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The volume and complexity of information about individual patients
is greatly increasing with use of electronic records and personal
devices. Potential effects on medical product development in the
context of this wealth of real-world data could be numerous and
varied, ranging from the ability to determine both large-scale and
patient-specific effects of treatments to the ability to assess how
therapeutics affect patients' lives through measurement of
lifestyle changes. In October 2016, the National Academies of
Sciences, Engineering, and Medicine held a workshop to facilitate
dialogue among stakeholders about the opportunities and challenges
for incorporating real-world evidence into all stages in the
process for the generation and evaluation of therapeutics.
Participants explored unmet stakeholder needs and opportunities to
generate new kinds of evidence that meet those needs. This
publication summarizes the presentations and discussions from the
workshop. Table of Contents Front Matter 1 Introduction 2 Improving
Evidence Generation for Decision Making on Approval and Use of New
Treatments: Some Stakeholder Priorities 3 Opportunities for
Real-World Data 4 Generating and Incorporating Real-World Evidence
into Medical Product Development and Evaluation: Building from
Successful Case Studies 5 Potential Strategies for a Way Forward
Appendix A: Bibliography Appendix B: Workshop Agenda Appendix C:
Participant Biographies Appendix D: Discussion Paper: Real-World
Evidence to Guide theApproval and Use of New Treatments
Since the 2014 Ebola outbreak many public- and private-sector
leaders have seen a need for improved management of global public
health emergencies. The effects of the Ebola epidemic go well
beyond the three hardest-hit countries and beyond the health
sector. Education, child protection, commerce, transportation, and
human rights have all suffered. The consequences and lethality of
Ebola have increased interest in coordinated global response to
infectious threats, many of which could disrupt global health and
commerce far more than the recent outbreak. In order to explore the
potential for improving international management and response to
outbreaks the National Academy of Medicine agreed to manage an
international, independent, evidence-based, authoritative,
multistakeholder expert commission. As part of this effort, the
Institute of Medicine convened four workshops in summer of 2015 to
inform the commission report. The presentations and discussions
from the Workshop on Research and Development of Medical Products
are summarized in this report. Table of Contents Front Matter 1
Introduction 2 Models and Incentives for Engagement 3 Discovery
Research 4 Development 5 Convergence of Regulatory Expectations,
Review, and Approval 6 Manufacturing, Stockpiling, and Distribution
7 Critical Considerations for Facilitating Medical Product Research
and Development 8 Crosscutting Themes and Closing Remarks Appendix
A: References Appendix B: Workshop Statement of Task Appendix C:
Workshop Agenda Appendix D: Workshop Speaker Biographies
There is growing recognition that the United States' clinical
trials enterprise (CTE) faces great challenges. There is a gap
between what is desired - where medical care is provided solely
based on high quality evidence - and the reality - where there is
limited capacity to generate timely and practical evidence for drug
development and to support medical treatment decisions. With the
need for transforming the CTE in the U.S. becoming more pressing,
the IOM Forum on Drug Discovery, Development, and Translation held
a two-day workshop in November 2011, bringing together leaders in
research and health care. The workshop focused on how to transform
the CTE and discussed a vision to make the enterprise more
efficient, effective, and fully integrated into the health care
system. Key issue areas addressed at the workshop included: the
development of a robust clinical trials workforce, the alignment of
cultural and financial incentives for clinical trials, and the
creation of a sustainable infrastructure to support a transformed
CTE. This document summarizes the workshop. Table of Contents Front
Matter 1 Introduction 2 Integrating Community Practice and Clinical
Trials 3 Improving Public Participation in Clinical Trials 4
Creating a New Business Model for Clinical Trials 5 Building an
Infrastructure to Support Clinical Trials 6 Suggesting an Agenda
for Transforming Elements of the Clinical Trials Enterprise
References Appendix A: Workshop Agenda Appendix B: Participant
Biographies Appendix C: Registered Workshop Attendees Appendix D:
Discussion Paper: The Clinical Trials Enterprise in the United
States: A Call for Disruptive Innovation Appendix E: Discussion
Paper: Developing a Robust Clinical Trials Workforce Appendix F:
Discussion Paper: Transforming the Economics of Clinical Trials
Appendix G: Discussion Paper: Developing a Clinical Trials
Infrastructure Appendix H: Discussion Paper: Canadian Strategy on
Patient-Oriented Research Appendix I: Discussion Paper: Health
Research as a Public Good Appendix J: Discussion Paper: Novel Ways
to Get Good Trial Data: The UK Experience Appendix K: IOM Staff
Paper: Context and Glossary of Select Terms Associated with the
Clinical Trials Enterprise
The field of endeavors known as "regulatory science" has grown out
of the need to link and integrate knowledge within and among basic
science research, clinical research, clinical medicine, and other
specific scientific disciplines whose focus, aggregation, and
ultimate implementation could inform biomedical product development
and regulatory decision making. Substantial efforts have been
devoted to defining regulatory science and communicating its value
and role across the scientific and regulatory ecosystems.
