Including the patient voice in the development and implementation of patient‐reported outcomes in cancer clinical trials

Addario, Bonnie; Geißler, Jan; Horn, Marcia K.; Krebs, Linda U.; Maskens, Deborah; Oliver, Kathy; Plate, Ananda; Schwartz, Erin; Willmarth, Nicole E.

doi:10.1111/hex.12997

Cited by 28 publications

(19 citation statements)

References 54 publications

(96 reference statements)

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“…Patient-reported data not only has the capacity to empower patients in managing their own health condition but also contributes to broader knowledge that can inform healthcare more generally, including HTAs ( 47 , 48 ). Patients have felt that they are peripheral to the HTA process and that their involvement takes place too late in the process to make any real difference.…”

Section: Are We Seeing Changes In Approach?mentioning

confidence: 99%

Can We Afford to Exclude Patients Throughout Health Technology Assessment?

Wale¹,

Chandler

Collyar³

et al. 2022

Front. Med. Technol.

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Health technology assessment (HTA) is intended to determine the value of health technologies and, once a technology is recommended for funding, bridge clinical research and practice. Understanding the values and beliefs expressed by patients and health professionals can help guide this knowledge transfer and work toward managing the expectations of end users. We gathered patient and patient group leader experiences to gain insights into the roles that patients and patient advocacy groups are playing. We argue that through partnerships and co-creation between HTA professionals, researchers and patient advocates we can strengthen the HTA process and better align with service delivery where person-centered care and shared decision making are key elements. Patient experiences and knowledge are important to the democratization of evidence and the legitimacy of HTAs. Patient preference studies are used to balance benefits with potential harms of technologies, and patient-reported outcomes (PROs) can measure what matters to patients over time. A change in culture in HTA bodies is occurring and with further transformative thinking patients can be involved in every step of the HTA process. Patients have a right to be involved in HTAs, with patients' values central to HTA deliberations on a technology and where patients can provide valuable insights to inform HTA decision-making; and in ensuring that HTA methodologies evolve. By evaluating the implementation of HTA recommendations we can determine how HTA benefits patients and their communities. Our shared commitment can positively effect the common good and provide benefits to individual patients and their communities.

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Section: Are We Seeing Changes In Approach?mentioning

confidence: 99%

Can We Afford to Exclude Patients Throughout Health Technology Assessment?

Wale¹,

Chandler

Collyar³

et al. 2022

Front. Med. Technol.

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“…Capturing patient's perspective during clinical trials in the oncology setting is an opportunity to collect unique information on the patient's experience of the disease, its treatment and, most importantly, the impact on their quality of life, which may contribute to develop more appropriate health care interventions (1)(2)(3). Patient-Reported Outcomes (PRO) provide a holistic approach of treatment effects, and have been increasingly recognized as an essential complement to clinical [e.g., overall survival (OS), progression-free survival (PFS) and safety] and laboratory-related (e.g., biomarker) outcomes (4).…”

Section: Introductionmentioning

confidence: 99%

“…The use of PRO measures (PROM) is encouraged both by the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) guidelines for anticancer medicinal products development (1), covering single and multi-dimension measures such as symptoms, feelings, functioning, well-being and treatment satisfaction (5)(6)(7). Inclusion of PRO as an endpoint in a clinical trial is of added value for various stakeholders beyond regulators, including the health technology assessment (HTA) bodies/payers and professional organizations, as they contribute to distinct among products with similar survival benefits (1)(2)(3)(6)(7)(8)(9).…”

