Comment Envisioning an IDEAL future for urological innovation Philipp Dahm Urology Section, Minneapolis VA Health Care System and Department of Urology, University of Minnesota, Minneapolis, MN, USA
Urologists take pride in standing at the forefront of cutting-edge innovation and being among the first to embrace new procedures and technologies. In fact, when talking to urology residency applicants, access to advanced technology is among the most frequently cited motivating factors for their career choice. This innovative spirit has allowed urologists to harness acoustic waves to treat nephrolithiasis, made us leaders in the use of miniaturised endoscopic equipment, and pioneers in the application of robot-assisted laparoscopic surgery. At the same time, we have to admit that much of what we do as urologists on a day-to-day basis is based on low or very low quality evidence. Although we should demand similar evidentiary standards for everything we do as clinicians, this is less of an issue for time-honoured surgical techniques and management approaches that have been in use for decades, making them the de facto standard of care. However, in a healthcare and regulatory environment increasingly focused on safety and costs, such low quality evidence will be increasingly problematic for surgical innovation, in particular game-changing, disruptive technologies for which the ‘true’ benefits are often unproven, the safety profile unclear, and the up-front costs are high. The plethora of poorly conducted research on surgical innovation that fills too many pages of our journals has been the driving force behind the work of the Idea, Development, Exploration, Assessment, Long-term follow-up (IDEAL) Collaboration, an international, Oxford-based group of
surgeons and methodologists determined to change the landscape of surgical research. At its core are the IDEAL recommendations (Table 1), which map the various stages of surgical innovation and related research objectives to the best possible study design, thereby providing a comprehensive framework for surgical innovation similar to that which exists for drug development [1]. Uptake of the IDEAL recommendations in the urological research community has been encouraging. One example is the development of robotic kidney transplantation (RKT) with regional hypothermia by Menon et al. [2]. In a series of papers, the investigators outlined a rational, IDEAL-guided approach to the development of this pioneering procedure [2–4]. Corresponding to a preclinical stage (stage 0), they reported on the feasibility of achieving renal hypothermia in the cadaver laboratory. This was followed by a prospectively planned, small case series of carefully selected patients in whom RKT was performed, which represented the ‘first-in-man’ experience and corresponded to the ‘Idea phase’ (stage 1). In a subsequent, prospectively planned cohort of 43 patients, they reported the technical modifications that arose from their increasing experience with this approach, reflecting a ‘tinkering’ that is characteristic of surgical innovation and occurs in the ‘Development phase’ (stage 2a). In a separate group of patients from two institutions, the investigators also reported on the use of statistical methods such as cumulative summation (CUSUM) techniques to prospectively monitor safety and assess learning
Table 1 IDEAL stages of surgical innovation. IDEAL stage Stage 1 Idea Stage 2a Development Stage 2b Exploration Stage 3 Assessment Stage 4 Long-term study
Aim Proof of concept Safety, efficacy Safety, efficacy Comparative effectiveness (including resource utilisation) Quality assurance
Question Can the procedure achieve physical or physiological goal? What is the optimal technique? What are outcomes of more widespread use at different centres? How does the new procedure compare to the established standard of care? What are the long-term outcomes?
© 2015 The Authors BJU International © 2015 BJU International | doi:10.1111/bju.13129 Published by John Wiley & Sons Ltd. www.bjui.org
Optimal study design Structured case report Prospectively planned, development study Prospectively planned, collaborative study Randomised controlled trial Registry
Number of patients involved Single to a few 10s 100s 100s+ 100–1000s
BJU Int 2015 wileyonlinelibrary.com
Comment
curves, thereby addressing some of the issues that define the IDEAL ‘Exploration phase’ (stage 2b). This stage ideally achieves consensus on how the procedure is best performed in preparation for a multicentre clinical trial (stage 3) to establish superiority of RKT over standard approaches with regards to patient-important outcomes such as time to recovery, quality of life and adverse events, which these investigators will hopefully tackle next. Stage 3 is also the phase where resource utilisation should be assessed to establish comparative effectiveness to alternative treatment options. Two other examples of early stage urological innovation relate to development of a robotic device to facilitate flexible ureteroscopy and the use of focal electroporation for the treatment of prostate cancer [5,6]. Each of these examples reflects the desired paradigm shift away from retrospective urological research towards a more meaningful use of prospective observational studies that are protocol-driven and transparently reported. The recently published randomised trial of open vs robotassisted radical cystectomy, as an IDEAL ‘Assessment phase’ study (stage 3), further documents that trials can be done in urology [7]. However, a common criticism that proponents of more stringent evidentiary standards in urological surgery face is that of rapidly changing technology and strong surgeon and patient preferences, which effectively negate the possibility of such trials in many arenas. For such situations, IDEAL proposes the creation of national and international registries that would allow the continuous evaluation of the outcomes to permit high quality comparative effectiveness research. Pioneering work by the Urological Surgery Quality Collaborative speak to the feasibility of this approach and its growing importance towards quality assurance and improvement [8]. Ongoing efforts of the IDEAL Collaboration include the development of a modified version of the framework referred to as IDEAL (D) that is specific to devices, as well as a practical algorithm or check list to guide investigators on how best to align research objectives with the most appropriate study design. For the sake of better urological research and, ultimately, better patient care, we encourage all urologists to
2
© 2015 The Authors BJU International © 2015 BJU International
learn more about the IDEAL approach at our website (http:// www.ideal-collaboration.net/) or reach out to us in person.
