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OCR for page 27
3
Improving Public Participation
in Clinical Trials
In previous Forum workshops in the clinical trials series (IOM, 2010a,
2012a), it has been repeatedly observed that increased public understand -
ing of, and commitment to, the importance of clinical trials is a key com -
ponent of enhancing the CTE and achieving a learning health system.
The participation of both clinicians and patients and healthy volunteers
has been considered in these discussions. The workshop’s second session,
“Developing a Robust Clinical Trials Workforce,” and the following key -
note session explored ways to identify and meet workforce needs and to
enhance public engagement in the CTE. Many speakers noted that there is
an insufficient supply of highly trained researchers to lead, conduct, and
analyze clinical trials and proposed how a shortfall of these professionals
might be overcome. Building on discussions from a previous IOM work-
shop (IOM, 2012a), participants further elaborated on ways to expand
people’s support for, and involvement in, clinical trials.
DEVELOPING A ROBUST CLINICAL TRIALS WORKFORCE
Should we go so far as to expect, not just encourage,
participation in research?
—Ann Bonham, Association of American Medical Colleges;
Robert Califf, Duke University Medical Center;
Elaine Gallin, QE Philanthropic Advisors; and
Michael Lauer, National Heart, Lung, and Blood Institute (NHLBI)
27
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28 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE
A Five-Tiered Structure1
A clinical trials workforce, organized in several dimensions that reflect
the broad mission of the CTE, the specific disciplines involved, and the
levels of desirable expertise, would consist of several overlapping groups.
A construct of five workforce groups was described in a Discussion Paper
prepared by Ann Bonham, Chief Scientific Officer, Association of Ameri-
can Medical Colleges; Robert Califf, Duke University Medical Center;
Elaine Gallin, Principal, QE Philanthropic Advisors; and Michael Lauer,
Director, Division of Cardiovascular Sciences, National Heart, Lung, and
Blood Institute (NHLBI), NIH. (See “Developing a Robust Clinical Trials
Workforce” in Appendix E.) The co-authors suggested that the structure
of the workforce groups necessary to meet the demands of the CTE might
resemble a pyramid with five tiers (see Figure 3-1):
• Public—the broadest base of the pyramid consists of patients, fami-
lies, and citizens who, in the final analysis, have the greatest stake
in research results. This tier consists of engaged citizens who sup -
port development of the CTE as a national resource or public good
and who enroll in trials on a volunteer or basis.
• Community practitioners—health professionals who participate in
trials as part of their clinical practices, or at least help enroll their
patients as participants. This group will include physicians, nurses,
pharmacists, social workers, physical therapists, respiratory thera -
pists, and other health professionals.
• Implementers—individuals who devote specified portions of their
professional efforts to serving as principal investigators or col-
laborating co-investigators, with primary responsibility for imple-
menting clinical trials at a hospital or research site. This workforce
group would include physician-scientists, nurse-investigators, clin-
ical pharmacologists, research-oriented social workers, operations
specialists, data managers, computer specialists, clinical research
coordinators, and research site managers.
• Investigators—leaders and designers of clinical trials and scientific
experts who develop tools and innovative approaches for conduct-
1 This section is based on the presentations and Discussion Paper by Ann Bonham, Chief
Scientific Officer, Association of American Medical Colleges (unable to attend workshop);
Robert Califf, Vice Chancellor for Clinical Research, Director of the Duke Translational
Medicine Institute, and Professor of Medicine in the Division of Cardiology at the Duke
University Medical Center; Elaine Gallin, Principal, QE Philanthropic Advisors; and Michael
Lauer, Director, Division of Cardiovascular Sciences, National Heart, Lung, and Blood
Institute (NHLBI), NIH. (See Appendix E for the Discussion Paper “Developing a Robust
Clinical Trials Workforce.”)
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29
IMPROVING PUBLIC PARTICIPATION IN CLINICAL TRIALS
Academicians Doing
Research on Clinical
Trial Methods and
Policies
Clinical Trial Investigators
Clinical Trial Implementers
All Health Care Providers
All Individuals
FIGURE 3-1 Five suggested tiers for the workforce to meet the demands of a
transformed clinical trials enterprise.
SOURCE: Bonham et al, 2012. Developing a Robust Clinical Trials Workforce. Discus-
R02159
sion Paper, Institute of Medicine. (See Appendix E.)