Investments are also being made in technology infrastructure,
regulatory systems, and workforce development to support and
advance this burgeoning discipline. In October 2015, the National
Academies of Sciences, Engineering, and Medicine held a public
workshop to facilitate dialogue among stakeholders about the
current state and scope of regulatory science, opportunities to
address barriers to the discipline's success, and avenues for
fostering collaboration across sectors. Participants explored key
needs for strengthening the discipline of regulatory science,
including considering what are the core components of regulatory
science infrastructure to foster innovation in medical product
development. This report summarizes the presentations and
discussions from the workshop. Table of Contents Front Matter 1
Introduction 2 Characterizing the Regulatory Science Landscape 3
Regulatory Science Applications: Using Case Studies to Focus on
Approaches to Advance the Discipline 4 Regulatory Science
Infrastructure and Workforce 5 Challenges and Opportunities in
Regulatory Science Appendix A: Bibliography Appendix B: Workshop
Agenda Appendix C: Participant Biographies
Despite the extensive body of evidence that informs regulatory
decisions on pharmaceutical products, significant uncertainties
persist, including the underlying variability in human biology,
factors associated with the chemistry of a drug, and limitations in
the research and clinical trial process itself that might limit the
generalizability of results. As a result, regulatory reviewers are
consistently required to draw conclusions about a drug's safety and
efficacy from imperfect data. Efforts are underway within the drug
development community to enhance the evaluation and communication
of the benefits and risks associated with pharmaceutical products,
aimed at increasing the predictability, transparency, and
efficiency of pharmaceutical regulatory decision making.
Effectively communicating regulatory decisions necessarily includes
explanation of the impact of uncertainty on decision making. On
February 12 and May 12, 2014, the Institute of Medicine's Forum on
Drug Discovery, Development, and Translation held public workshops
to advance the development of more systematic and structured
approaches to characterize and communicate the sources of
uncertainty in the assessment of benefits and risks, and to
consider their implications for pharmaceutical regulatory
decisions. Workshop presentations and discussions on February 12
were convened to explore the science of identifying and
characterizing uncertainty in scientific evidence and approaches to
translate uncertainties into decisions that reflect the values of
stakeholders. The May 12 workshop presentations and discussions
explored tools and approaches to communicating about scientific
uncertainties to a range of stakeholders in the drug development
process. Characterizing and Communicating Uncertainty in the
Assessment of Benefits and Risks of Pharmaceutical Products
summarizes the presentation and discussion of both events. This
report explores potential analytical and communication approaches
and identifies key considerations on their development, evaluation,
and incorporation into pharmaceutical benefit- risk assessment
throughout the entire drug development lifecycle. Table of Contents
Front Matter 1 Introduction 2 Identifying and Characterizing
Uncertainty 3 The Regulators' Challenge 4 Basic Methodologies and
Applications for Understanding and Evaluating Uncertainty 5
Communicating Uncertainty 6 Final Reflections on Ways to
Characterize and Communicate Uncertainty References Appendix A:
Workshop Agenda Appendix B: FDA Case Studies Appendix C:
Bibliography Appendix D: Participant Biographies
The development and application of regulatory science - which FDA
has defined as the science of developing new tools, standards, and
approaches to assess the safety, efficacy, quality, and performance
of FDA-regulated products - calls for a well-trained,
scientifically engaged, and motivated workforce. FDA faces
challenges in retaining regulatory scientists and providing them
with opportunities for professional development. In the private
sector, advancement of innovative regulatory science in drug
development has not always been clearly defined, well coordinated,
or connected to the needs of the agency. As a follow-up to a 2010
workshop, the IOM held a workshop on September 20-21, 2011, to
provide a format for establishing a specific agenda to implement
the vision and principles relating to a regulatory science
workforce and disciplinary infrastructure as discussed in the 2010
workshop. Table of Contents Front Matter 1 Introduction 2 The
Importance of Innovative Regulatory Science 3 Defining a Discipline
of Regulatory Science and Core Competencies for Its Workforce 4
Education and Training of a Regulatory Science Workforce 5 Career
Paths Within Academia and Industry 6 International Applications of
Regulatory Science 7 Collaborative Models and New Paradigms for
Supporting Regulatory Science Research and Practice References
Appendix A: Workshop Agenda Appendix B: Participant Biographies
Multidrug-resistant tuberculosis (TB) is caused by bacteria
resistant to isoniazid and rifampicin, the two most effective
first-line anti-TB drugs, originally developed and introduced in
the 1950 and 1960s. Since 2008, the Forum on Drug Discovery,
Development, and Translation of the Institute of Medicine has
hosted or co-hosted six domestic and international workshops
addressing the global crisis of drug-resistant TB, with special
attention to the BRICS countries - Brazil, Russia, India, China,
and South Africa. The Global Crisis of Drug-Resistant Tuberculosis
and Leadership of China and the BRICS is the summary of a workshop
convened to address the current status of drug-resistant TB
globally and in China. This report considers lessons learned from
high burden countries; highlights global challenges to controlling
the spread of drug-resistant strains; and discusses innovative
strategies to advance and harmonize local and international efforts
to prevent and treat drug-resistant TB. Additionally, the report
examines the problem of MDR TB and emergent TB strains that are
potentially untreatable with drugs available and considers the
critical leadership role of the BRICS countries in addressing the
threats and opportunities in drug-resistant TB.
