Multimodal management has been proposed as key to any effective drug intervention in cachexia. This article attempts to reflect on clinical and regulatory considerations of multimodal management treatment as a regulatory requirement in anticachexia drug therapy. To date, no European Union (EU) regulatory guidelines have been published and therefore this review could attempt to present and discuss some central issues to consider when developing an anticachexia drug.
The following themes are considered: EU regulatory pathways for drug approval (conditional and exceptional circumstances as well as adaptive licensing); selection criteria for randomized clinical trials allowing the identification and characterization of the population of interest that is an at-risk population with undisputable clinical need; issues related to primary and secondary outcome measures that are adequate to determine the efficacy of the intervention and the approach for the development of clinical biomarkers for cachexia.
Conversely, the incorporation of multimodal treatment in anticachexia drug therapy is expected to increase the effectiveness of intervention. This aspect is the aspect that appeals to pharmaceutical companies; however, at the same time, this raises regulatory and clinical issues that need to be kept in mind when designing randomised clinical trials.
aMedicines Authority, Rue D’Argens, Gzira, Malta
bDepartment of Biology, School of Pharmacy, University of Tor Vergata, Rome, Italy
cInnovative Clinical Trials, Department of Cardiology and Pneumology, University Medical Center Göttingen (UMG), Göttingen, Germany
dIRCCS San Raffaele, Roma, Italy
eSt George's University of London, London, UK
fDepartment of Pharmacy, University of Malta, Msida, Malta
gOncological Palliative Medicine, Clinic Oncology/Hematology, Department of Internal Medicine and Palliative Center, Cantonal Hospital, St. Gallen, Switzerland
Correspondence to John J. Borg, PhD, Medicines Authority, 203 Level 3, Rue D’Argens, Gzira, GZR 1368, Malta. Tel: +356 23439129; e-mail: email@example.com