The economics of moonshots: Value in rare disease drug development-Reference-Cited by-同舟云学术

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The economics of moonshots: Value in rare disease drug development

Published:2022-03-25 Issue:4 Volume:15 Page:809-812
ISSN:1752-8054
Container-title:Clinical and Translational Science
language:en
Short-container-title:Clinical Translational Sci

Author:

Yates Nathan¹,Hinkel Jennifer²

Affiliation:

1. ForwardView Consulting Clintwood Virginia USA

2. The Data Economics Company Los Angeles California USA

Publisher

Wiley

Subject

General Pharmacology, Toxicology and Pharmaceutics,General Biochemistry, Genetics and Molecular Biology,General Medicine,General Neuroscience

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/cts.13270

Reference10 articles.

1. Rare Disease Terminology and Definitions—A Systematic Global Review: Report of the ISPOR Rare Disease Special Interest Group

2. The Orphan Drug Act at 35: Observations and an Outlook for the Twenty-First Century

3. Evaluating The Impact Of The Orphan Drug Act’s Seven-Year Market Exclusivity Period

4. Orphan Cancer Drugs in the Era of Precision Medicine

5. How many rare diseases are there?

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1. Advanced Regenerative Medicines for Rare Diseases: A Review of Industry Sponsors Investment Motivations;Therapeutic Innovation & Regulatory Science;2024-09-10

2. Non-viral gene therapy for neuromuscular diseases including Duchenne muscular dystrophy using nanovesicles derived from human cells;Rare Disease and Orphan Drugs Journal;2024-09-02

3. Application of observational research methods to real-world studies for rare disease drugs: a scoping review protocol;2024-05-16

4. Strategic aspects for the commercialization of nanomedicines;Journal of Controlled Release;2024-05

5. Time for a new approach to drug development for rare systemic autoinflammatory diseases;Nature Reviews Rheumatology;2024-04-02

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