Harish Eswaran, MD^1*, Jamie Jarmul, MD PhD^2*, Amy Shaheen, MD MSc^1*, David Meaux, BA^2*, Tristan Long, MBA^2*, Derek Saccoccio, MBA^2* and Stephan Moll, MD¹

¹Department of Medicine, University of North Carolina School of Medicine, Chapel Hill, NC
²UNC Health, Chapel Hill, NC

Federico Germini, MD, MSc^1,2*, Suzanne O'Callaghan, BA^3*, Chatree Chai-Adisaksopha, MD, PhD^4,5, Randall Curtis, MBA^6*, Neil Frick^7*, Michael B. Nichol, PhD^8*, Declan Noone, MSc^9,10*, Brian O'Mahony^11,12*, David Page^13*, Jeffrey Stonebraker^14*, Mark W Skinner, JD^1,15* and Alfonso Iorio, MD, PhD^1,16

¹Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Canada
²Department of Medicine, McMaster University, Hamilton, Canada
³Haemophilia Foundation Australia, Melbourne, Australia
⁴Medicine, McMaster University, Muang, Thailand
⁵Division of Hematology, Department of Internal Medicine, Chiang Mai University, Chiang Mai, Thailand
⁶Hematology Utilization Group Study (HUGS), Walnut Creek, CA
⁷National Hemophilia Foundation, Berkeley
⁸University of Southern California, Los Angeles, CA
⁹Health Decisions Consultants, Dublin, Ireland
¹⁰European Haemophilia Consortium, Brussels, Belgium
¹¹Irish Haemophilia Society, Dublin, Ireland
¹²Trinity College Dublin, Dublin, Ireland
¹³Canadian Hemophilia Society, Montreal, Canada
¹⁴North Carolina State University, Poole College of Management, Raleigh
¹⁵Institute for Policy Advancement Ltd, Washington, DC
¹⁶McMaster-Bayer Endowed Research Chair in Clinical Epidemiology of Congenital bleeding Disorders, Department of Medicine, McMaster University, Hamilton, Canada

Joanne Wu, MD, MS^1*, Randall Curtis, MBA^2*, Megan M. Ullman, MA, MPH^3*, Judith Baker, DrPH, MHSA^4*, Duc Quang Tran Jr., MD⁵ and Michael B. Nichol, PhD^6*

¹University Of Southern California, Los Angeles, CA
²Factor VIII Computing, Berkeley, CA
³Gulf States Hemophilia & Thrombophilia Center, University of Texas Health Science Center at Houston, Houston, TX
⁴Center for Inherited Blood Disorders, Orange, CA
⁵Emory University, Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory, Atlanta, GA
⁶University of Southern California, Los Angeles, CA

Poy P. Theprungsirikul, MD and Sunil E. Saith, MD, MPH, FACP^*

NYU Langone Health/NYU Grossman School of Medicine, New York, NY

Paul McLaughlin, MSc, MMACP^1*, Cedric Hermans, MD, MRCP, PhD², Sohaib Asghar, MSc^3*, Tom Burke^3*, Francis Nissen^4*, Martynas Aizenas^5*, Oliver Meier^4*, Harpal Dhillon^3* and Jamie O'Hara, MSc^3,6*

¹Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free London NHS Foundation Trust, London, United Kingdom
²Division of Hematology, Cliniques Univ. Saint-Luc, Brussels, Belgium
³HCD Economics, Daresbury, United Kingdom
⁴F. Hoffmann-La Roche Ltd, Basel, Switzerland
⁵F. Hoffmann-La Roche Ltd, Basel, CA, Switzerland
⁶University of Chester, Chester, United Kingdom

Jeries Kort, MD¹, Paolo F Caimi, MD², Pingfu Fu, PhD^3*, Shufen Cao^3*, James Driscoll^3*, Marcos de Lima, MD² and Ehsan Malek, MD⁴

¹University Hospitals Cleveland Medical Center, Cleveland, OH
²Adult Hematologic Malignancies & Stem Cell Transplant Section, University Hospitals Seidman Cancer Center, Cleveland, OH
³Case Western Reserve University, Cleveland, OH
⁴Adult Hematologic Malignancies & Stem Cell Transplant Section, Seidman Cancer Center, University Hospitals Cleveland Medical Center, Cleveland, OH

