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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]

Policy Points With increasing public attention to cases of inaccurate and misleading laboratory‐developed tests, there have been calls for regulatory reform. To protect patients from faulty laboratory tests, we need a framework that balances comprehensive test review with laboratory flexibility. The Verifying Accurate Leading‐edge IVCT [In Vitro Clinical Test] Development (VALID) Act would have helped ensure laboratory test safety and validity through a much‐needed expansion of Food and Drug Administration (FDA) oversight. However, Congress did not pass the VALID Act in 2022, forcing the FDA to start the regulatory reform process on its own. With increasing public attention to cases of inaccurate and misleading laboratory‐developed tests, there have been calls for regulatory reform. To protect patients from faulty laboratory tests, we need a framework that balances comprehensive test review with laboratory flexibility. The Verifying Accurate Leading‐edge IVCT [In Vitro Clinical Test] Development (VALID) Act would have helped ensure laboratory test safety and validity through a much‐needed expansion of Food and Drug Administration (FDA) oversight. However, Congress did not pass the VALID Act in 2022, forcing the FDA to start the regulatory reform process on its own. [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]

The US government supports drug innovation. It is therefore crucial that it distinguish between high-value and low-value innovation in purchasing expensive prescription drugs and medical devices and ensure the continued discovery of transformative drugs and that patient and taxpayer funds are not wasted. [ABSTRACT FROM AUTHOR]

During the COVID-19 pandemic when access to in-person care was limited, U.S. regulations requiring in-person dispensing of mifepristone for medical abortions were relaxed. Using health care claims, this study estimated changes in the incidence of elective medical and procedural abortions reimbursed by commercial insurance in the United States after the COVID-19 pandemic began. Visual Abstract. Changes in Induced Medical and Procedural Abortion Rates in a Commercially Insured Population, 2018 to 2022: During the COVID-19 pandemic, access to in-person care was limited, and U.S. regulations requiring in-person dispensing of mifepristone for medical abortions were relaxed. Using health care claims, this study estimated changes in the incidence of elective medical and procedural abortions reimbursed by commercial insurance in the United States after the COVID-19 pandemic began. Background: During the COVID-19 pandemic, access to in-person care was limited, and regulations requiring in-person dispensing of mifepristone for medical abortions were relaxed. The effect of the pandemic and accompanying regulatory changes on abortion use is unknown. Objective: To estimate changes in the incidence rate of induced medical and procedural abortions. Design: Serial cross-sectional study with interrupted time-series analyses. Setting: Commercially insured persons in the United States. Participants: Reproductive-aged women. Intervention: Onset of the COVID-19 pandemic in March 2020 and subsequent regulatory changes affecting the in-person dispensing requirement for mifepristone. Measurements: Monthly age-adjusted incidence rates of medical and procedural abortions were measured among women aged 15 to 44 years from January 2018 to June 2022. Medical abortions were classified as in-person or telehealth. Linear segmented time-series regression was used to calculate changes in abortion rates after March 2020. Results: In January 2018, the estimated age-adjusted monthly incidence rate of abortions was 151 per million women (95% CI, 142 to 161 per million women), with equal rates of medical and procedural abortions. After March 2020, there was an immediate 14% decrease in the monthly incidence rate of abortions (21 per million women [CI, 7 to 35 per million women]; P  = 0.004), driven by a 31% decline in procedural abortions (22 per million women [CI, 16 to 28 per million women]; P  < 0.001). Fewer than 4% of medical abortions each month were administered via telehealth. Limitation: Only abortions reimbursed by commercial insurance were measured. Conclusion: The incidence rate of procedural abortions declined during the COVID-19 pandemic, and this lower rate persisted after other elective procedures rebounded to prepandemic rates. Despite removal of the in-person dispensing requirement for mifepristone, the use of telehealth for insurance-covered medical abortions remained rare. Amid increasing state restrictions, commercial insurers have the opportunity to increase access to abortion care, particularly via telehealth. Primary Funding Source: Health Resources and Services Administration. [ABSTRACT FROM AUTHOR]

