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The article discusses a 2024 study on the attitudes of patients 65 years and older on the use of routine clinical data in making treatment decisions. Results show participants' interest in such data and in how drugs work for them, and their concerns on confounders like populations' similarity of conditions with them, and on common minor side effects. Also noted are participants' common view that their physicians determine the evidence's applicability to them, and recommend a medication.

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. The primary outcome was FDA trust, defined as the mean score that each respondent assigned to the FDA across four prespecified axes: (1) competence and effectiveness; (2) commitment to acting in the best interests of the American public; (3) abiding by the rules and regulations set forth by policy or law; and (4) expertise in health, science, and medicine. On multivariable ordinal logistic regression, FDA mistrust was associated with female gender (odds ratio [OR] = 0.74, 95% confidence interval [CI] 0.62–0.88), rural community (OR 0.85, 95% CI 0.75–0.96), conservative political views (OR 0.77, 95% CI 0.74–0.81), worse self‐reported health (OR 0.89, 95% CI 0.80–0.98), lower satisfaction with health care received (OR 0.63, 95% CI 0.56–0.71), less attention to health and science news (OR 0.72, 95% CI 0.64–0.80), and not having children under the age of 18 (OR 0.72, 95% CI 0.60–0.86). These findings underscore the challenges faced by US political leaders in convincing a heterogeneous American public to trust the FDA. The FDA should develop and deploy targeted outreach strategies to populations with lower levels of trust and strengthen internal processes that minimize biases and ensure sound decision‐making. [ABSTRACT FROM AUTHOR]

Importance: The Federal Trade Commission's (FTC) oversight role in the pharmaceutical market is critical to the health of patients and the health care system. This study characterized the FTC's policy on the pharmaceutical market in recent decades, identifying the types of actions it has favored, barriers it has faced, and authorities that remain untested. Objective: To review FTC legal actions in the pharmaceutical market from 2000-2022. Evidence Review: Legal actions were determined through manual review of search results from the FTC's online Legal Library as well as a 2023 FTC report on pharmaceutical actions. The alleged misconduct, type of legal action taken, timing, and outcome were collected from press releases, complaints, orders, and other legal documents. Findings: From 2000-2022, the FTC challenged 62 mergers, brought 22 enforcement actions against allegedly unlawful business practices, and made 1 rule related to pharmaceuticals. Alleged misconduct in enforcement actions involved anticompetitive settlements in patent litigation (n = 11), unilateral actions by brand manufacturers to delay generic competition (n = 6), noncompete agreements (n = 4), and monopolization (n = 3), with 10 outcomes involving monetary payment, totaling $1.6 billion. Of the 62 mergers the FTC challenged, 61 were allowed to continue, 58 after divesting certain drugs to third-party competitors. The FTC's reliance on drug divestitures decreased from 18 drugs per year from 2000-2017 to 4.3 per year from 2017-2023. Conclusions and Relevance: The FTC brought about 1 enforcement action and 3 merger actions per year against pharmaceutical manufacturers from 2000-2022, pursuing a small fraction of the estimated misconduct and consolidation in the pharmaceutical marketplace. Although the FTC faces substantial legal and practical limitations, important tools remain untested, including a rule defining "unfair methods of competition," that may allow it to more effectively prevent repetitive patterns of anticompetitive behavior. This Special Communication examines the Federal Trade Commission's policy and legal actions related to alleged misconduct, consolidation, and anticompetitive behavior in the pharmaceutical market between 2000 and 2022. [ABSTRACT FROM AUTHOR]

This document presents a study on the impact of skinny-label generic competition on Medicare Part D spending from 2015 to 2021. Skinny-label generics are generic drugs that are approved for a subset of the indications of the brand-name drug. The study found that skinny-label generic competition saved Medicare approximately $14.6 billion during this period. The savings were highest for drugs such as Crestor, Lyrica, and Gleevec. The study suggests that skinny-label generics can improve patient access to lower-cost medications and recommends reinforcing the skinny-label pathway to encourage timely generic competition. [Extracted from the article]

Under the Inflation Reduction Act, Medicare will be able to negotiate prices for a limited number of high-cost prescription drugs. This article describes a loophole in the rules around the selection of drugs for negotiation. The authors propose a solution that may reduce perverse incentives for drug manufacturers and improve the ability of Medicare to optimize savings. [ABSTRACT FROM AUTHOR]

