In Italy, the Balduzzi Law (Law 189/2012, Article 12, paragraph 3) allows pharmaceutical companies to submit P&R Dossiers for orphan drugs just after the publication of a positive CHMP opinion, without the need to wait for the EC decision; clearly, the aim of such provision is the acceleration of time to reimbursement for orphan drugs – https://www.aifa.gov.it/en/farmaci-orfani .
According to an analysis we conducted on our database of Italian P&R procedures, 108 new orphan drugs have received NHS reimbursement (Class H or Class A/PHT) for the first time since 2019; in only 37 cases (34,3%) the P&R Dossier was submitted prior to the date of EC approval (data cut-off for the analysis: Oct 8, 2026).
It is therefore evident that only a minority of pharmaceutical companies take advantage of this opportunity offered by the Italian legislator to accelerate orphan drugs market access, although no clear rationale is immediately identifiable for this behavior.
We would welcome comments on this finding and the opportunity to discuss the above topic; in case you were interested, please contact us at the following email address: valuebox@valuevector.com .
