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Key Points: Question: What was the added therapeutic benefit of the 50 top-selling drugs in Medicare in 2020, as assessed by key non-US health technology assessment (HTA) organizations? Findings: More than half of top-selling drugs in Medicare did not receive a moderate or high added therapeutic benefit rating by the national HTA organization of France, Germany, or Canada; these 27 drugs accounted for $19.3 billion net spending, or 11% of Medicare net prescription drug spending in 2020. Meaning: Many top-selling Medicare drugs provided low added benefits; this information can contribute to price negotiation under the Inflation Reduction Act. Importance: The Inflation Reduction Act of 2022 authorizes Medicare to negotiate prices of top-selling drugs based on several factors, including therapeutic benefit compared with existing treatment options. Objective: To determine the added therapeutic benefit of the 50 top-selling brand-name drugs in Medicare in 2020, as assessed by health technology assessment (HTA) organizations in Canada, France, and Germany. Design, Setting, and Participants: In this cross-sectional study, publicly available therapeutic benefit ratings, US Food and Drug Administration documents, and the Medicare Part B and Part D prescription drug spending dashboards were used to determine the 50 top-selling single-source drugs used in Medicare in 2020 and to assess their added therapeutic benefit ratings through 2021. Main Outcomes and Measures: Ratings from HTA bodies in Canada, France, and Germany were categorized as high (moderate or greater) or low (minor or no) added benefit. Each drug was rated based on its most favorable rating across countries, indications, subpopulations, and dosage forms. We compared the use and prerebate and postrebate (ie, net) Medicare spending between drugs with high vs low added benefit. Results: Forty-nine drugs (98%) received an HTA rating by at least 1 country; 22 of 36 drugs (61%) received a low added benefit rating in Canada, 34 of 47 in France (72%), and 17 of 29 in Germany (59%). Across countries, 27 drugs (55%) had a low added therapeutic rating, accounting for $19.3 billion in annual estimated net spending, or 35% of Medicare net spending on the 50 top-selling single-source drugs and 11% of total Medicare net prescription drug spending in 2020. Compared with those with high added benefit, drugs with a low added therapeutic rating were used by more Medicare beneficiaries (median 387 149 vs 44 869) and had lower net spending per beneficiary (median $992 vs $32 287). Conclusions and Relevance: Many top-selling Medicare drugs received low added benefit ratings by the national HTA organizations of Canada, France, and Germany. When negotiating prices for these drugs, Medicare should ensure they are not priced higher than reasonable therapeutic alternatives. This cross-sectional study assesses Medicare spending on 50 top-selling drugs with limited added clinical benefits using ratings from health technology assessment organizations in Canada, France, and Germany. [ABSTRACT FROM AUTHOR]

PURPOSE Regulatory agencies have sought to speed up the review of new cancer medicines and reduce delays in approval between countries. We examined trends in regulatory review times and association with clinical benefit for new cancer medicines in six jurisdictions: United States (Food and Drug Administration [FDA]), European Union (European Medicines Agency [EMA]), Switzerland (Swissmedic), Japan (Pharmaceuticals and Medical Devices Agency [PMDA]), Canada (Health Canada), and Australia (Therapeutic Goods Administration). METHODS We studied all new cancer drugs approved in the six aforementioned jurisdictions from 2007 to 2020. We extracted all applicable expedited programs, total regulatory review times, and, for drugs first approved by the FDA, times to subsequent regulatory approval. Clinical benefit was assessed using the European Society for Medical Oncology-Magnitude of Clinical Benefit Scale value framework and ASCO-Cancer Research Committee's targets. Nonparametric Kruskal-Wallis test was used to compare total review times for high versus low clinical benefit drugs. RESULTS One hundred and twenty eight drugs received initial approval in at least one of the six included jurisdictions. Most drugs approved by the FDA (91%) and Health Canada (59%) qualified for at least one expedited program within those jurisdictions, compared with 46% of EMA approvals and 18% of PMDA approvals. The FDA was the first regulator to approve 102 (80%) drugs. Delays in submission accounted for a median of 20.2% (EMA) to 83.8% (PMDA) of the time to subsequent approval. There was no association between high clinical benefit and shorter total review times. CONCLUSION Most new cancer therapies were approved first by the FDA, and delays in submission of regulatory applications accounted for substantial delays in approving cancer drugs in other countries. Regulators should prioritize faster review for drugs with high clinical benefit. [ABSTRACT FROM AUTHOR]

