Literature DB >> 30940936

What compassionate use means for gene therapies.

Carolyn Riley Chapman1, Kenneth I Moch2, Andrew McFadyen3, Lisa Kearns4, Tom Watson5, Pat Furlong6, Alison Bateman-House4.   

Abstract

Mesh：

Year:  2019        PMID： 30940936     DOI： 10.1038/s41587-019-0081-7

Source DB:  PubMed          Journal:  Nat Biotechnol        ISSN： 1087-0156            Impact factor:   54.908


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  6 in total

1.  Regulating clinical trials in a resource-limited setting during the Ebola public health emergency in Sierra Leone.

Authors:  Onome T Abiri; Abdulai J Bah; Michael Lahai; Durodami R Lisk; James P Komeh; Joy Johnson; Wiltshire C N Johnson; Sheku S Mansaray; Joseph Sam Kanu; James B W Russell; Fawzi Thomas; Murtada M Sesay; Thomas A Conteh; Alphan Tejan-Kella; Mohamed Sesay; Manal Ghazzawi; Brian Thompson; Sorie Conteh; Gibrilla Fadlu Deen
Journal:  Trials       Date:  2022-06-06       Impact factor: 2.728

2.  Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations.

Authors:  Tobias B Polak; David G J Cucchi; Joost van Rosmalen; Carin A Uyl-de Groot; Jonathan J Darrow
Journal:  Front Pharmacol       Date:  2022-05-23       Impact factor: 5.988

3.  Gene therapy companies have an ethical obligation to develop expanded access policies.

Authors:  Lisa Kearns; Carolyn Riley Chapman; Kenneth I Moch; Arthur L Caplan; Tom Watson; Andrew McFadyen; Pat Furlong; Alison Bateman-House
Journal:  Mol Ther       Date:  2021-03-13       Impact factor: 11.454

4.  Financial considerations in expanded access policy for gene therapies: A tough nut to crack?

Authors:  Tobias B Polak; Eline M Bunnik
Journal:  Mol Ther       Date:  2021-05-08       Impact factor: 12.910

Review 5.  Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals.

Authors:  Tobias B Polak; David GJ Cucchi; Joost van Rosmalen; Carin A Uyl-de Groot
Journal:  BMJ Open       Date:  2022-01-06       Impact factor: 2.692

6.  Expanded Access as a source of real-world data: An overview of FDA and EMA approvals.

Authors:  Tobias B Polak; Joost van Rosmalen; Carin A Uyl-de Groot
Journal:  Br J Clin Pharmacol       Date:  2020-04-07       Impact factor: 4.335
  6 in total

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