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2
The Importance of Innovative
Regulatory Science
Key Messages
• ollaboration among federal agencies offers a particularly productive venue for
C
developing a regulatory science workforce.
• reating a supportive academic environment and training a new generation
C
of researchers who are skilled in such areas as clinical pharmacology can
contribute greatly to the development of regulatory science as an accepted
discipline.
• egulatory science is more than just developing new methods for understand-
R
ing and assessing risk; it also includes consideration of cultural and societal
issues relating to how individual patients and society view the tradeoff between
reward and risk.
Different stakeholders in the therapeutics development enterprise
may have somewhat different perspectives on regulatory science. In
the opening session of the workshop, representatives of the four key
“locuses” of the development and practice of innovative regulatory sci -
ence for therapeutics development (FDA, NIH, the pharmaceutical indus-
try, and academia) described these perspectives. All four emphasized the
importance of developing regulatory science as an academic discipline
to address the multiple challenges they face. At the same time, different
perspectives create opportunities for productive collaboration, though
5
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6 STRENGTHENING A WORKFORCE FOR INNOVATIVE REGULATORY SCIENCE
as some participants cautioned, also potentially create opportunities for
misunderstandings.
Vicki Seyfert-Margolis, Senior Advisor for Science Innovation and
Policy, Office of the Commissioner, FDA, presented FDA’s rationale for
regulatory science and the strategic plan the agency has developed to pur-
sue this science. Story Landis, Director, National Institute of Neurological
Disorders and Stroke (NINDS), NIH, described some of the collaboration
ongoing between FDA and NIH. Andrew Dahlem, Vice President and
Chief Operating Officer, Lilly Research Laboratories, Eli Lilly & Co., sum-
marized some of the challenges and needs facing industry in a radically
changed drug development environment. Ralph Snyderman, Chancellor
Emeritus, Duke University, discussed therapeutics development in the
broader context of personalized medicine. Ellen Sigal, Chair and Founder,
Friends of Cancer Research, provided a patient perspective on the impor-
tance of regulatory science and moderated a panel discussion among the
keynote speakers.
PERSPECTIVE FROM THE FOOD AND DRUG ADMINISTRATION1
Addressing the Product Development Ecosystem
Seyfert-Margolis noted that FDA can bring data, insight, and knowl -
edge to the therapeutics development ecosystem. In the months preceding
the workshop, FDA had been examining the medical products ecosystem
to determine how the agency can facilitate innovation. The agency’s inno-
vation initiative will include regulatory science as a key component.2
According to Seyfert-Margolis, a major finding from this review was that
all of the various players in this arena are experiencing new stresses and
challenges. The scientific and global market environments are moving
away from the traditional blockbuster drug development model. In aca-
1 This section is based on the presentation by Vicki Seyfert-Margolis, Senior Advisor for
Science Innovation and Policy, Office of the Commissioner, FDA.
2 FDA released a report on innovation, Driving Biomedical Innovation: Initiatives to Improve
Products for Patients, on October 5, 2011. The report defines the “medical product ecosystem”
in the following context:
Translating a new idea from a discovery into a medical product is a complex process
involving an entire ecosystem consisting of academia, industry, small businesses,
payors, physicians, government agencies, and patient and consumer groups. Each
member of the ecosystem has an important role to play in bringing a new medical
product to market, and each piece of the ecosystem is currently under stress, putting
America at risk of losing its competitive edge as the leader in scientific innovation.
For more information, see http://www.fda.gov/AboutFDA/ReportsManualsForms/
Reports/ucm274333.htm (accessed November 28, 2011).
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THE IMPORTANCE OF INNOVATIVE REGULATORY SCIENCE
demia, the demands of tenure and recognition do not always align well
with the team-oriented imperatives of clinical research and development.
At the same time, academics confront difficulties in moving their ideas
into the clinic given the challenges of securing funding.
