Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints-Reference-Cited by-同舟云学术

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Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints

Published:2015 Issue:1 Volume:10 Page:16
ISSN:1750-1172
Container-title:Orphanet Journal of Rare Diseases
language:en
Short-container-title:Orphanet Journal of Rare Diseases

Author:

Kakkis Emil D,O’Donovan Mary,Cox Gerald,Hayes Mark,Goodsaid Federico,Tandon PK,Furlong Pat,Boynton Susan,Bozic Mladen,Orfali May,Thornton Mark

Publisher

Springer Science and Business Media LLC

Subject

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

Link

http://link.springer.com/content/pdf/10.1186/s13023-014-0195-4.pdf

Reference5 articles.

1. Building A Stronger Foundation For A New Century Of Treatments And Cures

2. The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases

3. Surrogate End Points in Clinical Trials: Are We Being Misled?

4. Expedited programs for drug development and approval

5. Surrogate Endpoints And FDA’s Accelerated Approval Process

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3. Trends in orphan medicinal products approvals in the European Union between 2010–2022;Orphanet Journal of Rare Diseases;2024-02-27

4. Navigating the unknown: how to best ‘reflect’ standard of care in indications without a dedicated treatment pathway in health technology assessment submissions;Journal of Comparative Effectiveness Research;2024-02

5. The advantages of rare disease biobanking: A localised source of genetic knowledge to benefit the South African rare disease community and related stakeholders worldwide;South African Medical Journal;2023-12-04

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