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. Inadequate agency independence can diminish public confidence in government leaders and the FDA. To consider whether reform of the EUA process might be appropriate, we considered three possible sources of inspiration for balancing independence and accountability in government scientific decision-making: models in other countries, models in other US agencies, and models within the FDA itself. Strategies used in these settings include: (1) expanding the role of advisory committees, (2) increasing transparency of the agency's decision-making process and supporting rationale, and (3) improving management of internal agency disagreement. Such reforms could improve public trust in public health regulation both related to and separate from future emergencies. [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]

This document investigates the practices of the European Medicines Agency (EMA) in using independent advisors for drug approvals. The study compares the frequency and outcomes of advisory practices at the EMA to those of the US Food and Drug Administration (FDA). The findings show that the EMA consulted advisors for a minority of drug approval decisions, with a frequency and alignment similar to the FDA. The study suggests that the EMA and FDA could coordinate to identify best practices for incorporating recommendations into regulatory decision-making. [Extracted from the article]

The article focuses on the Accelerated Approval Program for cancer drugs and the need to strike the right balance between expediting drug approvals based on surrogate measures and ensuring clinical benefits are confirmed in subsequent trials. Topics include the challenges and issues with the current program, proposed changes to the program, and the importance of safeguarding patient interests while accelerating drug development.

Key Points: Question: How have manufacturers of brand-name glucagon-like peptide 1 (GLP-1) receptor agonists used the patent and regulatory system to extend periods of market exclusivity? Findings: Brand-name manufacturers obtained a median of 19.5 patents per GLP-1 receptor agonist and secured a median of 18.3 years of expected protection; more than half of all patents were obtained on the delivery devices rather than active ingredients. No generic competition has yet emerged on these products. Meaning: Long periods of market exclusivity on GLP-1 receptor agonists underscore the need for patent and regulatory reform on drug-device combinations. Importance: Glucagon-like peptide 1 (GLP-1) receptor agonists were first approved for the treatment of type 2 diabetes in 2005. Demand for these drugs has increased rapidly in recent years, as indications have expanded, but they remain expensive. Objective: To analyze how manufacturers of brand-name GLP-1 receptor agonists have used the patent and regulatory systems to extend periods of market exclusivity. Evidence Review: The annual US Food and Drug Administration's (FDA) Approved Drug Products With Therapeutic Equivalence Evaluations was used to identify GLP-1 receptor agonists approved from 2005 to 2021 and to record patents and nonpatent statutory exclusivities listed for each product. Google Patents was used to extract additional data on patents, including whether each was obtained on the delivery device or another aspect of the product. The primary outcome was the duration of expected protection from generic competition, defined as the time elapsed from FDA approval until expiration of the last-to-expire patent or regulatory exclusivity. Findings: On the 10 GLP-1 receptor agonists included in the cohort, drug manufacturers listed with the FDA a median of 19.5 patents (IQR, 9.0-25.8) per product, including a median of 17 patents (IQR, 8.3-22.8) filed before FDA approval and 1.5 (IQR, 0-2.8) filed after FDA approval. Fifty-four percent of all patents listed on GLP-1 receptor agonists were on the delivery devices rather than active ingredients. Manufacturers augmented patent protection with a median of 2 regulatory exclusivities (IQR, 0-3) obtained at approval and 1 (IQR, 0.3-4.3) added after approval. The median total duration of expected protection after FDA approval, when accounting for both preapproval and postapproval patents and regulatory exclusivities, was 18.3 years (IQR, 16.0-19.4). No generic firm has successfully challenged patents on GLP-1 receptor agonists to gain FDA approval. Conclusions and Relevance: Patent and regulatory reform is needed to ensure timely generic entry of GLP-1 receptor agonists to the market. This Special Communication used data from the US Food and Drug Administration to analyze how manufacturers of brand-name glucagon-like peptide 1 (GLP-1) receptor agonists have used patent and regulatory systems to extend periods of market exclusivity. [ABSTRACT FROM AUTHOR]

