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Review

A systematic literature review on the efficacy–effectiveness gap: comparison of randomized controlled trials and observational studies of glucose-lowering drugs

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Pages 41-51 | Published online: 23 Jan 2017

References

  • LuceBRDrummondMJonssonBEBM, HTA, and CER: clearing the confusionMilbank Q201088225627620579285
  • EichlerHGBloechl-DaumBAbadieEBarnettDKonigFPearsonSRelative efficacy of drugs: an emerging issue between regulatory agencies and third-party payersNat Rev Drug Discov20109427729120186141
  • SilvermanEEffectiveness/efficacy difference too often ignoredManag Care201322136
  • NordonCKarcherHGroenwoldRHGetReal consortiumThe “Efficacy-Effectiveness Gap”: historical background and current conceptualizationValue Health2016191758126797239
  • DavisCEGeneralizing from clinical trialsControl Clin Trials199415111148149768
  • BaileyKRGeneralizing the results of randomized clinical trialsControl Clin Trials199415115238149769
  • BrittonAMcKeeMBlackNMcPhersonKSandersonCBainCThreats to applicability of randomised trials: exclusions and selective participationJ Health Serv Res Policy19994211212110387403
  • DowdRReckerRRHeaneyRPStudy subjects and ordinary patientsOsteoporos Int200011653353610982170
  • KhanAYPreskornSHBakerBEffect of study criteria on recruitment and generalizability of the resultsJ Clin Psychopharmacol200525327127515876909
  • Van SpallHGTorenAKissAFowlerRAEligibility criteria of randomized controlled trials published in high-impact general medical journals: a systematic sampling reviewJAMA2007297111233124017374817
  • SorensenHTLashTLRothmanKJBeyond randomized controlled trials: a critical comparison of trials with nonrandomized studiesHepatology20064451075108217058242
  • LaupacisAMamdaniMObservational studies of treatment effectiveness: some cautionsAnn Intern Med20041401192392415172907
  • LawlorDADavey SmithGEbrahimSCommentary: the hormone replacement-coronary heart disease conundrum: is this the death of observational epidemiology?Int J Epidemiol200433346446715166201
  • FreidlinBKornELAssessing causal relationships between treatments and clinical outcomes: always read the fine printBone Marrow Transplant201247562663221625225
  • BoykoEJObservational research–opportunities and limitationsJ Diabetes Complications201327664264824055326
  • EichlerHGAbadieEBreckenridgeABridging the efficacy-effectiveness gap: a regulator’s perspective on addressing variability of drug responseNat Rev Drug Discov201110749550621720406
  • The National Institute for Health and Care Excellence (NICE) [homepage on the Internet]Managing blood glucose in adults with type 2 diabetes Available from: https://pathways.nice.org.uk/pathways/type-2-diabetes-in-adults#path=view%3A/pathways/type-2-diabetes-in-adults/managing-blood-glucose-in-adults-with-type-2-diabetes.xml&content=view-node%3Anodes-insulin-based-treatmentsAccessed November 25, 2016
  • BergenstalRLewinABaileyTEfficacy and safety of biphasic insulin aspart 70/30 versus exenatide in subjects with type 2 diabetes failing to achieve glycemic control with metformin and a sulfonylureaCurr Med Res Opin2009251657519210140
  • DaviesMHellerSSreenanSOnce-weekly exenatide versus once- or twice-daily insulin detemir: randomized, open-label, clinical trial of efficacy and safety in patients with type 2 diabetes treated with metformin alone or in combination with sulfonylureasDiabetes Care20133651368137623275363
  • NauckMHortonEAndjelkovicMT-emerge 5 Study GroupTaspoglutide, a once-weekly glucagon-like peptide 1 analogue, vs. insulin glargine titrated to target in patients with type 2 diabetes: an open-label randomized trialDiabet Med201330110911322937895
