Overview of Exploration Topic
Although Academic Medical Centers (AMCs) have developed many advanced therapies for rare and fatal diseases, access to AMCs remains limited due to outdated production methods and inadequate quality validation. To enhancing quality assurance technologies at AMCs, the Advanced Research Projects Agency for Health (ARPA-H) has initiated the Decentralized Engineering of Cells Informed by Dynamic Evidence (DECIDE) Exploration Topic (ET). Through DECIDE-ET, ARPA-H aims to improve access to curative cell therapies for pediatric patients with rare genetic diseases.
DECIDE-ET will optimize production, streamline regulatory approvals, and create sustainable pathways for therapies. It will address economic challenges in autologous cell therapy, which often faces high costs.
The initiative will develop technologies to assess production variability and establish quality criteria, enabling efficient regulatory processes. By aligning quality assurance with regulatory needs, ARPA-H aims to reduce costs and support effective treatments, partnering with the Food and Drug Administration (FDA), National Institute of Standards and Technology (NIST), and Centers for Medicare and Medicaid Services (CMS) for support.
Key Areas of Focus
ETs are short-duration, fast paced efforts. The DECIDE-ET is a 24-month, three-stage effort focused on a single Technical Area (TA). It aims to develop innovative approaches to identify and quantify production variability in decentralized, small-batch manufacturing of autologous cell therapies. The goal is to dynamically inform decision criteria for confidently assessing the quality of low-volume batches for rare therapies. The three stages are as follows:
- Stage I: Method Development
- Performers will simulate small-batch manufacturing to identify variability sources and develop statistical methods for risk-adjusted decision-making. After six months, an Independent Verification and Validation (IV&V) will assess these methods and the criteria for minimum batch quantities.
- Stage II: Testing and Prototyping
- Performers will identify unique signatures linked to variation causes and develop a signature library. They will also refine decision support tools for regulators to assess cell production quality. Fifteen months in, an IV&V will verify signature correlations and evaluate tool effectiveness based on FDA feedback.
- Stage III: Deployment and Validation
- Performers will apply variability signatures across different sites and contexts to validate their effectiveness in correcting production variations in subsequent batches. They will also test and validate decision support tools using real data sets for new cell therapies.
Proposals Should Address the Following
- Decentralized Small Batch Autologous Cell Production
- Variability Attribution
- Signature Identification
- Decision Support Tool
Important Deadlines
Proposal due date: November 18, 2024
If your company has considered applying for ARPA-H funding, your federal funding journey starts here.
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