Tribuna

Real-world evidence for coverage determination of treatments for rare diseases

Dayer VW, Drummond MF, Dabbous O, Toumi M, Neumann P, Tunis S, Teich N, Saleh S, Persson U, von der Schulenburg JG, Malone DC, Salimullah T, Sullivan SD
[Original]

Dra. Mariló Edo Solsona
Farmacéutica especialista, Servicio de Farmacia, Hospital La Fe de Valencia.
Dr. José Luis Poveda
Director Gerente, Hospital La Fe de Valencia.

Partial Orphan Cancer Drugs: US Food and Drug Administration Approval, Clinical Benefit, Trials, Epidemiology, Price, Beneficiaries, and Spending

Michaeli T, Michaeli DT.
[Original]

Dra. Mariló Edo Solsona
Farmacéutica especialista, Servicio de Farmacia, Hospital La Fe de Valencia.
Dr. José Luis Poveda
Director Gerente, Hospital La Fe de Valencia.

ODs with a positive TPR conclusion,
not subject to a conditional approval, and approved without requiring a PASS would be more likely to be reimbursed in Spain

Poveda, J.L., Gómez, C., Gil, A. et al.
[Original]

Dra. Mariló Edo Solsona
Farmacéutica especialista, Servicio de Farmacia, Hospital La Fe de Valencia.
Dr. José Luis Poveda
Jefe de Servicio de Farmacia, Hospital La Fe de Valencia.

Strategic discussion on funding and access to therapies targeting rare diseases in Spain: an expert consensus paper. Orphanet J Rare Dis 18, 41 (2023)

Zozaya N, Villaseca J, Abdalla F. et al.
[Original]

Dra. Mariló Edo Solsona
Farmacéutica especialista, Servicio de Farmacia, Hospital La Fe de Valencia.
Dr. José Luis Poveda
Jefe de Servicio de Farmacia, Hospital La Fe de Valencia.

An international comparative analysis of public reimbursement of orphan drugs in Canadian provinces compared to European countries. Orphanet J Rare Dis. 2022 Mar 4;17(1):113

Ward LM, Chambers A, Mechichi E, Wong- Rieger D, Campbell C.
[Original]

Dra. Mariló Edo Solsona
Farmacéutica especialista, Servicio de Farmacia, Hospital La Fe de Valencia.
Dr. José Luis Poveda
Jefe de Servicio de Farmacia, Hospital La Fe de Valencia.

The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis

Costa E, Girotti S, Pauro F, Leufkens HGM, Cipolli M.
[Original]

Dra. Mariló Edo Solsona
Farmacéutica especialista, Servicio de Farmacia, Hospital La Fe de Valencia.
Dr. José Luis Poveda
Jefe de Servicio de Farmacia, Hospital La Fe de Valencia.

The effect of adding real-world evidence to regulatory submissions on the breadth of population indicated for rare disease medicine treatment by the European Medicines Agency

Jandhyala R.
[Original]

Dra. Mariló Edo Solsona
Farmacéutica especialista, Servicio de Farmacia, Hospital La Fe de Valencia.
Dr. José Luis Poveda
Jefe de Servicio de Farmacia, Hospital La Fe de Valencia.

FDA approval, clinical trial evidence, efficacy, epidemiology, and price for non-orphan and ultra-rare, rare, and common orphan cancer drug indications: cross sectional analysis

Michaeli T, Jürges H, Michaeli DT.
[Original]

Dra. Mariló Edo Solsona
Farmacéutica especialista, Servicio de Farmacia, Hospital La Fe de Valencia.
Dr. José Luis Poveda
Jefe de Servicio de Farmacia, Hospital La Fe de Valencia.

How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes?

Blonda A, Denier Y, Huys I, Kawalec P, Simoens S. Front
[Original]

Dra. Mariló Edo Solsona
Farmacéutica especialista, Servicio de Farmacia, Hospital La Fe de Valencia.
Dr. José Luis Poveda
Jefe de Servicio de Farmacia, Hospital La Fe de Valencia.

A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act

Fermaglich LJ, Miller KL.
[Original]

Dra. Mariló Edo Solsona
Farmacéutica especialista, Servicio de Farmacia, Hospital La Fe de Valencia.
Dr. José Luis Poveda
Jefe de Servicio de Farmacia, Hospital La Fe de Valencia.

Orphan drugs’ clinical uncertainty and prices: Addressing allocative and technical inefficiencies in orphan drug reimbursement

Eichler HG, Kossmeier M, Zeitlinger M, Schwarzer-Daum B
[Original]

Dra. Mariló Edo Solsona
Farmacéutica especialista, Servicio de Farmacia, Hospital La Fe de Valencia.
Dr. José Luis Poveda
Jefe de Servicio de Farmacia, Hospital La Fe de Valencia.

Prices of Orphan Drugs in Four Western European Countries Before and After Market Exclusivity Expiry: A Cross-Country Comparison of List Prices and Purchase Prices

Dane A, Klein Gebbink AS, Brugma JD, Degrassat-Théas A, Hug MJ, Houlind MB, Paubel P, van der Kuy PHM, Uyl-de Groot CA
[Original]

Dra. Mariló Edo Solsona
Farmacéutica especialista, Servicio de Farmacia, Hospital La Fe de Valencia.
Dr. José Luis Poveda
Jefe de Servicio de Farmacia, Hospital La Fe de Valencia.

The effect of adding real-world evidence to regulatory submissions on the breadth of population indicated for rare disease medicine treatment by the European Medicines Agency

Jandhyala R.
[Original]

Dra. Mariló Edo Solsona
Farmacéutica especialista, Servicio de Farmacia, Hospital La Fe de Valencia.
Dr. José Luis Poveda
Jefe de Servicio de Farmacia, Hospital La Fe de Valencia.