Abstract

Aim: This study aimed to identify from different stakeholders the benefits and obstacles of implementing precision medicine in diabetic kidney disease (DKD) and to build consensus about a way forward in order to treat, prevent, or even reverse this disease. Methods: As part of an ongoing effort of moving implementation of precision medicine in DKD forward, a two-day consensus-building meeting was organized with different stakeholders involved in drug development and patient care in DKD, including patients, patient representatives, pharmaceutical industry, regulatory agencies representatives, health technology assessors, healthcare professionals, basic scientists, and clinical academic researchers. The meeting consisted of plenary presentations and discussions, and small group break-out sessions. Discussion topics were based on a symposium, focus groups and literature search. Benefits, obstacles and potential solutions toward implementing precision medicine were discussed. Results from the break-out sessions were presented in plenary and formed the basis of a broad consensus discussion to reach final conclusions. Throughout the meeting, participants answered several statement and open-ended questions on their mobile device, using a real-time online survey tool. Answers to the statement questions were analyzed descriptively. Results of the open-ended survey questions, the break-out sessions and the consensus discussion were analyzed qualitatively. Results and conclusion: Seventy-one participants from 26 countries attended the consensus-building meeting in Amsterdam, April 2019. During the opening plenary on the first day, the participants agreed with the statement that precision medicine is the way forward in DKD (n = 57, median 90, IQR [75–100]). Lack of efficient tools for implementation in practice and generating robust data were identified as significant obstacles. The identified benefits, e.g., improvement of the benefit-risk ratio of treatment, offer substantive incentives to find solutions for the identified obstacles. Earlier and increased multi-stakeholder collaboration and specific training may provide solutions to alter clinical and regulatory guidelines that lie at the basis of both obstacles and solutions. At the end of the second day, the opinion of the participants toward precision medicine in DKD was somewhat more nuanced (n = 45, median 83, IQR [70–92]) and they concluded that precision medicine is an important way forward in improving the treatment of patients with DKD.

Highlights

  • Interventions in the Renin-Angiotensin System (RAS), first the ACE inhibitors (ACEi) in 1993 and later the AngiotensinReceptor Blockers (ARBs) in 2001, were breakthrough therapies to slow the progression of renal disease in patients with type 2 diabetes mellitus (Lewis et al, 1993; Nathan et al, 1993; Brenner et al, 2001; Lewis et al, 2001)

  • The topics discussed during this symposium were elaborated on in a series of articles published in a special issue on precision medicine in diabetic kidney disease (DKD)

  • Stakeholder specific focus group discussions were organized with representatives of European regulatory agencies (n 7), health technology assessors (HTAs) (n 4), healthcare professionals (HCPs) (n 5), patients (n 4) and BEAt-DKD partners (n 10) that expanded on these perspectives on precision medicine in DKD

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Summary

Introduction

Interventions in the Renin-Angiotensin System (RAS), first the ACE inhibitors (ACEi) in 1993 and later the AngiotensinReceptor Blockers (ARBs) in 2001, were breakthrough therapies to slow the progression of renal disease in patients with type 2 diabetes mellitus (Lewis et al, 1993; Nathan et al, 1993; Brenner et al, 2001; Lewis et al, 2001). Despite this success, the residual renal and cardiovascular (CV) risk in this population remained extremely high (Heerspink and de Zeeuw, 2011). Excluding those patients who showed harmful responses in biomarkers of renal damage could turn a failed trial into a renoprotective trial (Chin et al, 2014)

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