The pharma game needs to change. Big Pharma shaped the way drugs are developed, regulated, manufactured, and brought to market. This needs to change. As the biotech scene becomes more and more specialised (whether in regenerative medicine, orphan indications etc.) the current pharmaceutical framework only constrains the industry further.
This is the message that rings out from World Stem Cells and Regenerative Medicine Congress 2014. If stem cell and regenerative products are to successfully make it to market AND become integrated into healthcare systems across the world, the stem cells industry needs to affect change within the wider pharmaceutical framework.
If you build it will they come? That was the title of Michael May’s presentation on day two, and it’s a question that has sparked debate throughout the conference. The reason is because cell therapies are expensive. At least, the sorts of targeted cell therapies that are in development are, and this raises big concerns. Many cell therapies combat common diseases and conditions, yet hold price tags in the hundreds of thousands of dollars. Whilst orphan indications can get away with the big price tags because of small populations, treating just UK heart attack patients with a cell therapy over one year could cost $10,000,000.
What is the chance that payers will buy into regenerative medicine at this price? It is low. With increasing number of orphan drugs, specialised medicines, and expensive cancer treatments, payers and healthcare systems are already pushing back.
Developers need to think long and hard about the potential for reimbursement for their products and they need to do this think as soon as possible. If they cannot get their price point down what can they do to make the product more appealing to payers?
Firstly, talk to regulators. As many regulators as you possibly can. As Sven Kili from Sanofi put it, ‘talk to as many regulators as you can, not just the one you think will give you the hardest time’. The regulations surrounding cell therapies vary across the world and talking to just one regulator, who may well have very stringent conditions, will not cover you for ever regulatory system.
But as well as learning how to pass a particular set of regulations, talking with regulators will allow you to design better, and more realistic clinical trials with end points that are more representative of your product.
Leading the world in the regulation of cell therapies is Japan, and this could well be because of the good relationship between the Japanese regenerative medicine industry and their regulators. The new regulations, due to be implemented in November 2014, will reduce the cost of clinical trials, increase market exclusivity, allow products to enter the market before the completion of a full phase 3 clinical trial. As Dr. Narayana put it: ‘check safety, assume efficacy’. All of this will decrease risk and no doubt dramatically increase the levels of investment in regenerative medicine in Japan, and across the world.
Secondly, comply at all times. ‘We don’t want to end up with the tarnished reputation pharma sometimes has, so we must be compliant at all times’.
On a related note to both reimbursement and regulation, in the interests of improving the effectiveness of your treatments and defining better clinical endpoints, developers need to look at identifying non-responders. BigPharma has avoided research into the identification of non-responders purely because, if 30% of patients do not respond to traditional medications, if the technology to identify these non-responders were available they would lose 30% of their market. For cell therapy developers though, the constant narrowing of their patient pool and the increasing efficiency of their product will increase the worth of their product to payers.
Collaboration, modularity, and flexibility. These are the principles cell therapy biotechs should built on if they want to successful make the transition from development through to commercialisation. Throughout the conference we have heard about how Biotechs should avoid the traditional pharma model and move towards a more flexible and collaborative model. As such we have heard from the likes of Cell therapy Catapult and Chemelot who are offering regenerative developers a modular set of services that developers can simply ‘plug into’ as and when they need. As Robert Preti from the CMO Progenitor cell therapy explained during Thursday’s morning session, it can cost up to 3 times as much, and take twice as long, to get therapies from research to market if you don’t have the correct infrastructure to begin with.