Considerations for clinical trial design and conduct in the evaluation of novel advanced therapeutics in neurodegenerative disease

Drew, Cheney J.G. ORCID: https://orcid.org/0000-0002-4397-6252 and Busse, Monica ORCID: https://orcid.org/0000-0002-5331-5909 2022. Considerations for clinical trial design and conduct in the evaluation of novel advanced therapeutics in neurodegenerative disease. Lane, Emma, Cheney, Drew and Lelos, Mariah, eds. Current Challenges in Cell Therapy for Neurodegenerative Diseases, International Review of Neurobiology, Elsevier, pp. 235-279. (10.1016/bs.irn.2022.09.006)

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Abstract

The recent advances in the development of potentially disease modifying cell and gene therapies for neurodegenerative disease has resulted in the production of a number of promising novel therapies which are now moving forward to clinical evaluation. The robust evaluation of these therapies pose a significant number of challenges when compared to more traditional evaluations of pharmacotherapy, which is the current mainstay of neurodegenerative disease symptom management. Indeed, there is an inherent complexity in the design and conduct of these trials at multiple levels. Here we discuss specific aspects requiring consideration in the context of investigating novel cell and gene therapies for neurodegenerative disease. This extends to overarching trial designs that could be employed and the factors that underpin design choices such outcome assessments, participant selection and methods for delivery of cell and gene therapies. We explore methods of data collection that may improve efficiency in trials of cell and gene therapy to maximize data sharing and collaboration. Lastly, we explore some of the additional context beyond efficacy evaluations that should be considered to ensure implementation across relevant healthcare settings.

Item Type:	Book Section
Date Type:	Publication
Status:	Published
Schools:	Centre for Trials Research (CNTRR) Biosciences
Publisher:	Elsevier
ISSN:	0074-7742
Date of First Compliant Deposit:	28 October 2022
Last Modified:	04 Jun 2024 15:54
URI:	https://orca.cardiff.ac.uk/id/eprint/153768

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