|
Showing 1 - 20 of
20 matches in All Departments
Because of the recent advances in embryo modeling techniques, and
at the request of the Office of Science Policy in the Office of the
Director at the National Institutes of Health, the National
Academies of Sciences, Engineering, hosted a 1-day public workshop
that would explore the state of the science of mammalian embryo
model systems. The workshop, which took place on January 17, 2020,
featured a combination of presentations, panels, and general
discussions, during which panelists and participants offered a
broad range of perspectives. Participants considered whether embryo
model systems - especially those that use nonhuman primate cells -
can be used to predict the function of systems made with human
cells. Presentations provided an overview of the current state of
the science of in vitro development of human trophoblast. This
publication summarizes the presentation and discussion of the
workshop. Table of Contents Front Matter 1 Introduction and
Overview 2 Mammalian Embryo Research and Pluripotent Stem Cells 3
Examining the Development of Extraembryonic Lineages 4 Stem
CellBased Models of Human Embryos 5 Comparative Embryonic
Development Across Species 6 Exploring Opportunities and Challenges
with Mammalian Embryo Model Systems References Appendix A: Workshop
Agenda Appendix B: Speaker Biographical Sketches Appendix C:
Statement of Task Appendix D: Registered Attendees
Consumer genomics, encompassing both direct-to-consumer
applications (i.e., genetic testing that is accessed by a consumer
directly from a commercial company apart from a health care
provider) and consumer-driven genetic testing (i.e., genetic
testing ordered by a health care provider in response to an
informed patient request), has evolved considerably over the past
decade, moving from more personal utility-focused applications
outside of traditional health care to interfacing with clinical
care in nontraditional ways. As consumer genomics has increasingly
intersected with clinical applications, discussions have arisen
around the need to demonstrate clinical and analytical validity and
clinical utility due to the potential for misinterpretation by
consumers. Clinical readiness and interest for this information
have presented educational and training challenges for providers.
At the same time, consumer genomics has emerged as a potentially
innovative mechanism for thinking about health literacy and
engaging participants in their health and health care. To explore
the current landscape of consumer genomics and the implications for
how genetic test information is used or may be used in research and
clinical care, the Roundtable on Genomics and Precision Health of
the National Academies of Sciences, Engineering, and Medicine
hosted a public workshop on October 29, 2019, in Washington, DC.
Discussions included such topics as the diversity of participant
populations, the impact of consumer genomics on health literacy and
engagement, knowledge gaps related to the use of consumer genomics
in clinical care, and regulatory and health policy issues such as
data privacy and security. A broad array of stakeholders took part
in the workshop, including genomics and consumer genomics experts,
epidemiologists, health disparities researchers, clinicians, users
of consumer genomics research applications, representatives from
patient advocacy groups, payers, bioethicists, regulators, and
policy makers. This publication summarizes the presentations and
discussion of the workshop. Table of Contents Front Matter 1
Introduction and Workshop Overview 2 Understanding Consumer
Genomics Use 3 Exploring the Role of Diversity and Health
Disparities in Consumer Genomics 4 Integration Within Scientific
and Medical Communities 5 Regulatory and Health Policy Issues 6 How
Can Consumer Genomics Be Better Integrated to Improve Health?
