Explore Orfenix Insights, where we share our latest thoughts, research findings, and perspectives on advancing affordable therapies for rare diseases. Dive into in-depth articles, case studies, and collaborative stories that reflect our commitment to innovative drug repurposing and socially responsible healthcare.
Webinar: How do you design a clinical study that is registration-ready?
From protocol to registration: learn what changes when your endpoint is approval, not publication. Practical guidance on study design, CTIS readiness, GCP execution, and key post-study obligations.
Funding socially responsible drug development
Learn how to design a funding mix that gets your socially responsible drug from lab to patient, without losing sight of access and fair pricing.
How does a public-private-partnerships in drug development work?
Learn what a Public-Private Partnership in drug development is, how it works in practice, and when it is the right structure for your project.
Webinar: how do you build an impact-driven spin-off out of academia?
Learn how to build a venture to turn science into reality. Socially. Responsible.
When Does Your Research Need a Venture? A Guide to Socially Responsible Drug Development
How do you get your therapy to patients? This guide helps academic researchers, clinicians, and principal investigators choose if they should consider setting up a venture for that specific goal.
Socially Responsible Drug Development: From Vision to Patients
Many projects stall between the lab and the clinic. Socially responsible drug development is a way to cross that gap with patient access built in.
How to Select the Right Academic Development and Manufacturing Support
Choosing the wrong development manufacturing partner can derail academic research with failed submissions and costly delays. Use these practical guidelines to choose the right development partner.
Paediatric Investigation Plan (PIP) Guidelines: What, Why, When, and Who
Learn more about Paediatric Investigation Plans (PIPs) and how they shape your regulatory strategy, clinical timeline, and EMA approval pathway.
Cystinosis: the past, present and future
Cystinosis is a rare hereditary disease that affects multiple organs, of which the kidneys and eyes are affected first. In conversation with Irene Kinds from the Cystinosis Patient Organisation Vlaanderen and The Netherlands, and mother of a 16-year-old living with cystinosis, we discuss what daily life with the disease looks like, why early diagnosis and adherence to therapy matter, and how new treatments could change the future for patients worldwide.
Things to get right when conducting investigator-initiated clinical trials
Investigator-initiated trials drive innovation but require careful planning. Success depends on clear protocols, compliance, data quality, safety, and transparency. This ensures credible, clinically significant results that complement industry research and advance patient care.
Do you want to make an impact on patients’ lives?
At Orfenix, you work with top professionals and collaborate with leading experts to bring forgotten treatments from the lab to patients.
If you’re passionate about transforming healthcare and thrive in a transparent, trust-driven environment, Orfenix is the place for you.