Key Points: Question: What is the clinical benefit of cancer drugs granted accelerated approval, and on what basis are they converted to regular approval? Findings: In this cohort study of cancer drugs granted accelerated approval from 2013 to 2017, 41% (19/46) did not improve overall survival or quality of life in confirmatory trials after more than 5 years of follow-up, with results not yet available for another 15% (7/46). Among drugs converted to regular approval, 60% (29/48) of conversions relied on surrogate measures. Meaning: Although accelerated approval can be useful, some cancer drugs do not end up demonstrating benefit in extending patients' lives or improving their quality of life. Importance: The US Food and Drug Administration's (FDA) accelerated approval pathway allows approval of investigational drugs treating unmet medical needs based on changes to surrogate measures considered "reasonably likely" to predict clinical benefit. Postapproval clinical trials are then required to confirm whether these drugs offer clinical benefit. Objective: To determine whether cancer drugs granted accelerated approval ultimately demonstrate clinical benefit and to evaluate the basis of conversion to regular approval. Design, Setting, and Participants: In this cohort study, publicly available FDA data were used to identify cancer drugs granted accelerated approval from 2013 to 2023. Main Outcomes and Measures: Demonstrated improvement in quality of life or overall survival in accelerated approvals with more than 5 years of follow-up, as well as confirmatory trial end points and time to conversion for drug-indication pairs converted to regular approval. Results: A total of 129 cancer drug–indication pairs were granted accelerated approval from 2013 to 2023. Among 46 indications with more than 5 years of follow-up (approved 2013-2017), approximately two-thirds (29, 63%) were converted to regular approval, 10 (22%) were withdrawn, and 7 (15%) remained ongoing after a median of 6.3 years. Fewer than half (20/46, 43%) demonstrated a clinical benefit in confirmatory trials. Time to withdrawal decreased from 9.9 years to 3.6 years, and time to regular approval increased from 1.6 years to 3.6 years. Among 48 drug-indication pairs converted to regular approval, 19 (40%) were converted based on overall survival, 21 (44%) on progression-free survival, 5 (10%) on response rate plus duration of response, 2 (4%) on response rate, and 1 (2%) despite a negative confirmatory trial. Comparing accelerated and regular approval indications, 18 of 48 (38%) were unchanged, while 30 of 48 (63%) had different indications (eg, earlier line of therapy). Conclusions and Relevance: Most cancer drugs granted accelerated approval did not demonstrate benefit in overall survival or quality of life within 5 years of accelerated approval. Patients should be clearly informed about the cancer drugs that use the accelerated approval pathway and do not end up showing benefits in patient-centered clinical outcomes. This study aims to determine whether cancer drugs granted accelerated approval from the US Food and Drug Administration ultimately demonstrate clinical benefit and to evaluate the basis of conversion to regular approval. [ABSTRACT FROM AUTHOR]

Context: The False Claims Act is the US federal government's primary tool for identifying and penalizing pharmaceutical fraud. The Department of Justice uses the False Claims Act to bring civil cases against drug manufacturers that allegedly obtain improper payment from federal programs. Methods: The authors searched the Department of Justice website for press releases published between 2006 and 2022 that announced fraud actions brought against drug companies. They then used the World Health Organization's Anatomical Therapeutic Classification index to identify the classes of prescription drugs implicated in fraud actions. Findings: During fiscal years 2006–2022, payments by six manufacturers amounted to more than 28% of total payments made as a result of federal False Claims Act actions. Nervous system and cardiovascular drugs were the classes of medications most commonly implicated in alleged fraud. Federal officials most frequently alleged that companies improperly promoted nervous system drugs and paid kickbacks to increase revenues from cardiovascular, antineoplastic and immunomodulating, and alimentary tract and metabolism drugs. Conclusions: Despite frequent pharmaceutical fraud settlements and penalties, incidence of alleged fraud among drug companies remains high. Alternative methods for preventing and deterring fraud could help safeguard our health systems and promote public health, and policy makers should ensure that effective fraud enforcement complements preventive public health regulation. [ABSTRACT FROM AUTHOR]

This study estimates public and private spending on genetically targeted treatments for Duchenne muscular dystrophy during years in which the drugs were marketed without completed confirmatory studies. [ABSTRACT FROM AUTHOR]

This cross-sectional study identifies the prevalence of patents on risk evaluation and mitigation strategies and their association with delaying generic competition. [ABSTRACT FROM AUTHOR]

Glucagon-like peptide-1 receptor agonists are effective for treating obesity, but the high cost of these medications endangers the financial viability of our health care system. To ensure that these drugs are available to Medicare beneficiaries, pharmaceutical manufacturers must lower their prices. [ABSTRACT FROM AUTHOR]

Patents prevent generic drug entry. Brand firms file new "method of use" patents for old drugs to prevent generic entry. Congress addressed this issue by creating the "skinny label" pathway, which allows generic firms to use the drug label to indicate that the old drug can only be used for non‐patented uses. This pathway is now in jeopardy due to a recent court case. This paper outlines the issues and suggests possible legislative solutions. [ABSTRACT FROM AUTHOR]

This study evaluates the frequency of terminal disclaimers filed by drug patent holders to obviate obviousness-type double patenting rejections associated with drug patent thickets. [ABSTRACT FROM AUTHOR]

This Viewpoint reviews regulations regarding FDA's handing of confidential commercial information, explains how these regulations serve as a barrier to disclosure of information in the interest of public health, and suggests how information could be carefully shared to improve health outcomes and advance research. [ABSTRACT FROM AUTHOR]

In this Viewpoint, the Supreme Court case FDA v AHM is used to illustrate the tension the FDA faces between science and politics, and state authority over abortion vs federal authority over which drugs may be marketed nationwide. [ABSTRACT FROM AUTHOR]

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." [ABSTRACT FROM AUTHOR]

The article emphasize the crucial role of laboratory-developed tests in patient care, particularly in academic medical centers and caution against restricting beneficial tests. They advocate for a balanced regulatory approach, acknowledging the need for improved safety and effectiveness while ensuring continued access to essential testing.

This article discusses the FDA's approach to assessing the bioequivalence of generic and brand-name drugs, specifically focusing on the case of fluticasone-salmeterol inhalers used to treat chronic obstructive pulmonary disease (COPD). The FDA requires extensive testing for generic drug-device combinations, such as inhalers, to ensure that patients receive similar disease control as those using brand-name versions. The article suggests that further testing of generic fluticasone-salmeterol may not be necessary, as rigorous post-approval studies have shown clinical equivalence between the generic and brand-name versions. The authors also advocate for the FDA to support more clinical outcome database studies to facilitate access to affordable medications for patients with asthma and COPD. [Extracted from the article]