Abstract 132P
Background
Patients with hormone receptor–positive, HER2–negative breast cancer who have residual invasive disease after neoadjuvant chemotherapy (NACT), are at high risk of relapse. PENELOPE-B was a double-blind, placebo-controlled, phase III study that investigated adding palbociclib (PAL) to adjuvant endocrine therapy (ET) in these high-risk patients. Clinical results showed no improvement in invasive disease-free survival with PAL compared to ET alone (Loibl S et al. JCO 2021). Here we evaluated the cost-effectiveness of PAL in PENELOPE-B.
Methods
A total of 1´250 patients from 10 countries and 221 centers were randomly assigned to receive 125 mg of PAL or placebo over 13 28-day cycles. Health status and medical resource use were assessed before, during, and after treatment for up to 72 months. The EQ-5D instrument was used to score the health-related quality of life. Direct medical costs were assessed from the German health system perspective. Costs and effects were discounted at 3%. The incremental impacts of PAL on costs and Quality-Adjusted Life Years (QALYs) were estimated with generalized linear models adjusting for randomization, between-country heterogeneity, and censoring. Scenario and sensitivity analyses explored uncertainty in patient pathways and costs. Subgroup analyses were performed for key prognostic risk factors.
Results
In the analysis based on all patients, PAL led to a marginal increase in QALYs per patient of 0.005 (bootstrap 95% confidence interval (CI) -0.049; 0.040) at an incremental cost of EUR 9´262 (CI -14´572; 33´096) and a cost-effectiveness ratio above EUR 2´000´000 per QALY gained. Analysis restricted to patients enrolled in Germany differed in the direction and the magnitude of the effects. Neither outcomes were statistically significant. Costs varied greatly among patients; scenario analysis indicated the therapy might be cost-saving in Germany. Findings from subgroup analyses by risk factor were consistent with main results.
Conclusions
One year of PAL added to ET is unlikely to be cost-effective in women with residual invasive disease after NACT but could be cost-saving in some settings.
Clinical trial identification
NCT01864746.
Editorial acknowledgement
Legal entity responsible for the study
German Breast Group.
Funding
Pfizer.
Disclosure
S. Loibl: Financial Interests, Institutional, Advisory Board, honorarium for Ad Boards & Lecture, paid to institute / Medical Writing: Pfizer; Financial Interests, Institutional, Advisory Board, honorarium for Ad Boards, paid to institute: AbbVie; Financial Interests, Institutional, Advisory Board, honorarium for Ad Boards, paid to institute: Amgen; Financial Interests, Institutional, Advisory Board, honorarium for Ad Boards & Lectures, paid to institute / Medical Writing: AstraZeneca; Financial Interests, Institutional, Advisory Board, honorarium for Ad Board, paid to institute: Bayer; Financial Interests, Institutional, Advisory Board, honorarium for Ad Boards, paid to institute / Medical Writing: BMS; Financial Interests, Institutional, Advisory Board, honorarium for Ad Boards, paid to institute / Medical Writing: Celgene; Financial Interests, Personal, Invited Speaker, lecture: Chugai; Financial Interests, Institutional, Advisory Board, honorarium for Ad Board & Lecture, paid to institute / Medical Writing: Daiichi-Sankyo; Financial Interests, Institutional, Advisory Board, honorarium for Ad Board, paid to institute: Eirgenix; Financial Interests, Institutional, Advisory Board, honorarium for Ad Board, paid to institute: GSK; Financial Interests, Institutional, Research Grant, paid to institute / Medical Writing: Immunomedics/Gilead; Financial Interests, Institutional, Other, paid to institute: Ipsen; Financial Interests, Institutional, Advisory Board, honorarium for Ad Boards, paid to institute: Lilly; Financial Interests, Institutional, Advisory Board, honorarium for Ad Boards, paid to institute: Merck; Financial Interests, Institutional, Advisory Board, honorarium for Ad Board & Lectures, paid to institute / Medical Writing: Novartis; Financial Interests, Institutional, Advisory Board, honorarium for Ad Board & Lecture, paid to institute: Pierre Fabre; Financial Interests, Institutional, Advisory Board, honorarium for Ad Board & Lecture, paid to institute: Prime/Medscape; Financial Interests, Institutional, Advisory Board, honorarium