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Importance: Recent discussion has focused on questions related to the repeal and replacement of portions of the Affordable Care Act (ACA). However, issues central to the future of health and health care in the United States transcend the ACA provisions receiving the greatest attention. Initiatives directed to certain strategic and infrastructure priorities are vital to achieve better health at lower cost.Objectives: To review the most salient health challenges and opportunities facing the United States, to identify practical and achievable priorities essential to health progress, and to present policy initiatives critical to the nation's health and fiscal integrity.Evidence Review: Qualitative synthesis of 19 National Academy of Medicine-commissioned white papers, with supplemental review and analysis of publicly available data and published research findings.Findings: The US health system faces major challenges. Health care costs remain high at $3.2 trillion spent annually, of which an estimated 30% is related to waste, inefficiencies, and excessive prices; health disparities are persistent and worsening; and the health and financial burdens of chronic illness and disability are straining families and communities. Concurrently, promising opportunities and knowledge to achieve change exist. Across the 19 discussion papers examined, 8 crosscutting policy directions were identified as vital to the nation's health and fiscal future, including 4 action priorities and 4 essential infrastructure needs. The action priorities-pay for value, empower people, activate communities, and connect care-recurred across the articles as direct and strategic opportunities to advance a more efficient, equitable, and patient- and community-focused health system. The essential infrastructure needs-measure what matters most, modernize skills, accelerate real-world evidence, and advance science-were the most commonly cited foundational elements to ensure progress.Conclusions and Relevance: The action priorities and essential infrastructure needs represent major opportunities to improve health outcomes and increase efficiency and value in the health system. As the new US administration and Congress chart the future of health and health care for the United States, and as health leaders across the country contemplate future directions for their programs and initiatives, their leadership and strategic investment in these priorities will be essential for achieving significant progress. [ABSTRACT FROM AUTHOR]

The author offers his views on the practice by medical and scientific journals of requiring authors of papers to declare any financial conflicts of interest (COI) related to their research. He suggests that COIs should focus not only on financial but also on non-financial associations. The importance of disclosing medical biases to the public is emphasized.

This Viewpoint summarizes a new report from the National Academies of Sciences, Engineering, and Medicine that encourages the inclusion of pregnant and lactating individuals in clinical research. [ABSTRACT FROM AUTHOR]

The article focuses on the use of digital multimedia in the promotion and transmission of the informed consent process in medical research. Topics discussed include the rule-making issued the Office for Human Research Protections (OHRP) on how information should be presented, the role of informed consent as a vehicle for disclosure of research information and the studies supporting the effectiveness of digital multimedia. The advantages of digital multimedia over paper media are mentioned.

The article emphasizes the need to reassess the criteria by which scientists and their teams are rewarded for their biomedical research efforts by funding agencies and host institutions. Topics include how to align criteria with the desired research outcomes, such as rewarding high-influence science, how sharing of data can be measured through indexing databases, and performing reproducibility checks. It discusses the need to consider the potential for gaming any appraisal and reward system.

The authors discuss a research of the benefits and challenges of using preprints for non-peer reviewed medical research reports. Topics discussed include the goals of preprints, aim of clinical medical research to improve patient outcomes and public health, hosting and archiving preprint manuscripts in preprint servers, analysis of the policies of preprint servers and review of content on the medRxiv preprint server and trends a year after it was launched in June 2019.