Figures 3-1 and E-1
vector, editable
ing trials. This group will include biostatisticians and informaticists,
among others.
• Methodologists—the apex of the pyramid consists of research-
minded experts who explore the methodologies of conducting clin-
ical trials and design the analysis portions of studies. This group
of academicians will include clinical investigators, biostatisticians,
epidemiologists, and health services researchers.
The co-authors postulated that a great deal of mobility and teamwork
would occur among and across these groups. Each tier, in this vision,
would be associated with appropriate education or training, geared to
individuals’ interests and capabilities. This would include training in
research cooperation, or teamwork, across disciplines and among work-
force groups. Table 3-1 suggests how other training essentials might match
up with the five described tiers. In general, according to the co-authors,
the size of each tier is currently inadequate to contribute enough knowl-
edge to achieve transformation of the CTE. Expanding the size of the three
top tiers (methodologists, investigators, and implementers) could require
development of more rewarding career paths.
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30 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE
TABLE 3-1 Education and Training Needs of Groups in the Clinical
Trials Workforce
Workforce Groupa Education and Training Needs
Public Awareness of how clinical research can lead to higher
quality care; information about available trials and concept
of equipoiseb; relevant informed consent policies. Marketing
techniques might be used to reach this group.
Community Introduction to clinical trials; core competencies in translational
Practitioners and clinical research and public health
Implementers Pragmatic training in management of research projects;
discipline-specific education and training
Investigators Education and training in clinical subspecialties (such as
cardiology and infectious diseases), biomedical research,
biostatistics, health services research, regulatory science,
epidemiology, computational biology, genomics, and new
technologies (such as imaging, cell and tissue engineering, and
nanotechnology)
Academicians or Doctoral-level education in health research disciplines
Methodologists
aSee Figure 3-1.
b Equipoise is the point at which a rational, informed person has no preference between
two (or more) available treatments (Lilford and Jackson, 1995). In clinical research, the ethi -
cal concept of equipoise is satisfied when genuine uncertainty exists as to the comparative
therapeutic benefits of the therapies in each arm of a clinical trial.
SOURCE: Bonham et al., 2012. Developing a Robust Clinical Trials Workforce. Discussion Paper,
Institute of Medicine. (See Appendix E.)
Across the CTE workforce, increased demographic diversity—namely,
greater levels of participation by racial and ethnic minorities—could help
ensure that research conduct is relevant to, and research results serve
the needs of, largely underserved communities. Those who experience
health disparities often do not participate in trials at rates high enough
to constitute subpopulations from which statistically significant findings
are produced (e.g., Pollack, 2011). Inasmuch as current U.S. Census projec-
tions suggest that by 2020 nearly one in three Americans will be African
American or Hispanic, improving the health of these populations is essen-
tial to maintaining and improving the health of the country as a whole.
Similarly, higher rates of participation in clinical trials by people over age
65, and especially over age 85, could make trial results more relevant to
the very large and expanding elderly population, given projections that 1
in 5 U.S. residents will be 65 or older by 2030 (Vincent and Velkoff, 2010).
Clinical trials participation by overweight or obese people, people with
chronic diseases, and residents of urban communities also will have to
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31
IMPROVING PUBLIC PARTICIPATION IN CLINICAL TRIALS
increase in order for research to be representative of the total popula -
tion. Women and racial minorities remain underrepresented in the ranks
of principal investigators. One study found that 16.9 percent of White
physicians participate as principal investigators in clinical trials compared
to 14 percent of African American physicians, 10.8 percent of Hispanic,
and 9.6 percent of Asian physicians (Tufts Center for the Study of Drug
Development, 2007). The study also found that 10.9 percent of female
physicians participate as principal investigators compared to 16.9 per-
cent of male physicians. The co-authors noted that, even if demographic
parity is not achieved in all areas of the workforce, the CTE could benefit
if researchers are skilled in engaging the community in clinical trials and
able to accommodate demographic trends in designing, implementing,
and analyzing clinical trials.
