How should we assess the clinical and cost effectiveness of histology independent cancer drugs?

Cooper, Sophie; Bouvy, Jacoline C.; Baker, Lawrence; Maignen, F.; Jónsson, Páll; Clark, Peter; Palmer, Stephen; Boysen, Meindert; Crabb, Nick

doi:10.1136/bmj.l6435

Cited by 17 publications

(11 citation statements)

References 7 publications

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“…This may limit the applicability and relevance of our conclusions to other healthcare settings. Yet, our observations are at least broadly in line with the positions and views expressed by other decision makers external to our study team (10).…”

Section: Discussionsupporting

confidence: 87%

Exploring the opportunities for alignment of regulatory postauthorization requirements and data required for performance-based managed entry agreements

Eichler

Adams

Andreassen

et al. 2021

Int J Technol Assess Health Care

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Performance-based managed entry agreements (PB-MEAs) might allow patient access to new medicines, but practical hurdles make competent authorities for pricing and reimbursement (CAPR) reluctant to implement PB-MEAs. We explored if the feasibility of PB-MEAs might improve by better aligning regulatory postauthorization requirements with the data generation of PB-MEAs and by active collaboration and data sharing. Reviewers from seven CAPRs provided structured assessments of the information available at the European Medicines Agency (EMA) Web site on regulatory postauthorization requirements for fifteen recently authorized products. The reviewers judged to what extent regulatory postauthorization studies could help implement PB-MEAs by addressing uncertainty gaps. Study domains assessed were: patient population, intervention, comparators, outcomes, time horizon, anticipated data quality, and anticipated robustness of analysis. Reviewers shared general comments about PB-MEAs for each product and on cooperation with other CAPRs. Reviewers rated regulatory postauthorization requirements at least partly helpful for most products and across domains except the comparator domain. One quarter of responses indicated that public information provided by the EMA was insufficient to support the implementation of PB-MEAs. Few PB-MEAs were in place for these products, but the potential for implementation of PB-MEAs or collaboration across CAPRs was seen as more favorable. Responses helped delineate a set of conditions where PB-MEAs may help reduce uncertainty. In conclusion, PB-MEAs are not a preferred option for CAPRs, but we identified conditions where PB-MEAs might be worth considering. The complexities of implementing PB-MEAs remain a hurdle, but collaboration across silos and more transparency on postauthorization studies could help overcome some barriers.

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Section: Discussionsupporting

confidence: 87%

Exploring the opportunities for alignment of regulatory postauthorization requirements and data required for performance-based managed entry agreements

Eichler

Adams

Andreassen

et al. 2021

Int J Technol Assess Health Care

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“…A publication from senior members of the NICE team and CDF outlining the challenges to HTA bodies of assessing clinical efficacy and value for money of tumour-agnostic drugs and the need for post-authorisation data collection paved the way for release of the NICE decision. 1 …”

Section: Mainmentioning

confidence: 99%

“…The recent experience with larotrectinib highlights the importance to pharmaceutical companies of working with HTA bodies as they develop tumour-agnostic drugs to ensure that they consider the evidence requirements that will allow timely access to patients. 1 Marketing authorisation of more than one tumour-agnostic drug in the same class (e.g. both larotrectinib and entrectinib for TRK fusions) will in future be important to the NHS through introducing an element of competitive pricing.…”

Section: Mainmentioning

confidence: 99%

Tumour-agnostic drugs in paediatric cancers

2020

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“…The appraisal of other innovative technologies such as advanced therapy medicinal products including gene and cell therapies and histology-independent cancer drugs also represent a challenge to HTA agencies given the lack of experience in appraising these technologies [40]. Horizon Scanning and Scientific Advice activities implemented by some HTA agencies have been useful in this context [2,41].…”

Section: Potential Application Of Sandboxes In Htamentioning

confidence: 99%

The Sandbox Approach and its Potential for Use in Health Technology Assessment: A Literature Review

Leckenby

Dawoud

Bouvy

et al. 2021

Appl Health Econ Health Policy

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Background The concept of the regulatory sandbox-a safe space for testing new regulatory processes-was first used within the financial technologies (FinTech) sector, but has since expanded into other sectors, including healthcare. Objectives This review aims to describe the extent of use of sandboxes in healthcare and assess the potential for the sandbox approach to be used to test and develop emerging health technology assessment (HTA) methods, policies and processes for innovative technologies. Methods A systematic literature review was undertaken to identify published papers and reports that described and/or assessed the use of sandboxes in the healthcare sector. Searches were conducted in Medline, Embase, Econlit, Social Policy and Practice, and Health Management Information Consortium databases from inception to March 2020. Free-text Google search was also conducted to identify relevant grey literature. Only papers and reports discussing or evaluating the use of sandboxes in healthcare settings and published in English were included. Included studies were qualitatively summarised using a thematic analysis approach. Results Overall, 46 papers and reports were included. The topics covered were classified into 4 major themes: history of the regulatory sandbox, the sandbox as a testing environment, the sandbox as a regulatory approach, examples of using sandboxes in healthcare. Findings show that the use of regulatory sandboxes in healthcare is relatively new and primarily used in highincome countries to support the adoption of new technologies, particularly those related to digital health. Recommendations are made based on these findings to guide its use in HTA policy and methods development. Conclusions Sandboxes are increasingly used within healthcare regulation. Despite its potential, this approach has not been used in HTA policy and methodological developments to date. HTA agencies should consider this approach to facilitate developing policies, methods and processes for innovative and disruptive health technologies. Transferability to low-and middle-income countries' settings, however, should be assessed.

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How should we assess the clinical and cost effectiveness of histology independent cancer drugs?

Abstract: Sophie Cooper, Jacoline Bouvy and colleagues discuss the challenges that histology independent cancer drugs will pose for NICE and the NHS

Cited by 17 publications

References 7 publications

Exploring the opportunities for alignment of regulatory postauthorization requirements and data required for performance-based managed entry agreements

Exploring the opportunities for alignment of regulatory postauthorization requirements and data required for performance-based managed entry agreements

Tumour-agnostic drugs in paediatric cancers

The Sandbox Approach and its Potential for Use in Health Technology Assessment: A Literature Review

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