Clearly, the drug development process is under significant stress. Vicki Seyfert-Margolis, Ph.D. former Senior Advisor for Science Innovation and Policy at the U.S Food and Drug Administration, recently visited the University of Utah Health Sciences to talk about some of the most significant challenges facing drug development and offer some possible solutions.
Regulation and innovation are sometimes thought of as diametrically opposed. How can regulation promote and not constrain innovation?
There are certain criteria that have to be met in a regulatory environment and some people may think of those as barriers. However if you didn’t meet those criteria for safety, efficacy, potency, and quality, most likely you would have significant problems in the market. Regulatory agencies can use their platform to help inspire science and development of new tools because we have a deep understanding of where things are working and where they aren’t.
What are the barriers constraining innovation in drug development?
The major barrier to entry is the cost and the time that it takes to bring a discovery that’s made in a laboratory to the clinic
Why doesn’t the traditional drug development model fit today’s science?
Right now, in a post-human genome era, science is driving toward targeted therapy development, so you have the right drug for the right population or person. The industry is going through a transition to developing these kinds of drugs, but the science and understanding the underlying mechanisms of disease are still big challenges. It still costs an enormous amount – $1 billion to $2 billion – to bring a drug to market, and companies need to recoup their investment in a niche market, which is where science is happening. That’s why we’re seeing increasing prices in pharmaceutical products and medical devices. Some of the new and innovative drugs coming on the market are very expensive, more than $100,000 per course per patient. (Kalydeco, a new drug for cystic fibrosis, costs around $290,000 a year and has to be taken lifelong). If we were to try to support cures for all the orphan diseases, it would consume the entire U.S. GDP. Clearly we need to do a better job in driving down development costs in order to not completely break the health care system.
You talk about the prohibitively high cost of clinical trials. How can we be smarter about designing them?
We need to think about establishing a national infrastructure for clinical trials. Right now, every time we do a clinical trial we recertify and restart a site. We need to develop a system that allows for enrollment of multiple clinical trials at sites certified for specific disease areas as opposed to individual clinical trials. The other thing we need to think about is how we design smarter, leaner, less expensive clinical trials using patient stratification and enrichment strategies that allow us to get just as much information about a therapy out of a much smaller trial.
Where does development break down from bench to bedside?
I think one of the major challenges right now in doing smart drug development is our inability to connect our understanding of genetics and biology that we’re developing in the labs with the actual point-of-care definition of disease. There is a 90-95 percent failure rate from the time a discovery is made in the lab to translation into a commercially viable product. Until we can connect the dots between what's going on in real-world treatment and what we thought we knew about a drug, we’re not going to be successful in developing drugs that actually impact patient care.
You talk about reverse engineering the system. What do you mean by that?
Currently, we start with discovery and then we try to extrapolate what’s going to happen in the real world with the products we develop. We spend little to no effort on taking what we know from clinical trials and real world performance and integrating—or reverse engineering— that knowledge back into the discovery or to next generation product development. I think we need to flip how we think about science and medical product development so that we are not just looking at what’s happening in a cell but we’re incorporating the end game data.
What is the primary role of consumers and why is it important?
Patients are treated as subjects instead of co-researchers, and because of that very little consumer opinion goes into drug development. We never ask patients what they think about a drug or what’s actually happening in the real world as they use these products. Our willingness to consider their reported outcomes is critically important for developing better, next-generation products that actually work for patients.*
Talk about the importance of “unlocking data” and the potential that has for innovation.
All of our data is pretty much under lock and key. We need to unlock that data and share our failures with one another to prevent hundreds of millions of dollars from being wasted. And we need to be smarter about integrating and leveraging the knowledge we already have. For example, if 12 companies are looking at the same molecule, we need to create a space for those scientists to work together.
What do researchers need to do right now?
It’s an exciting time and there are lots of opportunities to develop tools, software, and algorithms. The doors to opportunities are cracked, and it's your job to start to push them open. It will require a lot of good thought and science and risk-taking. Without taking a risk, we're not going to get there.
*To see how the University of Utah is reverse engineering consumer input back into research, check out the Pharmacotherapy Outcomes Research Center.
Amy Albo is the Publisher for University of Utah Health Sciences.