Analysis Group’s Real-World Data Study Supports FDA’s Recent ExkivityTM (Mobocertinib) Approval

Date:October 19, 2021
Location:Boston, MA
Contact:Eric Seymour
+1 978 273 6049

Analysis Group, a global leader in health economics and outcomes research (HEOR) and regulatory epidemiology, today announced its collaborative real-world data (RWD) study was part of Takeda’s successful bid for the accelerated approval of Exkivity (mobocertinib) by the US Food and Drug Administration (FDA) for the treatment of locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor (EGFR) exon 20 insertion mutations. The retrospective RWD analysis of a community-based oncology data source, initiated by both Takeda and Analysis Group’s researchers, complemented Takeda’s Phase 1/2 single-arm, international, open-label, multicohort clinical trial (NCT02716116), also known as Study 101.

Exkivity, which the FDA granted priority review as well as breakthrough therapy designation, fast track designation, and orphan drug designation, is the first and only approved oral targeted therapy specifically designed for this difficult-to-treat cancer. The FDA review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence, which provides a framework for the concurrent submission and review of oncology products among international partners.

“The FDA’s acceptance of RWD and real-world evidence (RWE) in approvals is changing how pharma thinks about R&D,” said Analysis Group Managing Principal Mei Sheng Duh, M.P.H., Sc.D. “Takeda is at the fore of this understanding, and retained us to collaborate on the plan of the RWD study after its end of Phase 1 Type B meeting with the FDA to ensure the RWD study would align with the single-arm trial in the regulatory submission. The RWD provides contemporaneous natural history information and a benchmark of current treatment. The totality of evidence supports the clinical trial outcome for regulatory consideration.”

While the FDA has issued guidance documents for the use of RWE to support new drug applications and biologics license applications, the devil is, so to speak, in the details. Understanding the FDA’s oncology therapy decision making was essential in designing an adequate RWE study for the Exkivity regulatory submission.

“The FDA wants to see fit-for-purpose data and study design to identify and analyze a clinical trial-aligned cohort and generate RWE that they can consider in making regulatory decisions. Otherwise, the FDA is almost certainly not going to consider the RWE,” said study coauthor and Analysis Group Vice President Maral DerSarkissian, Ph.D. “It’s also important to note that, for a RWE study to meet the regulatory standard, the study protocol and statistical analysis plan must be specified a priori for transparency and to avoid ‘cherry picking’ data sources and results.”

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About Analysis Group’s HEOR Practice

Founded in 1981, Analysis Group is one of the largest international economics consulting firms, with more than 1,000 professionals across 14 offices. Analysis Group’s health care experts apply analytical expertise to health economics and outcomes research, clinical research, drug safety, epidemiology, market access and commercial strategy, and health care policy. Analysis Group’s internal experts, together with its network of affiliated experts from academia, industry, and government, provide our clients with exceptional breadth and depth of expertise and end-to-end consulting services globally.