Welcome to RareDataEU

We are a small consultancy with a laser focus: helping our clients collect rare disease clinical data in Europe. 


A disease is defined as rare (or orphan) when it affects less than 1 in 2000 people. There are an estimated 36 million rare disease patients suffering with any of 6000 different rare diseases. Patients with specific diseases are hard to locate and diagnose. Good clinical data on these heterogenous diseases is even rarer. Sharing that data across Europe is very challenging yet essential to develop treatments and improve the lives of these exceptional patients.  This data sharing challenge was discussed in the Financial Times in March . 


Solving this challenge is our mission.


RareDataEU consultants  have unique Real World Experience in rare diseases obtained over the last ten years. During that time, we have worked with Pharma clients such as Vertex, Biogen, Shire and Vifor. We have advised on data programs including Cystic Fibrosis, Epidermolysis Bullosa, SMA, Haemophilia, Vasculitis, Pulmonary Fibrosis and Osteogenesis Imperfecta and have spoken at major conferences including EURORDIS, EUCOPE and the European Parliament. This website summarises the practical insights gained from this unique experience.

  • Good clinical data on rare diseases is rare.
  • Rare disease research is fragmented across Europe.
  • Rare disease health factors are not collected within hospital EHR systems.
  • Pharma, researchers and patients have different needs and restrictions making collaborating challenging.
  • Patients and clinicians are not motivated to enter registry data resulting in low patient participation.
  • Patient data has historically not been shared across borders.
  • Patient engagement cannot occur without recording patient identifiable data within the registry.
  • Hospital Ethical Review Boards (ERB) and GDPR regulations add onerous responsibilities to protect patient data.
  • Registries need to be upgradable to meet evolving healthcare needs.
  • Public funding programs concentrate on software development and do not provide sustainability funding.
  • Public funded rare disease programs such as European Reference Networks (ERN) discourage direct cooperation with Pharma.
  • Every country in Europe has different health care polices and budgets.

RareDataEU provides clients with strategic, tactical and operational advice, enabling them to share registry and Real World Data on rare diseases across Europe. Our unique approach allows orphan drug pharma, researchers, and patient organizations to collaborate in collecting clinical rare disease data.

Strategic

The Rare2030 report, released last year by key figures in the rare disease community www.rare2030.eu , had as one of  its main recommendation that there should be better European infrastructure for information sharing. Europe is a heterogenous collection of large/small and rich/poor counties with numerous conflicting health care policies. Developing an interoperable clinical data platform that operates across this space presents a unique challenge.  RareDataEU has direct registry experience in virtually every European country. 

Tactical

For many rare diseases there are only a few dozen worldwide clinical experts who record patient data on an ad hoc basis. Without planning, this quickly evolves into a series of incompatible tables commonly referred to as 'spreadsheet hell'.


Getting Key Opinion Leaders to agree on which clinical data is, or is not, important presents real challenges. RareDataEU brings real experience to this process.


Rare diseases are not well researched and finding useful clinical data is extremely challenging. Collaboration and sharing is simply vital. We help clients develop long term collaboration procedures that benefit all stakeholders. We set up agreements where pharma commit to funding a common data platform for registries and RWD studies. This provides a mechanism for proprietary data from funded studies to be shared with the registry. 

Operational

A registry should be designed to operate for 20-30 years. It should never be a static standalone “black box” solution that can only understood by an IT professional. It will need to be easily upgraded by clinicians as it expands and becomes increasingly valuable. 


A registry is only useful if it contains data on a large percentage of patients. We design “Smart Registries” that are useful for clinicians and patents and not just researchers.  This ensures a high level of enrolment and participation. 


The registry must allow sharing of common outcome data with international registries while meeting the unique needs of national researchers. Pulmonary Fibrosis has over sixty separate international registries; an obvious obstacle for clinical research. In contrast Cystic Fibrosis has a  common European dataset allowing patient outcomes to be compared across countries. This has led to life saving treatments and care for CF patients.
Continue to RWD & Registries
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