EU Regulation (EC) No 141/2000 - Orphan Medicinal Products

What it says

This Regulation establishes a procedure for designating medicines as orphan medicinal products for rare conditions and sets incentives for their development. Designation is granted on criteria that include the seriousness and rarity of the condition and the lack of satisfactory existing treatment or significant benefit. Authorized orphan products benefit from a period of market exclusivity.

Paraphrased for plain-language clarity. Always verify against the current published regulation text.

What it means in practice

• Designation is requested before the marketing authorization application.
• Sponsors must keep the criteria in mind, since designation can be reviewed at the time of authorization.
• Incentives include protocol assistance and market exclusivity.

What inspectors look for

• Whether the criteria for orphan status continue to be met.
• Whether commitments made at designation are followed.
• Whether data submitted are consistent.

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