Clay T Cohen, MD¹, Sarah E Sartain, MD¹, Haleh Sangi-Haghpeykar, PhD^2*, Kamlesh Kukreja, MD^3* and Sudhen B Desai, MD^3*

¹Department of Pediatrics, Section of Hematology-Oncology, Baylor College of Medicine/Texas Children's Hospital, Houston, TX
²Department of Obstetrics and Gynecology, Baylor College of Medicine, Houston, TX
³Section of Interventional Radiology, Baylor College of Medicine/Texas Children's Hospital, Houston, TX

Heidi E. Kosiorek, MS^1*, Amylou C. Dueck, PhD¹, John Mascarenhas, MD^2,3, Ruben A. Mesa, MD, FACP⁴, Ronald Hoffman, MD^2,5 and Blake T. Langlais, MS^1*

¹Department of Health Sciences Research, Mayo Clinic, Scottsdale, AZ
²Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY
³Division of Hematology/Medical Oncology, Icahn School of Medicine at Mount Sinai, Tisch Cancer Institute, New York, NY
⁴Mays Cancer Center at UT Health San Antonio, San Antonio, TX
⁵Tisch Cancer Institute, Division of Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai, New York, NY

Dvora Bauman, MD^1*, Adir Sommer, MD^2*, Dar Noy^3*, Tal Hammer^3* and Shoshana Revel-Vilk, MD, MSc^3,4

¹Department of Obstetrics and Gynaecology, Hadassah Hebrew University Medical Center, Jerusalem, Israel
²The IDF Medical Corps, Ramat Gan, Israel
³Faculty of Medicine, Hebrew University, Jerusalem, Israel
⁴Pediatric Hematology/Oncology Unit, Shaare Zedek Medical Center, Jerusalem, Israel

Alexander Röth, MD¹, Ulrich Jaeger, MD², Bernd Jilma³, Wilma Barcellini^4*, Anita Hill^5*, Quentin Hill^5*, David J. Kuter, MD⁶, Catherine M. Broome^7*, Marc Michel^8*, Sacha Sergio Zeerleder, M.D.^9*, Yasutaka Ueda^10*, Jaime Morales-Arias¹¹, Jun Su¹¹ and Sigbjørn Berentsen^12*

¹Department of Hematology, University Hospital Essen, University of Duisburg-Essen, Essen, Germany
²Department of Internal Medicine I, Medical University of Vienna, Vienna, AA, Austria
³Department of Clinical Pharmacology, Medical University of Vienna, Vienna, Austria
⁴Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
⁵Department of Haematology, St James’ University Hospital, Leeds, United Kingdom
⁶Hematology Division, Massachusetts General Hospital, Boston, MA
⁷Division of Hematology, MedStar Georgetown University Hospital, Washington, D.C.
⁸Henri-Mondor University Hospital, Assistance Publique-Hôpitaux de Paris, UPEC, Paris, France
⁹Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland
¹⁰Department of Hematology and Oncology, Osaka University Graduate School of Medicine, Osaka, Japan
¹¹Sanofi, Cambridge, MA
¹²Department of Research and Innovation, Haugesund Hospital, Haugesund, Norway

Zachary French, MD^1*, Victor Bilan, MD^2*, Holleh Husseinzadeh, MD², Douglass A. Drelich, MD² and Ruben Rhoades, MD²

¹Department of Medicine, Thomas Jefferson University, Philadelphia, PA
²Cardeza Foundation for Hematologic Research, Thomas Jefferson University, Philadelphia, PA

Diane J. Nugent, MD^1*, Jamie O'Hara, MSc^2,3*, Antony Martin, PhD^3*, George Morgan^3*, Tyler W. Buckner, MD, MSc⁴, Mark W Skinner, JD^5,6*, Brian O'Mahony^7,8*, Brendan Mulhern^9*, Eileen K Sawyer, PhD^10* and Nanxin Li, PhD¹⁰

¹Department of Hematology, Children's Hosp. of Orange County, Orange, CA
²University of Chester, Chester, United Kingdom
³HCD Economics, Daresbury, United Kingdom
⁴Divisions of Hematology and Pediatric Hematology/Oncology, Hemophilia and Thrombosis Center, University of Colorado School of Medicine, Aurora, CO
⁵Institute for Policy Advancement Ltd, Washington, DC
⁶Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Canada
⁷Irish Haemophilia Society, Dublin, Ireland
⁸Trinity College Dublin, Dublin, Ireland
⁹University of Technology Sydney, Sydney, NSW, Australia
¹⁰uniQure Inc., Lexington, MA

