Moving forward from drug-centred to patient-centred research

Lacombe, Denis; O’Moráin, Colm; Casadei, Barbara; hill, kate; Mateus, Elsa F; Lories, Rik; Brusselle, Guy

doi:10.1183/13993003.01870-2018

Cited by 16 publications

(17 citation statements)

References 26 publications

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“…Participants were asked about the current situation in drug development as well as the optimal features of treatment optimization studies. The interview questions were derived from relevant literature on the topics of treatment optimization and the aforementioned evidence gap (Mullins et al, 2014;Ioannidis, 2016;Kempf et al, 2017;Lieu and Platt, 2017;EORTC, 2019;Lacombe et al, 2019b;Lacombe et al, 2019a). The full questions are listed in the Supplementary Material.…”

Section: Methodsmentioning

confidence: 99%

“…In recent years however, this paradigm has faced criticism from authors in the field (Mullins et al, 2014;Ioannidis, 2016;Lacombe et al, 2019b;Wieseler et al, 2019) for being too drugcentered and/or for not sufficiently focusing on the patients who will eventually receive the treatment in real-world clinical practice. The existing clinical development framework allows the industry to primarily pursue regulatory approval of their products without taking into account the real needs of patients and society (Lacombe et al, 2019a).…”

Section: Introductionmentioning

confidence: 99%

“…Although the drug-driven approach to bring new treatments to the market should ideally be balanced with a more patientfocused strategy, the former tends to dominate treatment development in oncology. As a result, many important aspects relating to the use of novel antitumor therapies in real-world settings are neglected throughout the process (Kempf et al, 2017;Lacombe et al, 2017;Lacombe et al, 2019b), as displayed in Table 1. For now, such clinically important and patient-centered questions are being addressed in a non-systematic and voluntary manner in the post-approval stage by non-commercial entities (Kempf et al, 2017), including academic research teams and notfor-profit organizations such as the European Organisation for Research and Treatment of Cancer (EORTC), which conducts independent international clinical trials in the field of oncology.…”

Section: Introductionmentioning

confidence: 99%

“…This situation has led to calls for a transition toward a patientcentered paradigm that puts a strong emphasis on applied clinical research (Kempf et al, 2017;Lieu and Platt, 2017;Lacombe et al, 2019a;Lacombe et al, 2019b) Applied clinical research, which has also been described as treatment optimization (EORTC, 2019;Lacombe et al, 2019c), can be defined as optimizing the way treatments are utilized in realworld conditions through the conduct of studies set up to provide an answer to one or multiple of the research questions shown in Table 1 (Kempf et al, 2017;Lacombe et al, 2019a). It is not intended to replace the current drug development trials; instead, it seeks to deliver results complementing those of the registrational studies as part of a streamlined process that bridges the gap between the first and second stage research (Kempf et al, 2017;Lacombe et al, 2019b). While no definitive methodological TABLE 1 | Overview of research concepts and questions that remain underrepresented in clinical cancer research today (Kempf et al, 2017;Lacombe et al, 2017;Lacombe et al, 2019b).…”

Section: Introductionmentioning

confidence: 99%

“…It is not intended to replace the current drug development trials; instead, it seeks to deliver results complementing those of the registrational studies as part of a streamlined process that bridges the gap between the first and second stage research (Kempf et al, 2017;Lacombe et al, 2019b). While no definitive methodological TABLE 1 | Overview of research concepts and questions that remain underrepresented in clinical cancer research today (Kempf et al, 2017;Lacombe et al, 2017;Lacombe et al, 2019b). The conclusions which may emerge from studies that explore such topics are highly relevant for clinical practice and are shown for a given treatment A.…”

Section: Introductionmentioning

confidence: 99%

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Views of European Drug Development Stakeholders on Treatment Optimization and Its Potential for Use in Decision-Making

