Journal of Health Politics Policy and Law最新文献

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. They extracted data on the trial characteristics, endpoint types, and expedited regulatory pathways.

Findings: Among 48 publications eligible for inclusion, 30 covered trial characteristics, 23 covered surrogate measures, and 30 covered regulatory pathways. Trends point toward less frequent randomization, double-blinding, and active controls, with variation by drug type and indication. Surrogate measures are becoming more common but are not consistently well correlated with clinical outcomes. Drugs approved through expedited regulatory pathways often have less rigorous trial design characteristics.

Conclusions: The characteristics of trials used to approve new drugs have evolved over the past two decades along with greater use of expedited regulatory pathways and changes in the nature of drugs being evaluated. While flexibility in regulatory standards is important, policy changes can emphasize high-quality data collection before or after FDA approval.

More than 40 years have passed since the enactment of the Patent and Trademark Amendment (Bayh-Dole) Act, which authorized institutions to patent inventions arising from federally funded research. Although some experts have heralded the Bayh-Dole Act as ushering in a new era of technological advances, others have been less sanguine about its impact. In recent years, the high price of prescription drugs and the patenting of COVID-19 therapeutics and vaccines developed with substantial federal government support have rekindled the debate over whether companies should receive more restricted rights to products originating with government funding. This article traces the history leading to the enactment of the Bayh-Dole Act and critically assesses its strengths and weaknesses as well as unresolved questions concerning its scope. Based on this analysis, the authors propose reforms to better align the Bayh-Dole Act with public values and health outcomes, including clarifying government-use rights, making it easier to invoke march-in rights for failure to meet health and safety needs, increasing transparency in how patents are licensed, and testing different approaches to foster the development and application of inventions.

Consumer cost sharing is widely employed by payers in the United States in an effort to control spending. Most cost-sharing strategies set patient contributions on the basis of costs incurred by payers and often do not consider medical necessity as a coverage criterion. Available evidence suggests that increases in cost sharing worsen health disparities and adversely affect patient-centered outcomes, particularly among economically vulnerable individuals, people of color, and those with chronic conditions. A key question has been how to better engage consumers while balancing appropriate access to essential services with increasing fiscal pressures. Value-based insurance design (VBID) is a promising approach designed to improve desired clinical and financial outcomes, in which out-of-pocket costs are based on the potential for clinical benefit, taking into consideration the patient's clinical condition. For more than two decades, broad multistakeholder support and multiple federal policy initiatives have led to the implementation of VBID programs that enhance access to vital preventive and chronic disease medications for millions of Americans. A robust evidence base shows that when financial barriers to essential medications are reduced, increased adherence results, leading to improved patient-centered outcomes, reduced health care disparities, and in some (but not most) instances, lower total medical expenditures.