References Appendix A: Workshop Agenda Appendix B: Speaker
Biographical Sketches Appendix C: Statement of Task Appendix D:
Registered Attendees
The emerging multidisciplinary field of regenerative engineering is
devoted to the repair, regeneration, and replacement of damaged
tissues or organs in the body. To accomplish this it uses a
combination of principles and technologies from disciplines such as
advanced materials science, developmental and stem cell biology,
immunology, physics, and clinical translation. The term
"regenerative engineering" reflects a new understanding of the use
of tissue engineering for regeneration and also the growing number
of research and product development efforts that incorporate
elements from a variety of fields. Because regenerative engineered
therapies rely on live cells and scaffolds, there are inherent
challenges in quality control arising from variability in source
and final products. Furthermore, each patient recipient, tissue
donor, and product application is unique, meaning that the field
faces complexities in the development of safe and effective new
products and therapies which are not faced by developers of more
conventional therapies. Understanding the many sources of
variability can help reduce this variability and ensure consistent
results. The Forum on Regenerative Medicine hosted a public
workshop on October 18, 2018, in Washington, DC, to explore the
various factors that must be taken into account in order to develop
successful regenerative engineering products. Invited speakers and
participants discussed factors and sources of variability in the
development and clinical application of regenerative engineering
products, characteristics of high-quality products, and how
different clinical needs, models, and contexts can inform the
development of a product to improve patient outcomes. This
publication summarizes the presentation and discussion of the
workshop. Table of Contents Front Matter 1 Introduction and
Overview 2 Sources of Variability Associated with Regenerative
Therapies: Lessons from Case Studies 3 Factors Contributing to
Patient Variability 4 Addressing Variability in Donor Tissues and
Cells 5 Addressing Variability and Meeting Quality Expectations in
the Manufacturing Setting 6 Exploring Variability and Its Impact on
Product Regulation and Outcomes 7 Potential Next Steps to Consider
for Addressing Variability References Appendix A: Workshop Agenda
Appendix B: Speaker Biographical Sketches Appendix C: Statement of
Task Appendix D: Registered Attendees
Those involved in the drug development process face challenges of
efficiency and overall sustainability due in part to high research
costs, lengthy development timelines, and late-stage drug failures.
Novel clinical trial designs that enroll participants based on
their genetics represent a potentially disruptive change that could
improve patient outcomes, reduce costs associated with drug
development, and further realize the goals of precision medicine.
On March 8, 2017, the Forum on Drug Discovery, Development, and
Translation and the Roundtable on Genomics and Precision Health of
the National Academies of Sciences, Engineering, and Medicine
hosted the workshop Enabling Precision Medicine: The Role of
Genetics in Clinical Drug Development. Participants examined
successes, challenges, and possible best practices for effectively
using genetic information in the design and implementation of
clinical trials to support the development of precision medicines,
including exploring the potential advantages and disadvantages of
such trials across a variety of disease areas. This publication
summarizes the presentations and discussions from the workshop.
Table of Contents Front Matter 1 Introduction 2 Overarching
Considerations for Implementing Successful Genetics-Enabled Drug
Development 3 Case Studies in Precision Drug Development 4
Integrating Genetics into the Drug Development Pathway for Complex
Diseases 5 Finding Innovative Ways to Integrate Genetic Research
into the Drug Development Process 6 Reflecting Back and Looking
Forward: Key Themes and Potential Next Steps in Genetics-Enabled
Drug Development Appendix A: References Appendix B: Statement of
Task and Workshop Agenda Appendix C: Speaker Biographical Sketches
Appendix D: Registered Attendees
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
The Forum on Regenerative Medicine of the National Academies of
Sciences, Engineering, and Medicine convened a two-day virtual
public workshop to address knowledge gaps in the understanding of
promising approaches to manipulate the immune system and/or the
regenerative medicine product to improve outcomes of tissue repair
and regeneration in patients. The workshop, titled "Understanding
the Role of the Immune System in Improving Tissue Regeneration,"
explored the role of the immune system in the success or failure of
regenerative medicine therapies. Participants considered potential
strategies to effectively "prepare" patients' immune systems to
accept regenerative therapies and increase the likelihood of
successful clinical outcomes and also discussed risks associated
with modulating the immune system. This Proceedings of a Workshop
highlights the presentations and discussions that occurred during
the workshop.