OPPORTUNITIES TO CREATE A SUSTAINABLE
WORKFORCE FOR THE CLINICAL TRIALS ENTERPRISE
In a panel discussion, the session chair, panel presenters, Discus-
sion Paper co-authors, and audience members reacted to and built upon
the ideas contained in the Discussion Paper. Participants included ses -
sion chair Sherine Gabriel, William J. and Charles H. Mayo Professor
of Medicine and Epidemiology, Mayo Medical School; and panelists
Briggs Morrison, Senior Vice President and Head of Worldwide Medical
Excellence, Pfizer Inc.; and Rebecca Jackson, Associate Dean for Clinical
Research, Professor of Medicine and Director of the Center for Women’s
Health at The Ohio State University. This section provides an integrated
summary of their remarks and should not be construed as reflecting
consensus or endorsement by the workshop participants, the planning
committee, the Forum, or the National Academies.
The Public
The clinical trials “workforce” of the future may consist largely of
patients themselves. Public participation in clinical trials could increase
if biomedical knowledge is widely accepted as a “public good,” benefit -
ing everyone, so that people perceive a duty to contribute (Schaefer et al.,
2009). Already, many patients and patient advocates, including disease-
specific organizations, have exhibited a superior understanding of clinical
trials. For example, the National Marfan Foundation says on its website
that the organization “does not recommend switching from a beta blocker
to losartan,” pending results of a clinical trial of losartan still in progress
(National Marfan Foundation, 2011). The National Foundation for Infan-
tile Paralysis pioneered public support for clinical research in the early
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32 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE
20th century, partly through the March of Dimes, and this tradition con -
tinues with efforts such as the Love/Avon Army of Women, which enlists
participants in breast cancer trials in collaboration with the American
Association for Cancer Research and the National Breast Cancer Coali -
tion. In the rare diseases clinical research network involving 93 diseases,
patient advocacy groups participate in research through such activities as
protocol development, study steering committee membership, and review
of informed consent policies.
Formal credentialing of individuals also can facilitate broader public
participation. In Cleveland, efforts are under way to provide certification
in clinical research to community leaders, who then help develop specific
research projects.
The low level of health literacy in the U.S. population is well docu-
mented (IOM, 2004, 2011c; Rudd et al., 2007). Teaching a basic medical
vocabulary and the principles of clinical research in high schools could
better prepare people to participate as volunteers in clinical trials, while
also introducing more students, including minority students, to the pos -
sibility of careers in clinical research. Incorporating clinical research into
the high school curriculum could lead to students’ assisting their parents
with participation in clinical research studies, including clinical trials. Sev-
eral workshop participants noted that, given the pace of change in both
medical science and health care delivery, the ability to be an “informed
consumer” is essential to maintaining health and maximizing benefit from
the health care system.
Community Practitioners
Negative physician attitudes toward research, often manifested
through limited communication with patients about opportunities to par-
ticipate in clinical trials, was mentioned by some participants as a major
reason why clinical trials today struggle to recruit and retain patients.
Only about 7 percent of adult Americans report that a physician has
ever suggested to them that they participate in a clinical research study,
although three-fourths say they would be very likely or somewhat likely
to participate if asked (Research!America, 2007).
Physician behavior is a critical factor in applying research results
to health care. Lauer noted that in the Occluded Artery Trial (OAT), no
benefit was found to the common U.S. practice of using stents or balloon
angioplasty to open occluded arteries that had not been opened within
12 hours following acute myocardial infarctions (AMIs) (Hochman et al.,
2006). Clinical guidelines were revised in accordance with these findings,
yet practices generally did not change in the several years immediately
following release of the revised guidelines (Deyell et al., 2011). Moreover,
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33
IMPROVING PUBLIC PARTICIPATION IN CLINICAL TRIALS
according to Lauer, the study researchers experienced extraordinary dif-
ficulty recruiting U.S. physicians to take part in the study, and this recruit-
ment difficulty is attributed largely to the fact that the study challenged
the conventional wisdom about the benefits of opening persistently
occluded arteries. Another workshop participant added that physician
recruitment in OAT was further challenged by financial disincentives to
the extent that a clinician’s participation in the trial limited the number
of artery–opening procedures she or he would be able to perform in com -
pliance with the trial protocol. Another financial disincentive was that
if the trial revealed the procedure to be unsafe or ineffective, physician
caseloads and revenue would decrease.
