{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,12,31]],"date-time":"2025-12-31T20:00:59Z","timestamp":1767211259221,"version":"3.48.0"},"reference-count":24,"publisher":"BMJ","issue":"3","license":[{"start":{"date-parts":[[2023,3,14]],"date-time":"2023-03-14T00:00:00Z","timestamp":1678752000000},"content-version":"unspecified","delay-in-days":13,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["bmj.com"],"crossmark-restriction":true},"short-container-title":["BMJ Open"],"accepted":{"date-parts":[[2023,2,19]]},"published-print":{"date-parts":[[2023,3]]},"abstract":"<jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>To review the survival modelling used in cost-effectiveness studies evaluating an interventional procedure and to discuss implications for decision-makers.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design<\/jats:title>\n                    <jats:p>A case study of three economic evaluations that each used immature data from the EVEREST II High Surgical Risk (HSR) Study of transcatheter edge-to-edge repair (TEER) for patients with severe mitral regurgitation (MR) who were at high risk of surgery.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Setting<\/jats:title>\n                    <jats:p>Estimation of patient survival in cost-effectiveness studies.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Participants<\/jats:title>\n                    <jats:p>The EVEREST II HSR Study included 78 patients who had TEER of the mitral valve using the MitraClip device and a retrospectively identified control group of 36 patients who received medical management and were followed up for 12 months. Observed survival (TEER arm only) was updated at 5\u2009years.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Two studies used 12-month observed mortality from EVEREST II HSR to model survival over lifetime horizons. Observed and modelled survival were associated with considerable uncertainty due to short follow-up and small numbers of participants. Modelling control patients\u2019 survival required an approximate 10-fold extrapolation based on 12-month observation of only 38 patients. Observed 5-year survival in the TEER group differed from that less mature follow-up suggesting that survival modelling based on shorter follow-up was unsatisfactory. No public domain data for the control group are available beyond 12-month follow-up so meaningful estimates using mature data for both arms are currently not possible. A third study developed survival models using incompletely reported transitions between MR grades in EVEREST II HSR and mortality rates observed for different MR grades derived from a study in an unrelated population.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Modelling survival in such small samples followed up for only 12 months is associated with great uncertainty, and cost-effectiveness results based on these analyses should be viewed as premature and used cautiously in reimbursement decisions.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1136\/bmjopen-2021-060423","type":"journal-article","created":{"date-parts":[[2023,3,14]],"date-time":"2023-03-14T10:26:01Z","timestamp":1678789561000},"page":"e060423","update-policy":"https:\/\/doi.org\/10.1136\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":1,"title":["Potential impact on cost-effectiveness estimates of using immature survival data: a case study based on transcatheter edge-to-edge repair (TEER) used for patients with severe mitral regurgitation at high surgical risk"],"prefix":"10.1136","volume":"13","clinical-trial-number":[{"clinical-trial-number":"nct02715284","registry":"10.18810\/clinical-trials-gov"},{"clinical-trial-number":"nct01940120","registry":"10.18810\/clinical-trials-gov"}],"author":[{"given":"Martin","family":"Connock","sequence":"first","affiliation":[{"name":"Division of Health Sciences, Warwick Medical School, University of Warwick, Coventry, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Peter","family":"Auguste","sequence":"additional","affiliation":[{"name":"Warwick Medical School, University of Warwick, Coventry, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Aude","family":"Capelle","sequence":"additional","affiliation":[{"name":"Pharmacy Department, Saint-Etienne University Hospital Bellevue Site, Saint-Etienne, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anna-Maria","family":"H\u00e9naine","sequence":"additional","affiliation":[{"name":"Clinical Pharmacy Department, School of Pharmacy, Beirut, Lebanon"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jean-Fran\u00e7ois","family":"Obadia","sequence":"additional","affiliation":[{"name":"H\u00f4pital Cardiovasculaire Louis Pradel, Chirurgie Cardio-Vasculaire et Transplantation Cardiaque, Hospices Civils de Lyon and Claude Bernard University, Lyon, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1166-0453","authenticated-orcid":false,"given":"Xavier","family":"Armoiry","sequence":"additional","affiliation":[{"name":"Warwick Medical School, University of Warwick, Coventry, UK"},{"name":"School of Pharmacy (ISPB)\/UMR CNRS 5510 MATEIS\/ Edouard Herriot Hospital, Pharmacy Department, University of Lyon, Lyon, France"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"239","published-online":{"date-parts":[[2023,3,14]]},"reference":[{"key":"2025121515040437000_13.3.e060423.1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacc.2011.08.067"},{"key":"2025121515040437000_13.3.e060423.2","unstructured":"FDA . 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