Federal Trade Commission Actions on Prescription Drugs, 2000-2022.

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.

Patents on Risk Evaluation and Mitigation Strategies for Prescription Drugs and Generic Competition.

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

Outcomes of the 340B Drug Pricing Program: A Scoping Review.

Importance: The 340B Drug Pricing Program requires manufacturers to offer discounted drug prices to support safety net hospitals and clinics (covered entities) providing care to low-income populations. Amid expansion, the program has received criticism and calls for reform. Objective: To assess the literature on the foundations of and outcomes associated with the 340B program. Evidence Review: The databases searched in this scoping review included PubMed, Embase, EconLit, National Bureau of Economic Research (NBER), Westlaw, the Department of Health and Human Services Office of the Inspector General (HHS-OIG) website, the Government Accountability Office (GAO) website, and Google in February 2023 for peer-reviewed literature, legal publications, opinion pieces, and government agency and committee reports related to the 340B program. Findings: Among a collected 900 documents, 289 met inclusion criteria: 83 articles from PubMed, 12 articles from Embase, 2 articles from EconLit, 1 article from NBER, 28 articles from Westlaw, 23 legislative history documents, 103 documents from Google, 11 GAO reports, and 26 HHS-OIG reports. Included literature pertained to 4 stakeholders in the 340B program: covered entities, pharmacies, pharmaceutical manufacturers, and patients. This literature showed that hospitals, clinics, and pharmacies generated revenue and manufacturers have forgone revenue from 340B discounted drugs. Audits of covered entities found low rates of compliance with 340B program requirements, whereas mixed evidence was uncovered on how covered entities used their 340B revenue, with some studies suggesting use to expand health care services for low-income populations and others to acquire physician practices and open sites in higher-income neighborhoods. These studies were hampered by a lack of transparency and reporting on the use of 340B revenue. Studies revealed patient benefits from access to expanded health care services, but there was mixed evidence on patient cost savings. Although the review identified considerable research on 340B hospitals, pharmacies, and patients, less research was found evaluating the 340B program's effect on nonhospital covered entities, drug pricing, and racial and ethnic minority groups. Conclusions and Relevance: In this scoping review of the 340B program, we found that the 340B program was associated with financial benefits for hospitals, clinics, and pharmacies; improved access to health care services for patients; and substantial costs to manufacturers. Increased transparency regarding the use of 340B program revenue and strengthened rulemaking and enforcement authority for the Health Resources and Services Administration would support compliance and help ensure the 340B program achieves its intended purposes.

US public investment in development of mRNA covid-19 vaccines: retrospective cohort study.

OBJECTIVE: To estimate US public investment in the development of mRNA covid-19 vaccines. DESIGN: Retrospective cohort study. SETTING: Publicly funded science from January 1985 to March 2022. DATA SOURCES: National Institutes of Health (NIH) Report Portfolio Online Reporting Tool Expenditures and Results (RePORTER) and other public databases. Government funded grants were scored as directly, indirectly, or not likely related to four key innovations underlying mRNA covid-19 vaccines-lipid nanoparticle, mRNA synthesis or modification, prefusion spike protein structure, and mRNA vaccine biotechnology-on the basis of principal investigator, project title, and abstract. MAIN OUTCOME MEASURE: Direct public investment in research and vaccine development, stratified by the rationale, government funding agency, and pre-pandemic (1985-2019) versus pandemic (1 January 2020 to 31 March 2022). RESULTS: 34 NIH funded research grants that were directly related to mRNA covid-19 vaccines were identified. These grants combined with other identified US government grants and contracts totaled $31.9bn (£26.3bn; €29.7bn), of which $337m was invested pre-pandemic. Pre-pandemic, the NIH invested $116m (35%) in basic and translational science related to mRNA vaccine technology, and the Biomedical Advanced Research and Development Authority (BARDA) ($148m; 44%) and the Department of Defense ($72m; 21%) invested in vaccine development. After the pandemic started, $29.2bn (92%) of US public funds purchased vaccines, $2.2bn (7%) supported clinical trials, and $108m (<1%) supported manufacturing plus basic and translational science. CONCLUSIONS: The US government invested at least $31.9bn to develop, produce, and purchase mRNA covid-19 vaccines, including sizeable investments in the three decades before the pandemic through March 2022. These public investments translated into millions of lives saved and were crucial in developing the mRNA vaccine technology that also has the potential to tackle future pandemics and to treat diseases beyond covid-19. To maximize overall health impact, policy makers should ensure equitable global access to publicly funded health technologies.

