September 13, 2012
The Alliance for Regenerative Medicine (ARM) is pleased to comment on the draft report
developed by the Methodology Committee from the Patient-Centered Outcomes Research
Institute (PCORI).
ARM is a multi-stakeholder organization based in Washington, DC whose members represent
120 leading life sciences companies, patient advocacy groups, non-profit research organizations,
universities, and investors in regenerative medicine. While existing regenerative medicine
products have already helped millions of patients with a variety of conditions, products in
development have the potential to treat diseases such as diabetes, stroke, heart disease, and
cancer. They will do so in a manner that will "bend the cost curve" and reduce health care
expenditures.
Regenerative medicine products hold the promise of treating currently unmet medical needs,
improving patients' quality of life, and reducing overall health spending. The Alliance for
Regenerative Medicine (ARM), a multi-stakeholder organization dedicated to advancing
development of regenerative medicine therapies, believes that comparative effectiveness research
-- done properly -- will support uses of these technologies.
ARM supports efforts to make sure that doctors and patients have access to all necessary
information about proposed treatments. This includes the best information available to assess
relative benefits and risks of these treatments. Comparative effectiveness information is an
integral part of this data.
Despite its potential benefits to health care, we are concerned that comparative effectiveness
analysis will be used inappropriately, which will stifle innovation and hurt patient care.
Some general comments about the report follow.

As you know, the report was required by the Affordable Care Act that created PCORI
and it describes methodologies to prioritize the patient’s perspective in all phases of
research to be funded by the Institute. ARM supports prioritization of the patient
perspective and the intent of the paper's approach which is to produce trusted information
and lead to better healthcare decisions, and ultimately to better health.

It appears that the methodology committee consisted primarily of policy makers,
academics, and a couple of reimbursement/payer representatives—not the people that
would be impacted such as principal investigators, commercial sponsors, practicing
physicians, or other organizations that will have to implement outcomes research. In
addition, it doesn't appear that anyone from the Centers for Medicare and Medicaid
Services or FDA was involved. It is essential to ensure that FDA marketing approval
requirements and even CMS requirements do not conflict with PCORI requirements.

It is unclear how PCORI requirements and studies will be used in conjunction with
research data or standards from other countries. Since biomedical research often
transcends national borders, we encourage PCORI to consider this issue.

The report describes recommended actions for public registration of all PCORI studies,
including the sharing of protocols, statistical code, and data. It is unclear whether certain
exclusivity rights and drug/device/ other patents may be infringed by such disclosure or
whether this would entail disclosure of otherwise confidential information.

PCORI should consider developing its standards in the format of other standards (such as
ISO, AAMI, EN, etc) for consistency purposes as well as easier readability,
interpretation, and implementation.
The following are issues PCORI and other decision-makers should consider in crafting policies
related to comparative effectiveness:
Comparative effectiveness information should inform clinical judgment, providing
clinicians, patients, other stakeholders, and policymakers with important information
about treatments and not be used to contain costs. Comparative effectiveness research can
play a vital role in health care delivery by helping doctors and patients understand the relative
potential risks and benefits of treatment options and thereby make informed decisions. As
companies develop new and innovative technologies, having comparative effectiveness
information will be critical for physician-patient decision making. However, comparative
effectiveness research is sometimes viewed as an instrument to contain costs, rather than provide
health care value by improving patient health outcomes. If comparative effectiveness research
focuses simply on costs, it runs the risk of working at cross purposes with ensuring that
physicians, patients, and others have access to information about treatment options because new
technologies could be viewed negatively simply because of their price.
The legislation creating PCORI states in part that the "purpose of the Institute is to assist
patients, clinicians, purchasers, and policy-makers in making informed health decisions by
advancing the quality and relevance of evidence concerning the manner in which diseases,
disorders, and other health conditions can effectively and appropriately be prevented, diagnosed,
treated, monitored, and managed…" Thus, we are hopeful that PCORI's research will inform
clinical judgment and not be used to contain costs.
Comparative effectiveness research must account for clinical situations where there is no
"comparable" treatment. In many situations, when physicians and patients make healthcare
decisions, they may weigh the risks and benefits of two or more therapies. Comparative
effectiveness research is designed to facilitate that process. Sometimes, though, no comparator
exists. This is the case for many regenerative medicine technologies that are indicated for
conditions for which there is no alternative therapy. In those situations, researchers should
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compare the new treatment to existing patient care practices (even if these do not involve drugs
or biologicals) -- such as hospitalization -- in terms of clinical benefit, patient quality of life, and
other criteria.
Comparative effectiveness research should allow for sophisticated analyses of different
treatments. For example, many of the unique benefits provided by drugs and biologics, such as
increased safety and improved patient quality of life, are often not captured in comparative
effectiveness analyses. Increased worker productivity and savings to other parts of the health
care system are also important benefits that may not be reflected in studies conducted with a
narrow perspective that simply focuses on direct costs and benefits of a particular product. In
addition, comparative effectiveness research should also consider the impact of better health
outcomes on patients' families. When family members need to be caregivers, that could impact
their quality of life including emotional well-being or ability to work productively.
Further, even when two distinct therapies produce an equivalent benefit on average in a patient
population, and may even have a comparable safety profile on average, comparative
effectiveness research should explicitly recognize that individual patients may be more
responsive to one form of therapy, or may be more (or less) sensitive to side effects from one
particular form of therapy. Therefore, there are benefits to patients from having access to
multiple forms of treatment.
Comparative effectiveness research must not stifle innovation. Patients and their families are
counting on drug developers and researchers to develop cures and treatments for many currently
untreatable diseases and conditions. Regenerative medicine holds the promise of providing these
treatments. Historically, medical innovations cost more when first introduced into the market
and there is less data about how a new product is used in practice. These factors could lead to
unfair comparisons with older treatments. Comparative effectiveness analysis should not be
used to restrict access to such innovation simply because products have a higher price or are
recent market entries. That's especially important because innovations not only help current
patients, but also often lead to new breakthroughs that help future patients. Government policies
addressing comparative effectiveness need to ensure that comparative effectiveness research
does not lead to conclusions and decisions that discourage or impede medical advancements and
breakthroughs that can address unmet medical needs.
Comparative effectiveness research should be conducted through an open and transparent
process involving all stakeholders, starting from the research planning stage. Different
comparative effectiveness studies evaluating the same item or service can produce widely
disparate results, depending on the methods and assumptions used in the analysis. To enhance
the credibility and usefulness of any comparative effectiveness study, stakeholders should be
afforded the opportunity to provide meaningful input into all steps along the study process,
including the identification of priority areas to research, study design and research methods, and
dissemination of results. Getting stakeholder buy in increases the chances that the research will
be useful -- and used -- for clinical decision making.
ARM members remain committed to advancing innovation and developing new therapies to
meet unmet medical needs, improve patients' lives and "bend the cost curve" for health care.
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ARM welcomes the opportunity to work with policymakers and other stakeholders on ways to
ensure that comparative effectiveness research helps clinicians and patients get the information
they need to make informed care decisions.
We look forward to working with PCORI in the days ahead.
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