The U.S. Food and Drug Administration approved the first generic maintenance inhaler for asthma and chronic obstructive pulmonary disease (COPD) in 2019. This cohort study compared the effectiveness and safety of generic and brand-name fluticasone–salmeterol among patients with chronic obstructive pulmonary disease treated in routine care. Visual Abstract. Generic Versus Brand-Name Fluticasone–Salmeterol for COPD.: The U.S. Food and Drug Administration approved the first generic maintenance inhaler for asthma and chronic obstructive pulmonary disease in 2019. This cohort study compared the effectiveness and safety of generic and brand-name fluticasone–salmeterol among patients with chronic obstructive pulmonary disease treated in routine care. Background: In 2019, the U.S. Food and Drug Administration (FDA) approved the first generic maintenance inhaler for asthma and chronic obstructive pulmonary disease (COPD). The inhaler, Wixela Inhub (fluticasone–salmeterol; Viatris), is a substitutable version of the dry powder inhaler Advair Diskus (fluticasone–salmeterol; GlaxoSmithKline). When approving complex generic products like inhalers, the FDA applies a special "weight-of-evidence" approach. In this case, manufacturers were required to perform a randomized controlled trial in patients with asthma but not COPD, although the product received approval for both indications. Objective: To compare the effectiveness and safety of generic (Wixela Inhub) and brand-name (Advair Diskus) fluticasone–salmeterol among patients with COPD treated in routine care. Design: A 1:1 propensity score–matched cohort study. Setting: A large, longitudinal health care database. Patients: Adults older than 40 years with a diagnosis of COPD. Measurements: Incidence of first moderate or severe COPD exacerbation (effectiveness outcome) and incidence of first pneumonia hospitalization (safety outcome) in the 365 days after cohort entry. Results: Among 45 369 patients (27 305 Advair Diskus users and 18 064 Wixela Inhub users), 10 012 matched pairs were identified for the primary analysis. Compared with Advair Diskus use, Wixela Inhub use was associated with a nearly identical incidence of first moderate or severe COPD exacerbation (hazard ratio [HR], 0.97 [95% CI, 0.90 to 1.04]) and first pneumonia hospitalization (HR, 0.99 [CI, 0.86 to 1.15]). Limitations: Follow-up times were short, reflecting real-world clinical practice. The possibility of residual confounding cannot be completely excluded. Conclusion: Use of generic and brand-name fluticasone–salmeterol was associated with similar outcomes among patients with COPD treated in routine practice. Primary Funding Source: National Heart, Lung, and Blood Institute. [ABSTRACT FROM AUTHOR]

This document discusses the challenges and benefits of implementing low-dose anticoagulant use in patients with peripheral artery disease (PAD). The article highlights the prevalence and seriousness of PAD, as well as the underutilization of guideline-recommended therapies. The VOYAGER PAD and COMPASS trials have provided evidence supporting the use of low-dose anticoagulation in PAD patients, particularly those who have undergone endovascular revascularization procedures. The article emphasizes the need for further research, safety studies, and implementation efforts to ensure the effective use of low-dose anticoagulants in clinical practice. Additionally, barriers such as high drug prices and prescriber behavior are discussed, and potential interventions to increase anticoagulation use are suggested. [Extracted from the article]

The article discusses the potential impact of Medicare price negotiations on cancer drug launches. It mentions that the Inflation Reduction Act (IRA) could lead to delays in the release of new cancer drugs, especially those with limited indications.

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 article discusses the introduction of biosimilars as competition to the blockbuster drug Humira (adalimumab), which has been a top-selling drug for the past decade. It examines the potential impact of biosimilars on lowering drug prices and addresses the challenges in biosimilar market competition, including patent issues and financial incentives in the supply chain.

In an analysis of risk evaluation and mitigation strategies for teratogenic drugs, Ameet Sarpatwari, Beatrice Brown and Aaron Kesselheim explore the variation in primary and secondary prevention measures. [ABSTRACT FROM AUTHOR]

Background Antiretroviral (ARV) medications to treat human immunodeficiency virus (HIV) are a major contributor to Medicaid prescription drug spending. Despite having been used for over 3 decades, the first generic ARVs only recently became available, and many newer versions continue to be sold at high prices despite within-class competition. We estimated Medicaid spending on ARVs from 2007 through 2019. Methods Using public Medicaid State Drug Utilization data, we identified trends in ARV spending and use from 2007 through 2019. We estimated net spending and average prices (spending per 30-day supply), accounting for statutory Medicaid rebates, including a 15%–23% base rebate plus additional rebates if a drug's price increased faster than inflation. Results Among 48 ARVs, estimated net Medicaid spending from 2007 through 2019 was $25 billion for 17 million 30-day supplies. Annual use increased 118%, from 0.7 million 30-day supplies in 2007 to 1.6 million in 2019. During this time, estimated annual net spending increased 178%, from $1.1 billion to $3.0 billion, and average net prices increased 28%, from $1432 to $1830 per 30-day supply. Conclusions Annual Medicaid net spending on ARVs nearly tripled from 2007 to 2019, due to a combination of expanded use and rising prices. Medicaid did not extract expected benefits from its mandatory inflationary rebates because they were offset by use of newer, more expensive ARVs. To better control spending related to products with incremental innovation, the US government should be authorized to assure that launch prices for new drugs are aligned with the added benefit they offer over existing therapies. [ABSTRACT FROM AUTHOR]