According to some participants, the culture of research in North
American medical schools appears to be diminishing. Part of physicians’
reluctance to participate in, or apply findings from, clinical trials may
stem from attitudes rooted in the Flexner reforms of medical education
dating back a century ago, noted Califf. Under the Flexner model, the
basis of medical practice is knowledge of human biology. Today it may
make more sense for the basis of medical practice to consist of clinical evi-
dence (albeit with an understanding of biology). Another partial explana -
tion for inadequate attention to research in medical schools may be faculty
members’ relative lack of knowledge or comfort with research science.
According to Jackson, empiricism could be inculcated into under-
graduate students, medical students, residents, and other future health
professionals, such as dietitians, by teaching about the scientific inves -
tigations process and its relationship to health care, and by integrating
clinical research into other coursework. In this way, students would learn
to think of a working diagnosis as a hypothesis, and to view medical
examinations and tests as an experiment to test the diagnostic hypothesis.
It would be helpful for education to include use of research results and
interpretation of statistical findings. It was suggested that, to introduce
and take advantage of these changes, external pressures may be needed.
Individual workshop participants highlighted several possible strategies
for improving the education of community practitioners about research
to help enhance their future participation in clinical trials:
• Revision of curricula in some medical schools, which may provide
opportunities to introduce research problems into each educational
module;
• Inclusion of questions about research in national medical board
and nursing examinations; and
• Strengthening of medical school accreditation standards and
requirements related to the teaching of research methods with the
goal of increasing support for a culture of research.
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34 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE
An additional frontier of learning involves management of trials.
Although some practitioners are very adept at managing logistics—a key
skill in clinical research—others are not, and the teaching of management
skills is not yet included in most health professions’ educational curricula.
Implementers
Implementers are the people who conduct trials as a primary job func-
tion but are not engaged in the academic study of trial design methods.
They (and others) will benefit from being taught to think in innovative
ways and being trained to look at research problems from the perspective
of patients and clinicians. It would be beneficial if education programs
were refocused in order to meet not only gaps in knowledge but also gaps
in methodology, implementation, and application, and to create cultural
change. To this end, the NIH Clinical and Translational Science Awards
(CTSAs) are striving to improve and increase training through a network
of 60 medical research institutions. The program’s training component
includes a mentored clinical research scholar program at the postdoctoral
level and tailored clinical research training for predoctoral students.
One core constituency of the implementers group is clinical research
coordinators. Unfortunately, clinical research coordinators have been
found to leave the field within 3 years, a waste of training and talent that
apparently results from inadequate career paths. Indeed, retention of
clinical research coordinators is now often considered a greater problem
than attracting applicants to coordinator positions. Partly to promote the
field and imbue it with the advantages of a recognized profession, efforts
to develop an accreditation program for these professionals have begun at
the University of Pennsylvania, and a Society of Clinical Research Asso -
ciates has been created. With expertise in both management and clinical
sciences, clinical research coordinators conceivably could be educated
through master’s degree programs jointly developed by medical and
business schools.
Investigators
Investigators are the personnel charged with leading and designing
clinical trials. This group would be composed of M.D.-, M.D./Ph.D.-, and
Ph.D.-level investigators from a range of disciplines with the opportunity
to supplement their expertise with specific training in translational and
clinical research offered by the CTSAs. Paper co-authors suggested that
the future transformation of AHSSs into IHSs will bridge translational
gaps between discovery science and health care delivery and will require
more of this highly trained cadre of clinical investigators.
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35
IMPROVING PUBLIC PARTICIPATION IN CLINICAL TRIALS
Several workshop participants suggested that a sustainable and inter-
esting career path is at least as important as, if not more important than,
instituting formal training in the medical school curriculum. The rise of
medical informatics may be instructive. Medical informatics is emerging
as a board-certified specialty due to demand on the part of young physi -
cians attracted to a field that hospitals and delivery systems find essential.
Likewise, clinical research could become attractive if many more job posi-
tions were created.
In addition to bench researchers, it also is important to prepare people
for positions in regulatory science,2 notably within FDA. FDA regulations
ensure the safety, efficacy, quality, and performance of products that rep -
resent about 25 cents of every dollar spent by American consumers. In the
face of rapidly changing technology and increasingly complex products,
FDA and other regulatory agencies must maintain their staff’s scientific
and technical expertise while responding as rapidly and effectively as
possible to patients’ needs and to public health emergencies.
