Tribuna

2024

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
Coordinador científico de ObserveMHe

Tribuna

2024

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

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

Analysis of the first ten years of FDA’s rare pediatric disease priority review voucher program: designations, diseases, and drug development

HTA criteria adopted in different models of public healthcare systems for orphan drugs. A scoping review

Comparative policy analysis of national rare disease funding policies in Australia, Singapore, South Korea, the United Kingdom and the United States: a scoping review

2023

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

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

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

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

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

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

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

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

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

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

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

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