A number of interesting models are emerging to deal with these
challenges, said Seyfert-Margolis. Some of these models are being sup -
ported directly by academic institutions, while others involve venture
philanthropy. Small business plays an important role in the product
development ecosystem, but small businesses today are often under-
capitalized and are struggling with the complexities of the therapeutic
product development cycle. FDA has been engaged in discussions with
companies along the size spectrum—from small biotechnology to large
pharmaceuticals—to provide advice on how to move ideas forward suc -
cessfully. Seyfert-Margolis noted that the calculus of risks and benefits is
an important discussion to have as a scientific community and as a society
and needs to be taken into account in consideration of potential new regu-
latory pathways such as conditional or progressive approval strategies.
Physicians and patients have their own concerns regarding new tech -
nologies and therapeutics. Society’s tolerance for risk is low, Seyfert-
Margolis observed, so it is important to communicate information and
concepts about risks and rewards more effectively to patients and physi -
cians. Payers are focusing on the concepts of evidence- and value-based
medicine to align their reimbursement structures with the actual real-
world performance of drugs, which is usually less than that seen in clini -
cal trials. Once in the real world, the risk equation shifts as comorbidities
and other influences on drug performance become apparent.
The Rationale for Regulatory Science
According to Seyfert-Margolis, there are four major reasons why reg-
ulatory science is needed.
First, major investments and advances in basic sciences are not fully
translating into products to benefit patients. In part, this is because of the
so-called “valley of death” (Figure 2-1). The valley of death refers to
the gap in funding between, on the one hand, NIH, state, and foundation
grants that support discovery, preclinical development, and the earliest
stages of clinical development and, on the other hand, pharmaceutical
company involvement that supports the later stages of clinical develop -
ment and commercialization. This funding gap has a critical, negative
impact on the translation of discoveries into medical products and new
medicines. Seyfert-Margolis emphasized that regulatory science can play
an important role in helping to bridge this gap.
Second, product development is increasingly costly and uncertain
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www.biomedcentral.com - Figure 1/11/12 6:32
8 STRENGTHENING A WORKFORCE FOR INNOVATIVE REGULATORY SCIENCE
Figure 3. Resolution: standard / high
Cash flow 'Valley of Death' diagram. The cash flow 'Valley of Death' as a function of development stage (time)
FIGURE 2-1 Many discoveries stages (adapted from [12]). RAID, of death” from dis-
with typical funding sources at various fail to traverse the “valley Rapid Access to Interventional
covery to commercial product.Innovative Research; RAPID, Rapid Access to Preventive Intervention
Development; SBIR, Small Business
Development; STTR, Small Business Technology Transfer.
NOTE: RAID, Rapid Access to Interventional Development; RAPID, Rapid Access
o Preventive Intervention 2009 9(Suppl 1):S2 doi:10.1186/1471-2377-9-S1-S2
tSteinmetz and Spack BMC NeurologyDevelopment; SBIR, Small Business Innovative Re -
search; STTR, Small Business Technology Transfer; U01, Cooperative Agreement
Research Project Awards.
SOURCE: Seyfert-Margolis, 2011. Presentation at IOM workshop on Strengthen -
ing a Workforce for Innovative Regulatory Science in Therapeutics Development.
while success rates remain low. For example, according to Seyfert-Margolis,
the need for cost containment has driven clinical trials to other countries;
now, approximately 50 percent of the clinical trials data included in regula-
tory submissions to FDA is generated in India or China. These populations
differ from the average U.S. population in terms of nutrition, genetics, envi-
ronment, lifestyle considerations, and other factors. As a result, data from
those populations may not fully translate into the average U.S. population,
said Seyfert-Margolis, while noting that this issue needs further study. FDA
file:///Users/cbaum/Desktop/www.biomedcentral.com%20-%20Figure.webarchive Page 1 o
believes that uncertainties associated with differences among populations
lie more in the scientific than in the policy realm, pointing to the need for
regulatory science.
Third, development and evaluation tools and approaches have not
kept pace with and have not incorporated emerging technologies. Exam -
ples include the new fields of nanotechnology, where tools to evaluate
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9
THE IMPORTANCE OF INNOVATIVE REGULATORY SCIENCE
the safety and toxicity of nanoscale products and genetically engineered
foods are lacking. Shortcomings in scientific capacity in this area also
affect the ability of FDA to educate and communicate with the public
about the risks and benefits of new technologies.
