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The use of immune modulator therapeutics, a type of immunotherapy enhancing the body immune system response to cancer, was perceived as the beginning of a new era in cancer care. While still important and frequently used, some of these therapeutics produce uneven response rates, disease resistance, and serious side effects. The National Academies National Cancer Policy Forum, in collaboration with the Forum on Drug Discovery, Development, and Translation, hosted a public workshop to discuss challenges related to immunotherapy treatment resistance, as well as potential policy opportunities to improve the development of immunotherapies for cancer treatment.

To explore the current and future state of serious illness care research, the National Academies Roundtable on Quality Care for People with Serious Illness hosted a public workshop in November 2023. Speakers identified research gaps and challenges and explored strategies and opportunities to further develop the evidence base to improve care for all those living with serious illness, their families, and care partners.

At any one time, at least 30,000 people in the U.S. are living with amyotrophic lateral sclerosis (ALS), a rapidly progressive, fatal neurological disease affecting individuals, caregivers, at-risk genetic carriers, and others. In 2022, Congress directed the National Institutes of Health to commission a National Academies committee of experts to recommend key actions public, private, and nonprofit sectors should take to make ALS a livable disease within the next 10 years. The resulting report, Living with ALS, focuses on an integrated ALS multidisciplinary care and research system to help facilitate earlier diagnosis and connections to specialty care.

The National Academies Roundtable on Genomics and Precision Health, in collaboration with the National Cancer Policy Forum, hosted a public workshop that examined how genomic data are used in health care, outside of the traditional settings for clinical genetics. The workshop identified opportunities for advancement of precision health care delivery. The event also explored how patients, clinicians, and payers assess and act upon the risks and benefits of genomic screening and diagnostic testing. Discussions focused on strategies to ensure that genomic applications are responsibly and equitably adopted to benefit populations as well as individuals over time. This Proceedings of a Workshop summarizes content from the event.

Studies show that people commonly have psychotic symptoms for over a year before receiving treatment. Reducing this duration is critical, because early treatment is strongly associated with better outcomes. The National Academies Forum on Mental Health and Substance Use Disorders hosted a workshop in July 2022 to examine the current evidence on short- and long-term outcomes for people at high risk for psychosis. Speakers explored potential policy solutions and strategies that are most effective for coordinated, early-intervention specialty services. This Proceedings document summarizes workshop discussions.

Where people live can affect their health because of location-specific exposure to chemicals and toxicants, lifestyle, diet, immune function development, and social and historical determinants of health. A better understanding of the connections between environmental exposures and genetics that are associated with cancer can help inform efforts to identify risks and prevent potentially harmful outcomes. Recent scientific advancements have brought new insights into genomic and epigenomic biomarkers of cancer, which encompasses the toxicogenomic effects of environmental hazards, genetic susceptibility to environmental exposures, and the role of genetic and epigenetic changes in the development of cancer. Public communication around the potential risks of environmental hazards has also been evolving. The National Academies of Sciences, Engineering, and Medicine National Cancer Policy Forum convened a virtual public workshop in collaboration with the Roundtable on Genomics and Precision Health on April 13, 2023 to explore the state of the science in identifying potential genomic and epigenomic biomarkers of environmental exposures associated with cancers, with an emphasis on pediatric cancers. This proceedings document summarizes workshop discussions.

Heritable disorders of connective tissue (HDCTs) are a diverse group of inherited genetic disorders and subtypes. Because connective tissue is found throughout the body, the impairments associated with HDCTs manifest in multiple body systems and may change or vary in severity throughout an affected individual's lifetime. In some cases, these impairments may be severe enough to qualify an eligible child or adult for monetary benefits through the U.S. Social Security Administration's (SSA's) Social Security Disability Insurance or Supplemental Security Income program. SSA asked the National Academies of Sciences, Engineering, and Medicine to convene an expert committee that would provide current information regarding the diagnosis, treatment, and prognosis of selected HDCTs, including Marfan syndrome and the Ehlers-Danlos syndromes, and the effect of the disorders and their treatment on functioning. The resulting report, Selected Heritable Disorders of Connective Tissue and Disability, presents the committee's findings and conclusions.

The COVID-19 pandemic has led to dramatic adjustments in cancer care delivery and cancer research. To examine these changes, the National Cancer Policy Forum of the National Academies of Sciences, Engineering, and Medicine convened a virtual workshop, Innovation in Cancer Care and Cancer Research in the Context of the COVID-19 Pandemic, in July 2021. Workshop speakers considered the lessons learned from these adaptations in order to improve the delivery of high-quality cancer care and the conduct of cancer clinical trials in the post-pandemic era. This Proceedings of a Workshop highlights presentations and discussions from the workshop.

