Objective This study aimed to understand the impact of different efficacy endpoints on reimbursement decisions made by health technology assessment (HTA) bodies. Materials and methods European Medicines Agency (EMA) oncology product marketing authorizations were screened to identify products that completed review by 3 HTA bodies during 2016-2019: United Kingdom's National Institute for Health and Care Excellence, Germany's Gemeinsamer Bundesausschuss, and France's Haute Autorite de Sante. Each decision's endpoint information, including overall survival (OS) and progression-free survival (PFS), was extracted. Each endpoint's influence on added benefits rating (the degree of added benefit as judged by the HTA agency) and full reimbursement (i.e. reimbursed population to label) decisions was tested using bivariate analyses. Results An increasing trend was observed toward HTA submissions with immature OS data (36.8% and 71.4% in 2016 and 2019, respectively), which was a predictor of limited added benefit (p < .001). Regarding data availability, 63% of submissions provided OS, 2% provided PFS without OS; and 35% provided neither. OS availability significantly influenced added benefit (p < .001) but not full reimbursement (p > .05) decisions, whereas PFS without OS had no significant impact compared with either OS or PFS data for either outcome (p = .99). Conclusions The trend toward fewer products filing mature OS data over time suggests sponsors may be increasingly confident achieving reimbursement with surrogate endpoint data, although mature OS data provided the strongest correlation to positive reimbursement decisions. Notably, in some locally advanced settings, OS data maturity will take a long time to obtain. To expedite patient access to new medicines, payers should consider the acceptance of surrogate endpoints predictive of clinical benefit.
机构:
BresMed Hlth Solut Ltd, North Church House,84 Queen St, Sheffield S1 2DW, S Yorkshire, England
Univ Sheffield, Sheffield, S Yorkshire, EnglandBresMed Hlth Solut Ltd, North Church House,84 Queen St, Sheffield S1 2DW, S Yorkshire, England
Akehurst, Ronald L.
Abadie, Eric
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机构:
Hop Ambroise Pare, Div Cardiol, Paris, FranceBresMed Hlth Solut Ltd, North Church House,84 Queen St, Sheffield S1 2DW, S Yorkshire, England
Abadie, Eric
Renaudin, Noel
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机构:
APHP Hop Paris, Conseil Adm, Paris, France
Conseil Adm InCa, Paris, FranceBresMed Hlth Solut Ltd, North Church House,84 Queen St, Sheffield S1 2DW, S Yorkshire, England
Renaudin, Noel
Sarkozy, Francois
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机构:
FSNB Hlth & Care, Paris, FranceBresMed Hlth Solut Ltd, North Church House,84 Queen St, Sheffield S1 2DW, S Yorkshire, England
机构:
Univ Exeter, Sch Med, Inst Hlth Res, Evidence Synth & Modelling Hlth Improvement, St Lukes Campus, Exeter EX1 2LU, Devon, England
Bocconi Univ, Ctr Res Hlth & Social Care Management, Milan, ItalyUniv Exeter, Sch Med, Inst Hlth Res, Evidence Synth & Modelling Hlth Improvement, St Lukes Campus, Exeter EX1 2LU, Devon, England
Ciani, Oriana
Buyse, Marc
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Int Drug Dev Inst, Louuain La Neuue, Belgium
CluePoints Inc, Cambridge, MA USAUniv Exeter, Sch Med, Inst Hlth Res, Evidence Synth & Modelling Hlth Improvement, St Lukes Campus, Exeter EX1 2LU, Devon, England
Buyse, Marc
Drummond, Michael
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Univ York, Ctr Hlth Econ, York, N Yorkshire, EnglandUniv Exeter, Sch Med, Inst Hlth Res, Evidence Synth & Modelling Hlth Improvement, St Lukes Campus, Exeter EX1 2LU, Devon, England
Drummond, Michael
Rasi, Guido
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European Med Agcy, London, England
Univ Tor Vergata, Rome, ItalyUniv Exeter, Sch Med, Inst Hlth Res, Evidence Synth & Modelling Hlth Improvement, St Lukes Campus, Exeter EX1 2LU, Devon, England
Rasi, Guido
Saad, Everardo D.
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机构:
Dendrix Res, Sao Paulo, BrazilUniv Exeter, Sch Med, Inst Hlth Res, Evidence Synth & Modelling Hlth Improvement, St Lukes Campus, Exeter EX1 2LU, Devon, England
机构:
Univ Washington, Div Hematol, Inst Hlth Metr & Evaluat, Seattle, WA 98195 USAUniv Washington, Div Hematol, Inst Hlth Metr & Evaluat, Seattle, WA 98195 USA
机构:
Promed writing, Grillparzerstr. 7, Freiburg
Center for Cognitive Science, University of Freiburg, FreiburgPromed writing, Grillparzerstr. 7, Freiburg
机构:
Univ Oxford, John Radcliffe Hosp, Sch Med, Oxford OX3 9DU, EnglandUniv Oxford, John Radcliffe Hosp, Sch Med, Oxford OX3 9DU, England
Kemp, Robert
Prasad, Vinay
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机构:
Oregon Hlth & Sci Univ, Knight Canc Inst, Div Hematol Oncol, 3181 SW Sam Jackson Pk Rd, Portland, OR 97239 USA
Oregon Hlth & Sci Univ, Dept Publ Hlth & Prevent Med, 3181 SW Sam Jackson Pk Rd, Portland, OR 97239 USA
Oregon Hlth & Sci Univ, Ctr Hlth Care Eth, 3181 SW Sam Jackson Pk Rd, Portland, OR 97239 USAUniv Oxford, John Radcliffe Hosp, Sch Med, Oxford OX3 9DU, England
机构:
BresMed Hlth Solut Ltd, North Church House,84 Queen St, Sheffield S1 2DW, S Yorkshire, England
Univ Sheffield, Sheffield, S Yorkshire, EnglandBresMed Hlth Solut Ltd, North Church House,84 Queen St, Sheffield S1 2DW, S Yorkshire, England
Akehurst, Ronald L.
