Understanding the Drug Developer's Perception of Value

Bruce A. Feinberg, DO: There was a point in time where all of this driving was just to make an impact—just to help patients overcome suffering and live longer. We’re now at a point where we have cures. We have diseases which convert into chronic disease and we have therapies that can be administered with much less suffering. I think you get to a point where, as in a specific disease like testicular cancer, we can say, “Maybe we don’t need 6 cycles. Maybe we can actually give 4 or give 3.”

I think that, in a way, that value story may be a bit of an evolution. It was almost impossible to go down that road when all you’re doing is striving for some improvement in people who are suffering. Now, maybe, we’ve gotten to a point where there’s enough improvement that we can start to look at this.

Now we’re at a point where you look at the NCCN [National Comprehensive Cancer Network] Guidelines and see 16 different regimens listed for node positive HER-2-negative breast cancer. And you’ve got to ask yourself, “Can that much variance be a good thing?”

So, Ira, you’ve lived for years on that payer side, and now you’re on the pharmaceutical side. It’s a learning piece for you. Maybe you’re understanding a new perspective.

Ira Klein, MD, MBA, FACP: Yes. When I think about value in the pharmaceutical industry, I think of, “Can we define what we’re talking about?” I work in the brand space. Innovative medicines come from preclinical; through phase 1, 2, and 3; to FDA approval. Then, post market surveillance after that. So, there’s value on the generic side, but I’m not an expert on that.

If you look at other disease states and populations—for example, if you look at cardiovascular disease, death rates have gone down by half, in the past 20 years, and the therapy is pennies per day. We tend to forget about that. But, working in the brand space, I think about parsing this between, “What about introducing a new drug in a therapeutic class where there are multiple other drugs?” and, “Are we doing a lot for society?” I think about parsing that versus trying to explore new mechanisms, or greatly improved mechanisms of managing disease to get to what you said before, which is cure or amelioration of disease and the ability to translate that disease into a chronic disease.

I now look at how brand companies can think about putting drugs into the innovative space. There are some issues there because we’re talking about a point where we want to investigate this. So, are we going to investigate this using the tools we’ve used in the past? The tools we’ve used in the past are the ones that have been handed to us by the FDA that look at safety and efficacy. So, the tools of overall survival, progression free survival, and time to progression—those things sometimes work, but in breakthrough drugs may not be so accurate. Then, there are some surrogate measures that just don’t work at all.

I think we have to get better at the tools. What are the rulers? I would argue that in breakthrough treatments, sometimes, we don’t have overall survival data because the drug is too good and it’s too far in the future. That’s a good problem to have, but it’s a tough problem as we wrestle with the overall problem of the cost of drugs and how that nests within the total cost of care. We just don’t have a lot of data. The other thing is that we don’t have a lot of great data exchange.

I do think that this new paradigm that Mike talked about will hopefully include people being transparent not just about some aspects of how they do their business, but also about data sharing. We’ve got data that is locked up in multiple vaults that I think is not benefiting society because we’re not sharing it. That is a way to reduce costs.