Section: Introductionmentioning

confidence: 99%

A review of patient-reported outcomes used for regulatory approval of oncology medicinal products in the European Union between 2017 and 2020

et al. 2022

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IntroductionCancer and corresponding available treatments are associated with substantial symptoms and functional limitations. In this context, collection of patient-reported outcomes (PRO) in clinical trials gained special interest and is recommended by regulatory authorities. Within clinical trials framework, PRO may provide evidence to support medicines approval, labeling and marketing claims. This study aims to analyze the existing evidence based on PRO as part of new oncology indications receiving positive opinions issued by the European Medicines Agency (EMA) between 2017 and 2020 and to identify PRO related label claims granted.MethodologyOncology medicinal products and indications approved by the European Commission following a positive opinion from the EMA between 2017 and 2020 were identified. European Public Assessment Report (EPAR) and Summary of Product Characteristics (SmPC) were reviewed for each medicinal product to identify use of PRO and PRO label claims.ResultsA total of 128 oncology indications, corresponding to 76 medicines, were approved; of those, 100 (78.1%) included PRO in the confirmatory clinical trials. Thirty-seven indications were supported by double-blind randomized trials and the remainder 63 by open-label trials. Out of the 104 confirmatory trials analyzed, PRO were defined as a secondary endpoint in 60 studies (57.7%), exploratory in 31 (29.8%) and as both in 13 (12.5%). In total, 54 different PRO measures (PROM) were used, of those 41 (75.9%) were disease-specific measures. Nevertheless, PROM selected relied on the EORTC (41.3%), FACIT (17.1%) and EQ-5D (29.2%) measures. A total of 76 indications (59.4%) had PRO reviewers comments included in the EPAR, however only 22 indications (17.8%) included label claims in the SmPC. The reasons identified in the EMA assessment supporting the exclusion of PRO claims were described for 34 indications (44.7%).ConclusionsDespite growing recognition of the value of PRO data for the development of improved cancer therapies, PRO implementation remains challenging. The main reasons identified in our study are related with study design, missing data, study conduct and PROM selection.

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“…To improve the patient centricity of the tool development process, it was imperative to include a patient advocate [ 30 ]. Patient advocates provide a patient’s perspective, which is key to effective care and the development of systems that support that care [ 31 , 32 ]. Therefore, one patient advocate was selected for the expert group to provide “the patient voice”.…”

Section: Methodsmentioning

confidence: 99%

Consensus for a primary care clinical decision-making tool for assessing, diagnosing, and managing shoulder pain in Alberta, Canada

et al. 2021

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Background Shoulder pain is a highly prevalent condition and a significant cause of morbidity and functional disability. Current data suggests that many patients presenting with shoulder pain at the primary care level are not receiving high quality care. Primary care decision-making is complex and has the potential to influence the quality of care provided and patient outcomes. The aim of this study was to develop a clinical decision-making tool that standardizes care and minimizes uncertainty in assessment, diagnosis, and management. Methods First a rapid review was conducted to identify existing tools and evidence that could support a comprehensive clinical decision-making tool for shoulder pain. Secondly, provincial consensus was established for the assessment, diagnosis, and management of patients presenting to primary care with shoulder pain in Alberta, Canada using a three-step modified Delphi approach. This project was a highly collaborative effort between Alberta Health Services’ Bone and Joint Health Strategic Clinical Network (BJH SCN) and the Alberta Bone and Joint Health Institute (ABJHI). Results A clinical decision-making tool for shoulder pain was developed and reached consensus by a province-wide expert panel representing various health disciplines and geographical regions. This tool consists of a clinical examination algorithm for assessing, diagnosis, and managing shoulder pain; recommendations for history-taking and identification of red flags or additional concerns; recommendations for physical examination and neurological screening; recommendations for the differential diagnosis; and care pathways for managing patients presenting with rotator cuff disease, biceps pathology, superior labral tear, adhesive capsulitis, osteoarthritis, and instability. Conclusions This clinical decision-making tool will help to standardize care, provide guidance on the diagnosis and management of shoulder pain, and assist in clinical decision-making for primary care providers in both public and private sectors.

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Including the patient voice in the development and implementation of patient‐reported outcomes in cancer clinical trials

Cited by 28 publications

References 54 publications

Can We Afford to Exclude Patients Throughout Health Technology Assessment?

Can We Afford to Exclude Patients Throughout Health Technology Assessment?

A review of patient-reported outcomes used for regulatory approval of oncology medicinal products in the European Union between 2017 and 2020

Consensus for a primary care clinical decision-making tool for assessing, diagnosing, and managing shoulder pain in Alberta, Canada

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