Over the two decades you have served as OHE director, what changes have you noticed in the way Big Pharma operates?
Towse: One key transition is the move away from the randomized clinical trial as the sole benchmark for establishing clinical value.
The structure of the RCT often discounts important sources of information, skewing the way we look at some diseases and their
treatments. The OHE has been a leader in advocating increased use of observational studies as a supplement to the RCT. Our
work has helped payers and the industry evolve toward greater acceptance of diversity in the evidence base. The change is
profound, as observational studies center decisions in the real world, where the patient experience is paramount. Better medicines
are the result.
Another change—born of necessity—is the time and effort companies must devote to the conduct of studies post-launch. Such
studies are often required today as a condition for regulatory approval, and involve large pools of patients. They can be
both costly and time consuming—even extending beyond the life of the patent. As noted, our research strives to identify how
these commitments affect the R&D enterprise, particularly in making sure the study process works as efficiently as possible.
We are also examining the revenue and resource impact of certain regulatory changes that will accelerate the take up of post-marketing
commitments, such as the European Medicines Agency's proposal on "adaptive" licensing, which entails periodic review of clinical
efficacy over the life of a product.
Can you provide an example of how OHE research has prompted a shift in thinking on the role that medicine plays in health
financing and/or delivery?
Towse: OHE did much of the early work in defining and then explaining the positive economics of personalized medicine. By asserting
that the value of a drug intervention is enhanced if it can be targeted more to the individual profile of the patients, personalized
medicine represents a revolutionary break with the random, "hit or miss" approach to treatment. We led the field in showing
that a higher price for a medicine in a smaller market is an acceptable trade off because the outcome for patients tends to
be better. Our current work in this area goes even further by proving that additional value is obtained when a single medicine
can be priced differentially based on therapeutic indication. A good example can be found in cancer treatments like Herceptin,
whose value when used in early stages of the disease differs than when it is used after the cancer has advanced. In fact,
you can make the same rationale for many of the innovative biologics now entering the market, one where the industry has staked
its future. Making drug budgeters more aware of these important distinctions will, in my view, be of great service to innovative
companies and to patients.
In such a tightly regulated sector, business success in pharma ultimately depends on good policy. As an observer of the industry
for many years, how effective do you think it is in making its case to stakeholders?
Towse: I have always believed that this industry has to respect science and eschew polemics—as a research-driven enterprise, the
preference is to try to engage in policy debates on the basis of good evidence. The problem is that much of what companies
do is built around the mindset of the quarterly P&E report—it's all about the short term, and the policy model tends to reflect
that. If you insist on compiling facts and analysis today for a problem that needs solving by tomorrow, the end result is
going to be ammunition, not evidence. It strikes me that very few people in the industry have the time or inclination to make
that investment in understanding what's really over the horizon. And there is so much personnel turnover in companies today
that institutional memory, which consists of integrating the lessons of the past against an uncertain future, is not a skill
that can easily be found.