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This issue of Surgical Oncology Clinics of North America, edited by
Adam Berger, is devoted to Melanoma. Articles in this issue
include: Current Staging and Prognostic Factors in Melanoma;
Melanoma Pathology; Surgical Treatment of Primary and Recurrent
Melanoma; Sentinel Lymph Node Mapping and Its Importance for
Melanoma in the 21st Century; Lymph Node Dissection for Stage 3
Melanoma; Metastasectomy for Stage IV Melanoma; Local/Injectional
Therapies for Satellite and In-Transit Disease; Regional Therapies
for In-Transit Disease; Role for Radiation Therapy in Melanoma;
Update on Immunotherapy for Melanoma; Targeted Therapies in
Melanoma; and Evidence for Long-Term Follow-up of Melanoma
Patients.
Many health care providers do not have either the knowledge or the
tools they need in order to apply genetic information in their
day-to-day practices. This lack of support is contributing to a
substantial delay in the translation of genetic research findings,
when appropriate, into improvement in patient outcomes within the
health care system. Although the need to improve genetics knowledge
among health care providers is clear, the best approaches to
educating health care providers in a way that produces meaningful
changes in clinical practice are not, especially given the
competing coursework and training needs that exist in today's
increasingly complex health care settings. To examine the potential
and the challenges of providing genetics education, the Roundtable
on Translating Genomic-Based Research for Health of the Institute
of Medicine hosted a workshop on August 18, 2014. The workshop
examined a variety of approaches that could improve the teaching of
genetics in the graduate and continuing education of health
professionals; these approaches included online and interactive
instruction, just-in-time approaches, the development of clinical
decision-support tools, and the incorporation of genetics
requirements into licensing and accreditation. This report
summarizes the presentations and discussion of the event. Table of
Contents Front Matter 1 Introduction and Themes of the Workshop 2
Myths and Mistakes in Graduate and Continuing Medical Education 3
Educational Approaches 4 Graduate Health Professional Education and
Post-Graduate Training 5 Continuing Medical Education 6 Next Steps
to Achieve Effective Genetics Education for Health Professionals
References Appendix A: Workshop Agenda Appendix B: Speaker
Biographical Sketches Appendix C: Statement of Task Appendix D:
Registered Attendees
Many drug developers have examined new strategies for creating
efficiencies in their development processes, including the adoption
of genomics-based approaches. Genomic data can identify new drug
targets for both common and rare diseases, can predict which
patients are likely to respond to a specific treatment, and has the
potential to significantly reduce the cost of clinical trials by
reducing the number of patients that must be enrolled in order to
demonstrate safety and efficacy. A key component of the approval of
targeted therapeutics is the ability to identify the population of
patients who will benefit from treatment, and this has largely
hinged on the co-development and co-submission to the FDA of a
companion diagnostic test.The co-development process, or the
development of the test and drug for the simultaneous submission to
FDA, has led to a major alteration in the way that drugs are being
developed, with traditionally separate entities-pharmaceutical and
diagnostic companies-now working in close collaboration. Refining
Processes for the Co-Development of Genome-Based Therapeutics and
Companion Diagnostic Tests is the summary of a workshop held by the
Roundtable on Translating Genomic-Based Research for Health on
February 27, 2013 to examine and discuss challenges and potential
solutions for the codevelopment of targeted therapeutics and
companion molecular tests for the prediction of drug response.
Prior to the workshop, key stakeholders, including laboratory and
medical professional societies, were individually asked to provide
possible solutions to resolve the concerns raised about
co-development of companion diagnostic tests and therapies.