A 2012 IOM report suggested the wide range of core competencies
needed for professionals working in the regulatory sciences. In medicine,
these competencies might include bioengineering, bioethics, clinical phar-
macology, epidemiology, genetics, nutrition, public health, toxicology, and
many others, going well beyond the disciplines traditionally associated
with regulation, such as statistics and clinical research. In some cases,
needed competencies may even be found outside the biomedical sciences
in disciplines such as economics and sociology (IOM, 2012b).
Academicians and Methodologists
Academicians and methodologists are those who conduct research in
new clinical trial methods and policies. This group of clinical investiga-
tors would consist of biostatisticians, epidemiologists, and health ser-
vices researchers tasked with advancing and improving upon clinical trial
methodologies. The workforce group would reside primarily in AHSSs,
government, research institutes, and some larger industry groups that
could provide protected time for research.
Investigators and academicians and methodologists are not entirely
distinct from each other in that both design clinical trials. Ideally, these
workforce groups will be taught to focus on what clinicians and patients
need and will become proficient in behavioral skills and teamwork and
even will become “design thinkers.” According to several workshop
2 FDA has defined regulatory science as the “science of developing new tools, standards,
and approaches to assess the safety, efficacy, quality, and performance of FDA-regulated
products” (FDA, 2010).
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36 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE
participants, the CTSA program and other initiatives have spurred the
development of innovative research teams that tackle complex health
and research challenges to identify ways to turn their discoveries into
practical solutions to problems in patient care. Even at this high level,
lifelong learning can help maintain and improve competencies, especially
as scientists move from one career challenge to another in both private
and public sectors.
SUSTAINING INSTITUTIONAL SUPPORT AND
PATIENT ENGAGEMENT IN CLINICAL TRIALS
Clinical research gives the public hope. And you
cannot underestimate the power of that word.
—John Gallin, NIH Clinical Center
Models and Messages from the NIH Clinical Center3
An innovative and comprehensive approach to facilitating the con-
duct of clinical research has been developed by the NIH Clinical Center,
a 240-bed facility located on the NIH campus in Bethesda, Maryland. The
Clinical Center, which calls itself “America’s Research Hospital,” conducts
a robust program of clinical research aimed at improving the treatment
of both common and rare diseases and conditions. The approach was
detailed in a keynote address by John Gallin, Director, NIH Clinical Center.
Every patient at the Clinical Center is enrolled in a clinical study protocol
and receives care free of charge (see Glossary in Appendix K for definition
of protocol). The center’s research portfolio consists mostly of Phase I and
II investigations, rather than later-stage, confirmatory research. Studies are
divided roughly in half between natural history studies of disease patho -
genesis in patients with rare diseases and clinical trials.
NIH intramural clinical research benefits from the agency’s adher-
ences to seven “principles and processes” that it provides researchers:
clinical informatics, data management, and protocol tracking; biostatistics
support; quality assurance and quality improvement; protocol review;
human resources and physical plant; training and education; and research
participants, or research partners, as the term used at the Clinical Center.
The Clinical Center has established systems to meet these needs for
researchers, thereby substantially lightening investigators’ administrative
burdens (see Box 3-1). Some of the resources that NIH has developed to
support its own intramural clinical trials program are increasingly being
3 This section is based on the keynote address by John Gallin, Director, NIH Clinical
Center.
