The 21st Century Cures Act (Cures Act), signed into law on December 13, 2016, is designed to accelerate medical product development and bring new innovations and advances faster and more efficiently to the patients who need them. Among other provisions, the Cures Act added section 505F to the Federal Food, Drug, and Cosmetic Act (FD&C Act). Pursuant to this section, the Food and Drug Administration (FDA) has created a framework for evaluating the potential use of real-world evidence (RWE) to help support the approval of a new indication for a drug already approved under section 505(c) of the FD&C Act or to help support or satisfy drug post-approval study requirements – Framework for FDA’s Real World Evidence Program – December 2018

Below is a ‘snippet’ of the FDAs Framework for Evaluating RWD/RWE for Use in Regulatory Decisions

Regulatory Considerations for Study Designs Using RWD

The use of EHRs, mobile health technology, and other electronic data-capture technology, together with new trial and study designs using RWD, has the potential to streamline and improve the efficiency of clinical studies. Nevertheless, these advances may also raise new questions about the applicability of certain regulatory requirements, including requirements for informed consent and appropriate oversight and monitoring. To facilitate the electronic capture of data from health care systems for research purposes while maintaining adequate documentation for FDA to validate the source and reliability of the data, FDA has already provided guidance on the use of electronic source data, electronic signatures, and EHRs. FDA has also issued the guidance Use of Electronic Informed Consent Questions and Answers in December 2016 on using electronic informed consent. Additional guidance may be needed to address different study designs using RWD to generate RWE for effectiveness determinations.

Use of Electronic Source Data

There are several FDA guidance documents that address the use of electronic data in clinical investigations. In September 2013, FDA published the guidance Electronic Source Data in Clinical Investigations, which promotes capturing source data in electronic format and provides recommendations on the capture, review, and retention of electronic source data in FDA-regulated clinical investigations. In July 2018, FDA published the final guidance Use of Electronic Health Records in Clinical Investigations. This guidance provides recommendations on ensuring the integrity of EHR data that are collected and used as electronic source data in clinical investigations of medical products.

FDA’s Part 11 regulations (21 CFR part 11) focus on the quality, authenticity, and reliability of electronic records from their point of creation to their modification, maintenance, archiving, retrieval, or transmission. In June 2017, FDA published a draft guidance Use of Electronic Records and Electronic Signatures in Clinical Investigations Under 21 CFR Part 11 – Questions and Answers. This guidance discusses procedures to help ensure that electronic records and electronic signatures are trustworthy and reliable and proposes a risk-based approach when deciding to validate electronic systems, implement audit trails for electronic records, and archive records that are pertinent to clinical investigations.

Program Item. FDA will consider whether these guidance documents adequately address concerns relevant to different study designs using RWD to generate RWE for drug product effectiveness determinations or whether additional guidance on use of electronic source data is needed.

Read More: Framework for FDA’s Real World Evidence Program – December 2018