Kerstin Noëlle Vokinger

Harvard University | Harvard · Medical School

Migraine is one of the most prevalent and disabling disorders worldwide.1 In addition to triptans, which are used as abortive medications for acute migraine attacks, the US Food and Drug Administration (FDA) has approved several preventive treatments for migraine, including propranolol, topiramate, and onabotulinumtoxinA. Although most of these the...

Several sources of bias can affect the performance of machine learning systems used in medicine and potentially impact clinical care. Here, we discuss solutions to mitigate bias across the different development steps of machine learning-based systems for medical applications. Vokinger et al. discuss potential sources of bias in machine learning sys...

This chapter provides an overview of how the field and its methods can contribute to these larger themes in medical care delivery and health services research, from the perspective of academia. Underuse of needed drugs by patients is one of the most common medication‐related problems, and one that can be readily identified by pharmacoepidemiology....

Zusammenfassung Zusammenfassung Onkologika sind seit mehreren Jahren die umsatzstärkste Arzneimittelgruppe in Deutschland. Während 2014 unter den 30 umsatzstärksten patentgeschützten Arzneimitteln nur drei Onkologika mit Nettokosten zwischen 184 Mio. € und 259 Mio. € je Arzneimittel waren, befinden sich unter den führenden 20 Arzneimitteln nach Ne...

Background: Most anticancer drugs are approved by regulatory agencies based on surrogate measures. This article explores the variables associated with overall survival (OS), quality of life (QoL), and substantial clinical benefit among anticancer drugs at the time of approval and in the postmarketing period. Methods: Anticancer drugs approved by...

The regulatory landscape for artificial intelligence (AI) is shaping up on both sides of the Atlantic, urgently awaited by the scientific and industrial community. Commonalities and differences start to crystallize in the approaches to AI in medicine.

Legislators in the USA have been discussing reforms to reduce the high cost of brand-name drugs, which are much higher in the USA than in other industrialized countries. One solution is to actively negotiate prices based on drugs’ clinical benefits. We discuss two important complexities from such an approach: drugs that have been approved for multi...

IMPORTANCE The high cost of cancer medicines is a public health challenge. Policy makers in the US and Europe are debating reforms to drug pricing that would cover both the prices of new medicines when entering the market and price increases after they are launched. OBJECTIVE To assess launch prices, postlaunch price changes, and clinical benefit...

Der vorliegende Beitrag wagt aus medizinischer und aus rechtlicher Perspektive eine vorläufige Standortbestimmung zu den Langzeitfolgen einer SARS-CoV-2-Infektion. Auf Grundlage einer Würdigung der publizierten medizinischen Studien zu «Long Covid» oder «Post Covid» erfolgt eine juristisch-medizinische (bidisziplinäre) Diskussion, an die eine sozia...

Background Many European countries introduced (confidential) rebates in the past years. Authorities and manufacturers argue that this strategy allows reduction of spending on high-cost drugs, and quick access of innovative drugs. We evaluated these arguments using Switzerland as an example, one of the last countries with transparent rebates. Metho...

There has been a surge of interest in artificial intelligence and machine learning (AI/ML)-based medical devices. However, it is poorly understood how and which AI/ML-based medical devices have been approved in the USA and Europe. We searched governmental and non-governmental databases to identify 222 devices approved in the USA and 240 devices in...

Objective: To characterize the therapeutic value of new drugs approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) and the association between these ratings and regulatory approval through expedited programs. Design: Retrospective cohort study. Setting: New drugs approved by the FDA and EMA between 2007 and...

Den Behörden kommt ein Ermessensspielraum bei der Beurteilung zu, ob sie dem Gesuchsteller den Zugang zu amtlichen Dokumenten basierend auf dem (BGÖ) gewährt. Wir Öffentlichkeitsgesetz haben alle Bundesverwaltungs- und Bundesgerichtsentscheide (2006 – 2019) empirisch analysiert, die den Zugang zu amtlichen Dokumenten i. S. d. BGÖ zum Streitgegensta...

2077 Background: A key clinical outcome for new cancer drugs is improvement in overall survival (OS), defined as time from the date of randomization to the death from any cause. However, many cancer drugs are approved by regulators based on changes to surrogate measures of OS, such as progression-free survival or overall response rate. When surroga...

7052 Background: Initial drug approval is often based on surrogate endpoints. Definitive outcomes like Overall Survival (OS) or Quality of life (QoL) may not be available. Here, we evaluate changes in the magnitude of clinical benefit using the American Society of Clinical Oncology Value Framework (ASCO-VF) and European Society for Medical Oncology...

