|
Showing 1 - 12 of
12 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
Genomic applications are being integrated into a broad range of
clinical and research activities at health care systems across the
United States. This trend can be attributed to a variety of
factors, including the declining cost of genome sequencing and the
potential for improving health outcomes and cutting the costs of
care. The goals of these genomics-based programs may be to identify
individuals with clinically actionable variants as a way of
preventing disease, providing diagnoses for patients with rare
diseases, and advancing research on genetic contributions to health
and disease. Of particular interest are genomics- based screening
programs, which will, in this publication, be clinical screening
programs that examine genes or variants in unselected populations
in order to identify individuals who are at an increased risk for a
particular health concern (e.g., diseases, adverse drug outcomes)
and who might benefit from clinical interventions. On November 1,
2017, the National Academies of Sciences, Engineering, and Medicine
hosted a public workshop to explore the challenges and
opportunities associated with integrating genomics-based screening
programs into health care systems. This workshop was developed as a
way to explore the challenges and opportunities associated with
integrating genomics-based programs in health care systems in the
areas of evidence collection, sustainability, data sharing,
infrastructure, and equity of access. This publication summarizes
the presentations and discussions from the workshop. Table of
Contents Front Matter 1 Introduction 2 Evidence Considerations for
Integrating Genomics-Based Programs into Health Care Systems 3
Financial Considerations for Implementing Genomics-Based Screening
Programs 4 Exploring Approaches to Optimize Data Sharing Among
Early Implementers of Genomics-Based Programs 5 Understanding
Participant Needs and Preferences and Improving Diversity and
Equity 6 Improving Health Through the Integration of Genomics-Based
Programs: Potential Next Steps References Appendix A: Workshop
Agenda Appendix B: Speaker Biographies 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
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
|
|