EJP RD – Internal call for innovation project in CT methodology

Please note that this information is not binding and may be submitted to changes. Any modifications will be clearly indicated in the text. 

The European Joint Programme on Rare Diseases (EJP RD) will implement the present internal (open to EJP RD beneficiaries and their linked third parties) call for collaborative innovation projects to improve RD clinical trials methodologies in limited populations. 


Three collaborative demonstration projects aiming to validate innovative statistical design and analysis methods developed by Asterix, IDeAl and InSPiRe in small population groups have been already supported. However, EJP RD recognizes that these existing methods leave space for improvement and innovation in particular for limited populations as urgent needs remain 

In the context of this call, limited populations are considered as a limited patient population EU wide (number below the definition of populations in ultra-rare diseases of 1 affected in 50.000), either because the disease prevalence is low or because the population is restricted to a specific disease sub-group representing only a limited number of patients.  

The focus is to target limited populations where traditional methodologies to conduct clinical trials often fail.  


The innovation methodologies topics particularly include (but are not limited to): 

  • Development of a disease progression model from a natural history cohort or other observational studies.
  • Development and validation of a disease specific clinically meaningful outcome with special interest in PCOMs, or composite endpoints.
  • Development of a design and analysis procedure for a pharmacometric model and/or bridging study.
  • Development of a randomization-based model as an alternative analysis strategy and explore the level of evidence.

The call intends to privilege support of collaborations among groups of experts consisting of different stakeholders including methodological experts, clinicians, patients and industry (when relevant) who will jointly develop innovative methods to fill gaps of RD clinical trial methodologies. The aim is, to have ready to use methods, which will drive regulatory decision-making. 

Applicants for this call should belong to one of the (beneficiary) institutions of the EJP RD or their linked third-party institutionTo pre-identify if your institution is eligible, at this stage, please refer to the definitions provided in the internal call for demonstration projectsMore information will be provided after official opening of this call.  


The call is expected to be launched in the beginning of December 2020.

A networking meeting will be held on January 11th  2021 to consolidate the collaborative networks.

The foreseen call closing deadline is the beginning of March 2021. 

All proposals will be reviewed by a scientific evaluation committee (SEC) composed of independent scientific external reviewers.


For any further information please contact the call secretariat at: innovation.callsec@ejprarediseases.org.