Advances in technologies and knowledge are creating new avenues for
research and opportunities for the discovery and clinical
development of innovative therapies and diagnostics. However,
despite these opportunities, only a small fraction of
investigational products are successfully developed into cures and
therapies that can be accessed by patients. One response to the
ever-widening gap between the number and promise of basic
scientific discoveries and the translation of those discoveries
into therapies is a renewed emphasis on collaborative approaches
among federal agencies, academia, and industry, all directed at the
advancement of the drug development enterprise. The newly developed
Cures Acceleration Network (CAN)-a part of the National Center for
Advancing Translational Sciences (NCATS) within the National
Institutes of Health (NIH)-has the potential to catalyze widespread
changes in NCATS, NIH, and the drug development ecosystem in
general. On June 4-5, 2012, the IOM Forum on Drug Discovery,
Development, and Translation held, at the request of NCATS, a
workshop-bringing together members of federal government agencies,
the private sector, academia, and advocacy groups-to explore
options and opportunities in the implementation of CAN.
Accelerating the Development of New Drugs and Diagnostics:
Maximizing the Impact of the Cures Acceleration Network: Workshop
Summary summarizes the workshop. Table of Contents Front Matter 1
Introduction 2 Approaches to Accelerating Translational Science 3
Application of Matching Authority 4 Application of Flexible
Research Authority 5 Situating CAN Within the Drug Development
Ecosystem 6 Final Reflections on Ways to Maximize the Goals of CAN
References Appendix A: Workshop Agenda Appendix B: Public Health
Service Act, Title IV - National Institutes of Health Appendix C:
Participant Biographies
To effectively treat patients diagnosed with drug-resistant (DR)
tuberculosis (TB) and protect the population from further
transmission of this infectious disease, an uninterrupted supply of
quality-assured (QA), second-line anti-TB drugs (SLDs) is
necessary. Patients diagnosed with multidrug-resistant tuberculosis
(MDR TB)-a disease caused by strains of Mycobacterium tuberculosis
(M.tb.) resistant to two primary TB drugs (isoniazid and
rifampicin)-face lengthy treatment regimens of 2 years or more with
daily, directly observed treatment (DOT) with SLDs that are less
potent, more toxic, and more expensive than those used to treat
drug-susceptible TB. From 2000 to 2009, only 0.2-0.5 percent of the
estimated 5 million MDR TB cases globally were treated with drugs
of known quality and in programs capable of delivering appropriate
care (Keshavjee, 2012). The vast majority of MDR TB patients either
died from lack of treatment or contributed to the spread of MDR TB
in their communities. A strengthened global supply chain for SLDs
could save lives by consistently delivering high quality medicines
to more of the people who need them. This public workshop explored
innovative solutions to the problem of how to get the right SLDs
for MDR TB to people who critically need them. More specifically,
the workshop examined current problems and potential opportunities
for coordinated international efforts to ensure that a reliable and
affordable supply of high-quality SLDs is available. Developing and
Strengthening the Global Supply Chain for Second-Line Drugs for
Multidrug-Resistant Tuberculosis: Workshop Summary covers the
objectives of the workshop, which were to review: -To what extent
and in what ways current mechanisms are or are not effectively
accomplishing what is needed, including consideration of
bottlenecks. -The advantages and disadvantages of centralization in
the management of the global drug supply chain, and potential
decentralized approaches to improve operations of the supply chain.