Importance: Convalescent plasma is a potential therapeutic option for patients with coronavirus disease 2019 (COVID-19), but further data from randomized clinical trials are needed.Objective: To evaluate the efficacy and adverse effects of convalescent plasma therapy for patients with COVID-19.Design, Setting, and Participants: Open-label, multicenter, randomized clinical trial performed in 7 medical centers in Wuhan, China, from February 14, 2020, to April 1, 2020, with final follow-up April 28, 2020. The trial included 103 participants with laboratory-confirmed COVID-19 that was severe (respiratory distress and/or hypoxemia) or life-threatening (shock, organ failure, or requiring mechanical ventilation). The trial was terminated early after 103 of a planned 200 patients were enrolled.Intervention: Convalescent plasma in addition to standard treatment (n = 52) vs standard treatment alone (control) (n = 51), stratified by disease severity.Main Outcomes and Measures: Primary outcome was time to clinical improvement within 28 days, defined as patient discharged alive or reduction of 2 points on a 6-point disease severity scale (ranging from 1 [discharge] to 6 [death]). Secondary outcomes included 28-day mortality, time to discharge, and the rate of viral polymerase chain reaction (PCR) results turned from positive at baseline to negative at up to 72 hours.Results: Of 103 patients who were randomized (median age, 70 years; 60 [58.3%] male), 101 (98.1%) completed the trial. Clinical improvement occurred within 28 days in 51.9% (27/52) of the convalescent plasma group vs 43.1% (22/51) in the control group (difference, 8.8% [95% CI, -10.4% to 28.0%]; hazard ratio [HR], 1.40 [95% CI, 0.79-2.49]; P = .26). Among those with severe disease, the primary outcome occurred in 91.3% (21/23) of the convalescent plasma group vs 68.2% (15/22) of the control group (HR, 2.15 [95% CI, 1.07-4.32]; P = .03); among those with life-threatening disease the primary outcome occurred in 20.7% (6/29) of the convalescent plasma group vs 24.1% (7/29) of the control group (HR, 0.88 [95% CI, 0.30-2.63]; P = .83) (P for interaction = .17). There was no significant difference in 28-day mortality (15.7% vs 24.0%; OR, 0.59 [95% CI, 0.22-1.59]; P = .30) or time from randomization to discharge (51.0% vs 36.0% discharged by day 28; HR, 1.61 [95% CI, 0.88-2.95]; P = .12). Convalescent plasma treatment was associated with a negative conversion rate of viral PCR at 72 hours in 87.2% of the convalescent plasma group vs 37.5% of the control group (OR, 11.39 [95% CI, 3.91-33.18]; P < .001). Two patients in the convalescent plasma group experienced adverse events within hours after transfusion that improved with supportive care.Conclusion and Relevance: Among patients with severe or life-threatening COVID-19, convalescent plasma therapy added to standard treatment, compared with standard treatment alone, did not result in a statistically significant improvement in time to clinical improvement within 28 days. Interpretation is limited by early termination of the trial, which may have been underpowered to detect a clinically important difference.Trial Registration: Chinese Clinical Trial Registry: ChiCTR2000029757. [ABSTRACT FROM AUTHOR]

The article discusses the relation of health care payment reform in the U.S. to biomedical innovation. Topics include changes to fee-for-service (FFS) payment systems, the relation of payment reform to efforts to improve health care quality and value, and the adoption of technologies related to biomedical innovation. Problems with adverse consolidation are noted.

The article discusses the notion of targeted science in relation to research on brain disorders. Topics include strategies for funding medical research, the role of the U.S. federal government and private foundations in the funding the BRAIN Initiative for brain disorder research as a public-private partnership, and the banking of biospecimens as part of collaboration in research and data sharing.

The article discusses issues with interoperability in U.S. health information technology (HIT). Topics include possible cost benefits of electronic health records (EHRs), the use of health data in public health and medical research efforts, and the importance of data privacy and data security for building patient trust. Cyber security issues are addressed.

An editorial is presented which addresses the authors' views about scientific research-related authorship and team science collaborations, and it mentions biomedical and basic science research involving big data. According to the article, team science is another name for collaborative research involving multiple investigators and research groups. Biomedical research and the International Committee of Medical Journal Editors' authorship criteria are examined.

This section presents abstracts of research papers on medical topics, including "Promoting Safe Use of Isotretinoin by Increasing Contraceptive Knowledge," "Patient Demands and Requests for Cancer Tests and Treatments" and "Efficacy and Optimization of Palivizumab Injection Regimens Against Respiratory Syncytial Virus Infection".

