Market Authorization is the holy grail for the cell therapy sector, with pressure to achieve it as quickly and cheaply as possible. In a complicated field, with significant amounts of data and information, it’s a huge challenge to meet the requirements of regulators. Even more so with rare diseases and few patients. Christopher Bravery, Director of Consulting on Advanced Biologicals, and an ex-regulator himself, talks us through how regulators are putting systems in place to accelerate this process:
“If there’s a small number of patients that have a particular disease, where there’s no other therapy, then it is possible to either get a conditional, or in some cases, an ‘exceptional circumstances’ approval. This means that we could collect enough clinical data to demonstrate safety and efficacy, but that might take a hundred years because there’s almost no patients. So that was the example when Glybera was approved. That’s a very ultra-orphaned indication and so it would never have been possible for them to collect enough data in a reasonable timeframe.”
“More common now is the use of conditional marketing authorization. The situation there is that you’ve been able to collect some data, which is showing a good safety and efficacy signal, and therefore because there is an unmet need, it would be desirable to get that to patients as quickly as possible. So before they’ve collected the sort of data that you might normally expect from market data authorization, they can be given a conditional approval while they continue to collect the data.”
The industry watched with interest when South Korea, followed by Japan two years ago, tuned their regulatory infrastructures to allow this to happen. Japan passed a new law where the regulatory pathway for commercial cellular products was significantly accelerated by skipping of typical “Phase III trials”. Conditional market authorization was allowed, after demonstration of safety and some signs of efficacy. That law has yielded it’s first results with two cell products approved on the Japanese market in September 2015.
Important to remember with conditional market authorizations is that they are just that: conditional. In the years that follow market authorization, developers will be obligated to provide clinical data on efficacy and only if there is strong data will the product remain on the market.
Christopher Bravery also points out: “the thing that you need to remember if you take that route is it’s conditional on the clinical data, but not on the manufacturing and quality. That means you will need to fast track that because you need to be ready to show you can make the product consistently so that you can place it on the market. So if you’re considering that approach, you must remember that you’ll need to provide extra resources to fast track your manufacturing development.’
Christopher does see enormous potential in the fast-track approach: “I think the types of product we’re going to see, trying that approach very soon are these chimeric antigen receptor CAR T-Cell products which are showing outstanding efficacy in clinical trials. And there’s a race between a number of large players to be the first to get those submitted and approved.”