Fourth, without regulatory science the economic health of the bio-
technology and medical products industry is at risk. This is of particular
concern given the importance to the U.S. economy of maintaining and
building on the nation’s leadership role in this important technological
sector.
FDA now has several programs in place, including a formal partner-
ship with NIH, to advance regulatory science. For the first time in its his -
tory, FDA has a budgetary line item for regulatory science and has issued
direct funding solicitations for projects in reproductive toxicology and
biomarker research and qualification.
The FDA Strategic Plan for Regulatory Science
FDA has issued a strategic plan for regulatory science that was crafted
with input from all of its centers yet takes an agency-wide, rather than
center-specific, perspective (FDA, 2011). The strategic plan’s vision state -
ment says that “FDA will advance regulatory science to speed innovation,
improve regulatory decision-making, and get safe and effective products
to people in need. Twenty-first-century regulatory science will be a driv -
ing force as FDA works with diverse partners to protect and promote the
health of our nation and the global community.”
The strategic plan lays out eight priority areas:
1. Modernize toxicology to enhance product safety.
2. Stimulate innovation in clinical evaluation and personalized
medicine.
3. Support new approaches to improve product manufacturing and
quality.
4. Ensure FDA readiness to evaluate emerging technologies.
5. Harness diverse data through information sciences to improve
health outcomes.
6. Enable a prevention-focused food safety system.
7. Facilitate development of medical countermeasures (MCMs) to
protect U.S. and global health and security.
8. Strengthen social and behavioral science to help consumers and
professionals make informed decisions.
Seyfert-Margolis briefly described FDA’s intentions in each of these
eight areas (see Box 2-1).
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10 STRENGTHENING A WORKFORCE FOR INNOVATIVE REGULATORY SCIENCE
BOX 2-1a
FDA Strategic Plan for Regulatory Science
Modernize toxicology to enhance product safety. FDA plans to develop better
models of human adverse response, identify and evaluate biomarkers and end
points that can be used in nonclinical and clinical evaluations, and use and develop
computational methods and in silico modeling.
Stimulate innovation in clinical evaluation and personalized medicine. FDA
seeks to develop and refine clinical trial designs, end points, and analysis methods;
leverage existing and future clinical trial data; identify and qualify biomarkers and
study end points; increase the accuracy and consistency, and reduce interplatform
variability, of analytical methods to measure biomarkers; and develop a “virtual
physiologic patient.”
Support new approaches to improve product manufacturing and quality. FDA
plans to enable development and evaluation of novel and improved manufactur-
ing methods, develop new analytical methods, and reduce the risk of microbial
contamination of products.
Ensure FDA readiness to evaluate emerging technologies. FDA will stimulate
development of innovative medical products while concurrently developing novel
assessment tools and methodologies, develop assessment tools for novel thera-
pies, assure safe and effective medical innovation, and coordinate regulatory sci-
ence for emerging technology product areas.
Harness diverse data through information sciences to improve health out -
comes. FDA plans to enhance information technology infrastructure development
and data mining; develop and apply simulation models for product life cycles, risk
assessment, and other regulatory science uses; analyze large-scale clinical and
preclinical data sets; incorporate knowledge from FDA regulatory files into a data-
base integrating a broad array of data types; and develop new data sources and
innovative analytical methods and approaches.
Enable a prevention-focused food safety system. The agency will establish
and implement centralized planning and performance measurement processes,
improve information sharing internally and externally, maintain mission-critical sci-
ence capabilities, and cultivate expert institutional knowledge.
Facilitate development of MCMs to protect U.S. and global health and security.
FDA will develop, characterize, and qualify animal models for MCM development;
modernize tools to evaluate MCM product safety, efficacy, and quality; devel-
op and qualify biomarkers of diseases or conditions; and enhance emergency
communication.
