Clinical Benefit and Revenues of Drugs Affected by Rare Pediatric Disease Priority Review Vouchers, 2017-2023-Reference-Cited by-同舟云学术

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Clinical Benefit and Revenues of Drugs Affected by Rare Pediatric Disease Priority Review Vouchers, 2017-2023

Published:2024-12 Issue: Volume:275 Page:114211
ISSN:0022-3476
Container-title:The Journal of Pediatrics
language:en
Short-container-title:The Journal of Pediatrics

Author:

Liu Ian T.T.^ORCID,Kesselheim Aaron S.

Funder

Arnold Ventures LLC

Publisher

Elsevier BV

Reference33 articles.

1. From scientific discovery to treatments for rare diseases–the view from the National Center for Advancing Translational Sciences–Office of rare diseases research;Kaufmann;Orphanet J Rare Dis,2018

2. The IDeaS initiative: pilot study to assess the impact of rare diseases on patients and healthcare systems;Tisdale;Orphanet J Rare Dis,2021

3. Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer;Kesselheim;JAMA,2011

4. Estimating the clinical cost of drug development for orphan versus non-orphan drugs;Jayasundara;Orphanet J Rare Dis,2019

5. Patient access to orphan drugs;Cohen;Expert Opin Orphan Drugs,2017

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