-What can be learned from case studies and examples from other
diseases (e.g., the Affordable Medicines Facility-malaria (AMFm)
and the U.S. President's Emergency Plan for AIDS Relief [PEPFAR]) -
The current allocation of responsibilities and roles of the private
(including industry and nonprofit public health organizations) and
public sectors, and examination of opportunities for enhancing and
optimizing collaboration -Identification of potential innovative
solutions to the problem Table of Contents Front Matter 1
Introduction 2 Logistics, Supply, and Demand 3 Financing of MDR TB
SLDs 4 Innovative Suggestions and Potential Solutions References
Appendix A: Workshop Agenda Appendix B: Participant Biographies
Appendix C: Registered Workshop Attendees
An estimated 8.8 million people fell ill with tuberculosis (TB) in
2010 and 1.4 million died from the disease. Although antibiotics to
treat TB were developed in the 1950s and are effective against a
majority of TB cases, resistance to these antibiotics has emerged
over the years, resulting in the growing spread of
multidrug-resistant (MDR) TB. Due to challenges in timely and
accurate diagnosis of drug-resistant TB, length and tolerability of
treatment regimens, and expense of second-line anti-TB drugs,
effectively controlling the disease requires complex public health
interventions. The IOM Forum on Drug Discovery, Development, and
Translation held three international workshops to gather
information from local experts around the world on the threat of
drug resistant TB and how the challenges it presents can be met.
Workshops were held in South Africa and Russia in 2010. The third
workshop was held April 18-19, 2011, in New Delhi, India, in
collaboration with the Indian National Science Academy and the
Indian Council of Medical Research. The aim of the workshop was to
highlight key challenges to controlling the spread of
drug-resistant strains of TB in India and to discuss strategies for
advancing and integrating local and international efforts to
prevent and treat drug-resistant TB. This document summarizes the
workshop. Table of Contents Front Matter 1 Introduction 2
Drug-Resistant TB in India 3 The Global Burden of Drug-Resistant TB
4 Preventing Transmission of Drug-Resistant TB 5 Detecting Drug
Resistance and Strengthening Laboratory Capacity 6 Addressing TB
and Drug-Resistant TB in Vulnerable Populations 7 Combating
Drug-Resistant TB Through PublicPrivate Collaboration and
Innovative Approaches 8 Confronting Challenges to the Supply Chain
for SecondLine Drugs 9 Creating a Blueprint for Action References
Appendix A: Workshop Agenda Appendix B: Summary of a Joint Meeting
of the National Institute of Allergy and Infectious Diseases,
National Institutes of Health, and Indian Biomedical Research
Agencies, Held April 20-21, 2011, New Delhi, India Appendix C:
Participant Biographies
Clinical trials provide essential information needed to turn basic
medical research findings into patient treatments. New treatments
must be studied in large numbers of humans to find out whether they
are effective and to assess any harm that may arise from treatment.
There is growing recognition among many stakeholders that the U.S.
clinical trials enterprise is unable to keep pace with the national
demand for research results. The IOM, along with the Mount Sinai
School of Medicine, held a workshop June 27-28, 2011, to engage
stakeholders and experts in a discussion about possible solutions
to improve public engagement in clinical trials. Table of Contents
Front Matter 1 Introduction 2 Framing the Problem 3 Recruitment
Challenges in Clinical Trials for Different Diseases and Conditions
4 Models for Public Engagement 5 Messages and Methods for Public
Engagement 6 The Media 7 Novel Clinical Trial Designs 8 The Health
System's Structure and Culture 9 Toward a Patient-Centered Strategy
for Clinical Trials References Appendix A: Workshop Agenda Appendix
B: The Clinical Trials Process Appendix C: Participant Biographies
Whether or not the United States has safe and effective medical
countermeasures-such as vaccines, drugs, and diagnostic
tools-available for use during a disaster can mean the difference
between life and death for many Americans. The Food and Drug
Administration (FDA) and the scientific community at large could
benefit from improved scientific tools and analytic techniques to
undertake the complex scientific evaluation and decision making
needed to make essential medical countermeasures available. At the
request of FDA, the Institute of Medicine (IOM) held a workshop to
examine methods to improve the development, evaluation, approval,
and regulation of medical countermeasures. During public health
emergencies such as influenza or chemical, biological,
radiological/nuclear (CBRN) attacks, safe and effective vaccines,
treatments, and other medical countermeasures are essential to
protecting national security and the well being of the public.
Advancing Regulatory Science for Medical Countermeasure Development
examines current medical countermeasures, and investigates the
future of research and development in this area. Convened on March
29-30, 2011, this workshop identified regulatory science tools and
methods that are available or under development, as well as major
gaps in currently available regulatory science tools. Advancing
Regulatory Science for Medical Countermeasure Development is a
valuable resource for federal agencies including the Food and Drug
Administration (FDA), the Department of Health and Human Services
(HHS), the Department of Defense (DoD), as well as health
professionals, and public and private health organizations. Table
of Contents Front Matter 1 Introduction 2 MCM Enterprise and
Stakeholder Perspectives 3 Cutting-Edge Efforts to Advance MCM
Regulatory Science 4 MCM Regulatory Science Needs for At-Risk
Populations 5 Crosscutting Themes and Future Directions 6 Closing
Remarks References Appendix A: Workshop Agenda Appendix B:
Participant Biographies
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