Strengthen social and behavioral science to help consumers and professionals
make informed decisions. FDA seeks to know its audience, reach that audience,
ensure audience understanding, and evaluate the effectiveness of communication
about regulated products.
a Based on the presentation by Vicki Seyfert-Margolis, Senior Advisor for Science Inno-
vation and Policy, Office of the Commissioner, FDA, which drew directly from FDA’s recent
strategic plan for regulatory science (FDA, 2011).
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THE IMPORTANCE OF INNOVATIVE REGULATORY SCIENCE
Seyfert-Margolis also noted that applications were under review for a
new National Capitol Region Center for Excellence in Regulatory Science
and Innovation (CERSI). The goal of the new center is “to advance the
field of regulatory science (including laboratory, population, behavioral,
and manufacturing sciences) and the Critical Path Initiative toward more
effective and efficient product development and evaluation. CERSI efforts
will focus on promoting innovation in support of the development and
evaluation of safe and effective products through training, applied col -
laborative science, professional development and scientific exchanges.” 3
PERSPECTIVE FROM THE NATIONAL INSTITUTES OF HEALTH4
Landis illustrated the promise of regulatory science through the case
example of advancements over the past 15 years in the development of
human embryonic stem cells and new techniques allowing dedifferentia -
tion and differentiation, with extraordinary potential for application to
regenerative medicine. She commented that there is a need for regula -
tory science in thinking about how to understand, regulate, and enable
clinical trials using human embryonic stem cells. She noted that FDA has
embraced this innovative technology and enabled it to move forward,
with three clinical trials currently being conducted in the United States.
Landis also described an innovative new initiative in which NIH
is partnering with FDA and the Defense Advanced Research Projects
Agency (DARPA) to develop embryonic stem cells as microphysiological
systems—organs on a chip—that could be used instead of laboratory ani -
mals to screen for safe and effective drugs. This partnership has received
5 years of funding worth $140 million, a substantial investment that
addresses goals of modernizing toxicology and adopting new innovative
technologies.
3 According to the CERSI Request for Application (RFA), the CERSIs should be aca -
demic, M.D. and Ph.D. degree–granting institutions with both strong life science and
clinical medical science activities. FDA also specified that they should be located within
a 50-mile radius of FDA’s Silver Spring, Maryland, campus to facilitate and enable train -
ing, research, scientific exchanges, and other collaboration among CERSI institution staff,
students, and trainees and FDA staff scientists. See http://grants.nih.gov/grants/guide/
rfa-files/RFA-FD-11-033.html (accessed November 28, 2011). The CERSI awards, totaling
$2 million, were announced on October 26, 2011. The centers, which will be located at
the University of Maryland and Georgetown University, will focus on strengthening sci -
ence and training needed to modernize and improve the ways drugs and medical devices
are reviewed and evaluated. See http://www.fda.gov/NewsEvents/Newsroom/Press
Announcements/ucm277267.htm (accessed November 28, 2011).
4 This section is based on the presentation by Story Landis, Director, NINDS, NIH. Landis
also serves as one of the co-chairs of NIH’s regulatory science initiative and is a member of
the NIH-FDA Joint Leadership Council.
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12 STRENGTHENING A WORKFORCE FOR INNOVATIVE REGULATORY SCIENCE
NIH-FDA Collaborative Efforts and Regulatory Science Funded Projects
Landis noted that the NIH and FDA have a history of innovations in
therapeutics development, for example, the creation of the exploratory
Investigational New Drug (IND) application. The exploratory IND came
about through a partnership between the National Cancer Institute (NCI)
and FDA and aimed to address the fact that the behavior of anticancer
agents seen in animal models does not translate well into humans by
developing innovative strategies to screen possible therapeutic candidates
for cancer in humans. The exploratory IND is designed to inform deci-
sion making on target selection and dose. It assesses pharmacokinetics,
biodistribution, and drug engagement with the appropriate target using
very small doses of drug and with no therapeutic intent.
The formation of the NIH-FDA Joint Leadership Council in 2010
greatly enhanced the partnership between NIH and FDA, Landis noted.
The council has six working groups that review proposals for collabora-
tion in areas that include regulatory science, training, and education.
