The Advanced Research Projects Agency for Health (ARPA-H) has introduced the Decentralized Engineering of Cells Informed by Dynamic Evidence (DECIDE) Exploration Topic (ET), representing a crucial initiative designed to revolutionize the production and validation of autologous cell therapies. This ET targets significant enhancements in the production processes at Academic Medical Centers (AMCs), focusing on pediatric patients with rare genetic diseases. Below, we delve deeper into the technical requirements and program structure to aid potential applicants in preparing comprehensive proposals.
Overview of DECIDE ET
The DECIDE ET focuses on enabling pediatric patients with rare genetic diseases to access clinically validated curative cell therapies. The goal is to innovate quality assurance technologies and establish novel pathways at Academic Medical Centers (AMCs) that can right-size cell therapy production, ensuring these transformative treatments are both accessible and affordable. Key technical areas include:
- Variability Identification and Control:
- Objective: Develop and implement technologies to precisely identify, quantify, and control production variability in the manufacturing of small batch cell therapies.
- Challenge Addressed: Reducing the variability inherent in small-scale productions that can affect the consistency and efficacy of cell therapies.
- Technical Goals: Creation of sensor and monitoring systems that provide real-time data on production processes, enabling immediate adjustments to maintain product quality.
- Quality Assurance and Process Optimization:
- Objective: Innovate scalable and transferable quality assurance methods that align with current Good Manufacturing Practices (GMP) tailored for small batch processes.
- Challenge Addressed: Establishing robust quality controls that can adapt to the unique demands of small batch production without the economies of scale available to larger operations.
- Technical Goals: Development of modular quality assurance systems that can be integrated into existing processes to ensure product integrity and patient safety.
- Regulatory and Reimbursement Framework Development:
- Objective: Create automated systems for evidence collection and analysis that support regulatory submissions and reimbursement pathway development.
- Challenge Addressed: Streamlining the pathway for regulatory approval and reimbursement, which are often cumbersome and slow, particularly for novel therapies.
- Technical Goals: Implementing blockchain or similar technologies to securely and transparently track and verify data throughout the production and testing phases.
Program Structure
The DECIDE ET is structured as a 24-month program divided into three stages, focusing on method development, testing and prototyping, and deployment and validation.
- Stage 1: Method Development
- Focuses on simulating and analyzing small batch manufacturing processes to identify key sources of variability.
- Key deliverables include developing statistical models that support risk-adjusted decision-making scalable to commercial production levels.
- Stage 2: Testing and Prototyping
- Develops technologies and processes identified in Stage 1, creating prototypes that can be tested in real-world settings.
- Key deliverables include the creation of a signature library that correlates specific variability signatures with their causes, enhancing the predictability of the production process.
- Stage 3: Deployment and Validation
- Applies the validated methodologies across different production settings to ensure that they are robust and scalable.
Key deliverables focus on integrating these methodologies into regular production lines and demonstrating their effectiveness in maintaining product quality across multiple batches and sites.
Collaborative and Regulatory Partnerships:
A critical component of the DECIDE ET is its collaboration with regulatory bodies like the FDA and partnership with academic and industry stakeholders to ensure the developed technologies meet regulatory standards and are commercially viable.
Key Dates and Submission Details
- Draft Module Release Date: August 20, 2024
- Questions & Answers Deadline: October 4, 2024
- Proposal Due Date: October 22, 2024
The DECIDE ET draft solicitation offers a unique opportunity for researchers and institutions to contribute to groundbreaking advancements in cell therapy production. By addressing both technical and systemic challenges, ARPA-H aims to catalyze innovations that will bring life-saving treatments to children with rare diseases more quickly and cost-effectively. Prospective applicants are encouraged to thoroughly review the full solicitation details once they are released and prepare their proposals to align with the ambitious goals of this initiative. As this is a draft solicitation, a final version will be released soon, which should provide further clarity and potentially additional requirements or opportunities.
If your company has considered applying for ARPA-H funding, your federal funding journey starts here.
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