Regenerative medicine products, which are intended to repair or
replace damaged cells or tissues in the body, include a range of
therapeutic approaches such as cell- and gene-based therapies,
engineered tissues, and non-biologic constructs. The current
approach to characterizing the quality of a regenerative medicine
product and the manufacturing process often involves measuring as
many endpoints as possible, but this approach has proved to be
inadequate and unsustainable. The Forum on Regenerative Medicine of
the National Academies of Sciences, Engineering, and Medicine
convened experts across disciplines for a 2-day virtual public
workshop to explore systems thinking approaches and how they may be
applied to support the identification of relevant quality
attributes that can help in the optimization of manufacturing and
streamline regulatory processes for regenerative medicine. A broad
array of stakeholders, including data scientists, physical
scientists, industry researchers, regulatory officials, clinicians,
and patient representatives, discussed new advances in data
acquisition, data analysis and theoretical frameworks, and how
systems approaches can be applied to the development of
regenerative medicine products that can address the unmet needs of
patients. This publication summarizes the presentation and
discussion of the workshop. Table of Contents Front Matter 1
Introduction 2 Introduction to Systems Thinking Concepts 3
Exploring the Challenges of Critical Quality Attributes: The Role
of Systems Thinking 4 Challenges Associated with Data Collection,
Aggregation, and Sharing 5 Challenges and Opportunities Associated
with Systems-Level Analysis and Modeling 6 Addressing Regenerative
Medicine Manufacturing and Supply Chain Challenges with
Systems-Level Approaches 7 Exploring Issues of Workforce
Development Related to Systems Thinking References Appendix A:
Workshop Agenda Appendix B: Speaker Biographical Sketches Appendix
C: Statement of Task
On February 26, 2020, the Board on Health Sciences Policy of the
National Academies of Sciences, Engineering, and Medicine hosted a
1-day public workshop in Washington, DC, to examine current and
emerging bioethical issues that might arise in the context of
biomedical research and to consider research topics in bioethics
that could benefit from further attention. The scope of bioethical
issues in research is broad, but this workshop focused on issues
related to the development and use of digital technologies,
artificial intelligence, and machine learning in research and
clinical practice; issues emerging as nontraditional approaches to
health research become more widespread; the role of bioethics in
addressing racial and structural inequalities in health; and
enhancing the capacity and diversity of the bioethics workforce.
This publication summarizes the presentations and discussions from
the workshop. Table of Contents Front Matter 1 Introduction 2
Ethically Leveraging Digital Technology for Health 3 Ethical
Questions Concerning Nontraditional Approaches for Data Collection
and Use 4 Understanding the Impact of Inequality on Health,
Disease, and Who Participates in Research 5 Bioethics Research
Workforce 6 Reflecting on the Workshop and Looking to the Future
References Appendix A: Workshop Agenda Appendix B: Speaker
Biographical Sketches Appendix C: Statement of Task Appendix D:
Registered Attendees
Recognizing the potential design complexities and ethical issues
associated with clinical trials for gene therapies, the Forum on
Regenerative Medicine of the National Academies of Sciences,
Engineering, and Medicine held a 1-day workshop in Washington, DC,
on November 13, 2019. Speakers at the workshop discussed patient
recruitment and selection for gene-based clinical trials, explored
how the safety of new therapies is assessed, reviewed the
challenges involving dose escalation, and spoke about ethical
issues such as informed consent and the role of clinicians in
recommending trials as options to their patients. The workshop also
included discussions of topics related to gene therapies in the
context of other available and potentially curative treatments,
such as bone marrow transplantation for hemoglobinopathies. This
publication summarizes the presentation and discussion of the
workshop. Table of Contents Front Matter 1 Introduction and
Overview 2 Developing First-in-Human Gene Therapy Clinical Trials 3
Understanding the Complexities of Patient Selection, Enrollment,
and the Consent Process 4 Developing Endpoints for Gene Therapy
Clinical Trials 5 Integrating Gene-Based Therapies into Clinical
Practice: Exploring Long-Term Clinical Follow-Up of Patients 6
Reflections on the Workshop and Potential Opportunities for Next
Steps References Appendix A: Workshop Agenda Appendix B: Speaker
Biographical Sketches Appendix C: Statement of Task Appendix D:
Registered Attendees
Genomic medicine is defined as the routine use of genomic
information about an individual as part of his or her clinical care
as well as the health outcomes and policy implications of that
clinical use. It is one approach that has the potential to improve
the quality of health care by allowing practitioners to tailor
prevention, diagnostic, and treatment strategies to individual
patients. In recent years, research breakthroughs, technological
advances, and the decreasing cost of DNA sequencing have led to the
wider adoption of genomic medicine. However, as with the
introduction of new technologies into health care, there are
concerns that genetic and genomic testing and services will not
reach all segments of the population both now and in the near
future, and there remains a gap in knowledge regarding potential
health care disparities in genomic medicine and precision health
approaches. On June 27, 2018, the National Academies of Sciences,
Engineering, and Medicine hosted a public workshop to examine the
gaps in knowledge related to access to genomic medicine and to
discuss health care disparities and possible approaches to
overcoming the disparate use of genomic medicine among populations.