Frequency of Approval and Marketing of Biosimilars With a Skinny Label and Associated Medicare Savings.

This cohort study assesses the frequency of approval and marketing of skinny-label biosimilars and their savings to Medicare.

INTRODUCTION: Promoting Drug and Vaccine Innovation and Managing High Prices: Introducing a Special Symposium.

This special JLME symposium addresses ways that federal policy can incentivize innovation in medical therapeutics and make pharmaceuticals more financially accessible.

Public Returns on Public Investment: Moderna's Violation of the Social Contract.

In January 2023, Moderna announced its intent to increase the price of the COVID-19 vaccine it co-developed with the National Institutes of Health (NIH) by 400%. The federal government should pressure Moderna to change course and resume buying doses for all Americans, leveraging its purchasing power to obtain a fair price.

High-Priced Sickle Cell Gene Therapies Threaten to Exacerbate US Health Disparities and Establish New Pricing Precedents for Molecular Medicine.

Gene therapies to treat sickle cell disease are in development and are expected to have high costs. The large eligible population size - by far, the largest for a gene therapy - poses daunting budget challenges and threatens to exacerbate health disparities for Black patients, who make up the vast majority of American sickle cell patients.

Industry Payments to Physicians Are Kickbacks. How Should Stakeholders Respond?

Payments from the pharmaceutical industry to US physicians are common. In determining which payments rise to the level of an illegal kickback under the Anti-Kickback Statute (AKS), the Department of Health and Human Services' Office of Inspector General (OIG) has stated in nonbinding guidance that influencing or "swaying" physician prescribing is key. OIG has highlighted as a compliance standard the Pharmaceutical Research and Manufacturers of America Code on Interactions with Health Professions, which stipulates that permissible payments are those that do not interfere with prescribing. However, recent evidence has shown that most payments influence physician prescribing, driving higher prescription drug costs by increasing use of brand-name and low-value drugs. This evidence implies that many payments that are currently commonplace could be subject to prosecution under AKS. Given that these payments increase costs to patients and the health care system, there is a public interest in curtailing them. This article proposes a range of actions available to stakeholders-including industry, providers, regulators, and payers-to mitigate the cost-increasing effect of industry payments to physicians.

State Laws and Generic Substitution in the Year After New Generic Competition.

OBJECTIVES: Substitution of brand-name drugs with less expensive, equally effective interchangeable generics is an important strategy for promoting adherence and controlling prescription drug spending. US state laws govern generic substitution, but there is variability among states in how these laws are designed. We aimed to determine how different features of state laws regulating generic substitution are associated with use of generic drugs. METHODS: Using national claims databases, we studied individuals with commercial insurance or Medicare Advantage plans who newly initiated one of 34 prescription drugs during the year after new generic competition (2017-2018) to determine any association between generic use and 3 different features of state laws. We used multivariable logistic regression to adjust for demographic and clinical characteristics. RESULTS: Of 502 763 individuals who initiated one of the drugs, 409 856 (81.6%) received a generic version. Those in states requiring patient consent or notification had lower use of generics (81.1% vs 82.9%; adjusted odds ratio 0.89; 95% confidence interval 0.87-0.91; P < .001). By contrast, mandating versus permitting generic substitution and protecting pharmacists from liability did not appear to have significant effects. CONCLUSIONS: In this study of commercially insured and Medicare Advantage patients, patients in states requiring consent or notification for pharmacists to substitute Food and Drug Administration-certified interchangeable generics had lower use of generics. Laws in 39 states plus the District of Columbia could be amended to improve use of inexpensive and equally effective generic drugs.

Competition law and pricing among biologic drugs: the case of VEGF therapy for retinal diseases.

