Research in rare brain tumours including glioblastoma
Diagnostic Innovation
Novel diagnostic tests for rare diseases
IIR Program Impact
Since the launch of our IIR programmeme, Orphalan has supported independent investigators across multiple countries and therapeutic areas, contributing to the generation of real-world evidence in rare diseases.
Principal Investigators
Leading researchers and clinicians worldwide
Countries
International reach across Europe and beyond
WE PARTNER
Interested in Collaborating?
We welcome proposals from qualified investigators in rare disease research. To learn more about our IIR programmeme or to submit a proposal, please contact our team.