Abstract 3362: Factors driving industry participation in the collaborative I-SPY2 platform trial

Abstract

Abstract Background: Platform trials enable faster, less expensive, and more efficient clinical readouts by evaluating multiple products and combinations. This study explores the willingness of pharmaceutical companies to put experimental drugs into a platform trial, and examines the drivers and barriers to their participation. Methods: The I-SPY2 (Investigation of Serial studies to Predict YourTherapeutic Response with Imaging And molecular anaLysis2) pre-competitive collaborative platform trial was used as a case study to elucidate the motivation and constraints of the pharmaceutical industry. Rogers’ Diffusion of Innovation theory (ISBN 0029266718) was used as theoretical framework, primary interviews were conducted with 14 executive level representatives from 9 different pharmaceutical companies, and data was analyzed in ATLAS.ti to derive the influence factors. The I-SPY2 neo-adjuvant breast cancer platform trial is a partnership of 10 pharmaceutical and 5 biotech companies, alongside 18 academic institutions, patient advocates and the FDA, under the sponsorship of the non-profit Quantum Leap Healthcare Collaborative. I-SPY2 has entered 15 investigational agents of which 10 have completed evaluation and 5 are still ongoing. Results: Two factors were critical drivers for whether pharmaceutical companies were willing to put experimental drugs into a platform trial: the registration status of the agent and whether or not it was the primary indication for treatment. Drugs were more likely to be considered for these types of trials if other registration trials were completed or in progress and if the primary indication for the agent was in a different cancer organ type (e.g. not breast). Four types of perspectives positively influenced pharmaceutical company to participation: a gain in efficiency of drug evaluation, the opportunity for knowledge generation, innovativeness of the trial design, and the expertise and reputation of the consortium. Primary barriers that reduced the chance of participation included a loss of control over the evaluation of the drug and the organizational and governance structure. No significant differences were identified between company size and experience in a therapeutic area. Finally, regulatory guidelines specified by the FDA and EMEA as well as payer engagement were the most significant influence factors which could increase pharmaceutical company’s future participation in platform trials. Conclusion: This is the first study to examine the drivers and barriers to pharmaceutical company participation in platform trials and provides insight for why innovative companies participate in these groundbreaking studies. The FDA has expressed strong interest in promoting platform trials because of their efficiency and knowledge generation. Working to integrate a registration component as part of adaptive platform trials is likely to be a key driver of successful future adoption. Citation Format: Jurr M. van Ramshorst, Laura J. van 't Veer, Dave Mandelkern, Laura J. Esserman, Daniel Dornbusch. Factors driving industry participation in the collaborative I-SPY2 platform trial [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2019; 2019 Mar 29-Apr 3; Atlanta, GA. Philadelphia (PA): AACR; Cancer Res 2019;79(13 Suppl):Abstract nr 3362.