The Neuroscience Center of Excellence Act would – as the name implies – establish a Center of Excellence within FDA to focus on a broad range of neurological conditions, such as Alzheimer’s and personality disorders.
Overview of the legislation, by section:
- The bill is sponsored by Senators Susan Collins (R-ME) and Ben Ray Luján (D-NM). In the legislative text, the bill highlights the overwhelming impacts of neurological conditions on U.S. populations. It defines “neuroscience diseases and disorders” as 22 diseases and conditions ranging from addiction, behavioral health disorders and neurodevelopmental disorders (e.g., autism spectrum disorder, Down syndrome) to Alzheimer’s disease and traumatic brain injuries, including a provision for “rare diseases that impact the brain and central nervous system.” As the bill text notes, developing (and receiving FDA approval for) therapeutics to treat these conditions is demonstrably difficult, given uncertainty around clinically meaningful endpoints and the progression of certain conditions.
- The bill would establish a Center of Excellence (CoE) for neuroscience at the FDA. This CoE would focus on coordinating and advancing both agency regulatory activities and scientific understanding around neurological conditions and diseases. Under the bill, the FDA would have one year to stand up the CoE, under which a series of regulatory programs and initiatives would be run. It would be modelled after the FDA’s Oncology Center of Excellence (OCE).
- The Neuroscience Therapeutics Program, or NTP, would focus on both policymaking activities and public engagement and reporting requirements. Under this program, the CoE would be directed to issue new guidance on novel approaches to researching neurological condition therapeutics, including guidance on master protocols and incorporating clinical outcome assessments (CoAs). The CoE would also be tasked with convening regular meetings to solicit input on outstanding needs and emerging opportunities – including the development of new biomarkers and endpoints and novel drug development methods – and to report back to Congress on its findings.
- The Neuroscience Patient-Focused Drug Development (PFDD) program within the CoE would be intended as hub for “patient experience data” and its use in development and regulation. This program would also be tasked with issuing new guidance to support the collection (and regulatory use of) patient experience data and convening public meetings on the subject.
- The Neuroscience Natural History Studies Program would focus on understanding the baseline science of neurological diseases. This provision of the bill would direct the FDA to issue guidance on conducting natural history studies (i.e., a “preplanned observational study”) that can track and help categorize the course of the disease of interest. Natural History Studies are essentially a study assessing what the disease looks like ordinarily. This data can then be used as a sort of control group for future research (or more commonly, permit smaller study groups).
- The Digital Health Technologies Program would seek to advance the use of digital health technologies and digital endpoints in neurological condition research. The agency would be tasked with issuing guidance on how to incorporate digital research methods within two years. Interestingly, digital health technologies like wearables are increasingly being used to support research into patient-facing measures (sometimes termed patient-generated health data) such as activity levels or sleep, according to the Digital Medicine Society (DiME) Playbook of digital measures (note: DiME is the convener of the Digital Health Measurement Collaborative Community (DATAcc), in which CDRH is a participant). However, FDA recently declined a request from Verily Life Sciences to qualify a method using their wrist-worn study watch as a drug development tool to support research in Parkinson’s patients. Dedicated guidance on this topic would likely be a significant value-add to the field of digital research, particularly as neuroscience may lend itself well to behavioral indicators.
- The Ensuring Equity in Neuroscience Program would, as the name implies, seek to reduce disparities in neuroscience research, including through bolstering research participation for traditionally underrepresented groups. As with the other programs, the CoE would seek to accomplish these by hosting a public meeting to solicit input and issuing guidance on enhancing diversity in clinical trials.
- The CoE would also seek to investigate the intersection of Covid-19 and neurological conditions. As the text of the legislation explains, the Covid-19 pandemic has “exacerbated the prevalence and impact of psychiatric diseases and disorders,” including a significant uptick in reported symptoms of anxiety and/or depression. In addition, previous infection with the virus may be linked to neurological complications in the longer-term. The CoE would be tasked with convening a series of public meetings to assess the impact of the Covid-19 pandemic on neuroscience disease and disorder diagnoses, and strategies “for the rapid development of medical products to address the direct and indirect impacts” of the pandemic. FDA would be tasked with publishing a report on their findings.
- Of note, top FDA regulators have previously questioned the utility having condition specific CoEs. In a June 2021 interview, OCE Director Rick Pazdur urged caution on the creation of CoEs for their own sake, especially when there is not a strong foundation in place. According to Pazdur, the FDA doesn’t need more bureaucracy for its own sake. Rather, a center of excellence first needs a strong scientific framework to understand how things may best be regulated. Then, he said, it needs to have a base of talented people. Only after the science and talent are in place would the FDA need additional and appropriate structure, such as a center of excellence. Without the first two elements in place, Pazdur said, the structure won’t do anything to help accelerate regulation and development – and might even serve as a barrier to efficient development. In fact, the only CoE creation that Pazdur specifically supported at the time was a Center of Excellence in Biostatistics, especially with the advancing field of advanced analytics.
- For now, it’s not clear if neuroscience has such a “strong foundation,” or if the development of the related deliverables might be better accomplished by existing FDA infrastructure. As outlined in the bill, the “neuroscience diseases and disorders” on which the new CoE would focus include a broad range of conditions: Alzheimer’s, anxiety, ALS, bipolar disorder, epilepsy, headaches, multiple sclerosis, pain, personality disorders and chronic traumatic encephalopathy (CTE), to name a few. With such a broad mandate, this could serve to reduce the utility of a potential CoE – especially, as Pazdur noted, since the success of the CoE relies on having a strong scientific framework for the conditions of interest.
- What’s next? The bill has just been introduced and is not currently scheduled for a hearing in the Senate. It would authorize $25 million in funding for fiscal years 2023 through 2023 to be appropriated, which would be dependent on subsequent federal appropriations.