Systematic Review of Clinical Outcome Assessments (COAs) for Communication Brain-Computer Interface Devices (cBCIs) in Amyotrophic Lateral Sclerosis (ALS) (UH2/UH3) Clinical Trials Not Allowed | RFA FD 23 030

FAQs: FDA RFA-FD-23-030 (UH2/UH3) on Clinical Benefit Measurement for Communication BCIs in ALS

1) What is the main purpose of this funding opportunity?

This FDA opportunity (RFA-FD-23-030) supports research to improve how clinical benefits are measured for communication brain-computer interface devices (cBCIs) used by people with ALS, particularly individuals with severe speech and communication limitations. The goal is to strengthen the evidence base and clarify what outcomes should be measured and why those outcomes matter.

2) Is this opportunity meant to fund a clinical trial?

No. Clinical trials are explicitly not allowed under this announcement. The focus is on measurement research and patient-centered understanding that can guide the future development and selection of clinical outcome assessments (COAs) for evaluating cBCIs in ALS.

3) What type of award mechanism is used?

The funding instrument is a cooperative agreement using a two-phase, milestone-driven UH2/UH3 structure. Because it is a cooperative agreement, FDA is expected to have substantial programmatic involvement compared with a typical grant.

4) What does the UH2/UH3 structure mean in this program?

The project is split into two sequential phases. UH2 is a first-phase effort focused on a systematic landscape analysis of existing measurement approaches. UH3 is a second-phase effort focused on patient and caregiver input. Movement from UH2 to UH3 depends on meeting predefined milestones.

5) How long can the project last?

The total project period can be up to two years, with each phase limited to one year (UH2 up to one year and UH3 up to one year).

6) Do applicants submit separate applications for UH2 and UH3?

No. Applicants must submit a single combined UH2/UH3 application, and the application needs to clearly describe plans, methods, and milestones for both phases.

7) What happens if UH2 milestones are not met?

Progression from the UH2 phase to the UH3 phase depends on meeting predefined milestones. If milestones are not met, the project may not transition to UH3 as planned.

8) What is the focus of the UH2 phase?

UH2 is a systematic landscape analysis intended to map the current state of measurement in the field. It includes a systematic review of available literature and other relevant data sources, along with interviews of key opinion leaders (KOLs). The aim is to document which COAs have been used in clinical contexts or studies involving cBCIs and to identify gaps in what is currently measured.

9) What deliverables are expected from the UH2 phase?

A practical deliverable is an inventory and critique of existing measures, including what the measures assess (for example, communication speed, accuracy, usability, independence, quality of life) and where they fall short (such as limited relevance to late-stage ALS communication needs). UH2 also aims to identify measurement concepts that may need to be added or refined.

10) What is the focus of the UH3 phase?

UH3 centers on direct input from ALS patients and their caregivers. It focuses on conducting focus groups to gather qualitative information about symptoms, functional status, day-to-day communication challenges, and perceived benefits and risks of cBCIs.

11) Why are patient and caregiver focus groups central to UH3?

The intent is to ensure future COAs reflect outcomes that matter in real life. Examples include being able to reliably express needs, participate in family decisions, communicate in emergencies, maintain social connection, reduce caregiver burden, and preserve autonomy.

12) How do UH2 and UH3 fit together?

UH2 provides the evidence and expert landscape (what has been measured and what gaps exist). UH3 adds lived-experience perspectives (what changes patients and caregivers consider meaningful). Together, the phases are designed to help prioritize which outcomes should be measured and how those outcomes should be framed so they are clinically meaningful.

13) What is meant by "clinical outcome assessments (COAs)" in this opportunity?

Within the context provided, COAs are the measures used to evaluate outcomes related to cBCIs in ALS. The opportunity emphasizes identifying what outcomes are currently being measured, what is missing, and what outcomes better capture functional benefits for people with profound communication impairments.

14) What kinds of outcomes or concepts might be considered in the measurement inventory?

The opportunity mentions outcomes such as communication speed, communication accuracy, usability, independence, and quality of life. It also highlights real-world functional outcomes valued by patients and caregivers, such as reliable expression of needs, emergency communication, social connection, reduced caregiver burden, and preserved autonomy.

15) What is the activity category for this funding?

The activity category is research and development (science and technology).

16) What is the award ceiling?

The opportunity lists an award ceiling of $500,000.

17) Which agency is sponsoring this opportunity?

The Food and Drug Administration (FDA) is the sponsoring agency.

18) What is the CFDA number associated with this program?

The CFDA number listed is 93.103.

19) When was the opportunity created and when did it close?

The opportunity was created on March 16, 2023, and the original closing date was May 24, 2023.

20) Who is eligible to apply?

Eligibility is broad and includes domestic and non-domestic entities. Eligible applicants include public and private institutions of higher education; nonprofit organizations with or without 501(c)(3) status; for-profit organizations (including small businesses); and various government and community-based entities (state, local, and tribal governments; U.S. territories or possessions; public housing authorities; independent school districts; regional organizations; and faith-based or community-based organizations). Foreign (non-U.S.) entities are also listed as eligible.

21) Are any types of institutions specifically encouraged?

Institutions of higher education are encouraged, including HBCUs, HSIs, TCCUs, Alaska Native and Native Hawaiian-serving institutions, and AANAPISIs.

22) What is the intended impact of the work supported by this award?

The work is intended to strengthen the measurement foundation needed to evaluate cBCIs for ALS in a way that regulators, clinicians, researchers, and technology developers can use, while keeping the focus on outcomes that reflect meaningful communication ability and real-world function for people living with advanced ALS.

23) What is the role of FDA in a cooperative agreement like this?

The announcement notes that FDA is expected to have substantial programmatic involvement compared with a typical grant. This is also why the project is milestone-driven and why transition from UH2 to UH3 is tied to progress against predefined milestones.

24) What does it mean that this is "milestone-driven"?

It means the application must include predefined milestones, and progress (including the ability to transition from UH2 to UH3) is dependent on meeting those milestones.

25) What population is the work primarily meant to benefit?

The focus is on people with ALS who use (or may use) communication BCIs, especially those with severe speech and communication limitations, along with their caregivers.