In addition, the leadership council created cooperative research grants
to advance translational regulatory science, and four projects totaling
approximately $9 million over 3 years have been funded under this
initiative:
• Creating an organ on a chip that functions as a heartlung micro-
machine model to test the safety and efficacy of drugs
• Designing innovative, adaptive clinical trials for evaluating drugs
and devices used in the emergency care of patients with acute
neurological illness or injury
• Developing an understanding of how nanoparticles interact with
the complement system and developing a model that can predict
which nanoparticles might activate the complement cascade
• Developing a novel strategy to predict ocular irritancy
NCATS and NIH Training Initiatives
The NIH-proposed National Center for Advancing Translational
Sciences (NCATS), if appropriated and stood up, would assume respon-
sibility for the agency’s regulatory science initiatives, in a shift from the
distributed function currently residing in NINDS and the National Insti-
tute of Diabetes and Digestive and Kidney Diseases.5 The vision and mis-
sion of NCATS have evolved over the year that the proposed new center
5 NCATS was formally established by Congress in the FY 2012 Omnibus Appropriations
Bill, signed into law on December 23, 2011 (after the date of the workshop). The new center
has a budget of $575 million.
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THE IMPORTANCE OF INNOVATIVE REGULATORY SCIENCE
has been in the planning stages; although there may have initially been a
notion that NCATS was envisioned as the “center for cures,” as the mis-
sion has been clarified, the center will be focused on “the science of how
you do translation better.” The center will be charged with advancing the
discipline of translational science to catalyze the development and test-
ing of novel diagnostics and therapeutics across a broad range of diseases
and conditions. In this pursuit the proposed NCATS will approach the
drug development pipeline as a “scientific problem ripe for intervention,
experimentation, and process engineering, looking for bottlenecks and
creating solutions.” NCATS will also have a training function in transla-
tional science, including clinical pharmacology.
Landis noted that NIH currently carries out a training function
through the institutes and centers. The broad training environment cre -
ated by NIH-funded programs operated by the institutes and centers
represents a real opportunity for producing scientists and physician-
scientists who can think about regulatory science, she said. She cited three
examples of training programs of relevance to regulatory science:
• Pre and postdoctoral fellows programs that often feed trainees
into positions at FDA
• Programs in the National Institute for General Medical Sciences
for physician-scientists, together with institute-specific initiatives
targeted at physician-scientists
• The Clinical and Translational Science Awards (CTSAs) (currently
residing within the National Center for Research Resources; slated
to move to the new NCATS)
PERSPECTIVE FROM THE PHARMACEUTICAL INDUSTRY6
Views about the division of labor among NIH, academia, and indus-
try in drug discovery and development have changed dramatically over
the past 20 years, observed Dahlem. Historically, NIH has had responsi -
bility for basic research, academia discovered potential new targets, and
industry looked for the commercial potential of those targets, with FDA
providing regulatory oversight of new drug applications (NDAs). In a
shift, today, Dahlem said, NIH has become increasingly interested in drug
discovery and development, while researchers in academia have joined
their industry colleagues in searching for new drugs rather than limiting
their pursuits to basic discovery.
6 This section is based on the presentation by Andrew Dahlem, Vice President and Chief
Operating Officer, Lilly Research Laboratories, Eli Lilly & Co.
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14 STRENGTHENING A WORKFORCE FOR INNOVATIVE REGULATORY SCIENCE
Challenges Faced by Industry
Dahlem noted that drug development expenditures have increased
while approvals have decreased. The number of drugs that will go off
patent is increasing, which will cause companies to become even more
cost-conscious in the coming years. This will affect not only industry hir-
ing (and contribute to the shedding of jobs) but also the amount of money
industry will provide to academia to support research. This evolving
framework provides a backdrop for thinking about the workforce needs
and capacity.
Industry is acting to reduce the failure rate of drugs in the develop -
ment pipeline, said Dahlem. For example, in 1988, nearly 40 percent of
Lilly’s drugs failed because of pharmacokinetic problems; today that
number has been reduced to about 3 percent through advancements in
science. Now, the most common reason for a drug to fail in clinical trials
is that data from animal models are not good enough predictors of suc -
cess in humans.