2006 Background: Cancer drug costs are rising in the US and Europe. While drug manufacturers set prices without restriction in the US, European countries have regulations that allow national authorities to directly negotiate drug prices at launch and over time. We analyzed and compared the launch prices and price developments of cancer drugs in the...

As COVID-19 spreads across the globe, crowdsourced digital technology harbours the potential to improve surveillance and epidemic control, primarily through increased information coverage, higher information speed, fast case tracking and improved proximity tracing. Targeting those aims, COVID-19-related smartphone and web-based health applications...

Background: Increasing cancer drug prices are a challenge for patients and health systems in the USA and Europe. By contrast with the USA, national authorities in European countries often directly negotiate drug prices with manufacturers. The American Society of Clinical Oncology (ASCO) and the European Society for Medical Oncology (ESMO) developed...

Background: Approved drugs must be included on the so-called "special list" (SL) by the Federal Office for Public Health (FOPH) to be reimbursed by the social health insurance in Switzerland. The FOPH decides whether a drug may be included on SL and if so, negotiates the maximum price with the manufacturer. Time period between approval and inclusi...

Artificial intelligence– and machine learning (AI/ML)–based technologies aim to improve patient care by uncovering new insights from the vast amount of data generated by an individual patient and by the collective experience of many patients. Machine learning is an AI technique that trains software algorithms to learn from and act on new data to co...

Pharmacoepidemiologists in their daily work encounter many different issues that have legal implications. How practitioners interface with systems of rules set by tort law and contract law principles can be vital to their work. Product liability cases, a variant of tort law, allege failure to warn about adverse events linked to pharmaceutical produ...

Background Given rising cancer drug costs, US Medicare recently proposed to tie some drug prices to average prices paid by comparable countries. To understand the potential scope of this policy, we assessed differences in cancer drug prices in the US and European countries. We also evaluated the correlation between drug prices and their clinical be...

Objective To determine differences in the characteristics of cancer drugs designated as orphan drugs by the Food and Drug Administration (FDA) and European Medicines Agency (EMA). Design and setting Identification of all cancer drugs (initial or supplementary indication) with orphan status approved by the FDA between 2008–2017 based on publicly ac...

Gerichtsurteile werden häufig in anonymisierter Form öffentlich zugänglich gemacht. In der vorliegenden Studie haben wir untersucht, ob es mit der Me-thodik des «Linkage»-der Verbindung von verschiedenen, öffentlich zugäng-lichen Daten(banken)-möglich ist, Urteile zu re-identifizieren. Materiell in-teressierten uns die Fragestellungen, welche pharm...

Clinical Practice Guidelines: An Ambiguous Term With Potentially Unexpected Legal Consequences Abstract. The term "Clinical Practice Guidelines (CPG)" is not applied consistently in Switzerland. The FMH published a definition in 1999. However, documents not corresponding to the definition are sometimes also called CPG, while those corresponding to...

Social health insurance is mandatory in Switzerland and covers the costs of basic medical care. In general, with regard to medicines, the costs are only reimbursed if the drug is (1) approved by Swissmedic and (2) listed on the so-called Spezia­litätenliste (SL) by the Federal Office of Public Health (FOPH). However, the SL does not include all dru...

Background: Successful first-generation drugs can be converted with small alterations to "second-generation drugs," which are cheaper to develop and may pose less financial risk for manufacturers due to already validated action mechanism and a well-defined consumer market. Methods: We found four classes of cancer drugs for first- and second gene...

Background: Given rising cancer drug costs, Medicare recently proposed to tie some US drug prices to average prices paid by comparable countries. To understand the potential scope of this policy, we assessed differences in cancer drug prices in the US and selected European countries. We also evaluated the correlation between drug prices and their c...

Historically, patient experience, including symptomatic toxicities, physical function, and disease-related symptoms during treatment or their perspectives on clinical trials, has played a secondary role in cancer drug development. Regulatory criteria for drug approval require that drugs are safe and effective, and almost all drug approvals have bee...

Federal Supreme Court Decisions in Medical Law (2000-2017) - Overview and Selected Case Reports Abstract. Within the framework of an empirical and casuistic analysis of Federal Court decisions in medical law between 2000 and 2017, the goal was to describe tendencies and patterns of Federal Court decisions in medical law. Among other things, our res...