Context Methods Findings Conclusions Policy Points Health care systems around the world rely on a range of methods to ensure the affordability of prescription drugs, including negotiating prices soon after drug approval and relying on formal clinical assessments that compare newly approved therapies with existing alternatives. The negotiation framework established under the Inflation Reduction Act is far more limited than other frameworks explored in this study. Adding elements from these frameworks could lead to more effective price negotiation in the United States. Health care systems around the world rely on a range of methods to ensure the affordability of prescription drugs, including negotiating prices soon after drug approval and relying on formal clinical assessments that compare newly approved therapies with existing alternatives. The negotiation framework established under the Inflation Reduction Act is far more limited than other frameworks explored in this study. Adding elements from these frameworks could lead to more effective price negotiation in the United States. In 2022, Congress passed the Inflation Reduction Act, which allowed Medicare, for the first time, to begin negotiating the prices for certain high‐cost brand‐name prescription drugs. Many other industrialized countries negotiate drug prices, and we sought to compare and contrast key features of the negotiation process across several health systems. We focused, in particular, on the criteria for selecting drugs for price negotiation, procedures for negotiation, factors that influence negotiated prices, and how prices are implemented.We included four G7 countries in our analysis (Canada, France, Germany, and the United Kingdom [England]), two Benelux countries (Belgium and the Netherlands), and one Scandinavian country (Norway) with long‐established frameworks for drug price negotiation. We also analyzed the Veterans Affairs Health System in the United States. For each system, we gathered relevant legislation, government publications, and guidelines to understand negotiation frameworks, and we reached out to key drug price negotiators in each system to conduct semistructured interviews. All interviews were recorded, transcribed, and coded, and data were analyzed based on an internal assessment tool that we developed.All eight systems negotiate the prices of brand‐name prescription drugs soon after approval and rely on formal clinical assessments that compare newly approved drugs with existing therapies. Systems in our study differed on characteristics such as whether the body performing clinical assessments is separate from the negotiating authority, how added health benefit is assessed, whether explicit willingness‐to‐pay thresholds are employed, and how specific approaches for priority disease areas are taken.High‐income countries around the world adopt different approaches to conducting price negotiations on brand‐name drugs but coalesce around a set of practices that will largely be absent from the current Medicare negotiation framework. US policymakers might consider adding some of these characteristics in the future to improve negotiation outcomes. [ABSTRACT FROM AUTHOR]

In many countries, health technology assessment (HTA) organizations determine the economic value of new drugs and make recommendations regarding appropriate pricing and coverage in national health systems. In the US, recent policy proposals aimed at reducing drug costs would link drug prices to six countries: Australia, Canada, France, Germany, Japan, and the UK. We reviewed these countries' methods of HTA and guidance on price and coverage recommendations, analyzing methods and guidance documents for differences in (1) the methodologies HTA organizations use to conduct their evaluations and (2) considerations they use when making recommendations. We found important differences in the methods, interpretations of HTA findings, and condition-specific carve-outs that HTA organizations use to conduct evaluations and make recommendations. These variations have ethical implications because they influence the recommendations of HTA organizations, which affect access to the drug through national insurance and price negotiations with manufacturers. The differences in HTA approaches result from the distinct political, social, and cultural contexts of each organization and its value judgments. New cost-containment policies in the US should consider the ethical implications of the HTA reviews that they are considering relying on to negotiate drug prices and what values should be included in US pricing policy. [ABSTRACT FROM AUTHOR]

Background The official prescribing information document distributed with a prescription drug is a key source of safety information, but it may include excessive or insufficient details. Objectives To compare prescribing information approved by the US Food and Drug Administration with the UK, Canada and Australia to identify content differences in safety warnings. Methods For 20 top-selling prescription drugs, we used an automated natural language processing tool to calculate the number and severity of reported adverse drug reactions (ADRs). We fit hierarchical Poisson models and included fixed effects for other prescribing information characteristics. Separately, we analysed the appearance and content of 'black box' warnings. Results There was substantial variation in safety content of approved prescribing information. Canada had the highest median ADRs per drug (138 (IQR 86-234)) and the UK had the lowest (84 (IQR 51-111)). The number of ADRs reported was on average 50% higher in Canada compared with the USA (ratio of ADRs/ document: 1.5, 95% CI 1.4 to 1.6, p<0.001). By contrast, there were on average 15% fewer ADRs listed in the UK compared with the USA (ratio of ADRs/document 0.85 (95% CI 0.78 to 0.93, p<0.001), and 21% fewer ADRs listed in Australia compared with the USS (ratio of ADRs/ document 0.79, 95% CI 0.74 to 0.85, p<0.001). There were no variations in ADR severity. The presence and qualitative content of boxed warnings also showed substantial diversity. Conclusions International variations exist in the presentation of safety data in drug prescribing information, which may have important implications for patient safety. Better international coordination is necessary to enhance use of this information for patient decision-making. [ABSTRACT FROM AUTHOR]

