Rare disease intelligence & strategy
Understand the disease.
See the opportunities.
We build comprehensive, continuously updated rare disease intelligence for pharmaceutical companies, biotechs and patient organisations. We combine scientific, clinical, stakeholder and commercial evidence to support better medical and commercial decisions across Europe. AI widens the search. Specialists validate every finding.
The challenge
Rare diseases are complex and the evidence is fragmented.
Critical information is scattered across publications, clinical trials, healthcare systems and specialist networks. This makes it difficult to understand the full picture, identify the right experts and centres, and recognise opportunities or gaps in the current landscape.
Evidence scattered across sources
Critical insights are distributed across publications, trial registries, regulators, companies and patient communities.
Incomplete disease understanding
Patient pathways, unmet needs and treatment landscapes are often difficult to connect.
Different markets, different realities
Diagnosis, treatment pathways and access vary across European countries.
Intelligence quickly becomes outdated
New research and clinical developments can change the assumptions behind existing plans.
OUR APPROACH
We build a living evidence base.
Traditional research delivers a snapshot. We build comprehensive disease intelligence foundations designed to evolve as new evidence emerges.
TRADITIONAL RESEARCH
A report
- A report representing one moment in time
- Evidence becomes outdated
- Sources may be difficult to revisit
- New questions require additional research
- Updates require a new project
Systemix living intelligence
A living evidence base
- A comprehensive, maintained disease landscape
- Relevant developments are continuously monitored
- Findings are linked to traceable evidence
- An existing evidence foundation supports further analysis
- New evidence can be incorporated into the existing foundation
Evidence you can audit
Every claim traceable.
Every decision signed.
Our intelligence is built for decisions that have to hold up in a medical, market access or investment review. Every claim carries its own ID, links to a numbered source and is backed by an archived copy of that source. The IDs make each report a living dataset. We update single claims as the evidence moves and re-check all of when needed.
- Published research
- Guidelines & HTA
- Real-world evidence
- Primary interviews and surveys
- Conference abstracts
- Regulatory sources
finding and
screening
in the loop
Reviews, accepts
or rejects
every claim.
claim in report
- Claim ID
- Numbered source link
- Archived copy
- Change log
- Named reviewer and sign-off
Traceable
Each claim links to a numbered source and an archived copy.
Signed
A named person decides on every claim. AI only ranks and drafts.
Current
Every claim is re-checked twice a year, with a full change log.
Who we help
Ecosystem readiness for every launch decision.
Biotech
ExploreComprehensive disease intelligence for medical strategy, European expansion and launch planning
Pharma
ExploreComprehensive disease intelligence for medical strategy, European expansion and launch planning
MedTech & Diagnostics
ExploreIntelligence on diagnosis, clinical pathways and adoption barriers
Our services
Comprehensive disease intelligence.
Built to evolve.
Core offering
Rare disease landscaping
Comprehensive scientific, clinical, stakeholder and commercial intelligence that establishes a reliable understanding of the disease and its ecosystem.
Explore rare disease landscaping
Keep it current
Continuous disease intelligence
Ongoing monitoring and interpretation of relevant evidence, helping medical and commercial teams keep their disease understanding current.
Explore continuous disease intelligenceOur evidence foundations can also support targeted assessments, including patient journeys, evidence gaps, treatment pathways and market-specific questions.
Latest insights
FDA proposes one licence for many versions of a personalised gene-editing therapy
The FDA has published draft guidance that would let a developer license one gene-editing product and later add versions for other patients' mutations on laboratory data, without a new clinical trial.
Rare disease drug development is evolving. What does this mean for evidence strategies?
In 2025, half of the FDA's novel drug approvals were for rare diseases. How can sponsors turn evolving evidence approaches into successful development and patient access across international markets?
EU pharma package: orphan exclusivity and HTA evidence
Under the Council's text, orphan exclusivity would be nine years, or eleven for breakthrough medicines. What this means for HTA evidence teams.
When the intelligence says stop
A Phase 3 go/no-go decision that rested on evidence, not optimism.
Anonymised examples of rare disease work for biotech and pharma teams across Europe.
All client resultsRare diseaseexperts
Our team combines scientific, clinical and commercial expertise. AI widens the search, and our specialists validate every finding before it reaches you.
Better rare disease decisions start with better intelligence
Whether you're building a medical strategy, exploring European markets or seeking a comprehensive understanding of a rare disease, we can help establish the evidence foundation.