IMPORTANCE Medical research is a prerequisite of clinical advances, while health service research supports improved delivery, access, and cost. Few previous analyses have compared the United States with other developed countries. OBJECTIVES To quantify total public and private investment and personnel (economic inputs) and to evaluate resulting patents, publications, drug and device approvals, and value created (economic outputs). EVIDENCE REVIEW Publicly available data from 1994 to 2012 were compiled showing trends in US and international research funding, productivity, and disease burden by source and industry type. Patents and publications (1981-2011) were evaluated using citation rates and impact factors. FINDINGS (1) Reduced science investment: Total US funding increased 6% per year (1994-2004), but rate of growth declined to 0.8% per year (2004-2012), reaching $117 billion (4.5%) of total health care expenditures. Private sources increased from 46% (1994) to 58% (2012). Industry reduced early-stage research, favoring medical devices, bioengineered drugs, and late-stage clinical trials, particularly for cancer and rare diseases. National Insitutes of Health allocations correlate imperfectly with disease burden, with cancer and HIV/AIDS receiving disproportionate support. (2) Underfunding of service innovation: Health services research receives $5.0 billion (0.3% of total health care expenditures) or only 1/20th of science funding. Private insurers ranked last (0.04% of revenue) and health systems 19th (0.1% of revenue) among 22 industries in their investment in innovation. An increment of $8 billion to $15 billion yearly would occur if service firms were to reach median research and development funding. (3) Globalization: US government research funding declined from 57% (2004) to 50% (2012) of the global total, as did that of US companies (50% to 41%), with the total US (public plus private) share of global research funding declining from 57% to 44%. Asia, particularly China, tripled investment from $2.6 billion (2004) to $9.7 billion (2012) preferentially for education and personnel. The US share of life science patents declined from 57% (1981) to 51% (2011), as did those considered most valuable, from 73% (1981) to 59% (2011). g Editorials pages 143 and 145 ¡ J Supplemental contentât jama.com CONCLUSIONS AND RELEVANCE New investment is required if the clinical value of past scientific discoveries and opportunities to improve care are to be fully realized. Sources could include repatriation of foreign capital, new innovation bonds, administrative savings, patent pools, and public-private risk sharing collaborations. Given international trends, the United States will relinquish its historical international lead in the next decade unless such measures are undertaken. [ABSTRACT FROM AUTHOR]

IMPORTANCE Reanalyses of randomized clinical trial (RCT) data may help the scientific community assess the validity of reported trial results. OBJECTIVES To identify published reanalyses of RCT data, to characterize methodological and other differences between the original trial and reanalysis, to evaluate the independence of authors performing the reanalyses, and to assess whether the reanalysis changed interpretations from the original article about the types or numbers of patients who should be treated. DESIGN We completed an electronic search of MEDLINE from inception to March 9,2014, to identify all published studies that completed a reanalysis of individual patient data from previously published RCTs addressing the same hypothesis as the original RCT. Four data extractors independently screened articles and extracted data. MAIN OUTCOMES AND MEASURES Changes in direction and magnitude of treatment effect, statistical significance, and interpretation about the types or numbers of patients who should be treated. RESULTS We identified 37 eligible reanalyses in 36 published articles, 5 of which were performed by entirely independent authors (2 based on publicly available data and 2 on data that were provided on request; data availability was unclear for 1). Reanalyses differed most commonly in statistical or analytical approaches (n = 18) and in definitions or measurements of the outcome of interest (n = 12). Four reanalyses changed the direction and 2 changed the magnitude of treatment effect, whereas 4 led to changes in statistical significance of findings. Thirteen reanalyses (35%) led to interpretations different from that of the original article, 3 (8%) showing that different patients should be treated; 1 (3%), that fewer patients should be treated; and 9 (24%), that more patients should be treated. CONCLUSIONS AND RELEVANCE A small number of reanalyses of RCTs have been published to date. Only a few were conducted by entirely independent authors. Thirty-five percent of published reanalyses led to changes in findings that implied conclusions different from those of the original article about the types and number of patients who should be treated. [ABSTRACT FROM AUTHOR]

The article discusses sanctioned trial of mitochondrial replacement therapy (MRT) in Great Britain. Topics discussed include reports and articles that expressed concerns with MRT, comments from Sir Douglas Turnbull whose research group at Newcastle University is likely to go forward with the first MRT procedure, and a recommendation by the Human Fertilisation Embryology Authority that MRT be approved for cautious use in specific circumstances.

The authors reflect on the noted dominance of the narrative positing in biomedical funding and journal publications. Topics covered include details relating to the reported increase in the number of published articles about genome, gene therapy and stem cell, the noted major themes describing the genetic revolution in medicine, and the comprehensive reviews challenging the effective treatment based on stem cells. The need to re-evaluate medical research funding is also discussed.

The authors discuss the development of new hepatitis C virus (HCV) effective treatments. The authors agree about difficulties in determining the cell culture system despite the unlimited number of HCV genome ribonucleic acid (RNA). Also discussed are laboratory studies on cell clone mutations enhancing HCV RNA replication, the genetic system for HCV biology studies and focus of biotech and pharmaceutical companies on searching for HCV inhibitors.

The article reports on the reluctance of researchers to share clinical trial data. Topics discussed include the reasons behind the reluctance, the impact of this behavior on medical research and clinical trial results validation and the Institute of Medicine (IOM) issuing proposed standards for sharing clinical trial data. Also discussed are the benefits of sharing clinical trial data.

An introduction to a series of articles that offers explanations about statistical analytic approaches and methods used in research reported in the "Journal of the American Medical Association (JAMA)" articles.