The article reports that the U.S. prices of older generic medications such as albuterol sulfate, enalapril and fluoxetine have increased. Topics discussed include high prescription-drug spending market because of exclusivity and negotiating power, prohibition of prescription drugs importation under the Food, Drug, and Cosmetic Act, and need of alternative strategies to provide less expensive medications.

This article analyzes differences in prescription drug pricing transparency practices among 3 Organisation for Economic Co-operation and Development member nations: the United Kingdom, Germany, and Canada. Specifically, this article compares these countries' policies on list and net price disclosures and on how international reference pricing is used to evaluate merits and drawbacks of different pricing transparency approaches. Finally, the article summarizes what policymakers in the United States should learn from these comparisons., (Copyright 2022 American Medical Association. All Rights Reserved.)

Background: Patenting medicine-delivery devices (inhalers and pens) is controversial when it extends market protections beyond that of the underlying therapeutic agent. We evaluated how common device patenting is, internationally., Method: Using a product sample (n = 88) and an international patent database, we assessed the issue's scope., Results: When comparing the 88 patent portfolios for each product in each country, Canada was found to be among the most impacted, with 90% of the portfolios containing at least one device patent and 35% of the portfolios containing device patents exclusively., Conclusion: Patenting of delivery devices impacts major pharmaceutical manufacturing centres worldwide. International consensus among stakeholders (regulators and payors) is needed on which device modifications represent meaningful clinical value., (Copyright © 2022 Longwoods Publishing.)

Importance: The US Food and Drug Administration (FDA) created the exception from informed consent (EFIC) pathway in 1996 to allow some emergency trials to enroll patients without informed consent. To protect individual autonomy and preserve public trust, the FDA requires that EFIC trial investigators consult with community members before a trial may begin., Objectives: To analyze data from surveys conducted as part of community consultation ahead of EFIC trials and assess levels of public approval., Data Sources: All trials granted an EFIC must submit documentation of compliance with EFIC regulations to a publicly available docket at the FDA. Submissions between November 1, 1996, and October 23, 2017, were reviewed., Study Selection: Trials with survey data were included., Data Extraction and Synthesis: Data were extracted between January 2018 and June 2018 and were analyzed between June 2018 and August 2018. The quality and validity of data were assessed according to Preferred Reporting Items for Systematic Reviews and Meta-analyses (PRISMA) guidelines. A random-effects metaregression was used to assess the association of demographic characteristics with EFIC approval., Main Outcomes and Measures: The primary study outcome was EFIC approval., Results: The FDA docket contained 15 958 pages of material with survey data for 42 448 individuals submitted by 27 trials. Public approval of EFIC varied by question type, with more people willing to approve initiation of EFIC trials in their community (86.5%) than personal enrollment (73.0%), enrollment of a family member (68.6%), or the principle of enrollment without consent (58.4%) (P < .001 for all comparisons). In the United States, African American individuals made up 29.3% of those enrolled in EFIC trials that reported data on race (5064 of 17 302) but only 16.7% of those surveyed as part of community consultation. In the United States and Canada, men made up 42.9% of the surveyed population but 65.6% of those eventually enrolled in EFIC trials (29 961 of 45 694). Groups surveyed with higher proportions of African American and male respondents had lower rates of EFIC approval., Conclusions and Relevance: Public approval of EFIC trials varied by question type and by the respondents' reported race and sex. The demographic characteristics of those surveyed did not match the demographic characteristics of EFIC enrollees. The FDA could strengthen community consultation by standardizing survey instruments and reporting, requiring broader inclusion of African American and male respondents, clarifying the function of surveys in the development and modification of trial protocols, and building more public consensus around the acceptable use of EFIC.