Key Guidance Updates in 2022

Your first holiday gift of the season has arrived: updated FDA guidances from the past year—and one key update from the European Medicines Agency (EMA). These final guidances reflect the breadth of pharmaceutical and technology-related submissions that FDA now contends with. The agency even asks for help with its administrative tasks, such as asking stakeholders to put in submission cover letters whether there is real-world evidence or real-world data in the application.

This guidance replaces the 2007 version. It is written for businesses that FDA refers to as “small entity establishments,” stakeholders who create human cells, tissues, or tissue-based products. The guidance details the agency’s regulatory framework on the manufacturing of these products.

This guidance addresses creation of human gene therapy products for adults and children with neurodegenerative disease. Recommendations span the products’ development to clinical trial design.

This guidance was written to help stakeholders already in possession of an approved application, be it for a new drug application (NDA), an abbreviated new drug application (ANDA), or for a biologics license application (BLA), with making a chemistry, manufacturing, and controls (CMC) change, post-approval, by using a comparability protocol. The protocol is a plan to assess the identity, potency, purity, quality, and strength of the changed product.

Sponsors who are submitting NDAs, investigational drug applications, and BLAs—and including real-world data and/or evidence in their supporting documents—are encouraged to identify uses of such evidence in their cover letters. The agency says such identifications will help with internal tracking.

This final guidance focuses on clarifying the types of functions included in clinical decision support (CDS) software that up until now were outside the definition of non-device CDS criteria. FDA’s existing policies on digital health products policies still apply to software functions that meet its definition of a device.

FDA is separating software and its purpose, determining which devices will be subject to oversight, and which will not. Software used to display data, images, and waveforms, are included in the latter.

This guidance discusses the agency’s views on development programs and clinical trial designs for drugs and biologics intended to treat acute myeloid leukemia. It covers individual treatment phases.

This “internationally harmonized” guidance details those factors stakeholders can use to justify their reasons for collecting less types of data in specific interventional clinical trials.