This study uses annual Medicare Part B spending data to examine the potential savings from the drug pricing reforms in the Inflation Reduction Act of 2022. [ABSTRACT FROM AUTHOR]

The USA pays more for brand-name prescription drugs than any other country and new legislation from August 2022 gives Medicare the authority to directly negotiate certain drug prices with manufacturers starting in 2026—something the federal insurer had been prohibited from doing for its prior history. As the USA prepares for negotiations, we therefore surveyed how comparable industrialized countries use statutory requirements and procedures to negotiate brand-name drug prices. Guidance documents, regulations, government and academic publications were reviewed to identify the process of negotiating drug prices in peer countries that have been cited as potential examples for US payment reform: Australia, Canada, France, Germany, and the UK. Processes for arriving at a final price for a drug generally fall under three approaches: statutory rebates, setting a maximum price, and arbitration between national (public) insurers and manufacturers. Each approach to price negotiation could be adopted by Medicare and reduce spending even if Medicare does not adopt an exclusionary or closed formulary. Much remains to be determined about how the new price negotiation authority in the USA will be implemented, and policymakers can learn from comparator countries' statutory and regulatory strategies for price negotiation. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

New research and a government investigation have shed light on an anticompetitive practice called "Product Hopping" and specifically how it was employed in the case of the multiple sclerosis treatment glatiramer acetate beginning in 2014, which cost payers billions of dollars. We examine this case as well as a separate, impending instance of product hopping. [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]

Aims: To describe naloxone dispensing in Medicaid fee-for-service (FFS) and examine relationships between copays and coverage limits for naloxone and its dispensing rates. Methods: Cross-sectional study using Medicaid FFS State Drug Utilization Data to quantify the use of naloxone in 2018. The primary outcomes of this study were the proportion of naloxone prescriptions relative to all prescriptions and all opioid prescriptions dispensed in each state. We obtained drug benefit design information from the Medicaid Behavioral Health Services Database. The primary analysis examined the influence of copays (yes/no), copay amounts, and coverage limits on medication dispensing using simple linear regression, excluding states with no measurable use or less than 5% Medicaid FFS. Results: We found substantial variability across 50 states and DC in the proportion of prescriptions dispensed for Narcan and generic naloxone. We found a positive relationship between copay and copay amount and dispensing of generic naloxone. However, a sensitivity analysis including the broadest possible cohort of states failed to confirm this relationship. We found no other relationships between copays or coverage limits and dispensing of any naloxone formulation. Conclusions: Substantial variation exists between the rates of naloxone dispensing across the US for Medicaid patients, but we did not find a meaningful relationship between plan design and dispensing. Whether drug benefit designs in Medicaid influence naloxone use requires further evaluation to avoid limiting access to this life-saving medication. [ABSTRACT FROM AUTHOR]

The authors address government patent use to promote public health in the U.S. Topics discussed include statutes that protected new medications which includes the Hatch-Waxman Act, one way to facilitate public access to high-cost medications, and challenges facing government manufacture of patented drugs without a license.

The article reports that Health law is slow-moving accretive, but it also shapes the landscape of health care delivery in powerful ways. Since implementation of the law requires democratic deliberation, administrative oversight, and judicial enforcement, health law is often a lagging indicator of the norms of health care practice.

