Closed information loops
Sanjay Shah, Optimal Strategix Group: There is some way to go before we have truly integrated care in the United States. This is because actions by all parties—providers,
payers, and manufacturers—remain driven by disjointed financial incentives and misalignment on assumed risk that are hard
to reverse. Europe is further ahead, having embraced payment schemes that are built around performance and outcomes, expressed
via the accumulation of evidence of comparative effectiveness (which differs from efficacy established in well-controlled
clinical trials) through post-approval observational studies, in the real world setting. It is possible that policy pressures
in the expensive, high- profile specialty biologics segment might accelerate a similar transition here.
Barnett: Payers we interviewed will be demanding progressively higher substantiation of clinical effectiveness for these biologics.
They admit, however, that creating workable criteria to demonstrate value—criteria that are mutually acceptable to payers
and manufacturers—is still a work in progress. Support is strong on a conceptual level but the goal now is to move from that
to something that you can actually measure. If by uniformity you mean the United States will evolve toward a single federal
standard of cost effectiveness, I suspect this idea will be fiercely resisted, not just on methodological grounds but because
of the politics.
Kara Clinton, Eli Lilly & Co.: There is substantial discussion on how to manage and reduce costs in healthcare. Information and data will continue to be
critical as we examine disease and treatment costs holistically. Pharmaceutical prices are relatively transparent and have
been a major focus of reducing costs. However, drugs are often not the major driver of the expense of treating a patient.
To reduce healthcare costs, stakeholders should start with accurately measuring and comparing all costs with improved transparency
and data. It is also important to capture cost offsets and savings in different treatment options. What does it cost to treat
an individual throughout the full course of a disease? This is the key principle in aligning market access with treatment
outcomes, in an affordable way.
Joshua Parks, Valeant: The situation in Europe today suggests it may be naïve to assume that better information combined with the right clinical
profile will result in patient access to a new drug. The patient population is increasingly diverse, so it follows that there
is an equally diverse range of opinion on what constitutes improvement in the standard of care. All the industry's computer
models will have little influence on that determination, which most often ends up being, "we just can't pay for it." This
is the direction European countries are taking, and it will be interesting to see where that leaves the European-based industry
three to five years from now. Precedent shows that there will be less innovation and slower uptake of newer drugs.
Don't we need to start with an agreed definition among payers and manufacturers about what the concept of "effectiveness"
really means? Does it apply to clinical standards only or does it incorporate cost?
Smeeding: It carries a very broad scope. Some of the larger provider and managed care organizations have evolved their own internal
definitions. Efforts by the networked academic organizations like ISPOR have also brought the various interests together,
which is critical as the mechanics of effectiveness evaluations must make sense to the insurance providers first, because
they are the ones who pay the bill. But drug makers will confront price resistance, regardless of the degree of consensus
on standards. It's always been a contest of wills to wrest a good price from a payer, and I don't expect that to change very
Sanjiv Sharma, Duchesnay USA: Each provider wants to design their own cost effectiveness model because everyone in the business claims their covered patient
base is unique. The practical implication is a drug manufacturer is forced to do endless variations of the same thing to accommodate
Brendan Bertsch, Optimer Pharmaceuticals: In the United States, no standard for effectiveness can include a frank assessment of cost. Only clinical applications are
permissible; anything beyond that entails endorsement of rationed care, symbolized by the "death panels" that are supposed
to be implicit in the Obamacare legislation. European precedents don't travel well here.
Michael McLellan, Pfizer: Criteria for effectiveness in Europe seems to focus predominantly on cost, so much that one might suspect that in some countries,
the institutionalized process known as health technology assessment [HTA] is more of a political tool masquerading as an objective
evidentiary standard. It provides an opportunity for payers to tactically create negative assessments prior to negotiating
a reimbursement price. The lack of objective and scientific benefit assessments becomes clear when a manufacturer can submit
the same dossier of evidence for the same drug to HTA authorities in a number of countries and get a very different conclusion
in each one. It would be unfortunate if these assessments amounted to no more than a clever way to manage a fixed budget for
medicines, instead of identifying true innovations that benefit patients.
The situation could only change if the agency doing the benefit assessment was not so closely tied to the payer negotiating
reimbursement. There are efforts underway to create a pan-European assessment process on relative effectiveness. Opinions
differ, but my view is that this is unlikely to improve the current problem of HTA bias and lack of objectivity, in a way
that rewards drugs addressing unmet medical needs. That's because all reimbursement negotiations are inherently political;
payers in countries controlling access to new drugs are unlikely to relinquish direct control over the HTA process. The group
coordinating the pan-European HTA assessment pilot, the European Network for Health Technology Assessment [EUnetHTA], consists
of representatives of the national HTA agencies. You can't expect them to support any significant changes to the status quo
in their home countries, as it would adversely impact their prerogatives as the gatekeepers on patient access.
Sharma: Throughout my career, I've been closely involved in negotiations on clinical and cost effectiveness standards, in Canada as
well as the United States. My experience leads me to conclude that the only way industry can avoid making this process a narrow
budgetary and financial calculation is to engage the patient. Putting forward an articulate patient perspective is unsettling
to the payer—which actually confirms we are doing the right thing. Patient involvement tilts the discussion away from a focus
that is exclusively about the cost of therapy.