Randomized controlled trials and real-world evidence for market access and surveillance of high-risk products—The example of paclitaxel

C.‑A Behrendt,Kommission Pavk Und Diabetischer Fuß Der Dgg E V ,H Görtz,G Rümenapf,U Rother,D Böckler,C Uhl

doi:10.1007/s00772-020-00726-0

Abstract

AbstractIn 2018 and 2020, two meta-analyses using summary-level data from randomized controlled trials reported worse mortality following the application of paclitaxel-coated stents and balloons in femoropopliteal and crural arteries. These results initiated a heated global discussion concerning the validity of this association, while various observational studies using clinical and administrative registries proved the safety of coated devices. This article aimed to summarize the development and adoption of paclitaxel-coated balloons and stents for the treatment of peripheral arterial occlusive disease in clinical practice, research, and practice guidelines. It especially focusses on the European Unionʼs medical device regulation, which has far-reaching implications for the market approval and monitoring of high-risk medical devices.

Highlights

In recent years the legal framework of the European Union (EU) has been subject to far-reaching reforms, which have had an impact on care for patients suffering from cardiovascular diseases as well as research in this field
Following the discovery and isolation of the paclitaxel (Taxol) active ingredient from the bark of the Pacific yew tree in the 1960s and the preliminary description of its anticarcinogenic effects in the late 1970s, market access was granted for its use in coronary arteries in the early 2000s [25, 41]
Due to the superiority of sirolimus-based medical devices, paclitaxel failed to prevail in the cardiological sector, with just a handful of exceptions [16, 23, 31, 34, 40]

Summary

Introduction

In recent years the legal framework of the European Union (EU) has been subject to far-reaching reforms, which have had an impact on care for patients suffering from cardiovascular diseases as well as research in this field. An interesting example illustrating the importance of market access and the surveillance of high-risk medical devices is the use of paclitaxel-coated stents and balloons to treat atherosclerotic peripheral vascular diseases. This review examines the market introduction and rapid spread of paclitaxelcoated stents and balloons in vascular medicine It draws on the divergent findings from randomized and nonrandomized studies to debate the strengths and limitations of complementary study designs for market access and surveillance. Almost 3 years later, 3 balloons and 1 stent were approved in the USA for use in peripheral arteries [44] Following their market introduction and dissemination in Europe and the USA, countless successive guidelines have included recommendations or considerations

Conclusions for professional practice

Findings

Compliance with ethical guidelines