Approximately five percent of Medicare beneficiaries (2.2 million Americans) are living with serious illness, as are many other non-Medicare eligible individuals. This number is expected to grow rapidly as the population ages and the prevalence of progressive illness increases. In many communities, particularly urban and rural underserved communities, primary care clinicians are the main workforce caring for people with serious illness, which underscores the need to integrate high quality serious illness care into primary care delivery. To better understand the challenges and opportunities for integrating serious illness care into primary care settings, the National Academies of Sciences, Engineering, and Medicine's Roundtable on Quality Care for People with Serious Illness hosted a virtual workshop on June 10 and 17, 2021. The workshop, titled Integrating Serious Illness Care into Primary Care Delivery, explored the shared principles of primary and serious illness care, the interdisciplinary teams that power both disciplines, the policy issues that can act as barriers to or incentives for integration, and best practices for integrating primary care and serious illness care. This Proceedings of a Workshop summarizes the presentations and discussions that occurred during the workshop.

Every community is affected by traumatic brain injury (TBI). Causes as diverse as falls, sports injuries, vehicle collisions, domestic violence, and military incidents can result in injuries across a spectrum of severity and age groups. Just as the many causes of TBI and the people who experience it are diverse, so too are the physiological, cognitive, and behavioral changes that can occur following injury. The overall TBI ecosystem is not limited to healthcare and research, but includes the related systems that administer and finance healthcare, accredit care facilities, and provide regulatory approval and oversight of products and therapies. TBI also intersects with the wide range of community organizations and institutions in which people return to learning, work, and play, including the education system, work environments, professional and amateur sports associations, the criminal justice system, and others. Traumatic Brain Injury: A Roadmap for Accelerating Progress examines the current landscape of basic, translational, and clinical TBI research and identifies gaps and opportunities to accelerate research progress and improve care with a focus on the biological, psychological, sociological, and ecological impacts. This report calls not merely for improvement, but for a transformation of attitudes, understanding, investments, and care systems for TBI.

Patient-centered outcomes research (PCOR) studies consider the questions and outcomes that are meaningful to patients to compare the effectiveness of different prevention, diagnostic, and treatment options. PCOR also increases patient involvement in their care by providing them an opportunity to evaluate the quality, outcomes, and effectiveness of health care treatments and intervention, especially in areas where there is poor existing clinical evidence. The National Academies of Sciences, Engineering, and Medicine Board on Healthcare Services hosted a series of public workshops to explore ways of accelerating the use of PCOR findings in clinical practice to improve health and health care. Workshop discussions touched on the role of community health workers in helping care providers see and understand the whole picture of patient lives, the need for community engagement to ensure research is conducted and applied to practice equitably, and ways of measuring the impact of efforts to disseminate and implement new practices based on PCOR. This report summarizes the discussions that took place at these workshops.

The Board on Health Care Services of the National Academies of Sciences, Engineering and Medicine hosted a virtual workshop, funded by the Social Security Association, to examine disability associated with hematopoietic stem cell transplantation. The workshop, held on November 15, 2021, included presentations on the functional outcomes for individuals who have received hematopoietic stem cell treatment, as well as medical advances, developments, and research in this area. This Proceedings of a Workshop highlights the presentations and discussions that occurred in the workshop.

Astronauts face unique health-related risks during crewed space missions, and longer-duration missions that extend to greater distances in our solar system (including to the Moon and Mars) will likely increase those risks. Cancer risks due to ionizing radiation exposure are one of these health-related risks. Assessing, managing, and communicating radiation-induced cancer risks associated with spaceflight are challenging because of incomplete knowledge of the radiation environment in space, limited data on radiation-induced cellular damage mechanisms, lack of direct observations from epidemiological studies, and the complexities of understanding radiation risk. At the request of the National Aeronautics and Space Administration (NASA), an ad hoc committee of the National Academies of Sciences, Engineering, and Medicine convened to provide advice on NASA's proposed updates to their space radiation health standard, which sets the allowable limit of space radiation exposure throughout the course of an astronaut's career. Space Radiation and Astronaut Health: Managing and Communicating Cancer Risks provides the committee's recommendations and conclusions regarding the updated space radiation health standard, NASA's radiation risk communication strategies, and a process for developing an ethics-informed waiver protocol for long-duration spaceflight missions.