Conflicts of Interest None disclosed.
References 1 McCulloch P, Altman DG, Campbell WB et al. No surgical innovation without evaluation: the IDEAL recommendations. Lancet 2009; 374: 1105–12 2 Menon M, Abaza R, Sood A et al. Robotic kidney transplantation with regional hypothermia: evolution of a novel procedure utilizing the IDEAL guidelines (IDEAL phase 0 and 1). Eur Urol 2014; 65: 1001–9 3 Menon M, Sood A, Bhandari M et al. Robotic kidney transplantation with regional hypothermia: a step-by-step description of the Vattikuti Urology Institute-Medanta technique (IDEAL phase 2a). Eur Urol 2014; 65: 991–1000 4 Sood A, Ghani KR, Ahlawat R et al. Application of the statistical process control method for prospective patient safety monitoring during the learning phase: robotic kidney transplantation with regional hypothermia (IDEAL phase 2a–b). Eur Urol 2014; 66: 371–8 5 Saglam R, Muslumanoglu AY, Tokatli Z et al. A new robot for flexible ureteroscopy: development and early clinical results (IDEAL stage 1–2b). Eur Urol 2014; 66: 1092–100 6 Valerio M, Dickinson L, Ali A et al. A prospective development study investigating focal irreversible electroporation in men with localised prostate cancer: Nanoknife Electroporation Ablation Trial (NEAT). Contemp Clin Trials 2014; 39: 57–65 7 Bochner BH, Sjoberg DD, Laudone VP. Memorial Sloan Kettering Cancer Center Bladder Cancer Surgical Trials Group. A randomized trial of robot-assisted laparoscopic radical cystectomy. N Engl J Med 2014; 371: 389–90 8 Miller DC, Murtagh DS, Suh RS, Knapp PM, Dunn RL, Montie JE. Establishment of a urological surgery quality collaborative. J Urol 2010; 184: 2485–90
Correspondence: Philipp Dahm, Urology Section 112D, Minneapolis VA Health Care System, 1 Veterans Drive, Minneapolis, MN 55417, USA. e-mail: [email protected] Abbreviations: IDEAL Idea, Development, Exploration, Assessment, Long-term follow-up; RKT robotic kidney transplantation.
Urologists take pride in standing at the forefront of cutting-edge innovation and being among the first to embrace new procedures and technologies. In fact, when talking to urology residency applicants, access to advanced technology is among the most frequently cited motivating factors for their career choice. This innovative spirit has allowed urologists to harness acoustic waves to treat nephrolithiasis, made us leaders in the use of miniaturised endoscopic equipment, and pioneers in the application of robot-assisted laparoscopic surgery. At the same time, we have to admit that much of what we do as urologists on a day-to-day basis is based on low or very low quality evidence. Although we should demand similar evidentiary standards for everything we do as clinicians, this is less of an issue for time-honoured surgical techniques and management approaches that have been in use for decades, making them the de facto standard of care. However, in a healthcare and regulatory environment increasingly focused on safety and costs, such low quality evidence will be increasingly problematic for surgical innovation, in particular game-changing, disruptive technologies for which the ‘true’ benefits are often unproven, the safety profile unclear, and the up-front costs are high. The plethora of poorly conducted research on surgical innovation that fills too many pages of our journals has been the driving force behind the work of the Idea, Development, Exploration, Assessment, Long-term follow-up (IDEAL) Collaboration, an international, Oxford-based group of
surgeons and methodologists determined to change the landscape of surgical research. At its core are the IDEAL recommendations (Table 1), which map the various stages of surgical innovation and related research objectives to the best possible study design, thereby providing a comprehensive framework for surgical innovation similar to that which exists for drug development [1]. Uptake of the IDEAL recommendations in the urological research community has been encouraging. One example is the development of robotic kidney transplantation (RKT) with regional hypothermia by Menon et al. [2]. In a series of papers, the investigators outlined a rational, IDEAL-guided approach to the development of this pioneering procedure [2–4]. Corresponding to a preclinical stage (stage 0), they reported on the feasibility of achieving renal hypothermia in the cadaver laboratory. This was followed by a prospectively planned, small case series of carefully selected patients in whom RKT was performed, which represented the ‘first-in-man’ experience and corresponded to the ‘Idea phase’ (stage 1). In a subsequent, prospectively planned cohort of 43 patients, they reported the technical modifications that arose from their increasing experience with this approach, reflecting a ‘tinkering’ that is characteristic of surgical innovation and occurs in the ‘Development phase’ (stage 2a). In a separate group of patients from two institutions, the investigators also reported on the use of statistical methods such as cumulative summation (CUSUM) techniques to prospectively monitor safety and assess learning
Table 1 IDEAL stages of surgical innovation. IDEAL stage Stage 1 Idea Stage 2a Development Stage 2b Exploration Stage 3 Assessment Stage 4 Long-term study
Aim Proof of concept Safety, efficacy Safety, efficacy Comparative effectiveness (including resource utilisation) Quality assurance
Question Can the procedure achieve physical or physiological goal? What is the optimal technique? What are outcomes of more widespread use at different centres? How does the new procedure compare to the established standard of care? What are the long-term outcomes?
