What Is the Required Certainty of Evidence for the Implementation of Novel Techniques for the Treatment of Benign Prostatic Obstruction?

Abstract

Context

A large number of minimally invasive techniques have been developed for the surgical management of male lower urinary tract symptoms (LUTS) presumed to be secondary to benign prostatic obstruction (BPO) over the last 3 decades. Many have not stood the test of time often because they were overpromoted before there were sufficient data.

Objective

The scope of this paper is to consider whether new devices, for the treatment of male LUTS/BPO, have been implemented prematurely in the past. We also examine the relative certainty of evidence (CoE) that is currently available for newer developing technologies and make recommendations about the CoE that should be demanded in the future before widespread implementation.

Key messages

This evidence must provide adequate length of follow-up to allow proper information to be provided for patients before treatment choices are made and to be able to create recommendations in high-quality guidelines such as those of the European Association of Urology. It is not just within the domain of LUTS treatments that this is important, other urological devices, such as mesh devices, have been equally “guilty” and likewise devices in most other (surgical) specialities. We believe that there is a need for a set of requirements built around primary randomised controlled trials (RCTs) looking at both efficacy and safety, and secondary studies to confirm the reproducibility and generalisability of the first pivotal studies. Otherwise, there is a danger that a single pivotal study can be overexploited by device manufacturers. Studies that are needed include (1) proof of concept, (2) RCTs on efficacy and safety, as well as (3) cohort studies with a broad range of inclusion and exclusion criteria to confirm both reproducibility and generalisability of the benefits and harms. It is not the purpose of this paper to make judgements about individual treatments but simply to look at different treatments to provide verification for this debate.

Patient summary

Many new treatment devices have been developed over the last 20–30 yr, often with inadequate medium- to long-term results. Many have not stood the test of time, but were heavily promoted by manufacturers, the press, and some doctors when they were first released, meaning that many patients had unsatisfactory results. This paper proposes minimum standards for the investigation of new treatments before their widespread promotion to patients.

Introduction

It is a common perception that surgical rather than medical intervention becomes appropriate in patients with bothersome lower urinary tract symptoms (LUTS) due to benign prostatic obstruction (BPO), who are unwilling to try medical therapies, or in cases where medical therapies are ineffective or not well tolerated, and in cases of complicated LUTS. However, with the advent of more minimally invasive treatments, the threshold for surgical intervention has become much lower [1].

This paper is the opinion of the current European Association of Urology (EAU) male LUTS guideline development group and has been developed after a considerable review of the current literature on novel treatment devices.

A large number of minimally invasive techniques (MITs) have been developed for the management of male LUTS/BPO, in the last 3 decades or so. From the middle of the 1980s onwards, attempts to use balloon dilation of the prostate, prostatic stents, prostate hyperthermia and cryotherapy, neodymium:YAG-based therapies of the prostate-like interstitial laser coagulation or visual laser ablation of the prostate, transurethral needle ablation (TUNA), transurethral microwave therapy, and high-intensity focused ultrasound have all had strong advocates, but over time many devices have not stood the test of time and have fallen out of favour. These may have disappeared for a variety of reasons, such as lack of long-term efficacy, adverse events, poor cost effectiveness, or development of newer versions (the “industrial/commercial circle”), which again dilute the data available on the latest or current version of the device.

It is important to bear in mind, from the start, that patients’ and clinicians’ expectations of outcomes may differ. For instance, patients (compared with clinicians) may prefer treatment options that are less effective if they result in a lower risk of complications or a faster return to normal activities. There is little doubt that there is a desire from patients for MITs. Unfortunately, sometimes the eagerness for these new MITs, in the popular press—the commonest source of direct patient information—but sometimes also from the early adopters of the treatment, has meant that this enthusiasm has often outstripped the clinical evidence. Early and aggressive company marketing can also overpower clinical evidence. Unlike pharmaceutical interventions, implementation of novel devices and techniques into clinical practice is not governed by the same strict regulatory mechanisms [2].

Therefore, the quality of the studies must remain high: (1) there must be adequate control arms (eg, compared with sham treatment or other established comparable therapies); (2) outcomes, including patient-reported outcome measurements, should be important to patients; and (3) studies should include a relevant study population that is generalisable.

We also need to remember that behind the tool, there is also a concept: ablation, resection, enucleation, etc.; therefore, a new device for resection is not a new device for vaporisation. Some of the principles behind these “failed” treatments have been generated again using newer or more powerful devices. It is therefore important that unlike their predecessors, sufficient controlled trials are carried out before their widespread application. Certain concepts, however, such as (laser) enucleation, have resulted in a paradigm change in the surgical management of LUTS/BPO.