Framework of the Strengths and Challenges of Clinically Integrated Trials: An Expert Panel Report.

The limitations of the explanatory clinical trial framework include the high expense of implementing explanatory trials, restrictive entry criteria for participants, and redundant logistical processes. These limitations can result in slow evidence generation that is not responsive to population health needs, yielding evidence that is not generalizable. Clinically integrated trials, which integrate clinical research into routine care, represent a potential solution to this challenge and an opportunity to support learning health systems. The operational and design features of clinically integrated trials include a focused scope, simplicity in design and requirements, the leveraging of existing data structures, and patient participation in the entire trial process. These features are designed to minimize barriers to participation and trial execution and reduce additional research burdens for participants and clinicians alike. Broad adoption and scalability of clinically integrated trials are dependent, in part, on continuing regulatory, healthcare system, and payer support. This analysis presents a framework of the strengths and challenges of clinically integrated trials and is based on a multidisciplinary expert "Think Tank" panel discussion that included representatives from patient populations, academia, non-profit funding agencies, the U.S. Food and Drug Administration, and industry.

Lessons From COVID-19 for Pandemic Preparedness: Proceedings From a Multistakeholder Think Tank.

While the coronavirus disease 2019 (COVID-19) pandemic continues to present global challenges, sufficient time has passed to reflect on lessons learned and use those insights to inform policy and approaches to prepare for the next pandemic. In May 2022, the Duke Clinical Research Institute convened a think tank with thought leaders from academia, clinical practice, the pharmaceutical industry, patient advocacy, the National Institutes of Health, the US Food and Drug Administration, and the Centers for Disease Control and Prevention to share, firsthand, expert knowledge of the insights gained from the COVID-19 pandemic and how this acquired knowledge can help inform the next pandemic response. The think tank focused on pandemic preparedness, therapeutics, vaccines, and challenges related to clinical trial design and scale-up during the early phase of a pandemic. Based on the multi-faceted discussions, we outline 10 key steps to an improved and equitable pandemic response.

A Quantitative Framework to Identify and Prioritize Opportunities in Biomedical Product Innovation: A Proof-of-Concept Study.

Importance: Prioritization and funding for health initiatives, including biomedical innovation, may not consistently target unmet public health needs. Objective: To (1) develop a quantitative, databased framework to identify and prioritize opportunities for biomedical product innovation investments based on a multicriteria decision-making model (MCDM) that includes comprehensive measures of public health burden and health care costs, and (2) pilot test the model. Design, Setting, and Participants: The Department of Health and Human Services (HHS) convened public and private experts to develop a model, select measures, and complete a longitudinal pilot study to identify and prioritize opportunities for investment in biomedical product innovations that have the greatest public health benefit. Cross-sectional and longitudinal data (2012-2019) for 13 pilot medical disorders were obtained from the Institute for Health Metrics Global Burden of Disease database (IHME GBD) and the National Center for Health Statistics (NCHS). Main Outcome Measures: The main outcome measure was an overall gap score reflecting high public health burden (composite measure of mortality, prevalence, years lived with disability, and health disparities), or high health care costs (composite measure of total, public, and out-of-pocket health spending) relative to low biomedical innovation. Sixteen innovation metrics were selected to reflect the pipeline of biomedical products from research and development to market approval. A higher score indicates a greater gap. Normalized composite scores were calculated for public health burden, cost, and innovation investment using the MCDM Technique for Order of Preference by Similarity to Ideal Solution method. Results: Among the 13 conditions tested in the pilot study, diabetes (0.61), osteoarthritis (0.46), and drug-use disorders (0.39) had the highest overall gap score reflecting high public health burden, or high health care costs relative to low biomedical innovation in these medical disorders. Chronic kidney disease (0.05), chronic obstructive pulmonary disease (0.09), and cirrhosis and other liver diseases (0.10) had the least amount of biomedical product innovation despite similar public health burden and health care cost scores. Conclusions: In this cross-sectional pilot study, we developed and implemented a data-driven, proof-of-concept model that can help identify, quantify, and prioritize opportunities for biomedical product innovation. Quantifying the relative alignment between biomedical product innovation, public health burden, and health care cost may help identify and prioritize investments that can have the greatest public health benefit.

A call to action-stopping antimicrobial resistance.

Antimicrobial resistance is a public health emergency and represents an impending pandemic. Implementing the lessons learned from responding to the COVID-19 pandemic is essential in accelerating the development of new antimicrobials and therapeutic strategies, rapid diagnostics and improved vaccines. A rededicated, coordinated and collaborative global effort of all stakeholders in academia, healthcare, government agencies, industry, finance and philanthropy is essential to halt the continued spread of antimicrobial resistance and to prevent further morbidity and mortality from drug-resistant pathogens.