The Neuroscience Steering Committee, led by the FNIH and its co-chairs Dr. Linda Brady, Dr. Hartmuth Kolb, and the emeritus co-chair Dr. Bill Potter, brought together experts in the field of neuroscience from industry, NIH, FDA, and academia to present progress to date, next steps, and key obstacles that need to be addressed in order to drive biomarker development in a multitude of neuroscience focus areas. The 2-day agenda, was put together in collaboration with our federal colleagues at the NIH and FDA, was comprised of various sessions focusing on multimodal biomarkers, fluid and imaging biomarker qualification, clinical measures, and best practices in data management and sample collection.
The Neuroscience Symposium was held on April 30-May 1 in the Washington, D.C. area. Review the agenda here.
View videos from the Symposium below:
Session 1 – Multimodal Biomarkers in Neuroscience
Session 2 – Fluid Biomarkers
Session 3 – Imaging Biomarker State of Science
Session 4 – EEG Biomarker State of Science
Session 5 – Clinical Measures in Drug Development
On April 30 and May 1, 2019, in Bethesda, Maryland, the Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium Neuroscience Steering Committee (BC NSC) convened a meeting of public- and private-sector stakeholders, including representatives of the National Institutes of Health (NIH), the Food and Drug Administration (FDA), and various pharmaceutical companies and nonprofits, as well as academics, to (1) review the state of the science for biomarker discovery, validation, and qualification efforts across a broad range of indications and disease areas, and (2) discuss potential contexts of use (COUs) for different biomarkers, given that FDA can only qualify a biomarker for a pre-specified, well-defined purpose.
The agenda for the symposium was divided into five sessions, each focused on a different category of biomarker: (1) Multimodal, (2) Fluid, (3) Imaging, (4) Electroencephalography (EEG), and (5) Clinical measures. Each session consisted of several participant presentations, followed by panel discussions during which all presenters from that session provided additional thoughts and responded to questions from other participants.
Informed by outputs from this meeting, the BC NSC convened at a second face-to-face meeting in June 2019, during which participants prioritized and formed working groups to develop new projects to drive biomarker development efforts, aiming to maximally leverage the advantages of precompetitive partnerships.
In addition to the Symposium convening various stakeholders to assess the state of biomarker discovery and qualification, the Symposium served as a forum for the Neuroscience Steering Committee to assess potential new projects. The BC NSC convened at a second face-to-face meeting in June 2019, during which participants prioritized and formed 4 working groups to develop new projects to drive biomarker development effortsBased on the Symposium discussions, the aiming to maximally leverage the advantages of precompetitive partnerships:
- AD Bio sample-Data Collection Working Group (WG)
- Digital Biomarker Validation WG
- Pre-Competitive PET Tracer Development WG
- Validation of a Fluid Biomarker against Imaging WG
If interested in any of these working groups or learning more about joining the Neuroscience Steering Committee, please contact Wesley Horton, [email protected]
Other Outcomes: Participants of the Symposium closed the meeting by stressing the importance of the following activities:
- Producing multimodal phenotyping frameworks for various neuroscience therapeutic areas, similar to the ATN system created to identify Alzheimer’s Disease (AD) patient subgroups. The ATN system was designed for flexibility and can evolve by incorporating new biomarkers within existing ATN groups.
- Developing multicomponent biomarkers to capture disease complexity and to better understand the sequence of pathophysiological events.
- Adhering to “fit-for-purpose” principles, taking care to deliberately select and define what is being measured, and how. FDA can only evaluate the quality of a biomarker against the backdrop of a specific context of use (COU).
- Investigators should generally focus their biomarker discovery and development efforts on clinically useful COUs, such as patient enrichment, change in patient management, or improved outcomes. FDA advocates “purpose-driven” Research.
- Recognizing that different biomarker modalities can face different validation challenges. For example, imaging biomarkers can be validated using postmortem pathology, such as plaques and tangles for AD. By contrast, fluid and protein biomarkers are often far more complex to validate.
- Standardizing operating procedures such as data collection, measurement, and storage protocols.
- Learning from biomarker development efforts in other disease areas, such as oncology (e.g., the MammaPrint biosignature) and cardiovascular disease, that align with the BC NSC’s goals.
- Sharing and harmonizing data through open access platforms, as the Alzheimer’s Disease Neuroimaging Initiative (ADNI) does.