POSB211 Is an Early Access Program for an Oncology Drug an Asset in the French Pricing and Reimbursement Pathway?

Cogez, F; Gervais, Martin; Cardon, S.C.; Kachaner, I.; Bougeois, M

doi:10.1016/j.jval.2021.11.705

Cited by 1 publication

(1 citation statement)

References 0 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…Esistono poi alcune evidenze di impatto di schemi di accesso precoce finanziati a livello pubblico ( 5 ). Tali evidenze riguardano in particolare la Francia, paese che ha una lunga esperienza su tali schemi di accesso, e mostrano una maggiore probabilità di rimborso per i farmaci già inseriti in schemi di accesso precoce ( 6 , 7 ), una valutazione della dimensione temporale dell’accesso precoce ( 8 , 6 ) e gli effetti dell’inserimento in tali programmi sui tempi negoziali di Prezzo e Rimborso (P&R) ( 7 ).…”

Section: Introduzioneunclassified

The economic impact of 14 compassionate use programs for medicines in Italy, in the perspective of the National Health Service

Jommi,

Pantellini,

Giuliani

et al. 2024

Grhta

View full text Add to dashboard Cite

Background: Compassionate use programs (CUP) for medicines allow patients, not recruited in trials, accessing medicines before marketing approval. The evidence on the economic impact of CUP is limited to one paper that did not include the possible long-term economic impact of medicines used in CUP compared to alternative treatments, like avoided hospitalization costs. This paper aims at covering this information gap, updating and integrating the existing evidence, with a cost analysis in the perspective of the Italian National Health Service (SSN). Methods: Data were retrieved from the Roche CUP Database, the peer-reviewed literature (e.g. published cost-effectiveness studies, if any), the gray literature (e.g. regional documents for the alternative treatment cost) and unpublished data (e.g. cost-effectiveness analysis provided for price and reimbursement negotiation for medicines in CUP). Results: We retrieved data from 14 CUPs and 3,485 patients (70% affected by tumors) (Jan 2015-August 2022). The alternative treatment mean cost per patient ranged from € 11.1 k to € 18.8 k. The net economic benefit ranged from € 47 million to € 75 million (€ 13.5 k-€ 21.5 k per patient). Avoiding alternative treatments mostly contributes to the economic benefit. The long-term economic impact accounted for 20/30% of the net economic impact. The net economic benefit for cancer drugs ranged from € 12.6 million to € 40.0 million. Conclusions: Despite research limitations, this paper provides additional evidence on the economic impact of CUP in Italy. This could represent the first step of a broader analysis of the economic impact of early access schemes for drugs.

show abstract