Workshop speakers were charged with addressing these solutions in
their presentations by providing insight on (1) whether the
proposed solutions address the problems described, (2) whether
there are other solutions to propose, and (3) what steps could be
taken to effectively implement the proposed solutions. Table of
Contents Front Matter 1 Introduction 2 Regulatory Perspectives 3
Perspectives from Patients, Providers, and Laboratory
Representatives 4 Perspectives of Diagnostic Test and
Pharmaceutical Developers 5 Perspectives of Payers and Regulators 6
Concluding Observations References Appendix A: Workshop Agenda
Appendix B: Speaker Biographical Sketches Appendix C: Statement of
Task Appendix D: Registered Attendees
Rapid advances in technology have lowered the cost of sequencing an
individual's genome from the several billion dollars that it cost a
decade ago to just a few thousand dollars today and have
correspondingly greatly expanded the use of genomic information in
medicine. Because of the lack of evidence available for assessing
variants, evaluation bodies have made only a few recommendations
for the use of genetic tests in health care. For example,
organizations, such as the Evaluation of Genomic Applications in
Practice and Prevention working group, have sought to set standards
for the kinds of evaluations needed to make population-level health
decisions. However, due to insufficient evidence, it has been
challenging to recommend the use of a genetic test. An additional
challenge to using large-scale sequencing in the clinic is that it
may uncover "secondary," or "incidental," findings - genetic
variants that have been associated with a disease but that are not
necessarily related to the conditions that led to the decision to
use genomic testing. Furthermore, as more genetic variants are
associated with diseases, new information becomes available about
genomic tests performed previously, which raises issues about how
and whether to return this information to physicians and patients
and also about who is responsible for the information. To help
develop a better understanding of how genomic information is used
for healthcare decision making, the Roundtable on Translating
Genomic-Based Research for Health of the Institute of Medicine held
a workshop in Washington,DC in February 2014. Stakeholders,
including clinicians, researchers, patients, and government
officials, discussed the issues related to the use of genomic
information in medical practice. Assessing Genomic Sequencing
Information for Health Care Decision Making is the summary of that
workshop. This report compares and contrasts evidence evaluation
processes for different clinical indications and discusses key
challenges in the evidence evaluation process. Table of Contents
Front Matter 1 Introduction 2 How Evidence Is Gathered and
Evaluated 3 Patient Care and Health Decisions 4 The Development of
Practice Guidelines 5 How Insurers Decide Whether to Pay for
Testing 6 Addressing Challenges References Appendix A: Workshop
Agenda Appendix B: Speaker Biographical Sketches Appendix C:
Statement of Task Appendix D: Registered Attendees
Drug development can be time-consuming and expensive. Recent
estimates suggest that, on average, it takes 10 years and at least
$1 billion to bring a drug to market. Given the time and expense of
developing drugs de novo, pharmaceutical companies have become
increasingly interested in finding new uses for existing drugs - a
process referred to as drug repurposing or repositioning.
Historically, drug repurposing has been largely an unintentional,
serendipitous process that took place when a drug was found to have
an offtarget effect or a previously unrecognized on-target effect
that could be used for identifying a new indication. Perhaps the
most recognizable example of such a successful repositioning effort
is sildenafil. Originally developed as an anti-hypertensive,
sildenafil, marketed as Viagra and under other trade names, has
been repurposed for the treatment of erectile dysfunction and
pulmonary arterial hypertension. Viagra generated more than $2
billion worldwide in 2012 and has recently been studied for the
treatment of heart failure. Given the widespread interest in drug
repurposing, the Roundtable on Translating Genomic-Based Research
for Health of the Institute of Medicine hosted a workshop on June
24, 2013, in Washington, DC, to assess the current landscape of
drug repurposing activities in industry, academia, and government.
