Bibliography 273 A ♦ Part II – The usage of expanded access data 1. Polak TB, van Rosmalen J, Uyl-de Groot CA. Expanded Access as a source of real-world data: An overview of FDA and EMA approvals. Br J Clin Pharmacol. 2020 Sep;86(9):1819-1826. 2. Polak TB, Cucchi DGJ, van Rosmalen J, Uyl-de Groot CA. Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals. BMJ Open. 2022 Jan 6;12(1):e052186. 3. Polak TB, Cucchi DGJ, Schelhaas J, Ahmed SS, Khoshnaw N, van Rosmalen J, Uyl-de Groot CA. Results from Expanded Access Programs: A Review of Academic Literature. Drugs. 2023 Jun;83(9):795-805. 4. Polak TB, van Rosmalen J, Uyl-De Groot CA. Response to Open Peer Commentaries on “Making It Count: Extracting Real World Data from Compassionate Use and Expanded Access Programs” Am J Bioeth. 2020 Nov;20(11):W4-W5. ♥ Part III – Statistical inclusion of expanded access data 1. Polak TB, Labrecque J, Uyl-De Groot CA, van Rosmalen J. Augmenting treatment arms with external data through propensity-score weighted power-priors with an application in expanded access. Statistics in Medicine: Under revision. Preprint: arXiv:2306.01557 ♠ Part IV – Policy implications and ethical considerations 1. Polak TB, Cucchi DGJ, van Rosmalen J, Uyl-de Groot CA. The DRUG Access Protocol: access inequality and European harmonisation. Lancet Oncol. 2022 May;23(5):e202. 2. Polak TB, Cucchi DGJ, van Rosmalen J, Uyl-de Groot CA, Darrow JJ. Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations Front Pharmacol. 2022 May 23;13:913567. 3. Polak TB, Fernandez Lynch H. The Ethics of Expanded Access Research. JAMA. 2023 Apr 4;329(13):1057-1058.
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