Module 6

04 October 2024

Early support – PRIME designation (1)

Goal and scope of the PRIME scheme

Context Many patients with serious diseases have no or unsatisfactory therapeutic options. Aim To foster the development of Medicinal products of major public health interest and in particular from the viewpoint of therapeutic innovation.

Benefits for medicine developers

Benefits for Patients

Encourages developers to focus resources on medicines likely to make a difference to patients’ lives

Driven by patients’ needs

Fosters early dialogue with EMA to facilitate robust data collection and high quality MA

Focuses on medicines that address an unmet medical need

Bring promising treatments to patients earlier

Speeds up evaluation – earlier access for patients

How? Applications => Designation Building on existing framework: eligibility according to existing Accelerated Assessment criteria

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An Agency perspective: the success and failure of a medicinal product in the EU

Classified as public by the European Medicines Agency

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Early support – PRIME designation (2)

Entry points PRIME eligibility and required evidence

Nonclinical

Phase I

Exploratory

Confirmatory

Confirmation

Any sponsor

SMEs Academia

Proof of principle (For SMEs and academia only) ▪ Sound pharmacological rationale, convincing scientific concept ▪ Relevant nonclinical effects of sufficiently large magnitude and duration ▪ Tolerability in first in man trials

Proof of concept ▪ Sound pharmacological rationale ▪ Clinical response efficacy and safety data in patients (exploratory trials) ▪ Substantial improvement ▪ Magnitude, duration, relevance of outcomes to be judged on a case by case basis

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An Agency perspective: the success and failure of a medicinal product in the EU

Classified as public by the European Medicines Agency

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