for Ad Board, paid to institute: Puma; Financial Interests, Institutional, Advisory Board, honorarium for Ad Boards & Lectures, paid to institute / Medical Writing: Roche; Financial Interests, Institutional, Invited Speaker, honorarium for Lecture, paid to institute: Samsung; Financial Interests, Institutional, Advisory Board, honorarium for Ad Boards, paid to institute: Seagen; Financial Interests, Institutional, Writing Engagements, paid to institute / Medical Writing: Vifor; Financial Interests, Personal, Other, Method for predicting the response to cancer immunotherapy in cancer patients: EP18209672.7; Financial Interests, Personal, Other, Method for predicting the response to CDK4/6 inhibitor therapy in cancer patients: EP21152186.9; Financial Interests, Personal, Other, Method for predicting the response to an anti-HER2 containing therapy and/or chemotherapy in patients with breast cancer: EP15702464.7; Financial Interests, Institutional, Licensing Fees, VM Scope GmbH, paid to institute: VM Scope GmbH. P. Salari: Financial Interests, Institutional, Research Grant, Research funding for health economic part of the study provided by German Breast Group: German Breast Group; Financial Interests, Institutional, Research Grant: Novartis. F. Marmé: Financial Interests, Institutional, Research Grant: AstraZeneca; Financial Interests, Personal, Other: AstraZeneca; Financial Interests, Personal, Other: MSD; Financial Interests, Personal, Other: Clovis; Financial Interests, Personal, Other: GSK/Tesaro; Financial Interests, Personal, Other: Pfizer; Financial Interests, Personal, Other: Novartis; Financial Interests, Personal, Other: Lilly; Financial Interests, Personal, Other: Roche; Financial Interests, Personal, Other: Celgene; Financial Interests, Personal, Other: Seagen; Financial Interests, Personal, Other: Myriad; Financial Interests, Personal, Other: PharmaMar; Financial Interests, Personal, Other: Eisai; Financial Interests, Personal, Other: Janssen-Cilag. M. Martin: Financial Interests, Personal, Research Grant: Roche; Financial Interests, Personal, Research Grant: Novartis; Financial Interests, Personal, Other: DAIICHI SANKYO; Financial Interests, Personal, Other: Lilly; Financial Interests, Personal, Research Grant: Puma; Financial Interests, Personal, Other: AstraZeneca; Financial Interests, Personal, Other: Pierre Fabre; Financial Interests, Personal, Other: Pfizer. M. Untch: Financial Interests, Institutional, Other, All fees to the institution/employer: AbbVie; Financial Interests, Institutional, Other, All fees to the institution/employer: Amgen GmbH; Financial Interests, Institutional, Other, All fees to the institution/employer: AstraZeneca; Financial Interests, Institutional, Other, All fees to the institution/employer: BMS; Financial Interests, Institutional, Other, All fees to the institution/employer: Celgene GmbH; Financial Interests, Institutional, Other, All fees to the institution/employer: Daiichi Sankyo; Financial Interests, Institutional, Other, All fees to the institution/employer: Eisai GmbH; Financial Interests, Institutional, Other, All fees to the institution/employer: Lilly Deutschland; Financial Interests, Institutional, Other, All fees to the institution/employer: Lilly Int.; Financial Interests, Institutional, Other, All fees to the institution/employer: MSD Merck; Financial Interests, Institutional, Other, All fees to the institution/employer: Mundipharma; Financial Interests, Institutional, Other, All fees to the institution/employer: Myriad Genetics; Financial Interests, Institutional, Other, All fees to the institution/employer: Pfizer GmbH; Financial Interests, Institutional, Other, All fees to the institution/employer: Roche Pharma AG; Financial Interests, Institutional, Other, All fees to the institution/employer: Sanofi Aventis Deutschland GmbH; Financial Interests, Institutional, Other, All fees to the institution/employer: TEVA Pharmaceuticals Ind Ltd.; Financial Interests, Institutional, Other, All fees to the institution/employer: Novartis; Financial Interests, Institutional, Other, All fees to the institution/employer: Pierre Fabre; Financial Interests, Institutional, Other, All fees to the institution/employer: Clovis Oncology; Financial Interests, Institutional, Other, All fees to the institution/employer: Seattle Genetics; Financial Interests, Institutional, Other, All