There is a void in evidence-based information for making informed decisions on how to optimize care for older adults, particularly those 80 years and over. Because older adults are vastly underrepresented in clinical trials, there is a dearth of information about the appropriate use of drugs in this population. Yet older adults have higher rates of comorbidities and simultaneous use of multiple medications than the general population, and are the majority users of many medications. Additionally, age-related physiological and pathological changes, particularly for adults 80 years of age and older, can lead to significant differences in the pharmacokinetics (PK)2 and pharmacodynamics (PD)3 of a given drug compared to the general population. On August 5 and 6, 2020, the National Academies of Sciences, Engineering, and Medicine hosted a workshop titled Drug Research and Development for Adults Across the Older Age Span. The workshop was designed to examine the challenges and opportunities in drug research and development for older adult populations, explore barriers that impede safety and efficacy studies in these populations, and share lessons learned for better understanding clinical pharmacology for populations over age 65. This publication summarizes the presentation and discussion of the workshop.

New technologies and improved understanding of the genesis and progression of various cancers have added to the enthusiasm for potential new strategies to improve screening and early detection of cancer. Research is also under way to evaluate refinements in current screening approaches, including determining optimal screening intervals, the ages at which screening should begin and end, as well as more specific estimates of the potential risks and benefits of screening for certain populations, such as racial and ethnic minority populations and people who have elevated risk for specific cancers. However, there remain significant challenges to developing, validating, and effectively implementing new cancer screening approaches. Guidelines for screening issued by different organizations vary considerably with no clear way of deciding which guidelines are most trustworthy. There is also a need to improve access to high-quality cancer screening and follow-up care, particularly in low-resource communities and among populations who are underserved or have numerous barriers to receiving care. To examine the challenges and opportunities related to improving current approaches to cancer screening, as well as the evidence base for novel cancer screening methods, the National Cancer Policy Forum held a workshop, Advancing Progress in the Development and Implementation of Effective, High-Quality Cancer Screening, on March 2-3, 2020, in Washington, DC. This workshop convened a broad range of experts, including clinicians, researchers, statisticians, and patient advocates, as well as representatives of health care organizations, academic medical centers, insurers, and federal agencies. This publication summarizes the presentations and discussions of the workshop and highlights suggestions from individual participants regarding how to improve cancer screening.

On November 18 and 19, 2019, the National Academies of Sciences, Engineering, and Medicine hosted a public workshop in Washington, DC, titled Sharing Clinical Trial Data: Challenges and a Way Forward. The workshop followed the release of the 2015 Institute of Medicine (IOM) consensus study report Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk, and was designed to examine the current state of clinical trial data sharing and reuse and to consider ways in which policy, technology, incentives, and governance could be leveraged to further encourage and enhance data sharing. This publication summarizes the presentations and discussions from the workshop.

To explore the role of the National Institutes of Health (NIH) in innovative drug development and its impact on patient access, the Board on Health Care Services and the Board on Health Sciences Policy of the National Academies jointly hosted a public workshop on July 24-25, 2019, in Washington, DC. Workshop speakers and participants discussed the ways in which federal investments in biomedical research are translated into innovative therapies and considered approaches to ensure that the public has affordable access to the resulting new drugs. This publication summarizes the presentations and discussions from the workshop.

Temporomandibular disorders (TMDs), are a set of more than 30 health disorders associated with both the temporomandibular joints and the muscles and tissues of the jaw. TMDs have a range of causes and often co-occur with a number of overlapping medical conditions, including headaches, fibromyalgia, back pain and irritable bowel syndrome. TMDs can be transient or long-lasting and may be associated with problems that range from an occasional click of the jaw to severe chronic pain involving the entire orofacial region. Everyday activities, including eating and talking, are often difficult for people with TMDs, and many of them suffer with severe chronic pain due to this condition. Common social activities that most people take for granted, such as smiling, laughing, and kissing, can become unbearable. This dysfunction and pain, and its associated suffering, take a terrible toll on affected individuals, their families, and their friends. Individuals with TMDs often feel stigmatized and invalidated in their experiences by their family, friends, and, often, the health care community. Misjudgments and a failure to understand the nature and depths of TMDs can have severe consequences - more pain and more suffering - for individuals, their families and our society. Temporomandibular Disorders: Priorities for Research and Care calls on a number of stakeholders - across medicine, dentistry, and other fields - to improve the health and well-being of individuals with a TMD. This report addresses the current state of knowledge regarding TMD research, education and training, safety and efficacy of clinical treatments of TMDs, and burden and costs associated with TMDs. The recommendations of Temporomandibular Disorders focus on the actions that many organizations and agencies should take to improve TMD research and care and improve the overall health and well-being of individuals with a TMD.

