Establishing Patient Registries for Rare Diseases: Rationale and Challenges-Reference-Cited by-同舟云学术

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Establishing Patient Registries for Rare Diseases: Rationale and Challenges

Published:2020-03-25 Issue:3 Volume:34 Page:185-190
ISSN:1178-2595
Container-title:Pharmaceutical Medicine
language:en
Short-container-title:Pharm Med

Author:

Boulanger Vanessa,Schlemmer Marissa,Rossov Suzanne,Seebald Allison,Gavin Pamela

Funder

U.S. Food and Drug Administration

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Pharmacology

Link

http://link.springer.com/content/pdf/10.1007/s40290-020-00332-1.pdf

Reference40 articles.

1. Slade A, Isa F, Kyte D, Pankhurst T, Kerecuk L, Lipkin G, Calvert M. Patient reported outcome measures in rare diseases: a narrative review. Orphanet J Rare Dis. 2018;13(1):61.

2. Groft SC. Rare diseases research: expanding collaborative translational research opportunities. Chest. 2013;144(1):16–23.

3. GARD. FAQS about rare diseases. 2017. https://rarediseases.info.nih.gov/diseases/pages/31/faqs-about-rarediseases. Accessed 20 Feb 2020.

4. Richter T, Nestler-Parr S, Babela R, Khan ZM, Tesoro T, Molsen E, Hughes DA, International Society for Pharmacoeconomics and Outcomes Research Rare Disease Special Interest Groups. Rare disease terminology and definitions—a systematic global review: report of the ISPOR Rare Disease Special Interest Group. Value Health. 2015;18:906–14.

5. Kaufmann P, Pariser AR, Austin C. From scientific discovery to treatments for rare diseases—the view from the National Center for Advancing Translational Sciences—Office of Rare Diseases Research. Orphanet J Rare Dis. 2018;13(1):196.

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