Considerations for successful clinical trial design for MSC products for OA
osteoarthritis, OA
OSTEOARTHRITIS, EMA
Clinical trials investigating MSC treatments in OA should incorporate additional readouts on putative mechanisms of action to facilitate future regulatory approvals of MSC products in OA and to tailor next-generation MSC therapies that have specific therapeutic properties and CQAs that are tuned for relevance in OA (Fig. Levers for enabling successful mesenchymal stromal cell trials in osteoarthritis - Four aspects that need to be addressed to enable successful patient outcomes. PROMs patient-reported outcome measures, CPPs critical process parameters, CQAs critical quality attributes, MSCs mesenchymal stromal cells, OA osteoarthritisThe episodic nature of OA, termed “flare-ups”, is poorly understood etiologically [There are existing guidelines for clinical trial design considerations, including the selection of OA patient populations, as put out by the European Medicines Agency (EMA) [Cost is another consideration for the commercialization of successful MSC therapeutic products. The extent of the effect size and whether there are symptom-modifying or disease-modifying effects (as evaluated by a combination of PROMs, imaging, and biomarker and biomechanic assays) will determine the willingness to pay of the health care system and market. Using patient survey-based methods, Piuzzi et al. obtained pricing information for same-day minimally manipulated autologous cell preparations from 65 centers in the USA and found that the mean price was US$ 5156 ± 2446 for a single treatment [