Background: Adequate patient selection is the key to successful lung volume reduction in patients with pulmonary emphysema. Computed tomography (CT) enables a reliable detection of pulmonary emphysema and allows an accurate quantification of the severity. Our goal was to investigate the usefulness and reliability of color-coded (CC) CT images in c...

Transparency in the development of new medicines has the potential to improve the efficiency and quality of clinical research by allowing investigators and companies to learn from successes and failures of similar products. It also reflects the ethical responsibility of regulators and sponsors to inform patients and involve them in the translation...

Importance The US Department of Health and Human Services (HHS) has proposed to reform drug pricing in Medicare Part B, which primarily covers physician-administered drugs and biologic agents. One HHS proposal would shift coverage of certain drugs from Medicare Part B to Part D, which is administered by private prescription drug plans. Objective T...

Historically, patient experience, including symptomatic toxicities, physical function, and disease-related symptoms during treatment or their perspectives on clinical trials, has played a secondary role in cancer drug development. Regulatory criteria for drug approval require that drugs are safe and effective, and almost all drug approvals have bee...

Purpose To evaluate the diagnostic performance of bone texture analysis (TA) combined with machine learning (ML) algorithms in standard CT scans to identify patients with vertebrae at risk for insufficiency fractures. Materials and methods Standard CT scans of 58 patients with insufficiency fractures of the spine, performed between 2006 and 2013,...

Über einen Zeitraum von 18 Jahren analysiert die Studie unter Anwendung eines systematisch-statistischen Ansatzes und Artificial Intelligence insgesamt 387 Bundesgerichtsurteile, bei denen es um krankenversicherungsrechtliche Streitigkeiten über die Deckung medizinischer Leistungen ging. Die Auswertung zeigt, dass der Streitwert im Einzelfall tief...

Unfortunately, after publication of this article [1], it was noticed that the name of Urs J. Muehlematter was incorrectly displayed as Urs J. Mühlematter. The corrected author list can be seen above and the original article has been corrected to reflect this.

Background: Accurate attenuation correction (AC) is an inherent problem of positron emission tomography magnetic resonance imaging (PET/MRI) systems. Simulation studies showed that time-of-flight (TOF) detectors can reduce PET quantification errors in MRI-based AC. However, its impact on lesion detection in a clinical setting with 18F-choline has...

Unter Anwendung eines systematisch-statistischen Ansatzes und Artificial Intelligence wird die bundesgerichtliche Rechtsprechung zum Arztrecht der Jahre 2000–2017 empirisch untersucht. Dabei stehen folgende Fragestellungen im Zentrum: Wie ist das Verhältnis der Rechtsgebiete (Privatrecht, Öffentliches Recht, Strafrecht) in arztrechtlichen Verfahren...

Seit 2002 wird die Zulassung im ambulanten Sektor für die ärztliche Tätigkeit zulasten der obligatorischen Krankenversicherung reguliert. Am 5. Juli 2017 präsentierte der Bundesrat eine neue Lösung, um die Zulassung der Ärzte im Bereich der ambulanten Medizin zulasten der obligatorischen Krankenpflegeversicherung – dieses Mal unbefristet – zu regel...

Der Pflegeberuf ist vor dem Hintergrund der medizinischen, gesellschaftlichen und politischen Entwicklungen stark im Wandel: So haben etwa die Multimorbidität sowie hochprävalente, chronische Krankheiten in den letzten Jahren zugenommen.1 So leiden beispielsweise Patienten mit psychischen Erkrankungen regelmässig auch an kardiovaskulären Krankheite...

Increasing prescription drug expenditures in the United States are primarily driven by high brand-name drug prices. Although generic competition helps lower drug prices, manufacturers of brand-name drugs often work to delay the availability of generic versions of their products. Strategies to forestall generic competition include patenting peripher...

Gerade in der Radiologie wird der Einsatz von Artificial Intelligence bzw. Machine Learning es ermöglichen, die Arbeitslast der Ärzteschaft zu reduzieren und dadurch z.B. mehr Zeit für komplexere Fälle und für den direkten Patientenkontakt zu ermöglichen. Während sich zumindest die medizinische Forschung bereits intensiv mit dem Machine Learning un...

Tamoxifen is frequently used for the treatment of hormone receptor positive breast cancer (BC). Mainly CYP2D6 is responsible for the transformation to therapeutically active metabolites, but CYP2C19, CYP2C9 and CYP2B6 also are involved. We investigated the impact of polymorphisms within the genes encoding these CYP enzymes on the relapse-free time...