Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders-Reference-Cited by-同舟云学术

Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders

Published:2019-02-08 Issue:1 Volume:14 Page:
ISSN:1750-1172
Container-title:Orphanet Journal of Rare Diseases
language:en
Short-container-title:Orphanet J Rare Dis

Author:

Mulberg Andrew E.,Bucci-Rechtweg Christina,Giuliano Joseph,Jacoby David,Johnson Franklin K.,Liu Qing,Marsden Deborah,McGoohan Scott,Nelson Robert,Patel Nita,Romero Klaus,Sinha Vikram,Sitaraman Sheela,Spaltro John,Kessler Vivian

Funder

Amicus Therapeutics, Inc

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Genetics (clinical),General Medicine

Link

http://link.springer.com/content/pdf/10.1186/s13023-019-1017-5.pdf

Reference39 articles.

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2. Cremers S, Aronson JK. Drugs for rare disorders. Br J Clin Pharmacol. 2017;83:1607–13.

3. Cox GF. The art and science of choosing efficacy endpoints for rare disease clinical trials. Am J Med Genet A. 2018;176:759–72.

4. Clarke JT, Coyle D, Evans G, Martin J, Winquist E. Toward a functional definition of a "rare disease" for regulatory authorities and funding agencies. Value Health. 2014;17:757–61.

5. Gobburu J, Pastoor D. Drugs against rare diseases: are the regulatory standards higher? Clin Pharmacol Ther. 2016;100:322–3.

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