Dahlem also noted that, in any exercise to define and ascertain how
to develop a workforce, one must keep in mind that, from any student’s
perspective, a training program must provide opportunities that allow
trainees to generate sufficient income to repay debts assumed during
education and training.
Dahlem provided several observations about industry needs for the
regulatory science process. First, industry needs timely and predictable
reviews of NDAs, which could be achieved through enhanced collabo-
ration among industry, academia, and FDA. Second, Dahlem called for
unbiased third-party assessments of benefits and risks, especially when
drug candidates move from the preclinical setting to human clinical trials.
Such a mechanism could address real and perceived biases that may color
industry assessment of risk and benefit. Third, harmonization of global
regulatory expectations is also a critical need, as industry files for regula-
tory approvals around the world. Fourth, industry needs researchers and
regulators who are trained to make both science- and judgment-based
decisions. This is a particular concern today because of the increasing
number of experienced regulators and researchers—individuals who have
seen multiple iterations of problems and issues and who understand
not just the rules but potential exceptions to those rules that they have
uncovered over the course of their careers—who are retiring or leaving
the industry because of consolidations and mergers. Dahlem emphasized
that there is a critical need to capture the experiences of these individuals
and to convey, perhaps using information technology–based tools, their
experiences to new generations of researchers and regulators. Fifth, there
is a huge need to better educate the public about risks and benefits to
reduce public misperceptions about medications and vaccines.
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THE IMPORTANCE OF INNOVATIVE REGULATORY SCIENCE
Dahlem also listed specific scientific challenges that could be addressed
through collaborative regulatory science initiatives:
• Scientists who are trained to understand the preclinicaltoclinical
transition. In particular, he said, funding needs to be restored for
training scientists in the classical disciplines of whole-animal phar-
macology and physiology.
• Toxicology advances to provide better predictions of human clini-
cal outcomes. This will involve better selection of animal models to
ensure human safety, new methods to provide better correlations
between whole-animal studies and human disease outcomes, and
improved in silico modeling.
• In the clinical area, industry needs better clinical trial designs as
well as an improved understanding of clinical trial data, particu-
larly of the placebo effect. The biological origins of phenotypic
responses need to be better understood, as do the effects of drugs
in special populations.
PERSPECTIVE FROM ACADEMIA7
The magnitude of change that has occurred in medicine over the
past century is staggering, Snyderman observed. The first major trans-
formation occurred when science brought to medicine the understanding
that at least some diseases could be defined as being caused by specific
agents such as particular types of bacteria. This mechanism-based under-
standing of disease led to what he called the “find and fix it” philoso -
phy that currently guides medicine. Today, the application of genomics,
proteomics, metabolomics, systems biology, informatics, and micro- and
nanoprocessing is creating a “predict it and personalize it” philosophy,
one guided by understanding the complexity of both health and disease.
Also transformational has been the growing understanding that
all diseases develop over time. Some develop rapidly, but the major
chronic diseases typically develop over a long period of time. For those
diseases, intervention typically occurs late, when the underlying pathol-
ogy makes reversibility difficult and therapy expensive. Today, new
technologies are seeking to enable earlier interventions, before primary
symptoms develop. For example, genomics provides an understanding
of baseline risks through the interaction of biology with the environ -
ment. This new knowledge will lead to new products that will require
a new way of looking at regulation. FDA’s strategic plan for regulatory
7 This section is based on the presentation by Ralph Snyderman, Chancellor Emeritus,
Duke University.
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16 STRENGTHENING A WORKFORCE FOR INNOVATIVE REGULATORY SCIENCE
science is timely because it seeks to develop the regulatory science that
will be needed to deal with these new technologies and products, noted
Snyderman.
Snyderman reinforced the observation that, whereas 20 years ago
academic medical centers were focused largely on basic discovery work,
today scientists at these institutions participate in the entire range of drug
discovery and development activities. Moving forward, he said, these
activities will include regulatory science and the development of new
tools for measuring the safety and efficacy of not only drugs but also the
accompanying diagnostics that are being developed.