et al. 2020

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Background: The current drug development paradigm has been criticized for being too drug-centered and for not adequately focusing on the patients who will eventually be administered the therapeutic interventions it generates. The drug-driven nature of the present framework has led to the emergence of a research gap between the pre-approval development of anticancer medicines and their post-registration use in real-life clinical practice. This gap could potentially be bridged by transitioning toward a patient-centered paradigm that places a strong emphasis on treatment optimization, which strives to optimize the way health technologies are applied in a real-world environment. However, questions remain concerning the ideal features of treatment optimization studies and their acceptability among key stakeholders. Objectives: The aim of this study was to explore the views of key stakeholders in the drug development process regarding the concept of treatment optimization. Methods: Semi-structured interviews were conducted between December 2018 and May 2019 with 26 participants across ten EU Member States and six different stakeholder groups, including academic clinicians as well as representatives of patient organizations, regulatory authorities, health technology assessment agencies, payers, and industry. Results: Based on the input of the experts interviewed, clarification was obtained regarding the optimal features of treatment optimization studies in terms of their conduct, funding, timing, design, and setting. Moreover, a number of opportunities and challenges of undertaking such trials were identified. Inter-stakeholder discussion during their design was seen as desirable. There was also broad support among the participants for regulatory measures to facilitate treatment optimization, although there was no agreement on the optimal scale and nature of these initiatives. Furthermore, the interviewees believed that the evidence strength of well-designed treatment optimization studies performed according to rigorous quality standards is greater than or at least equal to that of classical clinical trials. In addition, there was a strong consensus

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Section: Methodsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Views of European Drug Development Stakeholders on Treatment Optimization and Its Potential for Use in Decision-Making

et al. 2020

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Design, organisation and impact of treatment optimisation studies in breast, lung and colorectal cancer: The experience of the European Organisation for Research and Treatment of Cancer

Saesen

Lacombe

Huys

2021

European Journal of Cancer

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Background: Treatment optimisation studies (TOSs) are clinical trials which aim to tackle research questions that are often left unaddressed within the current drug development paradigm due to a lack of financial and regulatory incentives to undertake them. Examples include comparative effectiveness, therapeutic sequencing and dose de-escalation studies. Trials of this nature have historically been primarily carried out by academic institutions and notfor-profit organisations such as the European Organisation for Research and Treatment of Cancer (EORTC). Objectives: Our objective was to conduct an in-depth analysis of the breast, lung and colorectal cancer TOSs that have been performed by the EORTC in the past four decades. Methods: We searched the EORTC clinical trials database for relevant studies and subsequently analysed them based on a number of predefined criteria relating to their design, organisation and scientific impact. Results: The 113 EORTC TOSs examined in this analysis were mainly standard-sized, international, multicentre phase III trials using a relatively simple, randomised, open-label design and comparing pharmacological combination regimens against standard-of-care treatments in terms of their potential to improve overall survival of patients with cancer. Although they were typically financially and/or materially supported by the industry, their legal sponsor was nearly always an independent party that did not benefit monetarily from their outcomes. If meaningful findings were obtained, their results, regardless of whether positive or negative, were published in high-impact journals, and the corresponding articles usually received a

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Patient-centricity in digital measure development: co-evolution of best practice and regulatory guidance

Aryal,

Blankenship,

Bachman

et al. 2024

npj Digit. Med.

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Digital health technologies (DHTs) have the potential to modernize drug development and clinical trial operations by remotely, passively, and continuously collecting ecologically valid evidence that is meaningful to patients’ lived experiences. Such evidence holds potential for all drug development stakeholders, including regulatory agencies, as it will help create a stronger evidentiary link between approval of new therapeutics and the ultimate aim of improving patient lives. However, only a very small number of novel digital measures have matured from exploratory usage into regulatory qualification or efficacy endpoints. This shows that despite the clear potential, actually gaining regulatory agreement that a new measure is both fit-for-purpose and delivers value remains a serious challenge. One of the key stumbling blocks for developers has been the requirement to demonstrate that a digital measure is meaningful to patients. This viewpoint aims to examine the co-evolution of regulatory guidance in the United States (U.S.) and best practice for integration of DHTs into the development of clinical outcome assessments. Contextualizing guidance on meaningfulness within the larger shift towards a patient-centric drug development approach, this paper reviews the U.S. Food and Drug Administration (FDA) guidance and existing literature surrounding the development of meaningful digital measures and patient engagement, including the recent examples of rejections by the FDA that further emphasize patient-centricity in digital measures. Finally, this paper highlights remaining hurdles and provides insights into the established frameworks for development and adoption of digital measures in clinical research.

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Moving forward from drug-centred to patient-centred research

Cited by 16 publications

References 26 publications

Views of European Drug Development Stakeholders on Treatment Optimization and Its Potential for Use in Decision-Making

Views of European Drug Development Stakeholders on Treatment Optimization and Its Potential for Use in Decision-Making

Design, organisation and impact of treatment optimisation studies in breast, lung and colorectal cancer: The experience of the European Organisation for Research and Treatment of Cancer

Patient-centricity in digital measure development: co-evolution of best practice and regulatory guidance

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