Abstract
Gene therapy trial results show potential to cure haemophilia A and haemophilia B. Securing broad access to a cure for a lifelong chronic disease is anticipated to face barriers at the individual and healthcare system levels, which can be partly mitigated by harmonized planning of clinical research studies. The aim of the coreHEM project was to determine the set of outcome measures required to evaluate efficacy, safety, comparative effectiveness and value of gene therapy for haemophilia. Modified Delphi consensus process, based on methods adapted from the COMET Initiative. Forty-nine participants (five patients, five clinicians, five researchers, four regulators, three research agencies, six health technology assessors, nine payers and 12 drug developers) took part in the study, with over 90% participation. The frequency of bleeds, factor activity level, duration of expression, chronic pain, healthcare resource use and mental health were identified as the core outcomes to be measured in addition to regulatory-mandated adverse effects. For the first time in haemophilia, a core outcome set has been developed, with the involvement of representatives of all relevant stakeholder groups. The core set has been expanded to include outc...Continue Reading
References
Feb 2, 2011·PLoS Medicine·Ian P SinhaPaula R Williamson
Oct 11, 2012·JAMA : the Journal of the American Medical Association·Carolyn R BroderickSimon A Brown
Oct 10, 2013·Trials·Jamie J KirkhamPaula R Williamson
Nov 20, 2014·The New England Journal of Medicine·Amit C NathwaniAndrew M Davidoff
Mar 2, 2016·Journal of Comparative Effectiveness Research·Sean R TunisPaula R Williamson
Dec 3, 2016·Orphanet Journal of Rare Diseases·Ghayath JanoudiTrevor Richter
Jun 2, 2017·Orphanet Journal of Rare Diseases·Jamie O'HaraDaniel-Anibal Garcia Diego
Jul 12, 2017·The New England Journal of Medicine·Johannes OldenburgMidori Shima
Aug 24, 2017·Haemophilia : the Official Journal of the World Federation of Hemophilia·G F PierceS W Pipe
Nov 4, 2017·Orphanet Journal of Rare Diseases·Thomas Morel, Stefan J Cano
Nov 18, 2017·PLoS Medicine·Jamie J KirkhamPaula R Williamson
Dec 7, 2017·The New England Journal of Medicine·Lindsey A GeorgeKatherine A High
Dec 12, 2017·The New England Journal of Medicine·Savita RangarajanK John Pasi
Citations
Jan 4, 2019·Haemophilia : the Official Journal of the World Federation of Hemophilia·Glenn F PierceUNKNOWN Members of the WFH Gene Therapy Round Table Program Committee and Organizing Committee
Nov 26, 2018·Clinical Pharmacology and Therapeutics·Robyn LimLinda Wilhelm
Jul 12, 2019·Haemophilia : the Official Journal of the World Federation of Hemophilia·Flora PeyvandiWing Y Wong
Jun 4, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Steven W Pipe
Jun 5, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Barbara A KonklePeter Marks
May 25, 2020·The Patient·Elizabeth ClearfieldDonna A Messner
Sep 11, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Glenn F PierceMark W Skinner
Apr 24, 2019·Research and Practice in Thrombosis and Haemostasis·Barbara A KonkleAlfonso Iorio
Feb 9, 2019·Trials·Sinead DuaneDeclan Devane
Jun 18, 2020·Research and Practice in Thrombosis and Haemostasis·Saunya DoverBrian M Feldman
Nov 15, 2019·Haemophilia : the Official Journal of the World Federation of Hemophilia·Mark W SkinnerErik Berntorp
Oct 15, 2019·Expert Review of Hematology·Emerito-Carlos Rodriguez-Merchan
Aug 15, 2020·The Patient·Tania C Vasquez-LoarteDeborah J Bowen
Nov 9, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Eline van OverbeekeIsabelle Huys
Nov 26, 2020·BMJ Open·Alice LeeAmber E Young
Nov 28, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Declan NooneGlenn F Pierce
Aug 3, 2020·Blood Reviews·Gaia SpadarellaGiovanni Di Minno
Nov 27, 2020·Drug Discovery Today·Eline van OverbeekeSteven Simoens
Feb 8, 2021·Haemophilia : the Official Journal of the World Federation of Hemophilia·Camila C AquinoAlfonso Iorio
Oct 30, 2020·Journal of Clinical Medicine·Heike DudaChristine Keipert
Oct 17, 2020·American Journal of Hematology·Stacy E CroteauAllison P Wheeler
Oct 30, 2020·Therapeutic Innovation & Regulatory Science·Kevin NamMatt W Courtney
Apr 24, 2021·Haemophilia : the Official Journal of the World Federation of Hemophilia·Wolfgang MiesbachMark W Skinner
Apr 20, 2021·Research and Practice in Thrombosis and Haemostasis·Evelien KrumbCedric Hermans
Nov 14, 2020·Blood Reviews·Robert F SidonioStacy E Croteau
May 25, 2021·Research and Practice in Thrombosis and Haemostasis·Erna C van BalenSamantha C Gouw
Jun 25, 2021·Biologics : Targets & Therapy·Emanuela MarchesiniLeonard Valentino
May 28, 2021·The Journal of Trauma and Acute Care Surgery·Philip C SpinellaUNKNOWN Hemostasis Trials Outcomes Working Group
Jul 8, 2021·Blood·Frank W G Leebeek, Wolfgang Miesbach
Oct 5, 2021·Expert Opinion on Emerging Drugs·Hande Kizilocak, Guy Young
Oct 8, 2021·Clinical Obstetrics and Gynecology·Marisa E Schwab, Tippi C MacKenzie
Dec 2, 2021·Pediatrics in Review·Omar Matuk-VillazonFernando F Corrales-Medina