12/07/2021
Photo by Massimo Botturi on Unsplash
The US Food and Drug Administration (FDA) released a draft guidance “Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products” for public comment in November 2021. This draft guidance is published in line with the 21st Century Cures Act (Cures Act). Part of the Cures Act is related to the use of real-world evidence (RWE) to help support regulatory decision making, including the approval of a new indication for an approved drug or to support post approval study requirements. According to the FDA, RWE is “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than traditional clinical trials” and the “clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real word data (RWD).” RWD comes from medical billing claims, electronic health records (EHR), clinical registries, patient-generated data including from in-home use, and other sources. RWE provides valuable information on the safety and efficacy of a drug, biologic, or device when it is used as intended, outside the constraints of a clinical trial.
Background
A good source of RWE, a registry is an “organized system that collects clinical and other data in a standardized format for a population defined by a particular disease, condition, or exposure.” Data can be directly entered into the registry (e.g., clinician reported outcomes) or linked to external sources such as laboratory results. When appropriate, data from registries may be used to inform the design and support the conduct of clinical trials or observational studies (e.g., a more informed approach to selection of appropriate study participants).
Summary of Draft Guidance
Some considerations for using registry data to support regulatory decisions depend on factors such as:
how the data are intended to be used for regulatory purposes
the enrolled patient population
which data are collected
how data are collected and analyzed, including if there are processes in place to ensure data integrity
if data are collected for one purpose, they may not be fit for use for a different purpose. For example
This draft guidance discusses considerations for:
A registries’ fitness for use based on its ability to support the collection of relevant and reliable data.
Fit for use does not have a specific regulatory definition but means quality data that are relevant and reliable.
Relevant means “availability of key data elements (patient characteristics, exposures, outcomes) and a sufficient number of representative patients for the study.”
Reliable means “data accuracy, completeness, provenance, and traceability.”
The draft guidance provides multiple examples of relevant and reliable data that may be included in a registry.
Linking a registry to another data source to supplement data
Supporting FDA review of submissions that include registry data
Registries have an advantage over other sources of RWE due to their structured approach to data entry, but they also have limitations since they may focus on a limited dataset (e.g., patients may be different from the target population, patients in the registry may have more severe illness) which may introduce bias. A good reminder is to focus on objective endpoints when possible. The draft guidance offers valuable approaches to meaningfully use registry RWE while minimizing pitfalls.
Comment now through February 28, 2022 HERE.
Using RWE to support applications for drug or biologics can be expected to expedite the data collection process, thus leading to safe and effective products getting to market faster. Additional guidance on the use of RWE can be expected.
For more information on the FDA’s framework for RWE, visit our blog “FDA Framework for Use of Real-World Evidence.”
In September, the FDA published a draft guidance “Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision Making for Drug and Biological Products.” Read our blog HERE for more information.
In October, the FDA published a draft guidance “Data Standards for Drug and Biological Product Submissions Containing Real-World Data.” Read our blog HERE for more information.
If you are preparing a regulatory submission and need assistance with utilizing RWE, Clinical Pathways can provide training as it is important to know how to apply these methods. Contact us at info@clinicalpathwaysresearch.com.
- The Clinical Pathways Team
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