Bridging the gap: Can International Consortium of Health Outcomes Measurement standard sets align outcomes accepted for regulatory and health technology assessment decision‐making of oncology medicines

Kalf, Rachel R. J.; Vreman, Rick A; Delnoij, Diana; Bouvy, Marcel L.; Goettsch, Wim

doi:10.1002/prp2.742

Cited by 10 publications

(18 citation statements)

References 22 publications

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“…Non-oncology conditions were selected given previous work has examined uptake across oncology HTAs. 12,13 and where no appropriate NICE HTAs were identified, alternative COS were selected, using the same criteria of publication between 2015 and 2019 with patient involvement. The most recently published reports which matched a COS were included, with outcomes compared to core outcomes from the corresponding COS.…”

Section: Methodsmentioning

confidence: 99%

“…However, it should be noted that these outcome sets are developed for routine care, unlike COS which are produced specifically for research so are more relevant for HTAs. 12 Additionally, a review of technology appraisal oncology scopes found that in the majority of cases there was complete overlap with those outcomes in COS; in a small number of exceptions the COS included two additional outcomes to those specified in the scope. 13 Other work has investigated a trial funder and member of INAHTA, the National Institute for Health Research Health Technology Assessment (NIHR HTA), examining their recommendation for applicants to search for a COS to include in their trial.…”

Section: Introductionmentioning

confidence: 99%

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A pilot study assessing the similarity between core outcome sets and outcomes included in health technology assessments

Cox

Williamson

2022

F1000Res

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Objective: Core outcome sets (COS) are an agreed standardised collection of outcomes created with representation from all key stakeholders (such as patients, clinicians, researchers), which should be reported as a minimum for all trials in that corresponding clinical area. There has been little research investigating the use of core outcomes in Health technology assessments (HTAs) and none in non-oncology HTAs. This study aimed to assess the similarity between COS and HTA outcomes. Methods: Ten COS published between 2015 and 2019 were selected, with patient participation taken as a proxy measure for a high quality COS. The INAHTA database was used as a source to identify relevant HTAs, which were accessed through the hyperlinks provided. Outcomes selected for these assessments were categorised as either a specific, partial or no match compared to the COS. An additional cohort of non-oncology HTAs published between 2019 and 2021 were identified from the NICE website and compared against a relevant COS. Results: Six hundred and fifty-one HTAs were matched to the ten COS areas, of which 119 were reviewed. Of a possible 1318 core outcome matches, there were 562 (43%) matches, 413 (31%) specific and 149 (11%) partial. NICE HTA matches against corresponding COS ranged from 44% to 100%, with a total of 78% (73/94) matches, 57 (61%) specific and 16 (17%) partial. Conclusion: Further work is required to promote the awareness and implementation of COS within HTAs. The degree of matching between COS and NICE HTA outcomes is encouraging, demonstrating acceptance of COS by HTA producers.

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Section: Methodsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

A pilot study assessing the similarity between core outcome sets and outcomes included in health technology assessments

Cox

Williamson

2022

F1000Res

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“…The outcomes included in these HTAs were compared to core outcomes included in the matching COS. Non-oncology conditions were selected given previous work has examined uptake across oncology HTAs. 12,13 The results are presented through tables, graphs and using descriptive statistics. The analysis was performed using Excel 2019.…”

Section: Methodsmentioning

confidence: 99%

A pilot study assessing the uptake of core outcome sets in health technology assessments

Cox

Williamson

2021

F1000Res

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Objective: Core outcome sets (COS) are an agreed standardised collection of outcomes created with representation from all key stakeholders (such as patients, clinicians, researchers), which should be reported as a minimum for all trials in that corresponding clinical area. There has been little research investigating the use of core outcomes in Health technology assessments (HTAs) and none in non-oncology HTAs. This study aimed to assess the similarity between COS and HTA outcomes. Methods: Ten COS published between 2015 and 2019 were selected, with patient participation taken as a proxy measure for a high quality COS. The INAHTA database was used as a source to identify relevant HTAs, which were accessed through the hyperlinks provided. Outcomes selected for these assessments were categorised as either a specific, partial or no match compared to the COS. An additional cohort of non-oncology HTAs published between 2019 and 2021 were identified from the NICE website and compared against a relevant COS. Results: Six hundred and fifty-one HTAs were matched to the ten COS areas, of which 119 were reviewed. Of a possible 1318 core outcome matches, there were 562 (43%) matches, 413 (31%) specific and 149 (11%) partial. NICE HTA matches against corresponding COS ranged from 44% to 100%, with a total of 78% (73/94) matches, 57 (61%) specific and 16 (17%) partial. Conclusion: Further work is required to promote the awareness and implementation of COS within HTAs. Improved uptake across NICE HTAs is encouraging, demonstrating acceptance of COS by HTA producers.

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“…and therefore will be captured as part or routine care by the provider. We are hopeful that ongoing international efforts at standardizing outcomes measurements 43 may help improve technical efficiencies. Where legally possible, regulators could help catalyze broad agreement on a given end point (see below).…”

Section: Alignment Of Stakeholder Interestsmentioning

confidence: 99%

Precision Reimbursement for Precision Medicine: Using Real‐World Evidence to Evolve From Trial‐and‐Project to Track‐and‐Pay to Learn‐and‐Predict

Eichler

Trusheim

Schwarzer-Daum

et al. 2021

Clin Pharma and Therapeutics

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Basic scientists and drug developers are accelerating innovations toward the goal of precision medicine. Regulators create pathways for timely patient access to precision medicines, including individualized therapies. Healthcare payors acknowledge the need for change but downstream innovation for coverage and reimbursement is only haltingly occurring. Performance uncertainty, high price‐tags, payment timing, and actuarial risk issues associated with precision medicines present novel financial challenges for payors. With traditional drug reimbursement frameworks, payment is based on an assumed randomized controlled trial (RCT) projection of real‐world effectiveness, a “trial‐and‐project” strategy; the clinical benefit realized for patients is not usually ascertained ex post by collection of real‐world data (RWD). To mitigate financial risks resulting from clinical performance uncertainty, manufacturers and payors devised “track‐and‐pay” frameworks (i.e., the tracking of a pre‐agreed treatment outcome which is linked to financial consequences). Whereas some track‐and‐pay arrangements have been successful, inherent weaknesses include the potential for misalignment of incentives, the risk of channeling of patients, and a failure to use the RWD generated to enable continuous learning about treatments. “Precision reimbursement” (PR) intends to overcome inherent weaknesses of simple track‐and‐pay schemes. In combining the collection of RWD with advanced analytics (e.g., artificial intelligence and machine learning) to generate actionable real‐world evidence, with prospective alignment of incentives across all stakeholders (including providers and patients), and with pre‐agreed use and dissemination of information generated, PR becomes a “learn‐and‐predict” model of payment for performance. We here describe in detail the concept of PR and lay out the next steps to make it a reality.

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Bridging the gap: Can International Consortium of Health Outcomes Measurement standard sets align outcomes accepted for regulatory and health technology assessment decision‐making of oncology medicines

Abstract: This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made.

Cited by 10 publications

References 22 publications

A pilot study assessing the similarity between core outcome sets and outcomes included in health technology assessments

A pilot study assessing the similarity between core outcome sets and outcomes included in health technology assessments

A pilot study assessing the uptake of core outcome sets in health technology assessments

Precision Reimbursement for Precision Medicine: Using Real‐World Evidence to Evolve From Trial‐and‐Project to Track‐and‐Pay to Learn‐and‐Predict

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