Literature DB >> 33964226

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

Tobias B Polak1, Eline M Bunnik2.   

Abstract

Entities:  

Mesh：

Year:  2021        PMID： 33964226      PMCID： PMC8178466          DOI： 10.1016/j.ymthe.2021.04.030

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   12.910


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  5 in total

1.  What compassionate use means for gene therapies.

Authors:  Carolyn Riley Chapman; Kenneth I Moch; Andrew McFadyen; Lisa Kearns; Tom Watson; Pat Furlong; Alison Bateman-House
Journal:  Nat Biotechnol       Date:  2019-04       Impact factor: 54.908

2.  Reimbursing the value of gene therapy care in an era of uncertainty.

Authors:  Declan Noone; Donna Coffin; Glenn F Pierce
Journal:  Haemophilia       Date:  2020-11-27       Impact factor: 4.287

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.  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

5.  Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countries.

Authors:  Jesper Jørgensen; Eve Hanna; Panos Kefalas
Journal:  J Mark Access Health Policy       Date:  2020-01-15
  5 in total
  1 in total

Review 1.  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
  1 in total

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