Stakeholders, including government officials, pharmaceutical
company representatives, academic researchers, regulators, funders,
and patients, were invited to present their perspectives and to
participate in workshop discussions. Drug Repurposing and
Repositioning is the summary of that workshop. This report examines
enabling tools and technology for drug repurposing; evaluates the
business models and economic incentives for pursuing a repurposing
approach; and discusses how genomic and genetic research could be
positioned to better enable a drug repurposing paradigm. Table of
Contents Front Matter 1 Introduction and Themes of the Workshop 2
The State of the Science 3 Enabling Tools and Technology 4 Value
Propositions for Drug Repurposing 5 Policy Approaches and Legal
Framework 6 Increasing the Efficiency and Success of Repurposing
References Appendix A: Workshop Agenda Appendix B: Speaker
Biographical Sketches Appendix C: Statement of Task Appendix D:
Registered Attendees
Stem cells offer tremendous promise for advancing health and
medicine. Whether being used to replace damaged cells and organs or
else by supporting the body's intrinsic repair mechanisms, stem
cells hold the potential to treat such debilitating conditions as
Parkinson's disease, diabetes, and spinal cord injury. Clinical
trials of stem cell treatments are under way in countries around
the world, but the evidence base to support the medical use of stem
cells remains limited. Despite this paucity of clinical evidence,
consumer demand for treatments using stem cells has risen, driven
in part by a lack of available treatment options for debilitating
diseases as well as direct-to-consumer advertising and public
portrayals of stem cell-based treatments. Clinics that offer stem
cell therapies for a wide range of diseases and conditions have
been established throughout the world, both in newly industrialized
countries such as China, India, and Mexico and in developed
countries such as the United States and various European nations.
Though these therapies are often promoted as being established and
effective, they generally have not received stringent regulatory
oversight and have not been tested with rigorous trials designed to
determine their safety and likely benefits. In the absence of
substantiated claims, the potential for harm to patients - as well
as to the field of stem cell research in general - may outweigh the
potential benefits. To explore these issues, the Institute of
Medicine, the National Academy of Sciences, and the International
Society for Stem Cell Research held a workshop in November 2013.
Stem Cell Therapies summarizes the workshop. Researchers,
clinicians, patients, policy makers, and others from North America,
Europe, and Asia met to examine the global pattern of treatments
and products being offered, the range of patient experiences, and
options to maximize the well-being of patients, either by
protecting them from treatments that are dangerous or ineffective
or by steering them toward treatments that are effective. This
report discusses the current environment in which patients are
receiving unregulated stem cell offerings, focusing on the
treatments being offered and their risks and benefits. The report
considers the evidence base for clinical application of stem cell
technologies and ways to assure the quality of stem cell offerings.
Table of Contents Front Matter 1 Introduction and Themes of the
Workshop 2 Stem Cell Therapies - Knowns and Unknowns 3 Patients'
Experiences 4 Comparative Regulatory and Legal Frameworks 5 The
Roles of Professional Societies 6 Moving Forward References
Appendix A: Workshop Agenda Appendix B: Speaker Biographical
Sketches Appendix C: Statement of Task Appendix D: Registered
Attendees Appendix E: Glossary
The sequencing of the human genome and the identification of links
between specific genetic variants and diseases have led to
tremendous excitement over the potential of genomics to direct
patient treatment toward more effective or less harmful
interventions. Still, the use of whole genome sequencing challenges
the traditional model of medical care where a test is ordered only
when there is a clear indication for its use and a path for
downstream clinical action is known. This has created a tension
between experts who contend that using this information is
premature and those who believe that having such information will
empower health care providers and patients to make proactive
decisions regarding lifestyle and treatment options. In addition,
some stakeholders are concerned that genomic technologies will add
costs to the health care system without providing commensurate
benefits, and others think that health care costs could be reduced
by identifying unnecessary or ineffective treatments. Economic
models are frequently used to anticipate the costs and benefits of
new health care technologies, policies, and regulations. Economic
studies also have been used to examine much more specific issues,
such as comparing the outcomes and cost effectiveness of two
different drug treatments for the same condition. These kinds of
analyses offer more than just predictions of future health care
costs. They provide information that is valuable when implementing
and using new technologies. Unfortunately, however, these economic
assessments are often limited by a lack of data on which to base
the examination. This particularly affects health economics, which
includes many factors for which current methods are inadequate for
assessing, such as personal utility, social utility, and patient
preference. To understand better the health economic issues that
may arise in the course of integrating genomic data into health
care, the Roundtable on Translating Genomic-Based Research for
Health hosted a workshop in Washington, DC, on July 17-18, 2012,
that brought together economists, regulators, payers, biomedical
researchers, patients, providers, and other stakeholders to discuss
the many factors that may influence this implementation. The
workshop was one of a series that the roundtable has held on this
topic, but it was the first focused specifically on economic
issues. The Economics of Genomic Medicine summarizes this workshop.
