Emea Medical Plausibility

Applying for orphan designation | European …

https://www.ema.europa.eu/en/human-regulatory/research-development/orphan-designation/applying-orphan-designation

In particular, when completing section A.3.2 'Plausibility of the orphan condition; rationale for use of the medicinal product', sponsors should clearly identify studies with the substance in a relevant model(s) of the condition and, if possible, preliminary clinical data in patients with the condition. Each application is assigned two ...

Committee for Orphan Medicinal Products (COMP): …

https://www.ema.europa.eu/en/documents/work-programme/comp-work-plan-2020_en.pdf

recommendation on elements required to support the medical plausibility and the assumption of significant benefit for an orphan designation, EMEA/COMP/436/01”, taking …

Establishing medical plausibility in the context of orphan …

https://ojrd.biomedcentral.com/articles/10.1186/s13023-014-0175-8

Establishing medical plausibility in the context of orphan …

https://pubmed.ncbi.nlm.nih.gov/25475155/

This requirement is commonly referred to as "medical plausibility" and the justification of this intention is assessed on the merits of each application by the COMP, …

EMEA: COMP Recommendation on elements required to support …

https://www.sciencepharma.com/news/2000002/emea-comp-recommendation-on-elements-required,p200000024

EMEA: Committee for Orhphan Medicinal Products Recommendation on elements required to support the medical plausibility and the assumption of significant …

Research and development | European Medicines Agency

https://www.ema.europa.eu/en/human-regulatory/research-development

SME support. SME office briefing meeting. Aim: to discuss the regulatory strategy of a product and navigate the range of procedures and incentives available to support …

Presentations - Orphan designation - Key concepts …

https://www.ema.europa.eu/en/documents/presentation/presentation-orphan-designation-key-concepts-evaluation-criteria-dr-jordi-llinares_en.pdf

Medical Plausibility. CHMP: Marketing Authorisation. COMP: Orphan Designation. Evidence of positive Benefit-Risk. Evidence of ... Reproduction and/or distribution of this …

Guideline on legal status - European Medicines Agency

https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/guideline-legal-status-supply-patient-centrally-authorised-medicinal-products_en.pdf

Page 5/8 ©EMEA 2006 • Medicinal product subject to restricted medical prescription, where the medicinal product: - is reserved for treatments which can only be followed in a …

Orphan designation: Overview | European …

https://www.ema.europa.eu/en/human-regulatory/overview/orphan-designation-overview

About 30 million people living in the European Union (EU) suffer from a rare disease. The European Medicines Agency (EMA) plays a central role in facilitating the development and authorisation of medicines …

Orphans: Regulatory and procedural guidance and forms

https://www.ema.europa.eu/en/human-regulatory/research-development/orphan-designation/applying-designation/orphans-regulatory-procedural-guidance-forms

SOP/H/3046. Commission Regulation (EC) No 847/2000 of 27 April 2000 laying down the provisions for implementation of the criteria for designation of a …