Shire has a reputation for being very patient centric, where it leverages extensive outreach to disease groups as well as
analytics around data detection, trial design, and indication profiling to secure timely registration and launch. Do you intend
to build on this as a driver of competitive differentiation?
Ornskov: Everyone at Shire has a story about patients—relating to them is part of our culture. In some ways, it comes with the turf:
you have to be patient centric if you are active in attention deficit hyperactivity disorder [ADHD], or in extremely rare
diseases, where our sales force gets to know many of the patients eligible to treat. I want to bolster this sentiment and
keep it as part of our own DNA.
From a strictly commercial standpoint, patients are important but in many ways the key link is to the physician. This is certainly
true in our clinical trial work, where physicians are the critical intermediary in the therapeutic segments where we are strong,
especially rare diseases. The close interactivity with clinicians is one factor behind our acquisition of SARcode Biosciences.
The company had a strong reputation for conducting excellent clinical trials based on real-world input from the most knowledgeable
practicing physicians. Again, I cite it: they were very customer-focused. As a physician myself, I could see their capacity
to make the connection, from the business to the physician to the patient.
How are you incorporating the new emphasis on payer value in your growth blueprint for Shire?
Ornskov: Value is the entry point for all discussions on P&R today. It is harder to obtain access to the market without a careful
consideration of cost-effectiveness, impact on overall health outcomes, and analysis of the various tradeoffs important to
stakeholders. With prices for many rare disease drugs so high, transparency is a given. At the same time, every negotiation
is different. The rarer the disease—and we have an estimated 7,000 such diseases where there are no available treatments—the
more important it is to get the value story right. Ultimately, that story has to be persuasive for the patient: does our medicine
have a significant impact on his or her quality of life? The industry has to demonstrate that its therapies are life altering.
Shire has many products that do precisely that, and the requirement on us is to furnish the evidence base to prove it. We
have to do it through better clinical trials but also in the many additional real-life observational studies we are committing
to as part of the registration process and through negotiations with the HTA authorities and payers.
As a European, what is your perspective on the growing reliance of governments on cost-effectiveness measures to control access
of new medicines to patients? Do the criteria being used in these evaluation systems adequately incorporate the special social
value characteristics of drugs for rare diseases, where Shire is strong?
Ornskov: I am in regular contact with governments and payer representatives in Europe. All countries in the region are grappling with
serious challenges to the sustainability of national health systems. There is no choice but to prioritize. It's fair for them
to seek to obtain maximum value for what they can spend. Our industry has to accept and work within that harsh budgetary assessment.
HTA is now part of the access process; it's another barrier to market entry that, through rigorous attention to evidence,
we can address and surmount. The onus is on Shire and all companies to provide the documentation needed to help payers make
the best judgment. Where Shire wants to play is at the highest level of innovation. Our goal is to be recognized as a company
that specializes in innovation and thus deserves a real opportunity to show a benefit to the health system. It is true that
for some of the very small patient populations we serve, it is difficult to make our arguments purely on the basis of cost-effectiveness.
We want to open payers to a broader discussion around issues like how to adapt insurance programs to serve rare disease patients
as well as solidarity and ethical concerns, which are very important in Europe. To varying degrees, we are succeeding in that.