Table of Contents Front Matter 1 Introduction and Overview 2
Genomics, Population Health, and Technology 3 The Intersection of
Genomics and Health Economics 4 Preconception Care and Sequencing 5
Unprovoked Deep Vein Thrombosis 6 Cancer Care 7 Panelists' and
Stakeholders' Perspectives References Appendix A: Workshop Agenda
Appendix B: Speaker Biographical Sketches Appendix C: Statement of
Task Appendix D: Registered Attendees
The process for translating basic science discoveries into clinical
applications has historically involved a linear and lengthy
progression from initial discovery to preclinical testing,
regulatory evaluation and approval, and, finally, use in clinical
practice. The low rate of translation from basic science to
clinical application has been a source of frustration for many
scientists, clinicians, investors, policy makers, and patients who
hoped that investments in research would result in improved
products and processes for patients. Some feel that the anticipated
deliverables from the Human Genome Project have not yet
materialized, and although understanding of human health and
disease biology has increased, there has not been a concomitant
increase in the number of approved drugs for patients over the past
10 years. Improving the Efficiency and Effectiveness of Genomic
Science Translation is the summary of a workshop convened by the
Institute of Medicine Roundtable on Translating Genomic-Based
Research for Health in December 2012 to explore ways to improve the
efficiency and effectiveness of the translation of genomic science
to clinical practice. The workshop convened academic researchers,
industry representatives, policy makers, and patient advocates to
explore obstacles to the translation of research findings to
clinical practice and to identify opportunities to support
improvement of the early stages of the process for translation of
genetic discoveries. This report discusses the realignment of
academic incentives, the detection of innovative ways to fund
translational research, and the generation or identification of
alternative models that accurately reflect human biology or disease
to provide opportunities to work across sectors to advance the
translation of genomic discoveries. Table of Contents Front Matter
1 Introduction 2 Connecting Basic Research and Health Care Needs 3
Moving Basic Science Forward 4 Industry and Venture Capital 5 Role
of Advocacy in Facilitating Translation of Basic Scientific
Research 6 Strategies for Change References Appendix A: Workshop
Agenda Appendix B: Speaker Biographical Sketches Appendix C:
Statement of Task Appendix D: Registered Attendees
Genome-Based Diagnostics: Demonstrating Clinical Utility in
Oncology is the summary of a workshop convened in May 2012 by the
Roundtable on Translating Genomic-Based Research for Health and the
Center for Medical Technology Policy of the Institute of Medicine
to foster the identified need for further sustained dialogue
between stakeholders regarding the clinical utility of molecular
diagnostics. The workshop brought together a wide range of
stakeholders, including patients, health care providers, policy
makers, payers, diagnostic test developers, researchers, and
guideline developers, to identify the challenges and opportunities
in advancing the development and use of molecular diagnostic tests
designed to guide the treatment and management of patients with
cancer. The sequencing of the human genome has greatly accelerated
the process of linking specific genetic variants with disease.
