How Guidelines Happen
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linical practice
guidelines serve as
guideposts for providing
evidence-based care for patients,
assisting clinicians in decision-making.
They may focus on providing the best value to
patients, and in an era of health-care reform,
they can drive funding decisions by policymakers or lead payer reimbursement decisions.
Practice guidelines seem to appear out of
nowhere, as if handed down from above as the
next Ten Commandments for treating patients.
But just how does a clinical recommendation or idea become a guideline? And how do
guideline writing panels sift through the mountains of sound and not-so-sound evidence to
craft trustworthy, useful recommendations? As
might be expected, it is an often time-consuming process that involves numerous stakeholders, a commitment to transparency, and a
thorough investigation of existing research.
Panels of experts often spend several years
developing clinical questions and diving into
research pertaining to those questions, to
ensure that recommendations are based on the
most current clinical science.
If done right, the process yields a well
thought-out, evidenced-based document that
serves as a guide to practicing clinicians that
highlights areas where additional research may
be needed and drives future research and funding decisions where data are lacking.
ASH Clinical News spoke with experts
involved in the development of guidelines for
hematologic disorders and cancers about how
evidence ultimately becomes a guideline.
Narrowing the Focus
As one might imagine, the first step in guideline development is deciding what to focus on.
With new evidence emerging each day about
hematologic conditions, deciding where to
direct guideline development resources isn’t a
simple process.
“There is a huge amount of evidence that
gets published every day, every week, every
month, every year,” Adam Cuker, MD, MS,
assistant professor at the Hospital of the Uni-
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ASH Clinical News
versity of Pennsylvania in Philadelphia,
told ASH Clinical News, explaining
the rationale behind clinical practice
guidelines. “It’s pretty much impossible for a busy clinician to keep
up with all of the new information. One of the major
roles of clinical practice
guidelines is to synthesize
and rigorously, systematically appraise this evidence and
distill it down to recommendations
for the practicing clinician.” Dr. Cuker
serves as chair of an expert panel that is
coordinating a new ASH effort to develop comprehensive new guidelines on venous thromboembolism (VTE) (SIDEBAR).
ASH has previously published guidelines on
a few topics, most recently on immune thrombocytopenia (in 2011), and the organization
has endorsed guidelines developed by others.
The effort to develop ASH guidelines on VTE is
distinguished by the use of a new, standardized
process; greater staff support; and dedicated
resources for a systematic, rigorous review of
available evidence by independent researchers.
In 2014, ASH formed a new Committee on
Quality, which is responsible for overseeing
all ASH quality-related activities. Reporting
to this committee, a new Guideline Oversight
Subcommittee will manage the organization’s
guideline development efforts. Julie Panepinto,
MD, MSPH, professor at the Medical College
of Wisconsin, chairs the new subcommittee.
“Part of our task will be to prioritize disease
areas for future guideline development. For
the near future, the Committee on Quality has
already selected some topics, including VTE
and a revision of the guidelines on immune
thrombocytopenia.”
According to Dr. Panepinto, a major effort
of the past year by the Committee on Quality has been to define standard procedures for
each step of guideline development to ensure
the same level of rigor and transparency is applied to all topics.
According to Anita Rajasekhar, MD, of the
University of Florida, who oversaw the creation
of ASH’s guideline development process and
serves on ASH’s Committee on Quality, the
Guideline Oversight Subcommittee takes
several factors into account before making any
decisions.
First, they consider the needs of ASH
members and which topics are in high demand,
including suggestions from other internal committees, professional societies, policymakers,
payers, patient groups, and government and
non-government agencies.
“Next, we consider the disease itself and its
characteristics,” Dr. Rajasekhar explained. “Is
it a highly prevalent disease? Is it a very costly
disease to treat and, if so, even though it may
be rare, is it something that could potentially
lower health-care costs if we have a good set of
treatment guidelines available?”
Other factors that may drive the decision
are whether there is large variation in clinical practice across the country, or if a clinical
area is seeing rapidly changing evidence. “This
is a common scenario in oncology, but it is
also happening in hematology, particularly
with new oral anticoagulants coming out,” Dr.
Rajasekhar added.
So, why is ASH focusing on VTE for its fi rst
guideline development effort? “We chose venous
thromboembolism because it is a very common
disease that hematologists are involved with.
There has also been rapid evolution of evidence
in recent years,” Dr. Cuker said.
Finding the Right Team
Once the guideline topic has been decided, it’s
time to assemble the guideline writing team.
Panel selection is a careful process that involves
multiple levels of organizational approval,
beginning with the Guideline Oversight Subcommittee and ending with the ASH Executive
Committee or its officers. An important goal is
to create a multidisciplinary group that is able
to look at the topic from all angles. “Despite
our rigorous development process, panels
must still use judgment to evaluate evidence
and form recommendations. For this reason,
we aim to balance expertise, perspective, and
background,” Dr. Panepinto said. Face validity
is also important, she acknowledges. “Readers
may trust a guideline on the basis of a strong
development process, but they also pay attention to who served on the panel.”
The process begins by identifying a panel
chair, which Dr. Rajasekhar says is usually
determined from a short list of candidates who
possess a certain mixture of characteristics.
“Obviously, they have to have content expertise, but they also have to have some expertise
in developing guidelines, because this process
is so involved, and we want someone who can
bring both skill sets to the table,” she said.
Once the chair of a guideline panel is
approved by the Executive Committee, the
Guideline Oversight Subcommittee and the
chair select the rest of the team. Dr. Panepinto
notes that panelists are not just “friends of
August 2015