REGULATORY CORNER

As a full-service CRO who helps many sponsors develop their clinical strategy and the associated study protocols, the release of an International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) protocol template is of great interest to PROMETRIKA. Step 2 of the ICH harmonized guideline on the clinical electronic structured harmonized protocol (CeSHarP) occurred in September 2022. This draft guidance was released for public consultation in December 2022, together with technical specifications and an electronic template. The guidance provides detailed information to be included in each section of a clinical protocol, expanding on the information currently included in the ICH GCP guidance. For those of us working in this regulated industry, it is a welcome addition.

The Cures Act of December 2016 added section 505F to the Federal Food, Drug, and Cosmetic Act (FD&C Act), requiring FDA to create a framework for the use of real-world evidence (RWE) in regulatory decision-making. In response, the draft FDA guideline entitled, “Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products,” issued in August 2023, provides very interesting and useful information for consideration in the planning of a marketing application (NDA/BLA) for a drug or biologic product based on RWE.

Earlier this year, the FDA granted approvals for two noteworthy gene therapy products. In one case, the product received accelerated approval with a review time of approximately 9 months and included an advisory committee meeting during the review cycle. The other product received full approval after a much longer review time that included a complete response letter and resubmission of the application.

The safety of patients receiving drug or biologic products is paramount in both the investigational and post-marketing settings. Preparing routine required safety updates can be an arduous and time-consuming process with different data lock points (DLPs), Reference Safety Information (RSI), and reporting timeframes for clinical studies and post-marketing reports. This month, I thought I would take a step back and talk about periodic safety reporting in both the investigational and post-marketing setting and how one can streamline the process. This blog will focus on routine required reports, not expedited reporting.

As many of us are well aware, while single arm trials (SATs) historically have been accepted by the US FDA as primary evidence of efficacy for marketing approval in the setting of accelerated approval (AA) of new oncology agents/indications, their acceptance in other therapeutic areas including rare diseases has been more selective.