In an industry as heavily regulated as cell and gene therapies, gaining approval for a product can be a complicated and lengthy process. As a former regulator who helped develop European guidelines for cell therapy products, Christopher Bravery is in a better position than most to offer advice on achieving market authorisation.
Now director at Consulting on Advanced Biologics, Bravery has a number of concerns with how developers approach regulators, often due to the fact that “many advanced therapies have evolved out of academia by developers who have no prior experience”. This lack of experience can lead to misunderstandings of the process, particularly around the implications of what success at clinical trials actually means.
Bravery describes how “Clinical trials are assessed in a very different way to a market authorisation; they consider the specific group of patients that are recruited for the trial and they consider the risk to that small patient population versus the potential benefits. The clinical trial application is assessed primarily on safety, it takes no consideration of your actual development strategy, or whether you’re on track for that.”
Such issues are further amplified if there are only a small number of patients where it “might take 100 years” to collect enough clinical data to demonstrate safety and efficacy. However, for these cases Bravery highlights the fast-track system of conditional market authorisation, which can be granted to therapies for a particular disease that has no other treatment.
“Before the developers have collected the data set you might normally expect for market authorisation they can be given a conditional approval where they continue to collect the data. Because there is an unmet need there, it is desirable to get that to patients as quickly as possible.” Bravery explains.
Such schemes though shouldn’t be seen as shortcuts and still require huge amounts of preparation: “The thing you need to remember is that it is conditional on the clinical data, but not on the manufacturing and quality, which means you would need to fast-track that. You need to show you can make the product consistently so that you can place it on the market.”
Watch our exclusive video interview with Christopher Bravery above for much more advice on market authorisation.
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