Workshop participants discussed research on access to genetics and
genomics services in medically underserved areas, model programs of
care for diverse patient populations, and current challenges and
possible best practices for alleviating health care disparities as
they relate to genomics-based approaches. This publication
summarizes the presentations and discussions from the workshop.
Table of Contents Front Matter 1 Introduction and Overview 2
Exploring the Barriers to Accessing Genomic and Genetic Services 3
The Role of Health Systems in Delivering Equitable Access 4 How Can
Providers and Payers Make Genomic Medicine More Accessible? 5
Exploring Innovative Solutions and Models of Success 6 Considering
Unmet Needs to Alleviate Disparities in Genomic Medicine References
Appendix A: Summary of the Pre-Workshop Twitter Chat Appendix B:
Workshop Agenda Appendix C: Speaker Biographical Sketches Appendix
D: Statement of Task Appendix E: Registered Attendees
On June 26, 2017, the Forum on Regenerative Medicine hosted a
public workshop in Washington, DC, titled Navigating the
Manufacturing Process and Ensuring the Quality of Regenerative
Medicine Therapies in order to examine and discuss the challenges,
opportunities, and best practices associated with defining and
measuring the quality of cell and tissue products and raw materials
in the research and manufacturing of regenerative medicine
therapies. The goal of the workshop was to learn from existing
examples of the manufacturing of early-generation regenerative
medicine products and to address how progress could be made in
identifying and measuring critical quality attributes. The workshop
also addressed the challenges of designing and adhering to
standards as a way of helping those who are working to scale up
processes and techniques from a research laboratory to the
manufacturing environment. This publication summarizes the
presentations and discussions from the workshop. Table of Contents
Front Matter 1 Introduction 2 Transitioning from Discovery and
Development to Manufacturing 3 Identifying and Measuring Critical
Quality Attributes 4 Designing Technologies to Meet the
Manufacturing Needs of New Regenerative Medicine Therapies 5
Considerations for Improving and Regulating Regenerative Medicine
Products 6 Potential Next Steps for Supporting the Development,
Manufacture, and Regulation of Regenerative Medicine Therapies
References Appendix A: Workshop Agenda Appendix B: Speaker
Biographical Sketches Appendix C: Statement of Task Appendix D:
Registered Attendees
Regenerative medicine holds the potential to create living,
functional cells and tissues that can be used to repair or replace
those that have suffered potentially irreparable damage due to
disease, age, traumatic injury, or genetic and congenital defects.
The field of regenerative medicine is broad and includes research
and development components of gene and cell therapies, tissue
engineering, and non-biologic constructs. Although regenerative
medicine has the potential to improve health and deliver economic
benefits, this relatively new field faces challenges to developing
policies and procedures to support the development of novel
therapies are both safe and effective. In October 2016, the
National Academies of Sciences, Engineering, and Medicine hosted a
public workshop with the goal of developing a broad understanding
of the opportunities and challenges associated with regenerative
medicine cellular therapies and related technologies. Participants
explored the state of the science of cell-based regenerative
therapies within the larger context of patient care and policy.
