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:
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Enable early access through agile, regulatory-aware distribution
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Personalise care with digitally enabled patient support programmes (PSPs) and real-world insights
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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.
About Sciensus
Sciensus is the UK’s leading provider of complex clinical care at home. For over 30 years, we have partnered with the NHS to deliver specialist medicines, clinical nursing and patient support to people living with cancer, rare diseases and complex long-term conditions – supporting more than 300,000 patients a year across 180+ NHS trusts.
Beyond the UK, Sciensus helps pharmaceutical and biotech companies reach patients across Europe, managing the regulatory, logistics and patient support complexity of launching medicines in multiple markets.