fees to the institution/employer: Seagen. S. Kim: Financial Interests, Personal, Research Grant: Novartis; Financial Interests, Personal, Research Grant: Sanofi-Aventis; Financial Interests, Personal, Research Grant: DongKook Pharm Co; Financial Interests, Personal, Advisory Board: Novartis; Financial Interests, Personal, Advisory Board: AstraZeneca; Financial Interests, Personal, Advisory Board: Lilly; Financial Interests, Personal, Advisory Board: Dae Hwa Pharmaceutical Co. Ltd.; Financial Interests, Personal, Advisory Board: ISU Abxis; Financial Interests, Personal, Advisory Board: Daiichi-Sankyo; Financial Interests, Personal, Stocks/Shares: Genopeaks; Financial Interests, Personal, Stocks/Shares: NeogeneTC. H.D. Bear: Financial Interests, Personal, Research Grant: NSABP Foundation; Financial Interests, Personal, Stocks/Shares, Own stock in Pfizer: Pfizer. N. McCarthy: Financial Interests, Personal, Advisory Board: Pfizer; Financial Interests, Personal, Advisory Board: Novartis; Financial Interests, Personal, Advisory Board: AstraZeneca; Financial Interests, Personal, Advisory Board: Roche; Financial Interests, Personal, Advisory Board: Specialised Therapeutics; Financial Interests, Personal, Advisory Board: Eisai; Financial Interests, Personal, Advisory Board: Amgen; Financial Interests, Personal, Other, Travel, Accommodations, Expenses: AstraZeneca; Financial Interests, Personal, Other, Travel, Accommodations, Expenses: Roche; Financial Interests, Personal, Other, Travel, Accommodations, Expenses: Novartis. K. Gelmon: Financial Interests, Personal, Other, Honoraria: Pfizer; Financial Interests, Personal, Other, Honoraria: Eli Lilly; Financial Interests, Personal, Other, Honoraria: AstraZeneca; Financial Interests, Personal, Other, Honoraria: Novartis; Financial Interests, Personal, Other, Honoraria: Seagen; Financial Interests, Institutional, Funding: Pfizer; Financial Interests, Institutional, Funding: AstraZeneca; Financial Interests, Institutional, Funding: BMS; Financial Interests, Institutional, Funding: Roche; Financial Interests, Institutional, Funding: Seagen; Financial Interests, Personal, Expert Testimony: Genentech. J.A. García-Saenz: Financial Interests, Personal, Other: Lilly; Financial Interests, Personal, Other: Pfizer; Financial Interests, Personal, Other: Novartis; Financial Interests, Personal, Other: Seagen; Financial Interests, Personal, Other: AstraZeneca; Financial Interests, Personal, Other: Daiichi Sankyo; Financial Interests, Personal, Other: MSD; Financial Interests, Personal, Other: Roche. T. Reimer: Financial Interests, Personal, Other: Pfizer. M. Toi: Financial Interests, Personal, Research Grant, Research grant, Lecture honoraria: Chugai; Financial Interests, Personal, Research Grant, Research grant, Lecture honoraria: Takeda; Financial Interests, Personal, Research Grant, Research grant, Lecture honoraria: Pfizer; Financial Interests, Personal, Research Grant, Research grant, Lecture honoraria, Advisory role for a drug development: Kyowa-Kirin; Financial Interests, Personal, Research Grant, Research grant, Lecture honoraria: Taiho; Financial Interests, Institutional, Research Grant, Research grant: JBCRG association; Financial Interests, Personal, Research Grant, Research grant, Lecture honoraria: Eisai; Financial Interests, Personal, Research Grant, Research grant, Lecture honoraria, Advisory role for a drug development: Daiichi-Sankyo; Financial Interests, Research Grant, Research grant, Lecture honoraria: AstraZeneca; Financial Interests, Invited Speaker, Lecture Honoraria, An advisory role: Eli Lilly; Financial Interests, Personal, Invited Speaker, Lecture Honoraria: MSD; Financial Interests, Invited Speaker, Lecture Honoraria: Exact Science; Financial Interests, Invited Speaker, Lecture Honoraria: Novartis; Financial Interests, Advisory Role, Honoraria for an advisory meeting: Konica Minolta; Financial Interests, Research Grant, Research grant: Astellas; Financial Interests, Advisory Role, Honoraria for an advisory meeting: BMS; Financial Interests, Research Grant, Research grant, Lecture Honoraria: Shimadzu; Financial Interests, Research Grant, Research grant, Lecture Honoraria: Yakult; Financial Interests, Invited Speaker, Research Fund and Honoraria for lecture: Nippon Kayaku; Financial Interests, Research Grant, Research grant: AFI technologies; Financial Interests, Advisory