Launch and Postlaunch Price Developments of New Drugs in the US, Germany, and Switzerland.

Importance: High prices of new drugs are concerning. What remains to be understood are price developments after entering the market because not only initial prices, but also postlaunch prices, contribute to drug spending.

Objective: To analyze launch and postlaunch price developments of new drugs in the US, Germany, and Switzerland.

Why effect sizes are systematically larger for progression-free survival than overall survival in cancer drug trials: Prognostic scores as a way forward.

Cancer drugs have accumulated the most approvals over the past years. Overall survival (OS) is considered the gold standard for cancer trial outcomes. However, its use has declined over the past years, in favor of surrogate endpoints, such as progression-free survival (PFS).

Comparative Analysis of Drug Shortages in the US and Germany (2016 - 2023).

This article analyzes trends in drug shortages in the US and Germany, the largest pharmaceutical market in Europe, between 2016 and 2023. It assesses the commonalities and differences between the countries in terms of active substances in shortage, time duration in shortage, and cyclic trends.

Drug Prices After Patent Expirations in High-Income Countries and Implications for Cost-Effectiveness Analyses.

Importance: Understanding how patent expirations affect drug prices is crucial because price changes directly inform accurate cost-effectiveness assessments. This study investigates the association between patent expirations and drug prices in 8 high-income countries and evaluates how the changes affect cost-effectiveness assessments.

Objective: To analyze how the expiration of drug patents is associated with drug price changes and to assess the implications of these price changes for cost-effectiveness evaluations.

Defining 'therapeutic value' of medicines: a scoping review.

Objectives: In recent years, discussions on the importance and scope of therapeutic value of new medicines have intensified, stimulated by the increase of prices and number of medicines entering the market. This study aims to perform a scoping review identifying factors contributing to the definition of the therapeutic value of medicines.

Design: Scoping review.

Approval and therapeutic value of gene therapies in the US and Europe.

Gene therapies are a fast-growing area of innovation and hold promise for the treatment of many diseases currently with unmet medical need. To better understand the clinical importance of the current landscape of approved gene therapies, we conducted a systematic analysis of the approved gene therapies and their added therapeutic value. Through December 2022, 13 gene therapies have been approved in the US, 15 in the EU, and 9 in Switzerland.

Regulatory Review Duration and Differences in Submission Times of Drugs in the United States and Europe, 2011 to 2020.

The speed of drug regulatory agencies in the United States and Europe is often a source of discussion. The objective of this research was to assess regulatory review duration of first and supplementary indications approved between 2011 and 2020 in the United States and Europe (European Union [EU] and Switzerland) and differences in submission times between the United States and Europe. Descriptive statistics were applied to review times between the jurisdictions and across the therapeutic areas.

FDA-cleared artificial intelligence and machine learning-based medical devices and their 510(k) predicate networks.

The US Food and Drug Administration is clearing an increasing number of artificial intelligence and machine learning (AI/ML)-based medical devices through the 510(k) pathway. This pathway allows clearance if the device is substantially equivalent to a former cleared device (ie, predicate). We analysed the predicate networks of cleared AI/ML-based medical devices (cleared between 2019 and 2021), their underlying tasks, and recalls.

The cancer premium - explaining differences in prices for cancer vs non-cancer drugs with efficacy and epidemiological endpoints in the US, Germany, and Switzerland: a cross sectional study.

Background: High treatment prices of new cancer drugs are a global public health challenge to patients and healthcare systems. Policymakers in the US and Europe are debating reforms to drug pricing. The objective of this study was to assess whether drug efficacy or epidemiological characteristics (prevalence, incidence, mortality) explain the gap in treatment prices between cancer and non-cancer drugs in the US, Germany, and Switzerland.

Therapeutic value of first versus supplemental indications of drugs in US and Europe (2011-20): retrospective cohort study.

Objective: To analyze the therapeutic value of supplemental indications compared with first indications for drugs approved in the US and Europe.

Design: Retrospective cohort study.

Setting: New and supplemental indications approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) between 2011 and 2020.

Determinants of cancer drug pricing and how to overcome the cancer premium.

The high and increasing prices of cancer drugs are a public health challenge. To disrupt the cancer premium and improve patient access to cancer drugs, different action steps are indicated: more transparency on the price determination process and actual prices, value-based pricing, and "price with evidence development."

Comparison of Uptake and Prices of Biosimilars in the US, Germany, and Switzerland.

Importance: Biologics account for a substantial proportion of health care expenditures. Their costs have been projected to reach US $452 billion in global spending by 2022. Given recent expiration of patent protection of biologics, a shift toward greater follow-on competition among biosimilars would be expected that would allow greater uptake and lower drug costs.

Therapeutic Value of Drugs Granted Accelerated Approval or Conditional Marketing Authorization in the US and Europe From 2007 to 2021.

Importance: The number of drugs approved through the accelerated approval or conditional marketing authorization pathways has increased with unclear evidence of their therapeutic value.

Objectives: To assess the therapeutic value of drug indications granted accelerated approval in the US or conditional marketing authorization in the European Union (EU) overall and for cancer indications.

Design, Setting, And Participants: This cohort study used the public databases of the US Food and Drug Administration and the European Medicines Agency to identify all drugs (initial and supplemental indications) granted accelerated approval in the US or conditional marketing authorization (initial indications only) in the EU between January 1, 2007, and December 31, 2021.