These findings have yielded a rapidly increasing number of
molecular diagnostic tests designed to guide disease treatment and
management. Many of these tests are aimed at determining the best
treatments for specific forms of cancer, making oncology a valuable
testing ground for the use of molecular diagnostic tests in
medicine in general. Nevertheless, many questions surround the
clinical value of molecular diagnostic tests, and their acceptance
by clinicians, payers, and patients has been unpredictable. A major
limiting factor for the use of these tests has been the lack of
clear evidence of clinical utility. Genome-Based Diagnostics
assesses the evidentiary requirements for clinical utility of
molecular diagnostics used to guide treatment decisions for
patients with cancer; discusses methodologies related to
demonstrating these evidentiary requirements that meet the needs of
all stakeholders; and considers innovative, sustainable research
collaborations for generating evidence of clinical utility
involving multiple stakeholders. Table of Contents Front Matter 1
Introduction 2 Setting the Context 3 Perspectives from Stakeholders
4 Tools for Generating and Synthesizing Evidence 5 Advancing
Molecular Diagnostics for Oncology References Appendix A: Workshop
Agenda Appendix B: Speaker Biographical Sketches Appendix C:
Statement of Task Appendix D: Registered Attendees
The number of new drug approvals has remained reasonably steady for
the past 50 years at around 20 to 30 per year, while at the same
time the total spending on health-related research and development
has tripled since 1990. There are many suspected causes for this
trend, including increases in regulatory barriers, the rising costs
of scientific inquiry, a decrease in research and development
efficiency, the downstream effects of patient expirations on
investment, and the lack of production models that have
successfully incorporated new technology. Regardless, this
trajectory is not economically sustainable for the businesses
involved, and, in response, many companies are turning toward
collaborative models of drug development, whether with other
industrial firms, academia, or government. Introducing greater
efficiency and knowledge into these new models and aligning
incentives among participants may help to reverse the trends
highlighted above, while producing more effective drugs in the
process. Genome-Based Therapeutics explains that new technologies
have the potential to open up avenues of development and to
identify new drug targets to pursue. Specifically, improved
validation of gene-disease associations through genomics research
has the potential to revolutionize drug production and lower
development costs. Genetic information has helped developers by
increasing their understanding of the mechanisms of disease as well
as individual patients' reactions to their medications. There is a
need to identify the success factors for the various models that
are being developed, whether they are industry-led, academia-led,
or collaborations between the two. Genome-Based Therapeutics
summarizes a workshop that was held on March 21, 2012, titled New
Paradigms in Drug Discovery: How Genomic Data Are Being Used to
Revolutionize the Drug Discovery and Development Process. At this
workshop the goal was to examine the general approaches being used
to apply successes achieved so far, and the challenges ahead. Table
of Contents Front Matter 1 Introduction 2 The Current Landscape 3
Case Studies 4 Emerging Technologies in Drug Development 5 Evolving
Paradigms 6 Forging Collaborative Strategies for the Development of
Personalized Medicine References Appendix A: Workshop Agenda
Appendix B: Speaker Biographical Sketches Appendix C: Statement of
Task Appendix D: Registered Attendees
The sequencing of the human genome and the identification of
associations between specific genetic variants and diseases have
led to an explosion of genomic-based diagnostic tests. These tests
have the potential to direct therapeutic interventions, predict
risk or onset of disease, or detect residual disease. As research
progresses and an increasing number of associations are found,
further tests will be developed that can aid in providing
personalized treatment options for patients. However, the adoption
of genomic diagnostic tests by health care providers has been
limited due to a lack of evidence regarding the clinical utility of
many tests. Health funders and practitioners lack the data
necessary to distinguish which tests can improve practice or the
clinical settings in which tests will provide the greatest value.
The Roundtable on Translating Genomic-Based Research for Health
held a workshop in November 2010 to determine what evidence is
needed and how it is viewed by different stakeholders in order to
develop genomic diagnostic tests of clinical value. Genome-Based
Diagnostics summarizes the presentations and discussions that took
place throughout the workshop. Two presentations, in particular,
sparked extensive discussion. One presentation proposed that all
genomic diagnostic tests be reviewed and approved by the Food and
Drug Administration. The other observed that venture capitalists
are no longer investing substantially in the development of genomic
diagnostic tests because of a lack of clarity surrounding
regulatory and reimbursement pathways. Both presentations suggested
the need for major changes in the systems used to develop,
regulate, and reimburse genomic diagnostic tests. The report also
presents the perspectives of different stakeholders in the
development of genomic diagnostic tests. Each stakeholder group has
a different set of needs and issues of importance, yet
commonalities among them are apparent, such as the need to put
patients and health outcomes at the center of discussion and
action. Table of Contents Front Matter 1 Introduction 2 Calls for
Change 3 Test Developers 4 Patients 5 Payers 6 Regulation,
Reimbursement, and Public Health 7 Discussion of Major Proposals
References Appendix A: Workshop Agenda Appendix B: Speaker
Biographical Sketches Appendix C: Statement of Task Appendix D:
Registered Attendees
Despite the many basic research discoveries in genetics, relatively
few gene-based treatments, drugs, or preventative measures have
been developed. One way to bridge this gap may be for industry,
academia, and government to develop partnerships that share
resources while distributing risk. However, intellectual property
protections and other barriers can inhibit collaborative efforts.