Neovascular age-related macular degeneration (AMD) is a progressive eye disease and is a leading cause of vision loss in the Western world. Vascular endothelial growth factor inhibitors have become a mainstay of treatment for this disease. Currently, treatment options include three originator biologics with approvals for neovascular AMD (aflibercept, ranibizumab, and brolucizumab-dbll) and one biologic that is commonly used off-label for the condition (bevacizumab). In the USA, Medicare spending on these drugs consistently surpassed $4 billion per year between 2015 and 2019, driven by high prices and varying off-label use of bevacizumab, which is substantially cheaper than the other biologics used to treat neovascular AMD. In this article, we discuss how legal reform can improve market competition for biologic drugs, using AMD therapies as a case study. We chose this group of drugs for their significant contribution to Medicare spending, the price difference between approved therapies and intravitreal bevacizumab, and because there currently exists a large biosimilar pipeline with many drug candidates in the final stage of development. We propose mechanisms for anticipating and facilitating the market introduction of biosimilars, as well as changes to the pricing model in Medicare that can promote use of cost-effective therapies. Reforms such as empowering Medicare to negotiate drug prices may help ensure that introduction of new biologics and biosimilars for AMD will lower spending and increase patient access.

Patient and Caregiver Experiences With and Perceptions of Risk Evaluation and Mitigation Strategy Programs With Elements to Assure Safe Use.

Importance: The US Food and Drug Administration (FDA) Amendments Act of 2007 authorized the FDA to impose safety requirements on drugs with important risks, such as prescriber certification or routine laboratory testing, to ensure that the benefits of use outweighed the risks. However, little is known about patient and caregiver experiences with these Risk Evaluation and Mitigation Strategy (REMS) programs with Elements to Assure Safe Use (ETASU). Objective: To understand patient and caregiver experiences with and perceptions of REMS programs with ETASU. Design, Setting, and Participants: This qualitative study included semistructured qualitative phone interviews conducted between 2016 and 2017, with initial analysis performed in 2017 and reanalysis performed in 2021. Adult patients prescribed natalizumab or sodium oxybate, adult patients or caregivers of adult patients prescribed vigabatrin, and adult female patients of reproductive age prescribed riociguat were included. Main Outcomes and Measures: Assessment of knowledge, decision-making, medication access, and perceptions of medical privacy. Results: Among 63 participants, 46 (73%) were female. Twenty-five participants (40%) had taken natalizumab, 10 (16%) riociguat, 15 (24%) sodium oxybate, and 10 (16%) vigabatrin. One participant had taken both natalizumab and vigabatrin; 4 (6%) were caregivers of patients using vigabatrin. Most participants expressed knowledge of REMS program requirements, but many lacked the insight that these requirements were part of an FDA-mandated special safety program and expressed difficulty understanding program education materials. REMS requirements made some participants more likely to initiate treatment. However, many reported burdens accessing medication, including the need to travel to certified prescribers or pharmacies. Manufacturer access to personal health information was also controversial, although some participants expressed an altruistic desire to assist others. Conclusions and Relevance: This qualitative study found that REMS programs with ETASU reassured patients and their caregivers about drug safety and helped support medication initiation. However, steps are needed to improve the quality of REMS educational materials, promote efficient medication access, and protect patient privacy.

Indication-Specific Generic Uptake of Imatinib Demonstrates the Impact of Skinny Labeling.

PURPOSE: Generic competition can be delayed if brand-name manufacturers obtain additional patents on supplemental uses. The US Food and Drug Administration allows generic drug manufacturers to market versions with skinny labels that exclude patent-protected indications. This study assessed whether use of generic versions of imatinib varied between indications included and excluded from the skinny labels. METHODS: In this cross-sectional study, we identified adult patients covered by commercial insurance or Medicare Advantage plans who initiated imatinib from February 2016 (first generic availability) to September 2020. Generic versions were introduced with skinny labels that included indications covering treatment of chronic myelogenous leukemia (CML) but excluded treatment of gastrointestinal stromal tumors (GISTs) because of remaining patent protections. Logistic regression was used to determine whether use of generic versus brand-name imatinib differed between patients with a diagnosis of CML or GIST, adjusting for demographics, insurance type, prior use of brand-name drugs, and calendar month. RESULTS: Among 2,000 initiators, 934 (47%) had CML and 686 (34%) had GIST. Within 3 years after generics entered the market, more than 90% of initiators in both groups used generic imatinib. Initiation of generic imatinib was slightly lower among patients with GIST than among patients with CML (85% v 88%; adjusted odds ratio 0.56; 95% CI, 0.39 to 0.80; P ≤ .001). CONCLUSION: Generic versions of imatinib were dispensed frequently for indications both included (CML) and excluded (GIST) from the skinny labeling, although patients with GIST were slightly less likely to receive a generic version. The skinny labeling pathway allowed generics to enter the market before patent protection for treating patients with GIST expired, facilitating lower drug prices.