Natural History and Real‐World Data in Rare Diseases: Applications, Limitations, and Future Perspectives-Reference-Cited by-同舟云学术

Natural History and Real‐World Data in Rare Diseases: Applications, Limitations, and Future Perspectives

Published:2022-12 Issue:S2 Volume:62 Page:
ISSN:0091-2700
Container-title:The Journal of Clinical Pharmacology
language:en
Short-container-title:The Journal of Clinical Pharma

Author:

Liu Jing¹,Barrett Jeffrey S.²,Leonardi Efthimia T.³,Lee Lucy³,Roychoudhury Satrajit¹,Chen Yong¹,Trifillis Panayiota³

Affiliation:

1. Pfizer, Inc. Groton Connecticut USA

2. Critical Path Institute Rare Disease Cures Accelerator Data Analytics Platform Tucson Arizona USA

3. PTC Therapeutics, Inc. South Plainfield New Jersey USA

Publisher

Wiley

Subject

Pharmacology (medical),Pharmacology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/jcph.2134

Reference116 articles.

1. US Food and Drug Administration.Rare Diseases: Natural History Studies for Drug Development: Draft Guidance for Industry.2019. Accessed March 4 2022.https://www.fda.gov/media/122425/download

2. European Medicines Agency.Procedural advice for orphan medicinal product designation: Guidance for sponsors.2021. Accessed March 4 2022.https://www.ema.europa.eu/en/documents/regulatory‐procedural‐guideline/procedural‐advice‐orphan‐medicinal‐product‐designation‐guidance‐sponsors_en.pdf

3. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database

4. US Food and Drug Administration.Importance of natural history studies in rare diseases.2019. Accessed March 4 2022.https://events‐support.com/Documents/Pariser.pdf

5. Drug Discovery and Development in Rare Diseases: Taking a Closer Look at the Tafamidis Story

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