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SESSION SUMMARY

“WHAT EVIDENCE DO PAYERS NEED?”


NPC’S ANNUAL FORUM
NOVEMBER 2008

Prepared by:

Rebecca Singer Cohen, MPP


Bryan R. Luce, PhD, MBA

June 28, 2009


2008 NPC Annual Forum Session Summary, “What Evidence do Payers Need?”

TABLE OF CONTENTS

INTRODUCTION ........................................................................................................................................... 3
Brian Sweet, BS Pharm, MBA, Chief Pharmacy Officer, WellPoint ........................................................... 3
Tamara Syrek Jensen, Deputy Director for the Coverage and Analysis Group at the Centers for
Medicare and Medicaid Services (CMS) ................................................................................................... 3
Dr. Alberto Colombi, MD, MPH, Corporate Medical Director, PPG Industries ........................................... 3

THEMES AND KEY POINTS ........................................................................................................................ 4

CONCLUSION .............................................................................................................................................. 8

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2008 NPC Annual Forum Session Summary, “What Evidence do Payers Need?”

INTRODUCTION
On November 14, 2008, the National Pharmaceutical Council convened its Annual Forum,
during which Dr. Bryan Luce of United BioSource Corporation moderated a panel session
entitled, “What Evidence to Payers Need?” Speakers on the panel included:
Brian Sweet, BS Pharm, MBA, Chief Pharmacy Officer, WellPoint
Mr. Sweet focused his presentation on two core evidence functions at WellPoint: WellPoint’s
outcomes-based formulary process and newly revised health technology guidelines.
Tamara Syrek Jensen, Deputy Director for the Coverage and Analysis Group at the Centers for
Medicare and Medicaid Services (CMS)
Ms. Jensen focused her presentation on CMS’ ability to integrate evidence into decision
making and statutory barriers they face when trying to collaborate with non-governmental
organizations.
Dr. Alberto Colombi, MD, MPH, Corporate Medical Director, PPG Industries
Dr. Colombi focused his presentation on the efforts PPG has made to try to demonstrate the
value of improving a workplace health culture and the savings his company has seen as a
result.
The panelists represented crucial stakeholders integrally involved as “consumers” of evidence,
including the private sector, public sector, and an employer, respectively. The panelists were
asked to speak about the evidence they need and use when making policy decisions, their efforts
to improve the health outcomes of the policy decisions they make, and overarching issues and/or
barriers that may exist that prevent the development or implementation of such evidence.
This session summary document reviews the evidence-based themes of all of the presentations
and discusses relevant policy issues. Three primary themes emerged from the session:
1. All three sector representatives valued an increased focus on patient-related or
communicated outcomes, rather than just clinical markers. Each considered patients to be
important stakeholders that are incredibly influential when it comes to issues such as
adherence and motivation, as well as outcomes that directly affect their health-related
quality of life.

2. Policy decision-makers want effectiveness information, not just safety and efficacy
information. Specifically, they want evidence that the decisions made are the appropriate
decisions in their constituent population, and are based on real-world, comparative
effectiveness data.

3. Formulary decision guidelines should be transparent so that data and evidence


expectations are clear and sponsors can work on providing the most desired data.

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2008 NPC Annual Forum Session Summary, “What Evidence do Payers Need?”

Each of these themes is discussed in more detail below within the context of the presentations
made during the session.

THEMES AND KEY POINTS


1. All three sector representatives valued an increased focus on patient-related or
communicated outcomes, rather than just clinical markers. Each considered patients to be
important stakeholders who are incredibly influential when it comes to their issues such as
adherence and motivation, as well as outcomes that directly affect their health related quality
of life.

