In Development

RUF projects support the FDA while incorporating voices of all stakeholders, including FDA, patients and consumer advocates, regulated industry, healthcare providers, academics, and others – with the ultimate goal of improving public health. Some areas of work currently in development are outlined below.

Expanded Access (EA) Navigator

RUF is working with the FDA and many other partners to develop an Expanded Access (EA) Navigator aimed at increasing understanding around the issue of individual patient EA to investigational drugs.

EA, also known as compassionate use, refers to access to an investigational drug prior to regulatory approval by FDA. There are several FDA-designated pathways that allow use of an investigational drug to treat patients with serious diseases or conditions, who cannot participate in a clinical trial and for whom appropriate clinical trials or alternative therapies do not exist. These pathways include access for individual patients, intermediate-size patient populations, and large patient populations.

Of these pathways, single patient access (“single patient INDs”), has garnered increasing attention in recent years. Several high-profile social media campaigns, the Right to Try movement, FDA activities, legislative actions and significant press coverage have resulted in a spotlight on single patient access. Despite these initiatives, overall awareness of EA remains low and the steps to successfully navigate the process are not always well understood by healthcare providers and patients.

The Navigator will provide information on the non-emergency single patient EA request process via a website and a call-center. The website will be comprised of two main components: (1) educational content and tools for health care providers and patients, and (2) a corporate directory including information on companies’ policies and point of contact for EA requests, as required by the passage of 21st Century Cures Act in December 2016.

The Navigator will be launched in early June.

For questions about the project, please contact Navigator@reaganudall.org.

If you are a representative from a biopharmaceutical company that is interested in contributing information to the corporate directory, please click here.

 

Regulatory Science Education & Fellowships

This work area aims to address the critical need for strengthening scientific capability and capacity, both within the FDA as well as externally. RUF is currently in the process of defining and developing our regulatory science education agenda. Ultimately, this will include work to foster greater understanding of and expertise in new scientific tools, diagnostics, and manufacturing techniques, and will serve to identify potential barriers to translating basic research into clinical and regulatory practice.