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The Office of New Drugs' Efforts to Expand Regulatory Science Research

CDER’s Office of New Drugs (OND) developed a research program in 2018 to centralize and enhance its regulatory science research activities. Recently, the OND Research Program (OND-RP) published its first two fiscal year annual reports, the Extramural Research Outcomes Report and the ORISE Fellowship Research Outcomes Report, which highlight the research projects that the OND-RP funded.

In this CDER Conversation, Laura Jaeger, PhD, associate director of the OND-RP, talks about the program’s important work.

Laura Jaeger
Laura Jaeger, PhD

Congratulations on the publication of the OND-RP’s first two reports. Can you tell me why OND started the research program?

Thank you. The reports are a celebration of how we contributed to the field of regulatory science research, which is research to help our agency make informed, evidence-based regulatory decisions. We funded a lot of impactful projects, and I am happy to share these research outcomes with the public.

Our research program was formed in 2018 to centralize and create an infrastructure for OND’s regulatory science research. Most project ideas come from OND’s scientific review staff, who evaluate a drug’s safety and efficacy profile to help decide if the treatment should be approved. OND reviewers also assess if additional measures are necessary for safe drug use or if some patient populations should not take the medication, among other regulatory decisions.

In reviewing new drug applications, OND reviewers may realize that they cannot fully assess a drug’s safety or effectiveness — and therefore cannot make an informed decision — because of knowledge gaps in our scientific understanding. Whenever OND reviewers identify a knowledge gap like this, they can reach out to us, and we work together on initiating a research project to answer this question.

How is the OND-RP funded regulatory science research different from other types of research?

Regulatory science research is much more targeted than basic scientific research, which provides fundamental knowledge about the nature and behavior of living systems. Basic scientific research asks broad questions that may take a long time to answer. The OND-RP, meanwhile, is focused on addressing specific scientific questions that will produce immediate impacts on how CDER and FDA make drug approval decisions. Because our research is so targeted, our projects are relatively short, about three to four years.

We have comparatively limited funding, so our research projects use creative methods to generate high-quality clinical or scientific information. A strategy we like to use is “data mining” of existing large data sets. For example, OND often repurposes raw data submitted in new drug applications to address questions. We also leverage electronic health records and patient registries to address clinical knowledge gaps. We hope the research produces tangible end-products that will support FDA’s mission, such as guidances for industry, internal review documents, updates to regulatory policies, and Drug Development Tools. Like most scientific researchers, we share our findings with the community through presentations and published journal articles.

What are the two main ways that the OND-RP funds research?

While our OND review staff typically identify the research question, they don’t have the bandwidth to complete the study alone. Instead, the OND-RP funds both extramural (investigators from outside FDA) and intramural (investigators from within FDA) research. That is why we published two reports — one highlighting our extramural research projects and another focused on intramural research.

On the extramural front, we use several mechanisms to fund research. These include FDA’s Broad Agency Announcement program, the agency’s Centers for Excellence in Regulatory Science and Innovation program, inter-agency agreements with other government entities, and public-private partnerships. We also use agreements such as memorandums of understanding, research collaboration agreements, and cooperative research and development agreements to formalize our research partnerships. Our external investigators provide the capabilities, expertise, or equipment to conduct extramural research. These investigators come from industry, academia, or other government agencies.

We fund intramural research primarily through the Oak Ridge Institute for Science and Education (ORISE) fellowship program. In this program, college students or recent graduates (up to five years after graduating) can conduct research with an FDA mentor on a regulatory science project. It’s a great opportunity for young investigators to get hands-on experience in regulatory science and contribute to our mission. People interested in the fellowship program can search and apply here. We regularly post new full-year ORISE fellowship opportunities.

What are examples of research projects that the OND-RP has funded?

One example from our extramural report is an effort to develop blood-based biomarkers, or molecules that signal a normal or abnormal biologic process, for traumatic brain injury. Drug development in this area is difficult because people’s symptoms vary and there are few objective ways to determine prognosis. A biomarker would help investigators sort individuals into clinical trials by disease severity, ultimately fostering drug development.

Another extramural research project focuses on the challenges in interpreting electrocardiograms (ECGs) in children, making it difficult to evaluate drugs targeting serious and life-threatening heart rhythm abnormalities. In this project, researchers are collecting real-world ECG data to better understand how to interpret readings and perform pediatric cardiac safety evaluations.

One project highlighted in our ORISE report focused on enhancing the evaluation of bulk drug substances. Compounders use these substances to create custom drugs for individual patients. For example, if a patient has an allergy to an ingredient in an FDA-approved drug, compounders can use bulk drug substances to compound a drug without the allergen. In this project, ORISE fellows helped create internal resources to streamline our review of bulk substances. This will enhance the safety of compounded drugs, which are not FDA-approved and don’t have the same safety assurances as approved drugs.

In another example, ORISE fellows contributed to the development of a list of molecular targets related to the growth or progression of pediatric cancers. This list is published on the FDA website, and it’s helping to spur discussion on initial Pediatric Study Plans (iPSPs) for more than 100 drug and biologic candidates. iPSPs are outlines of pediatric stud(ies) that drug sponsors plan to conduct to show that a drug can be used in children. This is important because if a drug will be used to treat children, FDA needs data from pediatric clinical trials to assess safety and efficacy. In oncology, some molecular targets are unlikely to be associated with the growth of pediatric cancers, meaning that studying the drug in children may not be necessary. Industry uses the lists created from this research project to inform their iPSPs for oncology drugs.

I encourage you to read our extramural and ORISE outcomes reports to learn more about our work in advancing regulatory science research. For more information about the OND’s regulatory science research, please visit Office of New Drugs Regulatory Science Research . If you have suggestions for regulatory science knowledge gaps in new drug development, send us an email at ONDResearch@fda.hhs.gov.

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