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37
IMPROVING PUBLIC PARTICIPATION IN CLINICAL TRIALS
BOX 3-1a
NIH Clinical Center Key Services for Researchers and Patients
• ervices of protocol navigators, writers, and translators
S
• ffice of Protocol Services support as a repository of NIH protocols that also
O
prepares all reports to Congress and provides quality review of protocol actions,
such as informed consent documents
• rotoType, a web-based protocol writing tool that can be used, for example, in
P
developing cost estimates and assessing regulatory compliance, and a “wizard”
tool to help determine whether an investigator must submit an Investigational
New Drug (IND) Application to FDA
• iomedical Translational Information System, a web-based integrator of clinical
B
and research data that offers investigators access to deidentified patient data,
integrates data sources from multiple institutes, and provides an ontology for
representation queries
• ssistance with recruitment and retention of study participants and with xternal
A e
communications, including media relations, use of social media, and outreach
efforts, such as vaccine mobile clinics to facilitate vaccination studies
• olunteer survey research on people’s satisfaction with participation in tudies;
V s
study participants are viewed as partners in clinical research
• pecialized services, including phenotyping, which is especially useful to
S
r searchers in rare diseases, as well as superior imaging services and phar-
e
macy services that can formulate specialized products
• raining services, including NIH Curriculum on Clinical Research (introduc-
T
tion to the principles and practices of clinical research, principles of clinical
pharmacology, and ethical and regulatory aspects of clinical research), an
online course for clinical investigators on standards in clinical research, and a
six-module clinical researcher sabbatical program lasting an average of three
and a half months
• ommunity outreach activities, including a van that travels around the
C
W
ashington metro area to make it easy for individuals who want to participate
in a vaccine clinical trial to get examined and provide blood for purposes of the
clinical trial
• nnual survey (focus group) of participants’ perceptions of their research expe-
A
riences. Aggregate survey data will identify best practices and drive improve-
ments at the Clinical Center, and beyond (Kost et al., 2011)
a This box is based on the presentation by John Gallin, Director, NIH Clinical Center.
made available to external researchers. These resources are particularly
useful for researchers who need more support than what their home
institutions can offer. Making them more widely available also helps
the Clinical Center fulfill its mandate to open its doors to extramural
investigators.
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38 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE
Protocol navigators are a special feature of the Clinical Center’s
approach. Although their specific functions vary from one NIH Institute
or Center to another, their role is to interface with components of NIH
and to help overcome regulatory barriers. Figure 3-2 displays the National
Institute of Allergy and Infectious Diseases (NIAID) navigators’ functions.
From 2009 to 2011, the Protocol Navigator Program at NIAID has con-
ducted 29 initial protocol reviews, 10 protocol amendments, and 7 inter-
national studies serving 29 investigators from 10 different NIAID labs.
The annual cost of the NIAID Protocol Navigator Program is $600,000
and staffs 1 navigation manager, 2 protocol navigators and 3 medical
writers. To generate a larger pool of protocol navigators for intramural
and extramural research support, the Clinical Center is developing a one-
year training program intended as an alternative career path for research
nurses, scientific writers, and IRB professionals to become versed in pro -
tocol navigation.
The Clinical Center’s new vision statement reads: “The role of the NIH
Clinical Center should be to serve as a state-of-the-art national resource,
with resources optimally managed to enable both internal and exter-
nal investigator use” (NIH, Scientific Management Review Board, 2010).
The expansion from strictly intramural to joint intra- and extramural
research activity is being introduced through a project to create part-
nerships between extramural and intramural researchers. A partnership
program between basic scientists and clinical investigators also is being
maintained, for the duration of current funding capacity.
Other Institutional Supports and Patient Engagement
This section is based on a panel discussion with John Gallin; Janet
Tobias, CEO, Ikana Health; Annetine Gelijns, Co-chair, Department of
Health Evidence and Policy, Mount Sinai School of Medicine; and Heather
Snyder, Senior Associate Director for Scientific Grants, Alzheimer’s Associ-
ation International Research Grant Program. In the discussion, individual
panelists and audience members identified challenges and opportunities
to improve engagement in, and support of, clinical trials at the institutional
and patient levels. This section provides an integrated summary of their
remarks and discussions.
Public engagement is essential to the CTE but may constitute a weak
link. Clinical trial recruitment and retention rates vary across disease area
and provider, with AHSSs showing high variability.
Some barriers to participation by the public in clinical trials include
• lack of awareness of the benefits of engaging in clinical trials and the
availability of relevant trials in which to participate (IOM, 2012a);
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39
IMPROVING PUBLIC PARTICIPATION IN CLINICAL TRIALS
Medical
Institutional
Writers
Review Board
Investigational
O ce
New Drug
O ce of Saf
Safety O ce /
Scienti c Review Technology Data and Safety
Development
APPROVED Monitoring
Board
CONCEPT
C linical
• Hypothesis
Protocol
• O ce
Endpoints Site
PROTOCOL
of Ethics
• Design Establishment /
NAVIGATORS
• Statistics Monitoring
Institutional
Biosafety
Committee /
National Institutes O ce Biotech Case Report
of Health Activities Form
Production
O ce of
O ce of
National Institute Research
Human Subject
of Allergy and Services
Recruitment
Infectious Diseases
and Protection
(NIAID)
NIAID Regulatory
Compliance and
Human Subjects
Protection Branch
FIGURE 3-2 The protocol navigator interface at the National Institute of Allergy
and Infectious Diseases (NIAID).
