FDA Clarifies Guidance on Use of Real-World Data

Makers of drugs and biologics can use real-world data to support regulatory decision-making, according to new Food and Drug Administration guidance

The Food and Drug Administration published new guidance Aug. 31, 2023, stating that some studies using real-world data (RWD) do not require investigational new drug (IND) applications and describing considerations for fulfilling postmarket study requirements.

FDA's publication of the guidance fulfills an agency requirement under the 21st Century Cures Act, a 2016 law crafted to help accelerate medical product development and innovations for patient use in a more timely manner. The guidance finalizes draft recommendations first released in December 2021.

New guidance may accelerate innovation and patient access

By delineating between non-interventional studies (i.e., cohort and case-control designs) and interventional studies (i.e., clinical trials), the guidance establishes that only the latter qualify as clinical investigations according to S312.3 of the 21st Century Cures Act. This means that non-interventional studies do not require an IND submission, which may accelerate the review and approval of marketed medical products for new indications.

The guidance further clarifies that RWD may include electronic health records, medical claims, registries, and data generated under an emergency use authorization (EUA), during which FDA may authorize special use of products for the protection of public health.

Collaboration and transparency highlighted as critical components

The agency's guidance underscores the importance of collaboration, both with FDA and outside data privacy experts, early in the marketing application process. Such conversations can help ensure appropriate study design and data source selection; support the evaluation of a study's feasibility; identify potential design and implementation challenges; and guide the review of relevant study documentation.

With respect to transparency — a requirement for RWD supporting a marketing application — the guidance also acknowledges that patient-level data may not be directly accessible to the sponsor. The guidance points to various regulatory pathways (Type V drug master files, pre-investigational new drug applications, etc.) that can be used to submit patient data if the drug manufacturer cannot use traditional channels. 

As with all FDA guidance, the document's recommendations reflect the agency's current thinking but remain non-binding; however, the guidance has significant implications for sponsors, as they discuss incorporating RWD without lengthening the regulatory submission process. As such, RWD planning can start as early as phase 2 and run alongside randomized control trial (RCT) activities to ensure an optimal regulatory submission package with gold-standard RCT data strengthened by RWD for additional context around endpoints in clinical practice, under-represented populations, and long-term or rare events.