A strategic framework for global access and personalised care

Rare diseases, while individually uncommon, collectively impact over 300 million people worldwide, representing approximately 3.5–5.9% of the global population. Despite this significant burden, more than 90% of rare diseases lack approved treatments, leaving patients with limited – or no – therapeutic options.

As science advances, rare disease treatments increasingly rely on longitudinal real-world data (RWD) to confirm therapeutic effectiveness, inform reimbursement decisions, and personalise care pathways.

Unlock access and personalised care with real-world evidence

In this paper, we cover how to:

Enable early access through agile, regulatory-aware distribution
Personalise care with digitally enabled patient support programmes (PSPs) and real-world insights
Demonstrate therapeutic value through patient insights

By integrating the patient voice into design and delivery, Sciensus ensures that clinical and operational priorities align with real-world needs – accelerating meaningful impact in rare disease care.

Download free white paper

About Sciensus

Sciensus is a proven life sciences solutions partner with more than 30 years’ experience in navigating and unlocking the complex European healthcare ecosystem to maximise patient reach. We help accelerate the development journey, bringing medicines into the market and directly to patients to improve health outcomes. Through cutting-edge patient engagement programmes, offering an exclusive combination of skilled in-person care and digital support, we help patients make the most of their treatment and generate real-world evidence that can be used to improve treatment pathways.

Learn more