RISK OF DISCONTINUATION OF ADVANCED THERAPY MEDICINAL PRODUCTS CLINICAL TRIALS

Author(s)

Hanna E1, Rémuzat C2, Toumi M1
1Faculté de Médecine, Laboratoire de Santé Publique, Aix-Marseille Université, Marseille, France, 2Creativ-Ceutical, Paris, France

OBJECTIVES: Advanced Therapy medicinal products (ATMPs) constitute a class of innovative products that encompasses: gene therapy, somatic cell therapy and tissue engineered products (TEP). There is an increased investment of commercial and non-commercial sponsors in this field. Randomized clinical trials (RCT) generate data to prove the efficacy of a new therapy, but the discontinuation of RCTs wastes scarce resources. Our objective is to identify the number and characteristics of discontinued ATMPs trials in order to evaluate the rate of discontinuation. METHODS: We searched for ATMPs trials conducted between 1999 to June 2015 using three databases: Clinicaltrials.gov, the International Clinical Trials Registry Platform (ICTRP) and the EudraCT. We selected the ATMPs trials after elimination of duplicates. We identified the disease areas and the sponsors as commercial or non-commercial organization. We classified ATMPs by type and trial status: ongoing, completed, terminated, discontinued, and prematurely ended. Then we calculated the rate of discontinuation. RESULTS: CONCLUSIONS: No failure risk rate per development phase is available for ATMPs. Discontinuation rate may prove helpful when assessing expected net present value to support portfolio arbitration. These results carry limitation as the reason for discontinuation is unknown. Further research about the reasons of discontinuation and the risk of negative results is needed to inform manufacturers and investors decisions.

Conference/Value in Health Info

2016-09, ISPOR Asia Pacific 2016, Singapore

Value in Health, Vol. 19, No. 7 (November 2016)

Code

PHP44

Topic

Economic Evaluation, Health Service Delivery & Process of Care

Topic Subcategory

Cost/Cost of Illness/Resource Use Studies, Health Care Research

Disease

Multiple Diseases

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