One reason expressed in surveys of people reporting coronavirus disease 2019 (COVID-19) vaccine hesitancy is how rapidly these vaccines have reached the market. To estimate the length of time the COVID-19 vaccine spent in research and development as compared to other novel vaccines, we apply previously established methods for estimating medical product development times, using the key associated patent filings cited by the manufacturer as the marker of when commercial development activity began. Applying these methods to a cohort of recently approved innovative vaccines and comparing them to the first-approved COVID-19 vaccine (BioNTech/Pfizer), we found key patent filings for the technology in this COVID-19 vaccine occurred 10.0 years prior to regulatory authorization. By this metric, the development timelines for innovative vaccines have been shortening since the 1980s, and the COVID-19 vaccine comfortably fits within this pattern. Vaccine development timelines have now even drawn to parity with many of the most commonly used drugs. [ABSTRACT FROM AUTHOR]

AIMS: To describe naloxone dispensing in Medicaid fee-for-service (FFS) and examine relationships between copays and coverage limits for naloxone and its dispensing rates. METHODS: Cross-sectional study using Medicaid FFS State Drug Utilization Data to quantify the use of naloxone in 2018. The primary outcomes of this study were the proportion of naloxone prescriptions relative to all prescriptions and all opioid prescriptions dispensed in each state. We obtained drug benefit design information from the Medicaid Behavioral Health Services Database. The primary analysis examined the influence of copays (yes/no), copay amounts, and coverage limits on medication dispensing using simple linear regression, excluding states with no measurable use or less than 5% Medicaid FFS. RESULTS: We found substantial variability across 50 states and DC in the proportion of prescriptions dispensed for Narcan and generic naloxone. We found a positive relationship between copay and copay amount and dispensing of generic naloxone. However, a sensitivity analysis including the broadest possible cohort of states failed to confirm this relationship. We found no other relationships between copays or coverage limits and dispensing of any naloxone formulation. CONCLUSIONS: Substantial variation exists between the rates of naloxone dispensing across the US for Medicaid patients, but we did not find a meaningful relationship between plan design and dispensing. Whether drug benefit designs in Medicaid influence naloxone use requires further evaluation to avoid limiting access to this life-saving medication. [ABSTRACT FROM AUTHOR]

The "right to repair" movement highlights opportunities to reduce health care costs and promote public health resilience through increased competition in the way in which medical devices are serviced and updated over their lifespan. We review legislative and legal facets of third-party repair of medical devices, and conclude with specific recommendations to help this market function more efficiently to the benefit of patients and health care systems. [ABSTRACT FROM AUTHOR]

The article presents the discussion on providing some insight into the future of tobacco regulation. Topics include FDA playing important roles beyond simply setting basic quality standards as a purveyor of advice; and decisions around medical products promoting the general public health as well as the health of individuals.

Brand-name prescription drug manufacturers use various strategies to extend their market exclusivity periods by delaying generic or biosimilar competition. Recent Congressional legislation has targeted four such tactics. We analyze these proposals and assess their likely effect on competition in the U.S. drug market. [ABSTRACT FROM AUTHOR]

The article presents the discussion on Food and Drug Administration (FDA) facing a regulatory decision. Topics include offering insight to agency leaders facing difficult decisions for approving a drug for marketing or permitting a drug to remain on the market; and consisting of a conclusory sentence stating the application not raising important safety or efficacy issues.

If Medicare purchased generic drugs in the maximum quantity supplied by MCCPDC, it could have saved $3.3 billion (36%) on 77 of 89 (87%) generic drugs. Graph: Generic Drugs With the Highest Estimated Medicare Part D Savings Based on MCCPDC Prices If Medicare had purchased drugs in the minimum quantity available from MCCPDC, it could have saved $1.5 billion (16%) on 41 of 89 (46%) drugs. We identified 2020 Medicare Part D spending for 89 (82%) drugs, excluding 20 drugs with multiple dosage forms (for example, cream and ointment) because average weighted Medicare unit prices by ingredient were not comparable to MCCPDC unit prices ([2]). [Extracted from the article]

The number of new medical devices cleared by the US Food and Drug Administration (FDA) through the 510(k) pathway that has subsequently been associated with safety risks has led to discussion of approaches to regulation and communication of device risks. As debate continues over whether the pathway needs to be altered, features of ethical use of 510(k)-cleared devices can include (1) heightened caution with respect to newly cleared 510(k) products until adequate data are gathered through postmarket surveillance, (2) facilitating informed consent by improving physician and patient knowledge of the 510(k) pathway, and (3) basing distribution of these devices on individual risk assessments while ensuring equitable access. [ABSTRACT FROM AUTHOR]