The Institute of Medicine held a workshop on July 22, 2010, to
explore these issues and develop solutions. Table of Contents Front
Matter 1 Introduction 2 A Lesson About Precompetitive Collaboration
3 Requisites for Successful Precompetitive Collaboration 4
Frameworks for Collaboration 5 The Use of Biospecimens in
Precompetitive Collaborations 6 Ethical Challenges in the Use of
Biospecimens 7 Toward Developing a Cultural, Legal, and Behavioral
Framework for Precompetitive Collaboration References Appendix A:
Workshop Agenda Appendix B: Speaker Biographical Sketches
Newborn screening samples are used to test more than 4 million
infants each year for life-threatening diseases that are treatable
if found at birth. These specimens also represent a potentially
invaluable resource for public health and biomedical research. The
IOM held a workshop to examine issues surrounding the use of
residual specimens for translational research.
Knowing one's genetic disposition to a variety of diseases,
including common chronic diseases, can benefit both the individual
and society at large. The IOM's Roundtable on Translating
Genomic-Based Research for Health held a workshop on March 22,
2010, to bring together diverse perspectives on the value of
genetic testing, and to discuss its use in clinical practice. Table
of Contents Front Matter 1 Introduction 2 Tumor-Based Screening for
Lynch Syndrome 3 Pharmacogenomic Testing to Guide Warfarin Dosing 4
Genomic Profiling 5 Closing Remarks References Appendix A: Workshop
Agenda Appendix B: Speaker Biographical Sketches Appendix C: Lynch
Syndrome Topic Brief Appendix D: Warfarin Topic Brief Appendix E:
Genomic Profiling Topic Brief
Scientific advances such as the sequencing of the human genome have
created great promise for improving human health by providing a
greater understanding of disease biology and enabling the
development of new drugs, diagnostics, and preventive services.
However, the translation of research advances into clinical
applications has so far been slower than anticipated. This is due
in part to the complexity of the underlying biology as well as the
cost and time it takes to develop a product. Pharmaceutical
companies are adapting their business models to this new reality
for product development by placing increasing emphasis on
leveraging alliances, joint development efforts, early-phase
research partnerships, and public-private partnerships. These
collaborative efforts make it possible to identify new drug
targets, enhance the understanding of the underlying basis of
disease, discover novel indications for the use of already approved
products, and develop biomarkers for disease outcomes or directed
drug use. While the potential benefits of collaboration are
significant, the fact that the relationships among development
partners are often financial means that it is vital to ensure trust
by identifying, disclosing, and managing any potential sources of
conflict that could create bias in the research being performed
together. Conflict of Interest and Medical Innovation is the
summary of a workshop convened by the Institute of Medicine
Roundtable on Translating Genomic-Based Research for Health in June
2013 to explore the appropriate balance between identifying and
managing conflicts of interest and advancing medical innovation. A
wide range of stakeholders, including government officials,
pharmaceutical company representatives, academic administrators and
researchers, health care providers, medical ethicists, patient
advocates, and consumers, were invited to present their
perspectives and participate in discussions during the workshop.
This report focuses on current conflict of interest policies and
their effect on medical innovation in an effort to identify best
practices and potential solutions for facilitating innovation while
still ensuring scientific integrity and public trust. Table of
Contents Front Matter 1 Introduction and Overview 2 Conflict of
Interest Policies: An Overview 3 Perspectives on Conflict of
Interest Policies 4 Public Perceptions of Conflict of Interest 5
Managing Conflict and Facilitating Innovation References Appendix
A: Workshop Agenda Appendix B: Speaker Biographical Sketches
Appendix C: Statement of Task Appendix D: Registered Attendees
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