There’s no question that real-world data (RWD) and real-world evidence (RWE) are playing an increasingly important role in health care decisions. Historically, FDA has primarily used RWE to inform regulatory decisions related to drug safety in the post-marketing setting. The health care community uses these data to support coverage decisions, and to develop guidelines and decision support tools for use in clinical practice. And drug and medical product developers use it to improve the efficiency of clinical trials.
The 21st Century Cures Act, passed in 2016, mandated that FDA establish a program to evaluate the potential use of real-world evidence (RWE) to support the approval of new indications for drugs, as well as to satisfy post-approval study requirements. And in December of 2018, FDA released a strategic framework to advance the use of RWE in regulatory decision making.
In our experience, many drug developers are interested in exploring ways to leverage RWD and RWE in their development programs but have questions about how to approach the opportunity.
If you’re interested in learning more about FDA’s approach to incorporating RWD and RWE into regulatory decision-making – I encourage you to check out our latest webinar. In the webinar, my colleagues – Dr. Bruce Feinberg and Dr. Jonathan Kish – and I share contemporary case studies of how RWD and RWE have been successfully leveraged by drug developers.
- Learn how Brineura® -- an orphan drug that treats CLN2 Batten Disease, a rare neurodegenerative disorder in children, leveraged RWE in the form of an external historical cohort to support FDA approval of the original marketing application.
- Learn how Lutathera® -- an orphan drug used to extend the life expectancy of patients with a rare, heterogeneous group of neuroendocrine tumors (NETs)– leveraged RWE collected under an expanded access protocol to support FDA approval of the original marketing application.
- Learn how Ibrance® -- originally approved for the treatment of postmenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer – leveraged RWE collected from electronic health records to support expansion of the indication to men.
In the webinar, we also share examples of developers who encountered significant challenges with inclusion of RWE in their marketing applications – and key clinical and regulatory lessons other developers can learn from those case studies.
Regardless of what stage of development your drug is in, it’s never too early to begin considering the role RWD and RWE can play in your development and post-approval programs. Doing so the right way – with the support of experts with years of regulatory and real-world evidence experience – can help you accelerate regulatory approvals and add significant value to your development programs.