A Model for Accelerating Identification and Regulatory Approval of Effective Investigational Agents

Esserman LJ, Barker AD, Woodcock J, Buxton M, Berry DA, Patterson R, King M, DeMichele A, Hylton N, Rubin EH, Parkinson D, Wholley D, Van't Veer L, Yee D, Park J, Tripathy D, Perlmutter J, Buetow K, Hogarth M, Gray JW, Dilts D

<p>A path for accelerated regulatory review of new drug and biomarker combinations is badly needed to transform the current clinical drug development process into an efficient, effective system that meets current and future healthcare needs. However, this type of radical transformation will not occur by layering regulatory change on existing clinical practice patterns. Here, we summarize a May 2011 I-SPY 2 TRIAL (Investigating Serial Studies to Predict Your Therapeutic Response with Imaging and Molecular AnaLysis) workshop that included key leaders from the Food and Drug Administration (FDA), academia, industry, advocacy, and clinical trial-focused foundations that addressed this critical need. The workshop identified key aspects of the organization, trial design, and regulatory and industry alignment required to rapidly move successful agents from trial to clinical care. The workshop specifically focused on providing input for the development of a new evidence-based regulatory path for review of drugs and biomarkers that derive from neoadjuvant, modular, adaptive phase 2 screening trials achieved through precompetitive collaborations. The workshop participants agreed that new models, exemplified by the I-SPY 2 TRIAL for breast cancer, could address the need to more efficiently review and advance new drug and biomarker combinations. A three-tier model for trial development best describes this process. As practiced in I SPY 2, integrating imaging and biomarker information obtained through adaptive trials in the neoadjuvant breast cancer care setting is the first step. The second tier is replicable structural processes, including real-time data collection. The third is partner alignment achieved through pre-competitive collaboration and the potential to position successful drugs for accelerated regulatory approval in the neoadjuvant setting, where patients are likely to have the greatest benefit. Many elements of this model were incorporated into a new FDA draft guidance document which was released for public comment on May 31, 2012.</p>

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