WellPoint actively seeks out data points and markers that are directly relevant to patients,
such as health-related quality of life data, rather than the usual clinical intermediate markers
(e.g., lowering blood pressure or LDL markers). As long as the studies are well-designed and the
instruments are validated, patient-reported outcomes are highly desired and crucial to
understanding what a patient is thinking and feeling. As opposed to information typically found
in AMCP dossiers, the focus at WellPoint is on clinical evaluations and systematic reviews that
deliver a picture of real world outcomes, modeling/budget/systems impacts, and medical cost
offsets.
PPG began to measure health performance as a business performance and implement
prevention and disease management programs. As an employer, PPG has seen the same
systematic health care issues that other sectors bemoan—misuse, overuse, under use—and
wanted to find evidence that would demonstrate what is efficacious, effective, and efficient.
According to Dr. Colombi, PPG has learned the importance of evidence when making policy
decisions, how to collect evidence that answers the right questions, and how to integrate this
evidence into decisions. During this process, PPG discovered that employee motivation and
“readiness to change” was just as, if not more, important than the potential savings from
expensive situations. Reducing stress may improve productivity, but employees were much more
interested in—and more successful adhering to—weight management and weight reduction. Dr.
Colombi emphasized that employee/patient-focused willingness, awareness and education
programs are needed to successfully deploy any health-related program.
From Dr. Colombi’s perspective, there are two sides to what he termed the “healthy culture”
effort: what employees are willing to change and what an employer can change. This can also be
described as the frequency of an expense vs. per unit cost. Savings can be found in both.
Additionally, costs can be prohibitive: too high a co-pay reduces short term adherence or
prevention, which results in more serious and expensive patient care in the long term. Thus, Dr.
Colombi believes that patients should be seen as an influential decision maker and engaged.
CMS is constantly on the search for evidence that is directly relevant to its core population.
Medicare and Medicaid have very specific statutory authority for covering medical technologies
for its population, as specified in 1862 a(1)a of the Social Security Act: a covered item or service
must be reasonable and necessary to treat and diagnose an illness, always within the context of a
population that is over the age of 65, or meets other eligibility criteria. This applies to both local

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and national decisions. Most national coverage decisions are suggested by an outside source,
such as a sponsor. Occasionally CMS will open a decision internally based on new evidence, or
if there is a potential overuse issue, or if a non-coverage policy should be reconsidered.

All panelists agreed that there is a lack of data regarding how to engage patients in disease
management programs and documenting health and productivity improvements, absenteeism,
presenteeism. Mr. Sweet mentioned that WellPoint would welcome studies from the
pharmaceutical industry that include productivity, as long as the measures and process are as
transparent as possible in order to validate the new research against existing research. Ms. Jensen
emphasized that CMS needs patient-focused data relevant to the Medicare population.

2. Policy decision-makers want effectiveness information, not just safety and efficacy
information. Specifically, they want evidence that the decisions made are the appropriate
decisions in their covered population, and are based on real-world comparative effectiveness
data.

All three major sectors (insurer, government, and employer) emphasized the need for real-world
evidence, the importance of evidence development, and the need for cross-sector collaboration
and partnership. Efforts should include payers, employers, medical product manufacturers, the
clinical community, and patient advocacy groups. All three speakers agreed that it is important to
understand the comparative value of products on an ongoing basis, and build a usable
infrastructure and collaborative efforts to put in place what IOM would term a “Learning Health
Care System.”
Each presenter described in some detail his or her perspective with regard to the need for real-
world evidence, and these ideas are summarized below.
WellPoint

In order to make P&T decisions, WellPoint is looking for more than evidence from clinical trials
on efficacy and safety. WellPoint values information from clinical trials where doctors and
patients are observed and the results are analogous to what each might do in the real world.
Essentially, WellPoint is looking for both health outcomes and effectiveness outcomes
information. To get at this evidence, WellPoint is utilizing their own claims data and health
outcomes research capabilities in order to understand changing health behaviors and changing
prescribing behaviors and if the formulary had a part to play in the way patients behaved.
More than the negotiated or unit drug price, WellPoint needs information on the total cost of
care. For instance, WellPoint has begun trying to collect information on productivity outcomes
(e.g., how do we get people back to work quickly, keep patients/employees from being absent,
and keep them more productive while on the job?). WellPoint believes this evidence is crucial
both to its own policy decision making process as well as to the employers who are purchasing
insurance products from WellPoint.
WellPoint prizes studies with active comparators in the usual care setting testing
effectiveness in the real world. As Mr. Sweet stated during the presentation, “we need to blend

