(360) Feasibility of Prospective Real-World Studies to Fulfill Regulatory Commitments and Requirements in Oncology within the United States (US)

Background: Postmarketing commitments/requirements (PMR) are common in oncology, especially with accelerated approvals. While retrospective databases can fulfill a small proportion of PMR, prospective, fit-for-purpose, data are often needed. Prospective real-world studies (PrwS), designed to leverage routinely collected clinical data and intentionally captured data, can improve the efficiency of PMR fulfillment using established patient cohorts and existing infrastructures.

Objectives: To assess the opportunity for PrwS to fulfill oncology PMR within the US regulatory environment.

Methods: We evaluated the applicability of PrwS to fulfill oncology PMR issued by the US FDA. Both drugs and biologic products were included. From the publicly available FDA database, we selected oncology PMR with a due date from 2020–2029. Based on the descriptions, we classified each PMR by study type. A PrwS was considered a feasible solution when the PMR required prospective evidence in adult patients that could be generated from non-treatment/observational studies. We focused on scenarios with a need for intentionally collected data, so PMR with purely retrospective designs were excluded. Pediatric, preclinical, or treatment studies (e.g., Phase 1–3 clinical trials) were deemed unfeasible for PrwS. Two or more PMR with identical descriptions for the same product were deduplicated, with only one distinct PMR retained for the analysis.

Results: Out of 1873 unique PMR, there were 511 (27%) for 148 unique oncology products. Of these, 164 oncology PMR (32%, 93 products) were potentially suitable for PrwS and were included for further analysis. The number of these PMR due each year from 2020–2029 were as follows: 1, 16, 16, 21, 20, 27, 32, 16, 11, 4. Of the 54 PMR due from 2020–2023, 15 (28%) were “fulfilled,” 16 (30%) were “submitted,” and 4 (7%) were “released.” The remaining 19 (35%) were “delayed,” “pending,” or “ongoing.”

Conclusions: This analysis found that many oncology PMRs due within the past four years are not yet fulfilled, possibly due to delays during site selection, site activation and/or patient enrollment. A more detailed assessment of each PMR specification is needed to confirm the feasibility of a PrwS design. When feasible, real-world studies can accelerate completion of these important obligations via partnerships between regulators, sponsors, and organizations/networks capable of efficiently executing PrwS. Opportunities for acceleration and/or efficiency with PrwS include site selection/activation, patient identification, and streamlining data collection through an EHR.