ERN: A great vision meets an inevitable bureaucracy

 ERNs were founded on the principle that many rare disease issues are pan-European, and any single Member State cannot solve them alone.  ERNs represent a unique Rare Disease innovation by the European Community to standardize and improve the treatment and research of patients with rare diseases across Europe. 


In 2016 George Reynolds, our Managing Partner managed the tender process for the ICT platform for the 24 European Reference Networks. This resulted in two of his clients jointly winning the €5M contract to deliver the platform to the European Community. This game changing system was deployed in 300 hospitals throughout Europe. 


Unfortunately, and perhaps inevitably since 2016, ERNs have become much more prescriptive and burocratic. Their day-to-day functioning are severely hampered by many challenges, including, lack of legal status as well as insufficient and unsustainable funding.  


The paper “European Reference Networks: challenges and opportunities” provides  further background.  This was published by several ERN Coordinators in 2021. The paper expresses their frustration with the slow pace of ERN progress.  Click button below to reveiw
ERN Challenges & Opportunities

ERNs severely limit Industry collaboration


In the context of registries, RWD and industry collaboration, there are several other ERN challenges:


In 2019 the policy arm of the ERNs (the ERN Board of Member States) published a policy guideline “Statement of on European Reference Networks (ERNs) & industry” These policies make direct pharma collaboration impractical:


  • “There should be no funding from industry directly allocated for management and running of the Network nor for …establishing and maintaining patient registries.”


  • “ERNs should preferably seek public funding but, once exhausted, they could also look for solutions enabling shared funding from more than one industry partner”


  • “ERNs are expected to gather patients’ data to facilitate research…. Conditions for access to this data have to be …. transparent and not provide preferential treatment to any researcher or any other actor “


There are additional challenges to Industry cooperating with ERNs:


  • Incorporation: ERNs are not incorporated. They operate as a voluntary association of members. Pharma’s are highly regulated entities; they can only sponsor a legal entity willing to commit to an enforceable legal agreement.


  • GDPR: As they are not legal entities, ERNs cannot be Data Controllers: a key GDPR requirement. The original hospital where the patient was treated remains the Data Controller, the legal body responsible for protecting the data subjects (patient) rights. To consent patients for a study, Industry must therefore reach agreement with typically 30 different hospital Data Processing Officers each with different polices. Clearly an impractical solution.



  • Focus: ERNs are responsible for hundreds of rare diseases within their clinical classification e.g., Lung, Skin. Pharma has a laser focus on only one disease.


The only ERN approved collaboration process is for industry to contribute to a pool of funds to be allocated independently to different ERNs. This doesn’t address pharma’s requirement to provide real world regulatory and safety data.

ERN Registry Data Element standardization and sharing


Registries are regarded as key instruments for rare diseases by the EC. There have been several initiatives to help in their development:


  • The European Platform on Rare Disease Registration, has been established to make registries' data searchable at EU level and to standardise data collection and data exchange.
  • Through the Joint research Centre (JRC), 16 basic static data elements, such as ORPHAcode for disease diagnosis, have been defined as essential for each registry. Registry owners can registry and upload this anonymized dataset to this platform. These data elements are fully supported by the Model Registry and can be uploaded where required. 


It is our view that sharing data across diseases may be useful for public health studies but is of limited use for researchers. However, sharing detailed outcome data on a specific disease across countries is vital for researchers.


For example, a Cystic Fibrosis researcher in Italy has little interest in a Haemophilia patient in Italy. However, she would be very interested in comparing the outcome of a Cystic Fibrosis patient in Germany.

Limits to ERN Registries

ERNs has received a limited budget from the EC to develop their own registry. However, each ERN is responsible for hundreds of rare diseases whereas a pharma has a laser focus on a specific disease.

  •  We understand that these ERN registries will be limited to collecting static population data with a small amount of customization for more common diseases.
  • As a static registry they will not provide outcome measures and cannot be used for safety and efficacy monitoring. 
  • There will be limited customization by rare disease or by country.
  • There will be no industry participation.
  • There will be no long-term funding for sustainability.

ERN collaboration policies


With the above issues in mind, our recommendation is that clients should cooperate with their applicable ERN by supporting their core dataset and uploading new patient data as it occurs. However, we cannot envisage an ERN registry offering anything like the features required in a pharma sponsored Federated Registry as outlined in our Data Collection Roadmap. 

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