  • Del PratoSCamisascaRWilsonCFleckPDurability of the efficacy and safety of alogliptin compared with glipizide in type 2 diabetes mellitus: a 2-year studyDiabetes Obes Metab201416121239124625132212
  • HigginsJPTGreenSCochrane Collaboration Cochrane Handbook for Systematic Reviews of InterventionsChichester, EnglandWiley – Blackwell2008
  • SterneJHigginsJReevesBA Cochrane Risk of Bias Assessment Tool: for Non-Randomized Studies of Interventions (ACROBAT-NRSI), Version 1.0.02492014 Available from: http://www.riskofbias.infoAccessed October 8, 2015
  • U.S. Department of Health and Human Services Food and Drug Administration Center for Drug Evaluation and Research (CDER)Guidance for Industry Diabetes Mellitus: Developing Drugs and Therapeutic Biologics for Treatment and PreventionCDERSilver Spring, MD, USA2008
  • DiamantMVan GaalLGuerciBExenatide once weekly versus insulin glargine for type 2 diabetes (DURATION-3): 3-year results of an open-label randomised trialLancet Diabetes Endocrinol20142646447324731672
  • DiamantMVan GaalLStranksSSafety and efficacy of once-weekly exenatide compared with insulin glargine titrated to target in patients with type 2 diabetes over 84 weeksDiabetes Care201235468368922357185
  • DiamantMVan GaalLStranksSOnce weekly exenatide compared with insulin glargine titrated to target in patients with type 2 diabetes (DURATION-3): an open-label randomised trialLancet201037597332234224320609969
  • ThayerSWeiWBuysmanEThe INITIATOR study: pilot data on real-world clinical and economic outcomes in US patients with type 2 diabetes initiating injectable therapyAdv Ther201330121128114024293131
  • HeineRJVan GaalLFJohnsDExenatide versus insulin glargine in patients with suboptimally controlled type 2 diabetes: a randomized trialAnn Intern Med2005143855956916230722
  • BarnettAHBurgerJJohnsDTolerability and efficacy of exenatide and titrated insulin glargine in adult patients with type 2 diabetes previously uncontrolled with metformin or a sulfonylurea: a multinational, randomized, open-label, two-period, crossover noninferiority trialClin Ther200729112333234818158075
  • BunckMCDiamantMCornerAOne-year treatment with exenatide improves beta-cell function, compared with insulin glargine, in metformin-treated type 2 diabetic patients: a randomized, controlled trialDiabetes Care200932576276819196887
  • DaviesMJDonnellyRBarnettAHJonesSNicolayCKilcoyneAExenatide compared with long-acting insulin to achieve glycaemic control with minimal weight gain in patients with type 2 diabetes: results of the helping evaluate exenatide in patients with diabetes compared with long-acting insulin (HEELA) studyDiabetes Obes Metab200911121153116219930005
  • InagakiNAtsumiYOuraTSaitoHImaokaTEfficacy and safety profile of exenatide once weekly compared with insulin once daily in Japanese patients with type 2 diabetes treated with oral antidiabetes drug(s): results from a 26-week, randomized, open-label, parallel-group, multicenter, noninferiority studyClin Ther20123491892190822884767
  • WeissmanPNCarrMCYeJHARMONY 4: randomised clinical trial comparing once-weekly albiglutide and insulin glargine in patients with type 2 diabetes inadequately controlled with metformin with or without sulfonylureaDiabetologia201457122475248425208756
  • Russell-JonesDVaagASchmitzOLiraglutide Effect and Action in Diabetes 5 (LEAD-5) met+SU Study GroupLiraglutide vs insulin glargine and placebo in combination with metformin and sulfonylurea therapy in type 2 diabetes mellitus (LEAD-5 met+SU): a randomised controlled trialDiabetologia200952102046205519688338
