My first months at Sciensus: reflections on building the next phase of rare disease care in Europe
A few months into a new role is often when perspective starts to shift. The early introductions and orientation fade into the background while the reality of how a business operates comes into focus.
What has struck me most is the strength of the operational foundations in place. Sciensus delivers a consistently high level of service, often for treating institutions and patients with ultra-rare conditions where there is little margin for error. I’ve seen it in the day-to-day delivery of medicines and nursing care. In whether things happen when they are meant to and in how patients are supported when treatment feels hard. Consistency makes it easier for patients to stay on therapy, and over time, it builds trust with patients and clinicians.
One aspect, not always fully understood outside the organisation, is the breadth of the end-to-end supply chain capability that already exists. From import, secondary packaging, QP release and early access supply; through to commercial competitive tendering and finished product delivery directly into hospitals and pharmacies, much of the complexity biopharma companies often manage across multiple partners is handled within a single integrated model. This reduces touchpoints, accelerates access and allows biopharma companies to focus on innovation, market expansion and value propositions, knowing the essential step of getting therapies to patients as efficiently and safely as possible is with a trusted partner.
At the same time, coming in with fresh eyes has clarified where the next phase of opportunity lies. Rare disease care is complex by nature, but it is also deeply personal. For patients with ultra-rare conditions, treatment does not sit in isolation. It affects families, routines and long-term quality of life. Coordinated, sustainable care matters because it reduces uncertainty and makes the journey more manageable for both patients and caregivers. When support is in place, the clinical experience and the long-term value of the therapy are better aligned.
This is where I see significant potential for Sciensus. Our clinicians spend time with patients in real-world settings and hear things rarely captured elsewhere. Conversations about side effects, daily impact and quality of life often surface insights that do not appear in formal datasets. When handled responsibly and fed back into the system, this insight can improve patient education, support healthcare professionals and help shape data and evidence for access and reimbursement.
Crucially, this insight only has value because it sits within an integrated model. Early access, clinical care, direct distribution and in-market support reinforce one another. Together, they enable earlier access to treatment, improved patient identification, more efficient and targeted launches and, ultimately, sustainable access models over time.
The broader European orphan medicines environment is also changing. Changes to market exclusivity duration are intended, including shorter exclusivity periods depending on the type of orphan medicinal product, increasing pressure on timelines mean companies will miss revenues if they treat Europe as an afterthought once the US is established. Value proposition, launch sequencing, early access planning and country-specific pathways across Europe need to be considered much earlier to maximise value creation, even for organisations planning to partner or out-license ex-US.
Looking ahead 18 months, my ambition for the rare and specialty business at Sciensus is to be recognised as a genuinely consultative value-adding partner. One that brings practical insight and evidence into every engagement. Which means bringing our access and clinical service capabilities, value storytelling and real-world insight together more deliberately, and helping partners focus on outcomes that matter to patients and caregivers, not just regulatory endpoints.
It also means continuing to get the basics right. Innovation only delivers impact when it is built on strong operations. What gives me confidence is those foundations are already in place. From here, the opportunity is to translate real-world experience into scalable, meaningful progress for patients, clinicians and partners alike.