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the good science of clinical trials and the good science of well-designed health outcomes
research studies to get to real world effectiveness data that means something.”
According to Mr. Sweet, the goal is to obtain better overall data. Product claims should be
continually monitored and validated, and revisions to coverage should be made based on what
appears to actually work (from a study or from internal observation) in a managed care setting
given medical and pharmacy costs, as well as quality of care impact. WellPoint’s ideal studies
are high quality comparative effectiveness and/or efficacy trials with a population
representative of the WellPoint population.
There are opportunities for system-wide collaboration to help monitor how well a drug is
performing on the market. WellPoint is currently in discussions with the FDA to contribute to
the Sentinel Initiative in order to help monitor and validate label claims over time. The FDA’s
Sentinel Initiative is a system-wide effort to develop a “national, integrated, electronic system for
monitoring medical product safety.” Similarly, WellPoint wants evidence development to be
standardized and transparent, moving toward an “engagement model” that fosters
collaboration between insurers and pharmaceutical companies for better science and better
evidence and toward effective evidence implementation.
CMS

CMS defines “reasonable and necessary” to mean that an item or service has adequate evidence
to confidently conclude that the item or service improves health outcomes in the Medicare
population. When considering covering an item or service, CMS is interested in what real life
difference this drug or test makes compared to the management without this drug or test.
According to Ms. Jensen, CMS’ “golden standard” for evidence would be adequate evidence that
a particular treatment strategy using a new therapeutic technology compared to any alternative
leads to improved health outcomes for the Medicare population. In other words, CMS is looking
for real-world comparative effectiveness evidence.

When considering evidence, CMS often finds that trials do not include Medicare beneficiaries in
the population due to their advanced age, or the science is not as rigorous as desired. CMS
developed the Coverage with Evidence Development (CED) National Coverage Determination
in order to encourage promising technologies that did not have the proper evidence for full
coverage. The coverage with evidence development determination has two arms, either of which
essentially results in coverage with restrictions. Coverage with Appropriateness
Determination requires registry enrollment. This is in order to collect information not found
in a claim, such as the potential for overuse, a specific population or a specific kind of provider.
The second kind of CED is Coverage with Study Participation, which requires clinical trial
enrollment. CMS pays for that item or service, all routine costs in the trial, as well as the related
services.

Because of the intricacy and complex nature of Medicare statutes, CMS finds it difficult to
implement CED; evidence development is limited to only registries or trials. The Medicare
Improvements for Patients and Providers Act of 2008 (MIPPA) gave CMS broader authority
around clinical trials, which CMS is in the process of interpreting. With broader authority, CMS
would like to implement needed payment and coding changes, as well as collaborate with
health plans on evidence development processes.

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2008 NPC Annual Forum Session Summary, “What Evidence do Payers Need?”

PPG

Beyond clinical treatment data and health insurance plan management/oversight, Dr. Colombi
believes that employers have a duty and an ethical responsibility to improve upon what is spent
and how on behalf of employees. To him, there is a clear interplay between economic recovery
and how much it costs to provide health insurance. PPG decided to look for opportunities to
become more efficient and rescue resources that would help keep their employees healthy,
productive and at work while minimizing the sick care cost.
Dr. Colombi described previous efforts his company made to reduce costs quickly by changing
the benefit plan design, but PPG’s plan design staff was “running out of ammunition.” Therefore,
PPG targeted modifying the company’s health culture, such as the lifestyle, behavior, and
the overall risk factor profile of all employees.

3. Formulary decision guidelines should be transparent so that data and evidence expectations
are clear and sponsors can work on providing the most desired data.