Sharing knowledge is what drives scientific progress - each new advance or innovation in biomedical research builds on previous observations. However, for experimental findings to be broadly accepted as credible by the scientific community, they must be verified by other researchers. An essential step is for researchers to report their findings in a manner that is understandable to others in the scientific community and provide sufficient information for others to validate the original results and build on them. In recent years, concern has been growing over a number of studies that have failed to replicate previous results and evidence from larger meta-analyses, which have pointed to the lack of reproducibility in biomedical research. On September 25 and 26, 2019, the National Academies of Science, Engineering, and Medicine hosted a public workshop in Washington, DC, to discuss the current state of transparency in the reporting of preclinical biomedical research and to explore opportunities for harmonizing reporting guidelines across journals and funding agencies. Convened jointly by the Forum on Drug Discovery, Development, and Translation; the Forum on Neuroscience and Nervous System Disorders; the National Cancer Policy Forum; and the Roundtable on Genomics and Precision Health, the workshop primarily focused on transparent reporting in preclinical research, but also considered lessons learned and best practices from clinical research reporting. This publication summarizes the presentation and discussion of the workshop.

As part of the overall disability determination process, the Social Security Administration uses a step-by-step approach to understand how severe an individual's condition is and whether it meets program criteria for disability. The use of various types of biomarkers has been suggested as a way to strengthen the amount and quality of objective evidence available to the review process. At the request of the Social Security Administration, the National Academies of Sciences, Engineering, and Medicine's Board on Health Care Services organized a virtual workshop on July 21, 2020, titled The State of the Science of the Use of Biomarkers to Establish the Presence and Severity of Impairments. Workshop discussions focused on the current and potential uses for biomarkers; explored legal and ethical implications associated with biomarker use in clinical decision making; and considered the possible uses of nongenetic biomarkers as tools for the diagnosis or prognosis of the severity of specific physical and mental impairments. This publication summarizes the presentations and discussion of the workshop.

Palliative care is the interdisciplinary specialty focused on improving quality of life for people with serious illness and their families. This interdisciplinary care is provided by doctors, nurses, social workers, chaplains and others who work together with the patient's other doctors to provide an extra layer of support. Such care is appropriate for people at any age and at any stage in a serious illness, and can be provided together with curative treatment to address clinical, emotional, psychosocial and spiritual concerns of the patient and their family. To better understand how the principles of palliative care can be integrated into the overall provision of care and services to those facing serious illness, the Roundtable on Quality Care for People with Serious Illness held a public workshop in April 2017. This publication summarizes the presentations and discussions from the workshop.

The increasing diversity of population of the United States presents many challenges to conducting health research that is representative and informative. Dispersion and accessibility issues can increase logistical costs; populations for which it is difficult to obtain adequate sample size are also likely to be expensive to study. Hence, even if it is technically feasible to study a small population, it may not be easy to obtain the funding to do so. In order to address the issues associated with improving health research of small populations, the National Academies of Sciences, Engineering, and Medicine convened a workshop in January 2018. Participants considered ways of addressing the challenges of conducting epidemiological studies or intervention research with small population groups, including alternative study designs, innovative methodologies for data collection, and innovative statistical techniques for analysis.

Pharmaceutical companies, academic researchers, and government agencies such as the Food and Drug Administration and the National Institutes of Health all possess large quantities of clinical research data. If these data were shared more widely within and across sectors, the resulting research advances derived from data pooling and analysis could improve public health, enhance patient safety, and spur drug development. Data sharing can also increase public trust in clinical trials and conclusions derived from them by lending transparency to the clinical research process. Much of this information, however, is never shared. Retention of clinical research data by investigators and within organizations may represent lost opportunities in biomedical research. Despite the potential benefits that could be accrued from pooling and analysis of shared data, barriers to data sharing faced by researchers in industry include concerns about data mining, erroneous secondary analyses of data, and unwarranted litigation, as well as a desire to protect confidential commercial information. Academic partners face significant cultural barriers to sharing data and participating in longer term collaborative efforts that stem from a desire to protect intellectual autonomy and a career advancement system built on priority of publication and citation requirements. Some barriers, like the need to protect patient privacy, pre- sent challenges for both sectors. Looking ahead, there are also a number of technical challenges to be faced in analyzing potentially large and heterogeneous datasets. This public workshop focused on strategies to facilitate sharing of clinical research data in order to advance scientific knowledge and public health. While the workshop focused on sharing of data from preplanned interventional studies of human subjects, models and projects involving sharing of other clinical data types were considered to the extent that they provided lessons learned and best practices. The workshop objectives were to examine the benefits of sharing of clinical research data from all sectors and among these sectors, including, for example: benefits to the research and development enterprise and benefits to the analysis of safety and efficacy. Sharing Clinical Research Data: Workshop Summary identifies barriers and challenges to sharing clinical research data, explores strategies to address these barriers and challenges, including identifying priority actions and'low-hanging fruit'opportunities, and discusses strategies for using these potentially large datasets to facilitate scientific and public health advances.