Researchers and policy makers in the US are exploring the implementation of health technology assessment and value-based pricing to negotiate drug prices and limit spending. Objections made to the quality-adjusted life-year (QALY), the most frequently used health economic outcome for such assessments, are a barrier to the adoption of these tools. This literature review identifies and addresses the range of criticisms made against QALYs. Methods-based criticisms require attention from stakeholders to address well-known shortcomings of the QALY and ensure consistency. Ethical criticisms, however, do not apply only to the QALY and require political decisions about societal values. Understanding and overcoming criticisms of the QALY to enable its use as part of health technology assessment and value-based pricing will be crucial as US policy makers seek to address high drug costs and health care spending. [ABSTRACT FROM AUTHOR]

High biologic drug prices have placed substantial strain on the US health care system. The Biologies Price Competition and Innovation Act (BPCIA), passed in 2010 as part of the Affordable Care Act, created an abbreviated approval pathway for biosimilars--versions of "originator" biologic drugs made by different manufacturers--to help address this issue. However, a decade after its passage, the BPCIA has spurred only limited competition. We examined the role that litigation has played in this muted success, reviewing all lawsuits related to the BPCIA filed between its enactment and August 1, 2020. Our review identified two key problems: noncompliance with steps in the complex litigation process established by the BPCIA and large numbers of patents enforced by originator manufacturers. Both actions have contributed to frequent confidential settlements between originator and biosimilar manufacturers that have delayed the availability of biosimilars. To facilitate more timely biosimilar entry, policy makers should consider limits on patent prosecution, compulsory public patent listing, and enhanced antitrust enforcement. [ABSTRACT FROM AUTHOR]

This study assesses the frequency of continuation patents on brand-name drugs approved by the US Food and Drug Administration from 2000 to 2015. [ABSTRACT FROM AUTHOR]

Over-the-counter (OTC) drugs are ubiquitous in the US. Policymakers have long debated how to modernize the system for making determinations of safety and effectiveness and addressing safety issues with OTC drugs. [ABSTRACT FROM AUTHOR]

Although generic drugs are typically inexpensive, rising prices among some generic drugs in recent years have raised concern. Using Medicaid data, we found that one in five generic drugs sold in the US experienced a price spike (defined as a doubling in price over the course of one year) initiated by at least one manufacturer during the period 2014-17. There was a trend toward fewer price spikes each year, from 7.8 percent of drugs in 2014 to 5.8 percent in 2017. Among drugs experiencing price spikes, 51 percent were injected products, 64 percent had three or fewer manufacturers, and 18 percent were in shortage at the time of the spike. Generic drug price spikes cost Medicaid $1.5 billion during 2014-16, representing 4.2 percent of all Medicaid generic drug spending in that period. The trend toward fewer price spikes over time may be due to increased public scrutiny and regulatory actions. However, price spikes can be very costly, and additional policies are needed to both ensure adequate competition and control prices among generic drugs. [ABSTRACT FROM AUTHOR]

Objectives: Many countries use international (or external) reference pricing-benchmarking prices against those in other countries-to manage spending on prescription drugs. By contrast, the United States (US) allows manufacturers to set drug prices freely. In December 2019, a major bill passed the House of Representatives that would introduce international reference pricing to reduce US drug spending. In September 2020, President Trump issued an executive order to apply international reference pricing for drugs purchased under Medicare. As US policymakers consider adopting reference pricing, it is important to recognize four key administrative issues that have complicated other countries' experiences.Methods: We analyzed the US policy proposals and literature on international experience with international reference pricing to identify implementation challenges and potential effects of US adoption of international reference pricing.Results: Four key administrative issues were identified: lack of price transparency, delays in market approvals, the frequency of price revisions, and the prevalence of cross-referencing.Conclusions: Failure to account for the key issues in the emerging US approach will lead to overspending from overestimation of prices. Policymakers also need to recognize the collateral effects that the US adoption of international reference pricing may have on other countries' prices. Given the size of the pharmaceutical market in the US and other market issues, US reference pricing will likely increase drug list and net prices in other countries. Because of limitations in implementation and collateral effects, US policymakers should consider international reference pricing as a supportive tool alongside other cost containment policies, such as value-based pricing or volume agreements. International reference pricing could limit drug spending in the US but faces implementation challenges and will negatively affect other countries. [ABSTRACT FROM AUTHOR]

