Professor Mark Lowdell, Professor of Cell & Tissue Therapy at UCL in London, presented on pre-clinical and phase I trial considerations to prevent failure at marketing authorization approval as part of Cell & Gene Therapy Manufacturing Digital Week. Watch the full webinar presentation, as well as seven other on-demand webinars, here.
As ISCT European Vice President, and Director of Cellular Therapy at the Royal Free London NHS Foundation Trust, Prof. Lowdell has an overview of the ATMP industry from multiple angles.
He begins his presentation by questioning the perceived paucity of licensed ATMPs worldwide, pointing out that the rate of commercialisation is actually higher than the historic rate of mAbs. However, though 'the rate of ATMPs is perfectly acceptable, we should still seek to speed that up further'.
He explains how there are a number of partners involved in developing an ATMP – something he visualises with the ‘virtuous cirlce’. He goes on to explain how ATMP stakeholders are all interdependent:
- Clinician wants to innovate treatments and NEEDS patients
- University NEEDS high impact publication and IP but not to fund phase III trials for MA
- Industry NEEDS clinician to innovate and University to support proof-of-principle
- University NEEDS Industry to buy its IP and commercialise it after clinical trials
- Governments NEED to ensure patient safety and maximum availability of novel therapies and commercialisation
- Patients need access to new, safe and effective treatments from Clinicians AND Industry
Prof Lowdell goes on to explore what considerations are key in early phase trails (I-II) to guarantee success. He emphasizes: ‘The first question you have to ask yourself as a drug developer or an academic developing these therapies is: “what’s your goal?”. Do you want a high impact paper then to walk away or to change clinical practice by delivering a therapy to be made available to the global population that needs it?’
After this, a whole host of questions need to be answered: How many patients would need to be treated annually to change clinical practice? How could the logistics for your therapy work? Hub and spoke manufacture? How much would your therapy be worth? How could you make it at a COG which was acceptable? Can you design a cheaper alternative?
Prof. Lowdell goes on to explore planning a strategy for procurement of starting materials, when to close the process and how to deliver the therapy - including labelling, packaging and supply chain.
CLICK HERE to watch the full 50-minute presentation, as well as seven other on-demand webinars from the 2017 Cell & Gene Therapy Manufacturing Digital Week.