© 2015 The Authors BJU International © 2015 BJU International | doi:10.1111/bju.13129 Published by John Wiley & Sons Ltd. www.bjui.org
Optimal study design Structured case report Prospectively planned, development study Prospectively planned, collaborative study Randomised controlled trial Registry
Number of patients involved Single to a few 10s 100s 100s+ 100–1000s
BJU Int 2015 wileyonlinelibrary.com
Comment
curves, thereby addressing some of the issues that define the IDEAL ‘Exploration phase’ (stage 2b). This stage ideally achieves consensus on how the procedure is best performed in preparation for a multicentre clinical trial (stage 3) to establish superiority of RKT over standard approaches with regards to patient-important outcomes such as time to recovery, quality of life and adverse events, which these investigators will hopefully tackle next. Stage 3 is also the phase where resource utilisation should be assessed to establish comparative effectiveness to alternative treatment options. Two other examples of early stage urological innovation relate to development of a robotic device to facilitate flexible ureteroscopy and the use of focal electroporation for the treatment of prostate cancer [5,6]. Each of these examples reflects the desired paradigm shift away from retrospective urological research towards a more meaningful use of prospective observational studies that are protocol-driven and transparently reported. The recently published randomised trial of open vs robotassisted radical cystectomy, as an IDEAL ‘Assessment phase’ study (stage 3), further documents that trials can be done in urology [7]. However, a common criticism that proponents of more stringent evidentiary standards in urological surgery face is that of rapidly changing technology and strong surgeon and patient preferences, which effectively negate the possibility of such trials in many arenas. For such situations, IDEAL proposes the creation of national and international registries that would allow the continuous evaluation of the outcomes to permit high quality comparative effectiveness research. Pioneering work by the Urological Surgery Quality Collaborative speak to the feasibility of this approach and its growing importance towards quality assurance and improvement [8]. Ongoing efforts of the IDEAL Collaboration include the development of a modified version of the framework referred to as IDEAL (D) that is specific to devices, as well as a practical algorithm or check list to guide investigators on how best to align research objectives with the most appropriate study design. For the sake of better urological research and, ultimately, better patient care, we encourage all urologists to
2
© 2015 The Authors BJU International © 2015 BJU International
learn more about the IDEAL approach at our website (http:// www.ideal-collaboration.net/) or reach out to us in person.
Conflicts of Interest None disclosed.
References 1 McCulloch P, Altman DG, Campbell WB et al. No surgical innovation without evaluation: the IDEAL recommendations. Lancet 2009; 374: 1105–12 2 Menon M, Abaza R, Sood A et al. Robotic kidney transplantation with regional hypothermia: evolution of a novel procedure utilizing the IDEAL guidelines (IDEAL phase 0 and 1). Eur Urol 2014; 65: 1001–9 3 Menon M, Sood A, Bhandari M et al. Robotic kidney transplantation with regional hypothermia: a step-by-step description of the Vattikuti Urology Institute-Medanta technique (IDEAL phase 2a). Eur Urol 2014; 65: 991–1000 4 Sood A, Ghani KR, Ahlawat R et al. Application of the statistical process control method for prospective patient safety monitoring during the learning phase: robotic kidney transplantation with regional hypothermia (IDEAL phase 2a–b). Eur Urol 2014; 66: 371–8 5 Saglam R, Muslumanoglu AY, Tokatli Z et al. A new robot for flexible ureteroscopy: development and early clinical results (IDEAL stage 1–2b). Eur Urol 2014; 66: 1092–100 6 Valerio M, Dickinson L, Ali A et al. A prospective development study investigating focal irreversible electroporation in men with localised prostate cancer: Nanoknife Electroporation Ablation Trial (NEAT). Contemp Clin Trials 2014; 39: 57–65 7 Bochner BH, Sjoberg DD, Laudone VP. Memorial Sloan Kettering Cancer Center Bladder Cancer Surgical Trials Group. A randomized trial of robot-assisted laparoscopic radical cystectomy. N Engl J Med 2014; 371: 389–90 8 Miller DC, Murtagh DS, Suh RS, Knapp PM, Dunn RL, Montie JE. Establishment of a urological surgery quality collaborative. J Urol 2010; 184: 2485–90
Correspondence: Philipp Dahm, Urology Section 112D, Minneapolis VA Health Care System, 1 Veterans Drive, Minneapolis, MN 55417, USA. e-mail: [email protected] Abbreviations: IDEAL Idea, Development, Exploration, Assessment, Long-term follow-up; RKT robotic kidney transplantation.