  • HortonESSilbermanCDavisKLBerriaRWeight loss, glycemic control, and changes in cardiovascular biomarkers in patients with type 2 diabetes receiving incretin therapies or insulin in a large cohort databaseDiabetes Care20103381759176520460445
  • PawaskarMLiQHoogwerfBJMetabolic outcomes of matched patient populations initiating exenatide BID vs. insulin glargine in an ambulatory care settingDiabetes Obes Metab201214762663322321776
  • HallGCMcMahonADDainMPWangEHomePDPrimary-care observational database study of the efficacy of GLP-1 receptor agonists and insulin in the UKDiabet Med201330668168623330649
  • KaragianniPPolyzosSAKartaliNZografouISambanisCComparative efficacy of exenatide versus insulin glargine on glycemic control in type 2 diabetes mellitus patients inadequately treated with metformin monotherapyAdv Med Sci2013581384323640946
  • BounthavongMTranJNGolshanSRetrospective cohort study evaluating exenatide twice daily and long-acting insulin analogs in a Veterans Health Administration population with type 2 diabetesDiabetes Metab201440428429125059703
  • NauckMAMeiningerGShengDTerranellaLSteinPPSitagliptin Study GEfficacy and safety of the dipeptidyl peptidase-4 inhibitor, sitagliptin, compared with the sulfonylurea, glipizide, in patients with type 2 diabetes inadequately controlled on metformin alone: a randomized, double-blind, non-inferiority trialDiabetes Obes Metab20079219420517300595
  • SeckTNauckMShengDSitagliptin Study 024 GroupSafety and efficacy of treatment with sitagliptin or glipizide in patients with type 2 diabetes inadequately controlled on metformin: a 2-year studyInt J Clin Pract201064556257620456211
  • FerranniniEFonsecaVZinmanBFifty-two-week efficacy and safety of vildagliptin vs. glimepiride in patients with type 2 diabetes mellitus inadequately controlled on metformin monotherapyDiabetes Obes Metab200911215716619125777
  • MatthewsDRDejagerSAhrenBVildagliptin add-on to metformin produces similar efficacy and reduced hypoglycaemic risk compared with glimepiride, with no weight gain: results from a 2-year studyDiabetes Obes Metab201012978078920649630
  • GökeBGallwitzBErikssonJHellqvistAGause-NilssonID1680C00001 InvestigatorsSaxagliptin is non-inferior to glipizide in patients with type 2 diabetes mellitus inadequately controlled on metformin alone: a 52-week randomised controlled trialInt J Clin Pract201064121619163120846286
  • GökeBGallwitzBErikssonJGHellqvistAGause-NilssonISaxagliptin vs. glipizide as add-on therapy in patients with type 2 diabetes mellitus inadequately controlled on metformin alone: long-term (52-week) extension of a 52-week randomised controlled trialInt J Clin Pract201367430731623638466
  • FoleyJESreenanSEfficacy and safety comparison between the DPP-4 inhibitor vildagliptin and the sulfonylurea gliclazide after two years of monotherapy in drug-naive patients with type 2 diabetesHorm Metab Res2009411290590919705345
  • FilozofCGautierJFA comparison of efficacy and safety of vildagliptin and gliclazide in combination with metformin in patients with type 2 diabetes inadequately controlled with metformin alone: a 52-week, randomized studyDiabet Med201027331832620536495
  • JeonHJOhTKComparison of vildagliptin-metformin and glimepiride-metformin treatments in type 2 diabetic patientsDiabetes Metab J201135552953522111045
  • SrivastavaSSaxenaGNKeshwaniPGuptaRComparing the efficacy and safety profile of sitagliptin versus glimepiride in patients of type 2 diabetes mellitus inadequately controlled with metformin aloneJ Assoc Physicians India2012602730
  • Arjona FerreiraJCCorryDMogensenCEEfficacy and safety of sitagliptin in patients with type 2 diabetes and ESRD receiving dialysis: a 54-week randomized trialAm J Kidney Dis201361457958723352379