SOURCE: Gallin, J. 2011. Presentation at IOM workshop on Envisioning a Trans-
formed Clinical Trials Enterprise in the United States: Establishing an Agenda for
2020. Figure created by H. Clifford Lane, NIAID, and publicly available.
R02159
Figure 3-2
• clinical trials designed without patient input and therefore lacking
vector, editable
acceptability to the patients expected to participate in these studies;
• patient preferences for one treatment over another or the availability
color
of a treatment outside the clinical trial setting;
• logistic hassles in participation (such as requirements of frequent
patient visits, lab tests, and questionnaires); and
• inadequate reimbursement (for example, Medicare has paid for
routine care in clinical trials since about 2000, but many private
payers do not) which might lead to out-of-pocket expenses for trial
participants.
Several strategies might be used to reduce these barriers. For exam -
ple, Gelijns noted that a national coalition of patients, providers, and
political leaders might promote change. On a more limited basis, an
opportunity may exist to broaden the existing clinical trial networks that
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40 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE
have been established by NIH to increase efficiency in research. These
networks could include advocacy groups, community practitioners, and
other stakeholders who could enrich trial design, logistics, recruitment,
payment, and other aspects of studies. Networks might revolve around
academic health and science center hubs and include community practi -
tioners, whose patients could participate in trials through visits to their
usual practitioner’s office rather than to a distant research site. Engaging
patients in the development of novel clinical trial designs and the determi-
nation of clinical end points may help in raising enrollment rates. Routine,
effective communication with trial participants about study results also
could improve people’s sense of the value of the research effort. Collabo -
ration between payers and the CTE, such as new FDA collaboration with
CMS on innovative medical device use, could prove fruitful in ensuring
both reimbursement and the application of research results to practice.
Clinical research gives people hope, noted John Gallin. To illustrate,
parents enroll children in cancer studies at very high rates, as much as
90 percent. Advocacy groups have proved instrumental in promoting
engagement in research on breast cancer, cystic fibrosis (CF), Alzheimer’s
disease, and other illnesses, especially when they have been viewed as
impartial rather than favoring one treatment or trial over another. It
might be useful to devote more resources to public engagement at earlier
stages of research efforts, and to employ greater expertise in these com-
munication efforts. Tobias noted that people are more willing to enroll in
studies involving diseases perceived as life-threatening, such as cancer,
and less willing to enroll in studies involving diseases, such as diabetes or
Alzheimer’s disease, that, even though they too are life-threatening and
highly prevalent, are not perceived as such. A workshop participant also
noted that beyond issues of disease severity, or perception of severity, a
patient’s interest in participating in a clinical trial is related to the avail -
ability of effective, acceptable treatments. A current trend in Alzheimer’s
disease research, according to Snyder, is to seek ways to identify patients
as early as possible and to view the disease as a continuum.
There may be various education and communication strategies that
could prove useful. Scientific education in elementary, middle, and high
schools could enhance people’s scientific literacy and improve physician–
patient interactions. As mentioned in Chapter 2, the clinical trials work -
force could also become skilled at incorporating the patient perspective
into the design of clinical trials so as to make participation more attractive
and acceptable to the public. Individually matching a patient with a clini -
cal trial is another approach. When a 5-city survey found that a large pro -
portion of primary care physicians were unaware of local trials and lacked
time to find out about them, the Alzheimer’s Association developed a
program that has matched 3,300 patients with trials so far, said Snyder.
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IMPROVING PUBLIC PARTICIPATION IN CLINICAL TRIALS
Enhancing public awareness might only be part of a larger remedy
for improving participation in clinical research. In large part, recruitment
is a matter of using the right language and communication techniques. To
illustrate, mere education about clinical research and randomization may
not suffice to increase recruitment rates, especially in the face of the heavy
burden of participation. An evaluation of the “Get Randomised” media
campaign in Scotland revealed that, although the campaign increased
public awareness about clinical research, it did not increase the willing -
ness of individuals to participate in clinical studies (Mackenzie et al.,
2010).
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