Challenges on Outcomes Selection Along Early to Late Phase Clinical Trials of Drug Development: Survey to Experts in Neurosciences Drug Development

Author(s)

Zaragoza Domingo S1, Alonso Caballero J2, Ferrer M3, Acosta MT4, Annas P5, Bishop K6, Butlen-Ducuing F7, Bennett B8, Berger AK9, Dorffner G10, Edgar C11, Engler J12, Harel B13, de Gracia Blanco M14, Harrison J15, Horan W16, Kottner J17, Tinoco D18, Vance M19, Yavorsky C20
1Neuropsychological Research Organization s.l (Neuropsynchro), Sant Joan Despi, B, Spain, 2IMIM (Hospital del Mar Medical Research Institute) and CIBERESP, Madrid, UK, 3IMIM (Hospital del Mar Medical Research Institute) and CIBERESP, Barcelona, Spain, 4NIH, Washington DC, WA, Spain, 5Alexion Pharmaceuticals Ltd., Copenhaguen, Denmark, Denmark, 6Global Pharma Consultancy,, Washington DC, WA, USA, 7European Medicines Agency, Amsterdam, Amsterdam, Netherlands, 8Jazz Pharmaceuticals, London, London, UK, 9Lundbeck A/S, Copenhaguen, Copenhaguen, Denmark, 10The Siesta Group, Vienna, Austria, 11PCOA Associates Ltd, London, USA, 12CRONOS, IQVIA, Washington DC, WA, Spain, 13Takeda, Washington, WA, USA, 14Universitat de Girona, Girona, Girona, Spain, 15Scottish Brain Sciences, Edinburgh, UK, 16Karuna Therapeutics, Washington DC, WA, USA, 17Charité-Universitätsmedizin, Berlin, Berlin, Germany, 18Worldwide Clinical Trials, Barcelona, Barcelona, Spain, 19SANTIUM, Toronto, ON, Canada, 20Vialis Bioscience, Washington, WA, USA

OBJECTIVES: Despite major advances in neuroscience, clinical trials for neurological and psychiatric conditions continue to have notoriously high failure rates. The use of innovative clinical outcome assessments (COA) grounded in translational research is key to maximize the likelihood of identifying promising new treatments in early phase clinical trials. However, the field has lacked standardized practice guidelines for optimal selection of COAs and also face a low acceptance of innovative outcomes by the regulator by eventually health agencies. For the recently developed 7-steps standard process for COA selection, we wanted to explore the existing challenges for its implementation by pharma and biotech drug developers.

METHODS: A survey was distributed to experts on outcomes research to solicit feedback on the proposed 7-Step process; level of agreement, endorsement and expected challenges when implementing the standards.

RESULTS: Twenty-six participants, 46% from pharma industry, 19% CROs, 18% Academia among others were collected. The most (81%) had experience working on setting strategies for COA in clinical trials, where 60% for more than ten years. The 7-Step process was accepted by all, and 30% suggested additional activities. There was a high variety of opinions about how much the industry is currently using standards. Suggestions to encourage its adoption included; to conduct dissemination/educational activities, to proof the method, to stress out the alignment with existing regulatory guidances and to promote the advantages to pharma industry in terms of time and cost-effectiveness.

CONCLUSIONS: The consensus-based 7-Step method for setting COAs strategy in neuroscience clinical is nowadays the first and a key reference for any type of research in drug development. Feedback obtained from experts is in favor or its adoption. It is worth to mitigate the risks of failure and to facilitate the interaction with regulatory agencies. From the answers several actions raised from the participants describe a roadmap.

Conference/Value in Health Info

2024-11, ISPOR Europe 2024, Barcelona, Spain

Value in Health, Volume 27, Issue 12, S2 (December 2024)

Code

CO159

Topic

Clinical Outcomes, Patient-Centered Research

Topic Subcategory

Clinical Outcomes Assessment, Clinician Reported Outcomes, Instrument Development, Validation, & Translation, Performance-based Outcomes

Disease

Drugs, Medical Devices, Mental Health (including addition), Neurological Disorders, Pediatrics

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