Physician-Patient Communication about Novel Drugs and High-Risk Medical Devices.

Background: After a new drug or medical device is approved by the US Food and Drug Administration (FDA), physician-patient communication about benefits and risks is critical, including whether the product was approved through an expedited pathway based on limited evidence. In addition, physician reporting of drug- and device-related adverse events in real-world use is necessary to have a complete safety profile. We studied physician-reported communication and safety-reporting practices related to drugs and devices.

Trust in the Food and Drug Administration: A National Survey Study.

Building trust in public health agencies like the US Food and Drug Administration (FDA) has become a key government priority. Understanding the roots of FDA mistrust is important if the agency is to develop targeted messaging and reforms aimed at building confidence in the agency. We conducted a survey of 2,021 respondents in the US probing attitudes toward the FDA.

Assessment of Preanalytical Cerebrospinal Fluid Handling and Storage Factors on Measurement of Aβ1-42, Aβ1-40, and pTau181 Using an Automated Chemiluminescent Platform.

Background: Standardizing cerebrospinal fluid (CSF) laboratory protocols will improve the reliability and availability of clinical biomarker testing required for prescription of novel Alzheimer disease (AD) therapies. This study evaluated several preanalytical handling and storage factors common to β-amyloid1-42 (Aβ1-42), β-amyloid1-40 (Aβ1-40), and phosphorylated tau (pTau181) concentrations including storage at different temperatures, extended cap contact, various mixing methods, and multiple freeze-thaw cycles.

Methods: Aβ1-42, Aβ1-40, and pTau181 concentrations were measured using LUMIPULSE G1200 automated assays.

Leah Z. Rand, Daniel P. Carpenter, Aaron S. Kesselheim, Anushka Bhaskar, Jonathan J. Darrow, and William B. Feldman Reply.

The authors respond to a letter by Mitchell Berger in the March-April 2024 issue of the Hastings Center Report concerning their essay "Securing the Trustworthiness of the FDA to Build Public Trust in Vaccines."

Physicians' Perspectives On FDA Regulation Of Drugs And Medical Devices: A National Survey.

Physicians' knowledge of Food and Drug Administration (FDA) approval processes is important in informing clinical decisions and patient discussions. Among a randomly selected national sample of 509 internists, cardiologists, and oncologists, 41 percent reported moderate or better understanding of the FDA's drug approval process, and 17 percent reported moderate or better understanding of the FDA's medical device approval process. Nearly all physicians thought that randomized, blinded trials that met primary endpoints should be very important factors required to secure regulatory approval.

Pharmaceutical Innovation in Latin America and the Caribbean.

This study assesses Latin America and Caribbean countries' capacity to innovate new pharmaceuticals, defined as developing new drugs and vaccines, repurposing existing drugs, and inventing around patents to produce new drug variations. Vaccine innovation includes reengineering existing vaccines, developing new manufacturing methods, and the clinical development of unapproved vaccine candidates initiated elsewhere.

Securing the Trustworthiness of the FDA to Build Public Trust in Vaccines.

The Covid-19 pandemic highlighted the need to examine public trust in the U.S. Food and Drug Administration (FDA) vaccine approval process and the role of political influence in the FDA's decisions.

Prioritizing Early Disease Intervention.

Scholars and practitioners have described how investing in health care earlier rather than later can be beneficial, from how "biomarkers" offer promise for early disease detection to healthcare system "incentives" that can promote early preventive medicine. Work by health economists has also made clear that the "health capital" of an individual depreciates over time in the absence of investments in health. Yet, our current policy makers and healthcare system continue prioritizing care of late-stage complex symptomatic illness, often when cure is impossible and disease reversal is improbable, thus exacerbating public health burdens.

Variation in Endpoints in FDA Medication Approvals: A Review of Acute and Preventive Migraine Medications.

Background And Objective: To assess the characteristics and extent of variation of the endpoints used in trials supporting the US Food and Drug Administration (FDA) approval of medications treating migraine.

Methods: Using the Drugs@FDA online database, we identified novel prescription medications approved by the FDA between January 2001 and September 2022, for migraine with or without aura, for both acute and preventive treatment, and for episodic and chronic presentations. For each medication, we used the most recent FDA-approved labeling to identify indication, mechanism of action, mode of administration, manufacturer, approval year, number of pivotal trials, trial design, and primary endpoints.

An Empirical Review of Key Glucose Monitoring Devices: Product Iterations and Patent Protection.

Introduction: Each year, people with diabetes and their insurers or governments spend billions of dollars on blood glucose monitors and their associated components. These monitors have evolved substantially since their introduction in the 1970s, and manufacturers frequently protect original medical devices and their modifications by applying for and obtaining patent protection.

Research Design And Methods: We tracked the product iterations of five widely used blood glucose monitors-manufactured by LifeScan, Dexcom, Abbott, Roche, and Trividia-from information published by the U.

Trust and Regulation: Assuring Scientific Independence in the FDA's Emergency Use Authorization Process.

Since 2004, the US Food and Drug Administration (FDA) has had the authority to allow access to unapproved medical products via the Emergency Use Authorization (EUA) pathway during times of emergency. It was rarely used until the COVID-19 pandemic, when concerns arose regarding the role of political pressure in the FDA's issuance of some EUAs, such as for hydroxychloroquine. Although US government officials should be responsive to the public, democratic accountability must be balanced against the need for thoughtful science-based decision-making.

CSF Alzheimer Disease Biomarkers: Time-Varying Relationships With MCI Symptom Onset and Associations With Age, Sex, and .

Background And Objectives: This study aimed to examine whether baseline CSF measures of Alzheimer disease (AD)-related pathology are associated with the time to onset of mild cognitive impairment (MCI) and whether these associations differ by age, sex, () status, and proximal (≤7 years) vs distal (>7 years) time to symptom onset.

Methods: Measures of amyloid (Aβ1-42 and Aβ1-40), phospho-tau (ptau181), and total tau (t-tau) were determined from CSF samples obtained at baseline from participants in an ongoing longitudinal project, known as the Biomarkers for Older Controls at Risk for Alzheimer Disease study (BIOCARD) study. The fully automated, Lumipulse G immunoassay was used to analyze the specimens.

Communication of Drug Efficacy Information via a Popular Online Platform.

Background: Open-source online information channels have become increasingly important to the dissemination of medical information, including information about pharmaceuticals. We sought to determine the extent to which one prominent source of online information, Wikipedia, presented quantitative efficacy data about drugs.

Methods: Using the Drugs@FDA website, we identified all new drugs approved by the Food and Drug Administration (FDA) from 1982 to 2020 and their associated Wikipedia pages, and used dummy variables to code for the presence of efficacy data, safety data, and usage data.

Trends in the Quality of Evidence Supporting FDA Drug Approvals: Results from a Literature Review.

Context: New drug approvals in the United States must be supported by substantial evidence from "adequate and well-controlled" trials. The Food and Drug Administration (FDA) has flexibility in how it applies this standard.

Methods: The authors conducted a systematic literature review of studies evaluating the design and outcomes of the key trials supporting new drug approvals in the United States.

CSF Biomarkers Predict Gait Outcomes in Idiopathic Normal Pressure Hydrocephalus.

Background And Objectives: The assessment of biomarkers in selecting patients with idiopathic normal pressure hydrocephalus (iNPH) for shunt surgery has been limited to small cohort studies and those with limited follow-up. We assessed the potential for CSF biomarkers in predicting immediate response to CSF tap test (TT) and long-term response after shunt surgery.

Methods: CSF was obtained from patients with iNPH referred for CSF TT after baseline assessment of cognition and gait.