Role, An advisory role: Athenex Oncology; Financial Interests, Advisory Role, An advisory role: Bertis; Financial Interests, Advisory Role, An advisory role: Terumo; Financial Interests, Research Grant, Research grant, an advisory role: Luxonus; Financial Interests, Research Grant, Research grant: Shionogi; Financial Interests, Research Grant, Research grant: GL Science; Financial Interests, Advisory Role, An advisory role: Kansai Medical Net; Financial Interests, Member of the Board of Directors: JBCRG association, Organisation for Oncology and Translational Research, Kyoto Breast Cancer Research Network. H.S. Rugo: Financial Interests, Research Grant, Research support for clinical trials through the University of California: Pfizer; Financial Interests, Research Grant, Research support for clinical trials through the University of California: Merck; Financial Interests, Research Grant, Research support for clinical trials through the University of California: Novartis; Financial Interests, Research Grant, Research support for clinical trials through the University of California: Lilly; Financial Interests, Research Grant, Research support for clinical trials through the University of California: Genentech; Financial Interests, Research Grant, Research support for clinical trials through the University of California: Odonate; Financial Interests, Research Grant, Research support for clinical trials through the University of California: Daiichi; Financial Interests, Research Grant, Research support for clinical trials through the University of California: Seattle Genetics; Financial Interests, Research Grant, Research support for clinical trials through the University of California: Eisai; Financial Interests, Research Grant, Research support for clinical trials through the University of California: Macrogenics; Financial Interests, Research Grant, Research support for clinical trials through the University of California: Sermonix; Financial Interests, Research Grant, Research support for clinical trials through the University of California: Boehringer Ingelheim; Financial Interests, Research Grant, Research support for clinical trials through the University of California: Polyphor; Financial Interests, Research Grant, Research support for clinical trials through the University of California: AstraZeneca; Financial Interests, Research Grant, Research support for clinical trials through the University of California: Immunomedics; Financial Interests, Other, Honoraria: Puma; Financial Interests, Other, Honoraria: Mylan; Financial Interests, Other, Honoraria: Samsung. M. Gnant: Financial Interests, Personal, Other, an immediate family member is employed by Sandoz.: Sandoz; Financial Interests, Personal, Invited Speaker: Amgen; Financial Interests, Personal, Invited Speaker: Daiichi Sankyo; Financial Interests, Personal, Invited Speaker: AstraZeneca; Financial Interests, Personal, Invited Speaker: Eli Lilly; Financial Interests, Personal, Invited Speaker: LifeBrain; Financial Interests, Personal, Invited Speaker: Nanostring; Financial Interests, Personal, Invited Speaker: Novartis; Financial Interests, Personal, Invited Speaker: TLC Biopharmaceuticals. A. Makris: Financial Interests, Invited Speaker, Lectures for Pfizer: Pfizer; Financial Interests, Advisory Board, Advisory Boards for Pfizer: Pfizer. M. Schwenkglenks: Financial Interests, Research Grant, Research funding for health economic part of the study provided by German Breast Group: German Breast Group; Financial Interests, Research Grant, Research funding via employment institution: Swiss Group for Clinical Cancer Research (SAKK); Financial Interests, Research Grant, Research funding via employment institution: Merck, Sharp & Dohme; Financial Interests, Research Grant, Research funding via employment institution: Novartis; Financial Interests, Research Grant, Research funding via employment institution: Immunocore; Financial Interests, Research Grant, Research funding via employment institution; personal fees < CHF 5'000: Amgen; Financial Interests, Research Grant, Research funding via employment institution; personal fees < CHF 5'000: BMS; Financial Interests, Research Grant, Research funding via employment institution: AbbVie; Financial Interests, Personal, Invited Speaker, Personal fees < CHF 5'000: Pfizer. All other authors have declared no conflicts of interest.