The growth in the regenerative medicine market has exploded with over a thousand new products in development and numerous product approvals are in place and more on the horizon. Center Director, Dr Peter Marks M.D., PhD (Oct 2019) has stated that in 2019 there will be a 50% growth in the number of clinical trials and that there will be over 40-60 new product launches by 2030.
This rapidly growing pace of development and commercialization is out of the normal development and commercial timelines. The downstream processes for scaling and adopting these new therapies has been slow to develop and gaps are appearing which could derail their development and commercial success.
One of the areas that could benefit from both standardization and adopting new technologies is downstream commercialization, i.e. logistics, storage, quality and pharmaco-vigilance systems. Most products will need to be cryogenically preserved, shipped, stored, thawed, administered and then stored for quality, PV and legal considerations.
Numerous protocols are being developed that all differ in time, material and scale with regards to handling, especially at the point of care facility.
The question of liability is paramount and how best to track, receive, deliver and store the products is of concern, with no universal standardized SOPs in place for many of these activities.
Shipping and tracking
- How is the shipment container tracked and validated?
- Was the container sterilized properly, has it been cross-contaminated or simply 'off specifications'? How can this easily be determined at the POC?
- What happens if the bag is damaged during shipment?
- How is the product tracked and monitored and are there mitigation plans in place should there be an accident and/or claim?
Processing at point of care
- What is the protocol for removing the product from the container, handling it and subsequently delivering it to the patient?
- More specifically, how do you properly thaw and track the product prior to delivery to the patient?
Role of the hospital, pharmacy and BMT processing labs
- Who is responsible for product storage, thawing, washing or other procedures at the hospital/POC?
- To date pharmacies have not been involved with cell products and do not want to be involved as they do not have the equipment or training. What would be the best work-around for this in the future? How would these work-arounds differ in various parts of the world?
- Will there be a need for the development of cellular pharmacies to enable delivery of cell products more universally?
- How do you administer the product to the patient and is the product complete with delivery apparatus?
- Who is storing a sample of the product that has been delivered to the patient, and for how long and what is the process?
- What system is being used to track patient outcomes and how are these data tied to the sample lots?
- Should the POC facility become the holding and storage facility for these samples?
- Should the POC facility become the short-term storage depot for multiple products on a scaling magnitude and build out cryo freezers in their pharmacies?
- Or should the process be a just in time model but with advanced scheduling and tracking systems that are integrated into the POC and biomanufacturer?
- What about the future of home delivery systems and healthcare providers once the cytokine storm issue is resolved?
Target of workshop session
- Identify the critical success factors in the downstream finished product commercialization process for advanced therapies:
- (cryogenic) storage aspects
- point-of-care processing and delivery to patient
- Identify the key critical success factors for scaling and standardizing processes into specific categories
- Break down the broad specific categories further with issues still to be resolved
- Establish broad recommendations to develop the solutions for each of these categories and who best to approach these issues
Who should attend?
- Hospital staff
- Therapeutic biotechs
- Supply chain and logistics vendors
- Standards agencies
Phacilitate is currently undertaking a scoping exercise to understand the optimal route of progression for this initiative. We are interested in hearing from any stakeholder with an interest in the development of such standards, and who may or may not wish to participate in a working group at Phacilitate Leaders World in January.