Drug Development Tools (DDTs) play an important role in bringing new therapies to the market. DDTs include biomarkers, which are defined biological characteristics; clinical outcome assessments (COAs), which describe how patients feel, function, or survive; and certain animal models. DDTs that FDA has “qualified” (or accepted) can be relied upon to have a specific interpretation and application in medical product development and regulatory review.
There are many novel DDTs that incorporate the latest advances in science and technology but may not have a clear path forward for use in drug development. To encourage use of these novel DDTs, FDA is launching the Innovative Science and Technology Approaches for New Drugs (ISTAND) Pilot Program. This program will create a “home” for new technologies aimed to support, streamline and potentially speed up development of drugs and biologics.
In this CDER Conversation, Steve Berman, MPH, a science policy analyst in the Office of New Drugs and lead for ISTAND, discusses the pilot program and the importance of novel DDTs.
Why is FDA launching the ISTAND pilot program?
We are launching this program to provide a pathway for the most novel DDTs to be used in regulatory applications for new medical products. In other words, we wanted to give these newest-of-the-new tools a regulatory home. Biomarkers, COAs and certain animal models already have a well-established pathway forward, but these novel DDTs do not — yet. While staff within certain CDER divisions have spoken with developers of these tools casually or as part of drug and biologic applications, we have not had a formal program in place until now.
We believe novel DDTs can support and possibly speed up therapeutic development — ultimately delivering treatments to patients sooner. It’s no secret that lead times are long, often taking years or even decades for companies to bring treatments to the market. So, it is important from a clinical standpoint to have DDTs that incorporate the latest science and technological advances.
And there is a lot of innovation taking place right now. For instance, the scientific community is making great strides with artificial intelligence and microphysiological models — which are miniature models of human organs or connected systems. These both could be types of DDTs that lead to rapid advances in developing new drugs.
Can you talk about the biomedical community’s interest in DDTs and ISTAND?
Yes, there is a lot of interest in moving the needle on novel DDTs and, more generally, speeding up therapeutic development. In particular, the 21st Century Cures Act, which was enacted in December 2016, aims to bring innovative treatments to patients faster.
For DDTs specifically, the Cures Act created a process — known as a qualification process — that DDT developers can leverage to engage with FDA outside of a specific drug development effort. Once a tool is qualified for a specific use — the regulatory term is a “context of use” — then it can be used by anyone for that purpose. Qualification lets people use the DDT without having to prove its worth, because the qualification process has already done that.
Of course, a DDT does not have to be qualified to be used in a product development program, and, in fact, we think some that go through ISTAND will not be good fits for the qualification pathway. In these cases, we may be able to encourage the development of these DDTs through other mechanisms, such as FDA guidances, public meetings to identify key issues, meetings with agency staff, or “white papers” that discuss implementation.
How will ISTAND work?
We plan to evaluate novel DDTs similar to how we currently evaluate biomarkers and COAs, but with more feedback from FDA when needed. After a triage process where we will consider tool’s potential value and our resources (we’re a pilot program, after all), we will decide if qualification makes the most sense. If so, we can help move it through the DDT qualification process.
Other ISTAND projects may not be good candidates for qualification, but still could be useful in drug development, as I mentioned before. In these cases, we will work with the DDT developer to find the best path forward — whether that is FDA meetings, white papers or other avenues.
What are examples of submissions that FDA would consider for ISTAND?
I’ll start with a caveat — we do not want to box ourselves in here, because we don’t know what is going to cross our plate. So, these are some examples, but there certainly are other topics that we would consider:
- Data collection or analysis methods that differ from current standard approaches
- Tissue chips, which are miniature models of human organs, for safety assessments
- Certain digital health technologies, including artificial intelligence or wearable sensors, used during clinical trials
Submissions can touch on any area of medicine or disease. When reviewing them, we will prioritize ones that focus on a gap or need in drug development and are most feasible to implement.
I’ll end with a caveat too — while we are very much open to submissions that use all sorts of new science and technology, our regulatory standards remain unchanged. These submissions need to provide analytically and clinically meaningful information for us to accept them.
How do you apply to ISTAND?
People interested in applying to ISTAND should review the ISTAND Pilot Program webpage, which has information about the submission process. We expect to accept two to four ISTAND projects per year, but we are flexible on that number.
What is in store for the future of ISTAND?
Right now is the future! We hope these submissions represent the latest and greatest science and technology. We are excited to see what we receive and if any patterns emerge. We’re currently a pilot program working to help bring therapies to patients faster. We look forward to seeing how our pilot works and hopefully contributes to FDA’s and CDER’s public health mission.