Snyderman analogized the development of a discipline of regula -
tory science to efforts to advance translational research and the conduct
of clinical trials within academic medical centers. There is now a need
to approach the regulatory sciences in a concerted, organized way, to
define the discipline and competencies associated with its conduct, and
to define it as an innovative science that is a valid career path for young
scientists.
Snyderman listed the following as necessary elements in the fostering
of the regulatory scientist-investigator:
• Recognition and role models within the academic institution
• Training and research training programs
• Appropriate tenure tracks
• Research infrastructure where scientists reside
• Means of support, including federal funding
• Respect of their colleagues—regulatory science being viewed as
“worthwhile”
Snyderman made the following recommendations for the develop-
ment and advancement of regulatory science:
• Recognize it as a discipline
• Define the discipline
• Define the qualifications
• Define educational needs
• Create academic homes and promotion/tenure tracks
PATIENT PERSPECTIVE8
Sigal reminded the workshop participants that the ultimate respon -
sibility of regulatory science is to meet the needs of the patient. Among
8 This section is based on remarks given by Ellen Sigal, Chair and Founder, Friends of
Cancer Research.
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17
THE IMPORTANCE OF INNOVATIVE REGULATORY SCIENCE
the questions patients ask are the following: Will this new drug work for
me? What are my risks? Sigal also noted that, until recently, FDA was not
perceived to be a science-based agency. It had the burden of approving
drugs and diagnostics, but it did not have the ability to do the science
needed to guide decision making for the benefit of patients, which was
instead assumed to be the province of NIH.
Sigal remarked that a greater sense of urgency is needed to reinforce
that regulatory science is crucial to delivering therapies for patients. She
remarked that the agency has not had sufficient resources to accomplish
the science needs and added that the resources that are needed to advance
regulatory science include not just a budget line item but also a sup -
portive scientific ecosystem to continue the collaborative advancements,
notwithstanding a heavily resource-constrained environment.
PRINCIPLES AND THEMES
In a panel discussion, the keynote speakers and audience members
identified what they saw as the key needs for strengthening regulatory
science. The discussion included individual observations about the prin -
cipal barriers as well as the most promising opportunities. This section
provides an integrated summary of their remarks and discussions with
workshop participants during the panel and should not be construed as
reflecting consensus or endorsement by the workshop participants, the
planning committee, the Forum, or the National Academies:
• Among the biggest barriers to advancement of the science is a
shortage of experienced regulatory scientists and the lack of experi-
ence among the scientists now being trained.
• A key element of the practice of regulatory science is an under-
standing of societal and personal tolerance for risk and how society
and individuals experience the benefits of new drugs and technolo-
gies. Participants called for a more developed approach to benefit-
risk assessment that takes patient perspectives into account.
• It is important for FDA scientists to be able to participate as sci-
entists in academic settings or at NIH. It also is important that
academic and NIH scientists be allowed and encouraged to work
at FDA.
• Having an academic culture that views regulatory science as an
inherently collaborative enterprise and that recognizes the need
for development of people in academia to contribute to the science
would contribute greatly to development of the workforce.
• Safe harbors could support and protect collaborations not only
among the federal government and academia but also including
the pharmaceutical industry and patients.
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18 STRENGTHENING A WORKFORCE FOR INNOVATIVE REGULATORY SCIENCE
• Although progress has been made in providing opportunities for
continuing formal interactions between industry and FDA during
the drug development process, more work is needed to be done
to enhance and increase access to resources at FDA, including
increased informal interactions with FDA scientists and staff.
• Some participants called for definition as to what each sector (aca-
demia, industry, the agencies) needs to do to improve and enhance
the support and practice of regulatory science.
• A nextlevel, precise definition of regulatory science—and the com-
ponents of its practice—would spur academic medical centers to
create programs in this area. This exercise would include identifica-
tion of specific regulatory science competencies and definition of
collaborative mechanisms established to connect academic training
programs to relevant programs at NIH and FDA.
