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Results from Expanded Access Programs: A Review of Academic Literature
Tobias B. Polak
,
David G J Cucchi
,
Jasmin Schelhaas
,
Syed S Ahmed
,
Naima Khoshnaw
,
Joost van Rosmalen
,
Carin A Uyl-de Groot
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Results from Expanded Access Programs: A Review of Academic Literature
BACKGROUND: Although expanded access is an increasingly used pathway for patients to access investigational medicine, little is known …
Tobias B. Polak
,
David G. J. Cucchi
,
Jasmin Schelhaas
,
Syed S. Ahmed
,
Naima Khoshnaw
,
Joost van Rosmalen
,
Carin A. Uyl – de Groot
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DOI
The Ethics of Expanded Access Research
This Viewpoint discusses the benefits of expanded access research, the usefulness of expanded access data, the issues surrounding cost …
Tobias Boy Polak
,
Holly Fernandez Lynch
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Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations
Patients with rare diseases often have limited or no options for approved treatments or participation in clinical trials. In such …
Tobias B. Polak
,
David G. J. Cucchi
,
Joost van Rosmalen
,
Carin A. Uyl – de Groot
,
Jonathan J. Darrow
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DOI
The DRUG Access Protocol: Access Inequality and European Harmonisation
Tobias B Polak
,
David G J Cucchi
,
Joost van Rosmalen
,
Carin A Uyl-de Groot
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Incremental Benefits of Novel Pharmaceuticals in the UK: A Cross-Sectional Analysis of NICE Technology Appraisals from 2010 to 2020
Tobias B Polak
,
David GJ Cucchi
,
Jonathan J Darrow
,
Matthijs M Versteegh
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Real-World Data from Expanded Access Programmes in Health Technology Assessments: A Review of NICE Technology Appraisals
Tobias B Polak
,
David GJ Cucchi
,
Joost van Rosmalen
,
Carin A Uyl-de Groot
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The Predictive Value of a Positive Phase II ASH Abstract for Peer-Reviewed Publication and Progression to Phase III
David GJ J. Cucchi
,
Tobias B. Polak
,
Gert J. Ossenkoppele
,
Jacob M. Rowe
,
Elihu H. Estey
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DOI
The Wider Perspective: Twenty Years of Clinical Trials in Myelodysplastic Syndromes
Carolien Duetz
,
David G. J. Cucchi
,
Tobias B. Polak
,
Jeroen J. W. M. Janssen
,
Gert J. Ossenkoppele
,
Elihu H. Estey
,
Arjan A. Loosdrecht
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Financial Considerations in Expanded Access Policy for Gene Therapies: A Tough Nut to Crack?
Tobias B. Polak
,
Eline M. Bunnik
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