FDA’s Expedited Programs for Serious Conditions
Do you think your product may fall into the category of drugs covered by this incentive program? Are you seeking expedited approval for an applicable drug product? We can help you achieve a successful interaction with FDA. To learn more about our services and capabilities, contact us today.
Helping You Get Your Product to Market Faster
Pharmaceutical and biotechnology companies seeking to utilize at least one of the four FDA expedited programs—fast track designation, accelerated approval pathway, priority review designation, and most recently breakthrough therapy designation —can leverage our experience and expertise to bring their products to the market faster while staying in compliance with regulatory requirements.
Breakthrough Development Programs May Put CMC/GMP Activities on Critical Path
Timelines for completing CMC/GMP activities for a breakthrough product will be driven by the design of the development program. Each development program can vary depending on the complexity of the product, how soon accelerated CMC development activities begin, availability of platform technology, relevant prior knowledge, and timing of designation. Therefore, development of breakthrough drugs necessitates risk-based approaches to product and process development, commercial readiness, and regulatory filings, with a focus on a reliable supply of quality product available to meet and sustain market demand.
MANUFACTURING CONSIDERATIONS FOR BREAKTHROUGH DRUG DEVELOPMENT
DSI can support all critical product and process characterization activities that should be addressed earlier and can help facilitate manufacturing readiness for breakthrough products. To learn more manufacturing considerations for expedited drug development programs, the following approaches could be considered for discussion and agreement with FDA.
Process and Formulation Development Considerations
Expedited clinical development programs for breakthrough therapy products will shorten the time available to optimize phase III and commercial manufacturing processes. This will necessitate prioritization of development efforts on process reliability over yield and cost of goods. As a result, process and formulation optimization may need to be deferred to post-approval; if it can be determined, there is no impact on patient safety or product availability.
Manufacturing Scale and Launch Site Considerations
Clinical manufacturing facilities, used for launch, would need to meet the same quality/GMP expectations as commercial manufacturing facilities. This will necessitate prioritization on gaining concurrence on comparability strategy/protocol for post-approval site changes in advance and may lend confidence to manufacturer’s ability to ensure sustained supply post-launch, particularly when expediting launch upon initial approval.
Process Validation Considerations
Process characterization/process validation (PC/PV) studies impacting patient safety must be complete prior to filing. In addition, sufficient process characterization data from clinical and pilot scale lots should be completed to assure process capability and reliability for providing commercial product supply at launch until further PC/PV activities are completed.
Analytical Development Considerations
Analytical Method Development strategies for front-loading of analytical understanding to balance more limited process robustness and support future comparability exercises. Focused on high priority assays, including but not limited to potency for biologics and content, impurities, and dissolution for small molecules to ensure suitability for control system.
Control Strategy Considerations
Control Strategy, based on limited manufacturing experience, but ensuring patient safety and efficacy. Launching with a provisional control system that ensures consistent product and upgrading the control system post approval with more manufacturing experience and completion of process validation.
Stability Data Considerations
Accelerated development timelines may limit availability of real-time stability data, thus launching with reduced real- time stability for commercial material may require. Leverage stability from early development and clinical batches when formulation remains unchanged and product comparability is demonstrated.
Quality System Alignment with BT Product Development Considerations
Quality System requirements must be adhered to for breakthrough product development while providing appropriate flexibility to accommodate accelerated activities for breakthrough product development timelines. Thus, the accelerated development Quality System strategy for each product will be unique, as it depends on the timing of the BT designation.