This publication summarizes the presentations and discussions from
the workshop. Table of Contents Front Matter 1 Introduction 2 Skin
and Musculoskeletal Tissues 3 Hematologic and Immunologic
Applications 4 Neurological and Ophthalmological Tissues 5
Cardiovascular and Lung Tissues 6 Renal Tissue 7 Looking Toward the
Future: Concluding Thoughts References Appendix A: Workshop Agenda
Appendix B: Speaker Biographical Sketches Appendix C: Statement of
Task Appendix D: Registered Attendees
The process of discovering and developing a new drug or therapy is
extremely costly and time consuming, and recently, it has been
estimated that the creation of a new medicine costs on average more
than $2 billion and takes 10 years to reach patients. The
challenges associated with bringing new medicines to market have
led many pharmaceutical companies to seek out innovative methods
for streamlining their drug discovery research. One way to increase
the odds of success for compounds in the drug development pipeline
is to adopt genetically guided strategies for drug discovery, and
recognizing the potential benefits of collecting genetic and
phenotypic information across specific populations, pharmaceutical
companies have started collaborating with healthcare systems and
private companies that have curated genetic bioresources, or large
databases of genomic information. Large-scale cohort studies offer
an effective way to collect and store information that can be used
to assess gene?environment interactions, identify new potential
drug targets, understand the role of certain genetic variants in
the drug response, and further elucidate the underlying mechanisms
of disease onset and progression. To examine how genetic
bioresources could be used to improve drug discovery and target
validation, the National Academies of Sciences, Engineering, and
Medicine hosted a workshop in March 2016. Participants at the
workshop explored the current landscape of genomics-enabled drug
discovery activities in industry, academia, and government;
examined enabling partnerships and business models; and considered
gaps and best practices for collecting population data for the
purpose of improving the drug discovery process. This publication
summarizes the presentations and discussions from the workshop.
Table of Contents Front Matter 1 Introduction and Themes of the
Workshop 2 Maximizing Discovery Capabilities Through Cohort Design
3 Discovery Activities Related to Genetic Bioresources 4 Business
Models That Support Bioresource Discovery and Collaboration 5
Potential Next Steps in Using Genomics to Advance Drug Discovery
References Appendix A: Workshop Agenda Appendix B: Speaker
Biographical Sketches Appendix C: Statement of Task Appendix D:
Registered Attendees
Although it is becoming increasingly more common for clinicians to
use genomic data in their practices for disease prevention,
diagnosis, and treatment, the process of integrating genomic data
into the practice of medicine has been a slow and challenging one.
Some of the major barriers impeding the incorporation of new
genomic technology into clinical practice are: the difficulty of
changing routine medical practices to account for the use of
genetic testing, the limited knowledge of patients and providers
about genomic medicine, assessing sufficient evidence to support
the use of genetic tests, privacy and data security issues, and
uncertainty about reimbursement. The field of implementation
science may be able to provide insights concerning efficient ways
to incorporate genomic applications into routine clinical practice.
The focus of implementation science studies is to identify
integration bottlenecks and optimal approaches for a given setting
and ultimately to promote the up-take of research findings. To
explore the potential of implementation science to improve the
integration of genomics into medicine, the National Academies of
Sciences, Engineering, and Medicine held a workshop in Washington,
DC, in November 2015. Participants explored the challenges and
opportunities of integrating genomic advances into the clinic
through the lens of implementation science. This report summarizes
the presentations and discussions from the workshop. Table of
Contents Front Matter 1 Introduction and Themes of the Workshop 2
Implementation Science: Methods and Approaches 3 Engaging Large and
Diverse Populations for Analysis 4 Generating Evidence During
Implementation 5 Genomics and Implementation at the Level of
Population Health 6 Achieving the Vision References Appendix A:
Workshop Agenda Appendix B: Speaker Biographical Sketches Appendix
C: Statement of Task Appendix D: Registered Attendees Appendix E:
Implementation Science: A Background Appendix F: Large Genetic
Cohort Studies: A Background
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 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
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
|
You may like...
Top Five
Rosario Dawson, Cedric The Entertainer, …
Blu-ray disc
R40
Discovery Miles 400
|