Abadie, Eric
论文数: 0引用数: 0
h-index: 0
机构:
Hop Ambroise Pare, Div Cardiol, Paris, FranceBresMed Hlth Solut Ltd, North Church House,84 Queen St, Sheffield S1 2DW, S Yorkshire, England
Abadie, Eric
Renaudin, Noel
论文数: 0引用数: 0
h-index: 0
机构:
APHP Hop Paris, Conseil Adm, Paris, France
Conseil Adm InCa, Paris, FranceBresMed Hlth Solut Ltd, North Church House,84 Queen St, Sheffield S1 2DW, S Yorkshire, England
Renaudin, Noel
Sarkozy, Francois
论文数: 0引用数: 0
h-index: 0
机构:
FSNB Hlth & Care, Paris, FranceBresMed Hlth Solut Ltd, North Church House,84 Queen St, Sheffield S1 2DW, S Yorkshire, England
机构:
Univ Exeter, Sch Med, Inst Hlth Res, Evidence Synth & Modelling Hlth Improvement, St Lukes Campus, Exeter EX1 2LU, Devon, England
Bocconi Univ, Ctr Res Hlth & Social Care Management, Milan, ItalyUniv Exeter, Sch Med, Inst Hlth Res, Evidence Synth & Modelling Hlth Improvement, St Lukes Campus, Exeter EX1 2LU, Devon, England
Ciani, Oriana
Buyse, Marc
论文数: 0引用数: 0
h-index: 0
机构:
Int Drug Dev Inst, Louuain La Neuue, Belgium
CluePoints Inc, Cambridge, MA USAUniv Exeter, Sch Med, Inst Hlth Res, Evidence Synth & Modelling Hlth Improvement, St Lukes Campus, Exeter EX1 2LU, Devon, England
Buyse, Marc
Drummond, Michael
论文数: 0引用数: 0
h-index: 0
机构:
Univ York, Ctr Hlth Econ, York, N Yorkshire, EnglandUniv Exeter, Sch Med, Inst Hlth Res, Evidence Synth & Modelling Hlth Improvement, St Lukes Campus, Exeter EX1 2LU, Devon, England
Drummond, Michael
Rasi, Guido
论文数: 0引用数: 0
h-index: 0
机构:
European Med Agcy, London, England
Univ Tor Vergata, Rome, ItalyUniv Exeter, Sch Med, Inst Hlth Res, Evidence Synth & Modelling Hlth Improvement, St Lukes Campus, Exeter EX1 2LU, Devon, England
Rasi, Guido
Saad, Everardo D.
论文数: 0引用数: 0
h-index: 0
机构:
Dendrix Res, Sao Paulo, BrazilUniv Exeter, Sch Med, Inst Hlth Res, Evidence Synth & Modelling Hlth Improvement, St Lukes Campus, Exeter EX1 2LU, Devon, England
机构:
Univ Washington, Div Hematol, Inst Hlth Metr & Evaluat, Seattle, WA 98195 USAUniv Washington, Div Hematol, Inst Hlth Metr & Evaluat, Seattle, WA 98195 USA
机构:
Promed writing, Grillparzerstr. 7, Freiburg
Center for Cognitive Science, University of Freiburg, FreiburgPromed writing, Grillparzerstr. 7, Freiburg
机构:
Univ Oxford, John Radcliffe Hosp, Sch Med, Oxford OX3 9DU, EnglandUniv Oxford, John Radcliffe Hosp, Sch Med, Oxford OX3 9DU, England
Kemp, Robert
Prasad, Vinay
论文数: 0引用数: 0
h-index: 0
机构:
Oregon Hlth & Sci Univ, Knight Canc Inst, Div Hematol Oncol, 3181 SW Sam Jackson Pk Rd, Portland, OR 97239 USA
Oregon Hlth & Sci Univ, Dept Publ Hlth & Prevent Med, 3181 SW Sam Jackson Pk Rd, Portland, OR 97239 USA
Oregon Hlth & Sci Univ, Ctr Hlth Care Eth, 3181 SW Sam Jackson Pk Rd, Portland, OR 97239 USAUniv Oxford, John Radcliffe Hosp, Sch Med, Oxford OX3 9DU, England