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]

The Food and Drug Administration (FDA) approves vaccines when their benefits outweigh the risks for their intended use. In this article we review the standard FDA approach to vaccine evaluation, which underpins its current approaches to assessment of vaccines to prevent Coronavirus disease 2019 (COVID-19). The FDA has established pathways to accelerate vaccine availability before approval, such as Emergency Use Authorization, and to channel resources to high-priority products and allow more flexibility in the evidence required for approval, including accelerated approval based on surrogate markers of effectiveness. Among the thirty-five new vaccines approved in the US from 2006 through October 2020, about two-thirds of their pivotal trials used the surrogate outcome of immune system response, and only one-third evaluated actual disease incidence. Postapproval safety surveillance of new vaccines, and particularly vaccines receiving expedited approval, is crucial. This has generally been accomplished through such mechanisms as the Centers for Disease Control and Prevention (CDC) and FDA Vaccine Adverse Event Reporting System, the CDC Vaccine Safety Datalink, and the CDC Clinical Immunization Safety Assessment Project. Adverse events detected in this way may lead to changes in a vaccine's recommended use or withdrawal from the market. Regulatory oversight of new vaccines must balance speed with rigor to effectively address the pandemic. [ABSTRACT FROM AUTHOR]

Mayookha Mitra-Majumdar and Aaron Kesselheim reflect on steps taken to combat reporting bias in clinical trials over the last two decades. [ABSTRACT FROM AUTHOR]

There is popular and bipartisan support for legalizing the importation of lower-cost medicines from Canada to help reduce the high prescription drug costs that Americans pay. Despite the wide interest in this policy, attempts over the last sixteen years to create a formal system for large-scale prescription drug importation in the United States have failed. The Trump Administration recently issued a final rule to enable the legal importation of prescription drugs from Canada, but the rule has important design flaws and seems destined to suffer a similar fate as previous efforts. In this Article, we argue that prescription drug importation is a form of international regulatory engagement that can work, but not in the manner that recent congressional legislation or the Trump Administration has proposed. Importation of prescription drugs, even foreign versions of already-approved drugs, requires the importing nation to accept the marketing approval standards, processes, and product-specific decisions of the exporting nation as equivalent to domestic regulation. The FDA, however, has made far fewer determinations of foreign regulatory equivalence than its counterpart regulators. As a result, the statutory requirements for the FDA maintaining direct oversight over prescription drug imports from Canada are onerous and unlikely ever to be fulfilled. Examining U.S. prescription drug importation as a form of reliance on the equivalence of foreign regulation is, as far as we can determine, a novel inquiry, and it offers useful insights. Foreign equivalence determinations have been successfully used in pharmaceutical regulation in two contexts: (1) trade initiatives and (2) circumstances in which regulatory agencies were unable to fulfill their core institutional mandates without relying on the decisionmaking of their foreign counterparts. The FDA has not fit neatly into either of these contexts. In contrast to many of its foreign counterparts, the FDA has consolidated authority over pharmaceutical regulation, which it sustains through its reputation among its constituents--appropriators, consumers, pharmaceutical product sponsors, and the relevant medical and scientific communities--for overseeing the safety, efficacy, and quality of medicines. The FDA has resisted risking any harm to that gatekeeper reputation that might follow from its pursuit of other policy objectives, such as lowering drug prices or facilitating trade. Furthermore, FDA officials describe themselves as "the gold standard" for drug review--more thorough and rigorous about regulation than their counterparts--and, until recently, as able to fulfill their core institutional mandates without the cooperation of foreign counterparts. Based on this analysis of the political economy of pharmaceutical regulation and international regulatory cooperation at the FDA, we propose that U.S. prescription drug importation could be successfully used to reduce generic drug shortages, a persistent public health problem that the FDA has struggled to solve independently. We argue that the same analysis can help identify other circumstances when the FDA might usefully engage foreign counterparts, such as foreign manufacturer inspections for the rapidly increasing volume of U.S. drug imports, the growing complexity of global pharmaceutical supply chains, and the technological advances in personalized medicines. [ABSTRACT FROM AUTHOR]