When thinking about Regenerative Medicine, it is difficult to pin point where the industry sits on the learning curve.  While the science has made great strides, there are other things to think about such as manufacturing and figuring out how to make them accessable to patients across demographics.  We will not be able to fully take advantage of Regenerative Medicine therapies until we have the entire continuum vetted out. In this second installment of our Regenerative Medicine panel, our experts discuss their views on where we are on the learning curve.  Below is a condensed and edited version of Part 2 of a panel discussion moderated by Kevin Hampton, Chief Commercial Officer at Kineticos.

Panelists

  • Susan Nichols – CEO, Falcon Therapeutics
  • Flagg Flanagan – CEO & Chairman, DiscGenics 
  • Bob Hutchens – CEO, StemBioSys

Kineticos: We all know science is very complicated and complex. Where do you think we are on the learning curve? Is it another 5 years? 10 years? Do we fully understand things like efficacy, safety, etc.?

Flagg: CAR-T leads the way. I truly believe that, over time, we can cure cancer. Cancer is a very broad term, but the regenerative medicine field is doing amazing things with blood cancers with the CAR-T technology. From a cell therapy standpoint, we are just getting into the question about long-term safety, durability, and how these therapies work as a more curative opportunity. That is the key.

Also, we are at the beginning phases of exploring how we are going to pay for these potentially curative therapies. There is going to be thinking around how reimbursement works by taking a long-term view of the curative aspect and paying more money upfront for a better quality of life. Payers and developers alike are already thinking about that quite a bit and it is going to disrupt a lot of other therapies and modalities that have been heavily funded due to high capital intensity. I predict that technology will continue to evolve and that we will soon have more disruptive and curative options that will change the way we think about reimbursement for medicine and personalized care.

Kineticos: It’s tough for the public, the patient, and everyone involved to swallow some of these prices we’ve seen as of late. It will take some time and we will have to figure out what works and what doesn’t.

Bob: I want to pick up the point on the reimbursement. It is interesting because one of the challenges in the US is, we have a system that would seemingly rather pay for a therapy for 2-3 years towards the end of a patient’s life as opposed paying a premium to keep someone alive for 40-50 years.

You have to wonder if we’re going to have to do something on the payer side so that we can look at the lifetime cost as a society. It will be an interesting thing for the industry to contend with and makes you stop to think about other ways to do things.

Flagg: At DiscGenics, we’re developing a cell-based therapy for degenerative disc disease, a major cause of chronic low back pain, which costs more than $100B to treat per year in the U.S. alone. We believe we may have a curative solution in the mild to moderate phase that would address, and potentially arrest, disease progression before the rock rolls too far down the hill. If we’re successful, we could have a huge opportunity to take significant cost out of the system. We still could fail, of course, but we are on the cusp of really finding out what is going to work and what’s not going to work. We are already exploring generation 2 products. It really is a fascinating time and there are going to be a lot of winners and a lot of losers. We are in another 15 to 30-year transition in this whole area of curative products coming into the space. The regulatory bodies are trying to evolve to help, too.

Susan: The bar has been raised. As therapy companies, the FDA supplies guidelines to help us with our safety and toxicity profiles and articulating how our therapies will translate to human trials.  This is part of that evolution.  There is more competition for the capital out there and those companies who have good data packages are perceived as good investment targets thus high patient impact.

On the patient topic, the patient voice is stronger than ever these days. The single thing that motivates me most is looking at a patient who is practically destroyed by chemo. There has to be a better way and we have to work as a community. There will be failures and lots of learning, but the key is figuring out how can we modify and adjust best to serve patients. I want to see the day when somebody is diagnosed with cancer and you go get an off-the-shelf IV and your cancer is gone. That’s the day I’m waiting for.

Kineticos: I think you’re right, Susan.  The key is fail fast and move on to the next thing. Too many times, companies have a failed trial, see something in the data that might be encouraging, and they keep chasing something that isn’t really there.  That mentality, while I recognize it’s easy for me to say in my position, doesn’t do anything for these patients.


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