How can we get better, cheaper therapies from bench to bedside?

Vicki Seyfert-Margolis, Ph.D. former senior advisor for Science Innovation and Policy at the U.S Food and Drug Administration says trying to cure every orphan disease would consume the entire U.S. GDP. How can we lower drug and device development costs?


I think the regulatory agencies can use their platform to help inspire science and development of new tools that will provide better, safer, more effective, higher quality products, which is really what we're trying to do in terms of getting good therapies to patients.

The major barrier to entry in the market is the cost and the time that it takes to bring a discovery that's made in the laboratory actually to the clinic.

On average, right now, to bring a new drug to the market, it's probably somewhere between one and two billion dollars. If you've made a $2 billion investment in a new product, what do you have to price it at in order to get your return on investment?

If we were to try to support cures for all the orphaned diseases, it would consume the entire U.S. GDP. Clearly, we need to do a better job in driving down development costs so that we can drive down what these products are listed at in order to not completely break the healthcare system.

All of these different trends that are happening in the industry, the kind of merger and acquisition, the loss of capital, the direction that science is driving toward smaller markets, all of this is really converging to a point where this industry is under significant stress.

I think one of the things we need to do is think about establishing a national infrastructure for clinical trials. The other thing we need to think about is how we design smarter, I'll call them leaner, less expensive clinical trials that allow us to get just as much information about a therapy out of a much smaller trial.

We spend little to no effort on actually taking what knowledge we have, in terms of real world performance and things that we know about in prior studies, and integrating it back, or reverse engineering into the discovery or to the next generation product development.

Probably, most importantly, we don't include the patient and the consumer perspective. What are their opinions? What are their experiences, which are going to be critically important.

I think the academic medical centers are the drivers, or the engines of discovery and innovation, in terms of bringing basic fundamental mechanisms of knowledge of disease forward.

I think what academic medical centers could do, that might improve the situation, is take those findings a little bit further down that path of product development.

One of the problems, of course, is that they're not funded to do so. So the NIH isn't really funding product development, and that's something that I think, that the FDA is trying to change through its Regulatory Science Initiative, but it may necessitate other types of partnerships between academic institutions and private industries.