European Commission Horizon Europe Framework Programme (HORIZON)

The European Commission is funding a call on the development of new effective therapies for rare diseases with the aim of “Tackling diseases diseases and reducing disease burden.” The topic will support proposals covering several different stages in the continuum of the innovation pathway ( translational, preclinical, clinical research, validation in the clinical and/or real-world setting etc.), as relevant.

To that end, proposals under this topic should aim for delivering results that are directed, tailored towards and contributing to some of the following expected outcomes:

  •  Researchers and developers make the best use of the state-of-the-art knowledge and resources for a fast and effective development of new therapies for rare diseases.
  •  Researchers and developers increase the development success rate of therapies for rare diseases by employing robust preclinical models, methods, technologies, validated biomarkers, reliable patient reported outcomes and/or innovative clinical trials designs.
  • Developers and regulators move faster towards market approval of new therapies for rare diseases (with currently no approved treatment option) due to an increased number of interventions successfully tested in late stages of clinical development.
  • Healthcare professionals and people living with a rare disease get access to new therapeutic interventions and/or orphan medicinal products.

The deadline for this call is a two-stage model, including 1 February 2022 and 06 September 2022.

The EJPRD is offering services to help applicants for this call, notably in the fields of Data FAIRification, Clinical Studies Support Office and Mentoring.