This is the default registry for countries without a sustainable well-established registry. These would typically be smaller countries (for example within Scandinavia or Benelux). The registry is based on:

• Static data collected when the patient enrols in the registry.

• Encounter data which measures the patient’s treatment and outcomes over time. 

Encounters can be either:

• A single annual assessment. 

• Multiple date-based assessments recorded at each clinical encounter. 

A small number of fields (the Minimum Dataset) are defined by the Registry Scientific Working Group as mandatory. A record cannot be completed until data is entered for all mandatory fields. 

It is important to keep in mind that registries are completed by “busy doctors under pressure”. Care must be taken to reduce the mandatory data fields in the annual assessment to a minimum.  

The Model Registry should never be designed as an electronic version of a paper form. Many paper-based annual assessment reports are completed in the last days of the year with no opportunity to check missing and inconsistent data. 

In contrast to a passive paper-based data collection, an online registry is active and can assist doctors during a patient visit. Data validations and prior entry prompts (for example prior medication and dosage) will reduce frequency of data errors.  

An online registry will support a much more rigorously defined protocol where the clinician could be alerted if for example there was an unusual change in blood pressure from a prior encounter. 

The Model registry can support separate patient pathways to reflect different patient needs. A seriously ill patient needing multiple hospital encounters over a year will generate a lot more relevant data that a healthy patient – however data from both cohorts will need to be fully interoperable. Specifically, a process has to be defined to summarise multiple encounters into one annual summary.

Independent registries can join the Federated Registry by agreeing to support the Minimum Dataset. Independent registries are typically from larger countries such as the UK, France or Germany.  Linking future sponsorship will provide a major incentive for existing registries to supporting the minimum dataset.  

The Federated Registry will integrate annual assessment data from existing sustainable registries and Model Registries and publish annual reports comparing the outcome of patients in different countries.  These reports can be invaluable in providing evidence base data for increasing patient care to public health authorities. 

Benchmarking provides an incentive for larger countries with existing sustainable registries to share patient data.

A cohort of patients can be selected for a real-world study that could be monitored by an independent CRO. 

This architecture was first developed while partnering with Vertex Pharma a few years ago. Vertex planned to run a Phase 4 trail for 4,000 Cystic Fibrosis patients in Europe (10% of the patient population). 

The patient organization made access to these patient’s conditional on the data from the study being released and being compatible with the existing registry. The solution was to extend the registry dataset and make the additional data collected private until after it was  published by Vertex. 

We could clearly see that this cooperative solution could be offered as a general framework for collaboration between pharma and researchers.    

Rare patients are hard to find. Rare Registries identify more target patients for treatment and provide more granular safety and efficacy data to national funding agencies. This can assist in approving individual or small patient cohorts for reimbursement. 

Orphan drugs typically cost well over $100K per patient-year. Having a European wide dataset with interoperable diagnostic, treatment and outcome data will substantially increase pharma revenue and provide more lifesaving drugs to critically ill patients.

Assume there is a patient with a rare comorbidity in a small country like Norway. The submission for reimbursement approval could include retrospective safety and efficacy data from patients with that comorbidity in other European countries. There could also be a commitment to provide prospective interoperable data to justify continual reimbursement. 

• NICE, the UK agency responsible for drug reimbursement, required that PTC’s Duchenne muscular dystrophy treatment Translarna at an average cost of $319K per patient-year be made available under a “managed access” scheme. This requires that patients achieve a five-year efficacy measure.  
• G-BA now requires all orphan drug reimbursements to be conditional on annual access to safety and efficacy RWE from registry data. 

Pharma can run multiple RWD safety and efficacy studies on top of the Model Registry. Scaling as required between low-cost unverifiable registry data to higher cost FDA approved RWD studies. 

The Data Collection Roadmap outlines which 3rd party research funding proposals pharma will support.  Linking future sponsorship will provide a major incentive for existing registries to supporting the minimum dataset.  

The Federated Registry will provide a common study platform to meet the EMA and national HTA requirements such as German G-BA requirements for a registry to support orphan drug reimbursement.

Patient organizations receive a sustainable, advanced registry for a minimal cost

Patient organizations own the registry and control access to it. Pharma cannot influence the scientific direction of the Model or Federated registry

Registries can be extended to support national data collection & separate clinical research

Benchmarking: The Federated registry can publish vital annual patient outcome reports.  This allows funding and standards of care to be compared across countries. Click on button below to review example of annual reports published by the European Cystic Fibrosis registry