  • Arjona FerreiraJCMarreMBarzilaiNEfficacy and safety of sitagliptin versus glipizide in patients with type 2 diabetes and moderate-to-severe chronic renal insufficiencyDiabetes Care20133651067107323248197
  • DerosaGCiceroAFFranzettiIGA randomized, double-blind, comparative therapy evaluating sitagliptin versus glibenclamide in type 2 diabetes patients already treated with pioglitazone and metformin: a 3-year studyDiabetes Technol Ther201315321422223427864
  • RosenstockJWilsonCFleckPAlogliptin versus glipizide monotherapy in elderly type 2 diabetes mellitus patients with mild hyperglycaemia: a prospective, double-blind, randomized, 1-year studyDiabetes Obes Metab2013151090691423531118
  • KimHSShinJALeeSHA comparative study of the effects of a dipeptidyl peptidase-IV inhibitor and sulfonylurea on glucose variability in patients with type 2 diabetes with inadequate glycemic control on metforminDiabetes Technol Ther2013151081081624050737
  • AhrénBJohnsonSLStewartMHARMONY 3 Study GroupHARMONY 3: 104-week randomized, double-blind, placebo- and active-controlled trial assessing the efficacy and safety of albiglutide compared with placebo, sitagliptin, and glimepiride in patients with type 2 diabetes taking metforminDiabetes Care20143782141214824898304
  • DerosaGBonaventuraABianchiLVildagliptin compared to glimepiride on post-prandial lipemia and on insulin resistance in type 2 diabetic patientsMetabolism201463795796724874591
  • ShimodaSIwashitaSSekigamiTComparison of the efficacy of sitagliptin and glimepiride dose-up in Japanese patients with type 2 diabetes poorly controlled by sitagliptin and glimepiride in combinationJ Diabetes Investig201453320326
  • MorganCLPooleCDEvansMBarnettAHJenkins-JonesSCurrieCJWhat next after metformin? a retrospective evaluation of the outcome of second-line, glucose-lowering therapies in people with type 2 diabetesJ Clin Endocrinol Metab201297124605461223076348
  • LeeYKSongSOKimKJGlycemic effectiveness of metformin-based Ddual-combination therapies with sulphonylurea, pioglitazone, or DPP4-inhibitor in drug-naive Korean type 2 diabetic patientsDiabetes Metab J201337646547424404518
  • GittAKBramlagePBinzCKreklerMDeegETschopeDPrognostic implications of DPP-4 inhibitor vs. sulfonylurea use on top of metformin in a real world setting – results of the 1 year follow-up of the prospective DiaRegis registryInt J Clin Pract201367101005101423981060
  • GökeRGruenbergerJBBaderGDworakMReal-life efficacy and safety of vildagliptin compared with sulfonylureas as add-on to metformin in patients with type 2 diabetes mellitus in GermanyCurr Med Res Opin201430578578924328429
  • PeinemannFTushabeDAKleijnenJUsing multiple types of studies in systematic reviews of health care interventions – a systematic reviewPLoS One2013812e8503524416098
  • AnglemyerAHorvathHTBeroLHealthcare outcomes assessed with observational study designs compared with those assessed in randomized trialsCochrane Database Syst Rev20144MR000034
  • AhrénBMathieuCBaderGSchweizerAFoleyJEEfficacy of vildagliptin versus sulfonylureas as add-on therapy to metformin: comparison of results from randomised controlled and observational studiesDiabetologia20145771304130724682379
  • MathieuCBarnettAHBrathHEffectiveness and tolerability of second-line therapy with vildagliptin vs. other oral agents in type 2 diabetes: a real-life worldwide observational study (EDGE)Int J Clin Pract2013671094795623961850
  • SirisESSelbyPLSaagKGBorgstromFHeringsRMSilvermanSLImpact of osteoporosis treatment adherence on fracture rates in North America and EuropeAm J Med2009122Suppl 2S3S13