Mr. Sweet described in some detail the effort WellPoint has made to remodel and publicize their
formulary decision process, which involves a multi-tiered evidence consideration process. He
believes that objective consideration of the best evidence available from multiple perspectives
will result in a more thorough and effective formulary guideline.
First, WellPoint’s Clinical Review Committee reviews data from clinical trials, focusing on
safety, efficacy, and tolerability. This first committee is not allowed to look at any cost data, not
even any pharmacoeconomic data because it may include cost information. Second, the
Outcomes Advisory Committee reviews the information. This committee is made up of
physicians who conduct pharamcoeconomic, health economics, or outcomes studies. The
purpose of this committee is to supplement the recommendations from the first committee, and
specifically comment on whether research is appropriate and relevant to clinical care. Third,
recommendations from both committees are then handed to WellPoint’s actuaries who create
total cost to care models projected out over about five years. Lastly, the Value Assessment
Committee, made up of pharmacy and medical directors from the WellPoint system and business
leaders who own much of the cost to care and quality care metrics, review the data. This
committee makes the final formulary decision.
The WellPoint HTA Guidelines (available on the WellPoint NextRx website) are intended to
support applications for both new drugs as well as ongoing therapeutic class reviews. The
guidelines provide a framework for recommended evidence and analysis from the
pharmaceutical sponsor with comparative clinical cases where available, comparative cost
effectiveness, and budget system impact. WellPoint believes that these guidelines ought to be an
industry standard for review, and are publicizing them to demonstrate transparency and
encourage collaboration with sponsors.
Dr. Colombi reacted favorably to the WellPoint formulary decision process and guidelines, and
recommended that researchers consider pursuing value trials rather than more clinical trials in
order to demonstrate where the greatest value can be found given a variety of situations and
interventions. Companies and employers have to search for value maximization because of the
dominance of particular insurance providers or hospital systems.

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2008 NPC Annual Forum Session Summary, “What Evidence do Payers Need?”

According to Ms. Jensen, statutory barriers prevent Medicare innovation in formulary


development or transparency, as well as research and implementation. CMS wants to be able to
participate in some of the innovative collaboration occurring in the private sector but is often
limited in what they can do. Nonetheless, Ms. Jensen says that they welcome sponsor meetings
and discussions to try to discuss the available research in support of any particular medical
technology.

All three presenters enthusiastically supported the notion that information is needed to inform
decisions throughout the health care system, and stakeholders should become more sophisticated
partners to collaborate on better decision making processes. In particular, insurers and
manufacturers should work together to develop a process that is transparent and open to make
desired evaluation criteria clear in order to encourage maximally useful information and
research.
There is also movement, such as at WellPoint, to adopt coverage with evidence development
programs in the private sector. By marrying clinical trials, claims data analysis, outcomes
research, dossiers, and other data sources, it may be possible to build in productivity evidence
into decision making.

CONCLUSION
At the conclusion of the session, it was clear that all of the speakers agreed on several major
points. First, after evidence generation, there is a breakdown in use. Even though research may
be published in a highly respected journal, practitioners may not use the evidence, policy or
formulary developers may disregard evidence that is not immediately relevant, patients may
doubt it, or patients may not follow recommendations of the literature or of their doctors. From a
policy perspective, stakeholders also need to resolve how to use evidence to make changes to the
formulary or benefit design and achieve a desired outcome. Several employers are experimenting
with value-based insurance design, and if crafted appropriately, this may be an important and
practical policy initiative.

Additionally, there is a need for common evidence standards and expectations, including at CMS
and FDA. FDA has stringent rules on proprietary information that prevents full disclosure and
transfer of information outside the agency, appropriately. Nonetheless, both agencies need to
engage sponsors in developing efficient evidence methods.

Several questions remained unanswered. How can stakeholders collaborate broadly,


transparently and effectively across sectors? How can they connect and build on existing
resources across sectors?

There is a need for partnerships for both the development of evidence and the underlying
infrastructure to facilitate this development of evidence.

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