How do I obtain scientific advice for regulatory agencies?
Which is the difference between biomarker qualification and validation?
How do I get authorisation of administration of a drug under compassionate use?
How do I progress from preclinical to clinical trials?
Why are orphan drugs so expensive?
What kind of information should a package leaflet include to identify the medicine as an advanced therapy medicinal product?
How do I prepare a Paediatric Investigation Plan (PIP)?
How do I obtain orphan drug designation?
The toolbox has been developed through funding from the EJP RD project.
The European Joint Programme on Rare Diseases is an initiative that has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement N°825575