Making the right decision in socially responsible drug development starts with one question: 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.

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.

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.

Learn more about Paediatric Investigation Plans (PIPs) and how they shape your regulatory strategy, clinical timeline, and EMA approval pathway.

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.

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.

Developing a new medicine is a long, costly journey—especially in rare diseases or drug repurposing—where traditional funding models often fall short. Public-Private Partnerships (PPPs) offer a powerful alternative, bringing academics, industry, and patient groups together to share risks, combine expertise, and keep patient access at the heart of development.

Drug repurposing gives existing medicines a second life, but only when the business model clears the path. At Orfenix, we structure Intellectual Property strategically to align investor incentives, guarantee patient access, and deliver sustainable impact. Discover how our IP approach protects innovation and connects commercial potential with public health outcomes.

Orfenix proudly supports the development of guanabenz for Vanishing White Matter—a rare, progressive brain disease in children. Learn how repurposed drugs and dedicated research offer hope.