A Paradigm Shift Towards Patient Involvement in Medicines Development and Regulatory Science: Workshop Proceedings and Commentary

Borup, Gitte; Bach, Karin Friis; Schmiegelow, Merete; Kildemoes, Helle Wallach; Bjerrum, Ole J.; Westergaard, Niels

doi:10.1177/2168479015622668

Cited by 21 publications

(16 citation statements)

References 17 publications

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“…Instrumental benefits are related to improving the relevance of assessment to making better quality decisions, for example higher accuracy in measuring needs and preferences of patients and better quality of assessment and relevance of reports to the local context . Study uptake benefits refer to the usefulness of assessments for decision makers and the uptake of evidence by decision makers, for example gaining regulatory approval . Developmental benefits include, for example, increasing the public's understanding of HTA and openness of decision processes …”

Section: Resultsmentioning

confidence: 99%

Evaluating the “return on patient engagement initiatives” in medicines research and development: A literature review

Vat

Finlay

Schuitmaker-Warnaar

et al. 2019

Health Expectations

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Background Showing how engagement adds value for all stakeholders can be an effective motivator for broader implementation of patient engagement. However, it is unclear what methods can best be used to evaluate patient engagement. This paper is focused on ways to evaluate patient engagement at three decision‐making points in the medicines research and development process: research priority setting, clinical trial design and early dialogues with regulators and health technology assessment bodies. Objective Our aim was to review the literature on monitoring and evaluation of patient engagement, with a focus on indicators and methods. Search strategy and inclusion criteria We undertook a scoping literature review using a systematic search, including academic and grey literature with a focus on evaluation approaches or outcomes associated with patient engagement. No date limits were applied other than a cut‐off of publications after July 2018. Data extraction and synthesis Data were extracted from 91 publications, coded and thematically analysed. Main results A total of 18 benefits and 5 costs of patient engagement were identified, mapped with 28 possible indicators for their evaluation. Several quantitative and qualitative methods were found for the evaluation of benefits and costs of patient engagement. Discussion and conclusions Currently available indicators and methods are of some use in measuring impact but are not sufficient to understand the pathway to impact, nor whether interaction between researchers and patients leads to change. We suggest that the impacts of patient engagement can best be determined not by applying single indicators, but a coherent set of measures.

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Section: Resultsmentioning

confidence: 99%

Evaluating the “return on patient engagement initiatives” in medicines research and development: A literature review

Vat

Finlay

Schuitmaker-Warnaar

et al. 2019

Health Expectations

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“…The healthcare industry is undergoing a paradigm shift towards a more patient-centric approach as patients take centre stage in the contemporary patient-focused drug development (PFDD) process [1,2]. For successful development of meaningful therapies, it has become vital to determine what matters most to patients living with the respective condition.…”

Section: Introductionmentioning

confidence: 99%

Gathering Structured Patient Insight to Drive the PRO Strategy in COPD: Patient-Centric Drug Development from Theory to Practice

et al. 2019

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We illustrate our experience of gathering patient insights on the most patient-relevant symptoms in chronic obstructive pulmonary disease (COPD) via a structured and systematic approach towards 'patient-centric' drug development, leveraging recent advances in digital technologies using online platforms. The fourstep approach comprised the following: literature search, social media listening (SML) study, online bulletin board (OBB) exercise, and design of an online patient preference study (PPS). The initial online studies (SML and OBB) revealed that, besides dyspnoea and exacerbations, patients perceive cough and mucus production as equally important aspects of disease management for COPD. To further build and quantify patients' understanding of the importance of these symptoms, an online patient preference survey is underway. Based on these findings, we have elected to include the Cough and Sputum Assessment Questionnaire or CASA-Q, a validated instrument to collect patient-reported outcomes (PRO), besides the use of the COPD assessment test or CAT to assess the severity and impact of COPD in drug development studies for COPD. Additionally, to capture movement and sleep disturbance, we consider the inclusion of actigraphy as a digital evidence-capture end point. Lastly, in a phase II trial, a survey questionnaire on incontinence will be administered to evaluate the importance of this issue among patients. We believe that integrating insights derived from ''online'' studies (SML, OBB, and PPS) into drug development offers an opportunity to truly listen to patients' voices in early product design ensuring relevance of end points selected for the clinical trial program. This approach also has the potential to complement conventional qualitative and quantitative data collection requirements for PRO instrument development. While awaiting final guidance from the US Food and Drug Administration, or FDA, the recently released draft documents on collecting representative patients' input reference social media as a tool to collect qualitative patient preference data and these developments suggest that patient preference data can influence future clinical trial design, end point selection, and regulatory reviews.

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“…Our study results are consistent with those reported in Europe; specifically, uncertainty around the engagement process, the public’s lack of knowledge and interest in research and development (and the need to be proactive in education efforts), and industry’s lack of knowledge, interest, and receptivity to patient engagement. 12 , 13 …”

Section: Discussionmentioning

confidence: 99%

The Rules of Engagement: CTTI Recommendations for Successful Collaborations Between Sponsors and Patient Groups Around Clinical Trials

Bloom

Beetsch²,

Harker³

et al. 2018

Ther Innov Regul Sci

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Objective:To identify the elements necessary for successful collaboration between patient groups and academic and industry sponsors of clinical trials, in order to develop recommendations for best practices for effective patient group engagement.Methods:In-depth interviews, informed by a previously reported survey, were conducted to identify the fundamentals of successful patient group engagement. Thirty-two respondents from 3 sectors participated: patient groups, academic researchers, and industry. The findings were presented to a multistakeholder group of experts in January 2015. The expert group came to consensus on a set of actionable recommendations for best practices for patient groups and research sponsors.Results:Interview respondents acknowledged that not all patient groups are created equal in terms of what they can contribute to a clinical trial. The most important elements for effective patient group engagement include establishing meaningful partnerships, demonstrating mutual benefits, and collaborating as partners from the planning stage forward. Although there is a growing appreciation by sponsors about the benefits of patient group engagement, there remains some resistance and some uncertainty about how best to engage. Barriers include mismatched expectations and a perception that patient groups lack scientific sophistication and that “wishful thinking” may cloud their recommendations.Conclusions:Patient groups are developing diverse skillsets and acquiring assets to leverage in order to become collaborators with industry and academia on clinical trials. Growing numbers of research sponsors across the clinical trials enterprise are recognizing the benefits of continuous and meaningful patient group engagement, but there are still mindsets to change, and stakeholders need further guidance on operationalizing a new model of clinical trial conduct.

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A Paradigm Shift Towards Patient Involvement in Medicines Development and Regulatory Science: Workshop Proceedings and Commentary

Cited by 21 publications

References 17 publications

Evaluating the “return on patient engagement initiatives” in medicines research and development: A literature review

Evaluating the “return on patient engagement initiatives” in medicines research and development: A literature review

Gathering Structured Patient Insight to Drive the PRO Strategy in COPD: Patient-Centric Drug Development from Theory to Practice

The Rules of Engagement: CTTI Recommendations for Successful Collaborations Between Sponsors and Patient Groups Around Clinical Trials

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