Publications from the National Academies of Sciences, Engineering, and Medicine provide objective and straightforward advice to decision makers and the public. This site includes Health and Medicine Division (HMD) publications released after 1998. A complete list of HMD’s publications from its establishment in 1970 to the present is available as a PDF.
Examining the Impact of Real-World Evidence on Medical ...
Released: February 12, 2018
Randomized, controlled clinical trials (RCTs) have traditionally served as the gold standard for evidence generation in support of medical product development and evaluation. However, it is increasingly recognized that RCTs have inherent limitations, particularly with regard to generalizability, and time and monetary investment. Data from sources supplemental to RCTs, such as safety surveillance, observational studies, registries, claims, or patient-centered outcomes research, would be valuable to support biomedical research, including medical product development and evaluation.
The Drug Development Paradigm in Oncology: Proceedings ...
Released: July 24, 2017
Advances in cancer research have led to an improved understanding of the molecular mechanisms underpinning the development of cancer and how the immune system responds to cancer. This influx of research has led to an increasing number and variety of cancer therapies in the drug development pipeline. Compared with standard chemotherapies, these new cancer therapies may demonstrate evidence of benefit at an earlier stage of development.
Enabling Precision Medicine: The Role of Genetics in Clinical ...
Released: July 10, 2017
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 hosted the workshop Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development.
Real-World Evidence Generation and Evaluation of ...
Released: February 15, 2017
The traditional process for evaluating new therapeutics does not produce the evidence that patients, clinicians, and payers need for real-world decisions. 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 are numerous and varied, ranging from the ability to determine both large-scale and patient-specific effects of treatments to assessing how therapeutics affect patients’ lives through measurement of lifestyle changes.
Advancing the Discipline of Regulatory Science for Medical ...
Released: April 20, 2016
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 medicine, and other specific scientific disciplines whose focus, aggregation, and ultimate implementation could inform biomedical product development and regulatory decision making. On October 20–21, 2015, the Forum on Drug Discovery, Development, and Translation held a public workshop to facilitate dialogue among stakeholders about the current state and scope of regulatory sciences, opportunities to address barriers to the discipline's success, and avenues for fostering collaboration across sectors. This report is a summary of the workshop.
Rapid Medical Countermeasure Response to Infectious ...
Released: October 12, 2015
On March 26 and 27, 2015, the Institute of Medicine (IOM) Forum on Medical and Public Health Preparedness for Catastrophic Events; Forum on Drug Discovery, Development, and Translation; and Forum on Microbial Threats co-convened a workshop in Washington, DC, to discuss how to achieve rapid and nimble MCM capability for new and emerging threats.
Financial Incentives to Encourage Development of Therapies ...
Released: July 06, 2015
The Institute of Medicine (IOM) Forum on Neuroscience and Nervous System Disorders, in collaboration with the IOM Forum on Drug Discovery, Development, and Translation, convened a workshop on January 20–21, 2015, to explore policy changes that might increase private sector investment in research and development (R&D) innovation that fills unmet medical needs for CNS disorders.
Characterizing and Communicating Uncertainty in the ...
Released: September 26, 2014
On February 12 and May 12, 2014, the Institute of Medicine’s (IOM’s) Forum on Drug Discovery, Development, and Translation held public workshops at FDA Headquarters in White Oak, MD, 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. This report is a summary of the February 12 and May 12, 2014 workshops.
Characterizing Uncertainty in the Assessment of Benefits and ...
Released: April 07, 2014
On February 12, 2014, the Institute of Medicine’s (IOM’s) Forum on Drug Discovery, Development, and Translation (the Forum) held a public workshop as the first of a two-part series 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, as well as their implications for pharmaceutical regulatory decisions.