Ashwin Gupta¹, Jonathan R Day, MD, PharmD^2*, Michael B. Streiff, MD³, Clifford Takemoto, MD^4,5*, Kyungsuk Jung, MD, MPH^6*, Calvin Abro, MD^6*, Eric Gehrie, MD^7*, Evan Bloch, MD, MS^7*, Aaron Tobian, MD, PhD^7* and Ruchika Goel, MD, MPH^6,7

¹Vanderbilt University, Nashville, TN
²Department of Internal Medicine, University of Iowa Carver College of Medicine, Iowa City, IA
³Division of Hematology, Department of Medicine & Pathology, Johns Hopkins University School of Medicine, Baltimore, MD
⁴Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
⁵Division of Hematology, Department of Pediatrics, The Johns Hopkins University, Baltimore, MD
⁶Division of Hematology/Oncology, Simmons Cancer Institute, SIU School of Medicine, Springfield, IL
⁷Division of Transfusion Medicine, Department of Pathology, Johns Hopkins University, Baltimore, MD

Tom Burke^1*, Sohaib Asghar, MSc^1*, Natalia Misciattelli^2*, Sharmila Kar^2*, George Morgan^1*, Harpal Dhillon^1* and Jamie O'Hara, MSc^1,3*

¹HCD Economics, Daresbury, United Kingdom
²Freeline Therapeutics, London, United Kingdom
³University of Chester, Chester, United Kingdom

Olga A. Gavrilina, MD, PhD^1*, Kristina Zakurdaeva^2*, Vladimir I. Vorobyev, MD, PhD³, Anastasia N. Vasileva^4*, Lev S. Butaev^5*, Alena Sukhareva^6*, Lubov Gavrilova^7*, Olga Samoylova, MD^8*, Maria Grishunina, MD^9*, Ochsana Ochirova^10*, Alina Karpova^11*, Olga Panteleeva^12*, Mikhail A. Kunst^13*, Vasily Shuvaev, MD^14,15, Anzhelika Rakhmani^16*, Tatiana V. Gaponova, MD, PhD^4*, Gennadiy M. Galstyan, MD, PhD^4*, Vera V. Troitskaya, MD, PhD^1*, Anton V. Luchkin, MD^4*, Elena N. Parovichnikova, MD, PhD^1* and Valery Savchenko, MD, PhD, Academician⁴

¹National Research Center for Hematology, Moscow, Russian Federation, Moscow, Russian Federation
²RakFond, Moscow, Russia
³Hematology Department, S.P.Botkin City Hospital, Moscow, Russia
⁴National Research Center for Hematology, Moscow, Russian Federation
⁵Hematology Department, S.P.Botkin City Hospital, Moscow, Russian Federation
⁶S.P. Botkin City Hospital, Moscow, Russian Federation
⁷State Budgetary Healthcare Institution of the Republic of Mordovia "Republican Clinical Hospital No4" of the Ministry of Health of the Russian Federation, Mordovia, Russian Federation
⁸Regional Clinical Hospital N.A. Semashko, Nizhniy Novgorod, Russian Federation
⁹Regional Hospital, Nigniy Novgorod, Russian Federation
¹⁰Republican Clinical Hospital named after NA Semashko of the city of Ulan-Ude, Ulan-Ude, Russian Federation
¹¹NA Semashko Rpublican Clinical Hospital, Ulan-Ude, Russian Federation
¹²Regional Clinical Hospital, Vladimir, Russian Federation
¹³Republican Clinical Hospital of Tatarstan, Kazan, Russian Federation
¹⁴City Clinical Hospital n.a. V.V. Veresaev of the Moscow Department of Healthcare, Moscow, Russian Federation
¹⁵Federal State Budget Institution “Russian Scientific-Research Institute of Hematology and Transfusiology of Federal Medical-Biological Agency”, St. Petersburg, Russia
¹⁶City Clinical Hospital n.a. V.V. Veresaev, Moscow, Russian Federation

Arielle L Langer, MD, MPH¹, Adam Binder, MD^2* and Eileen Scigliano, MD³

¹Hematology Division, Brigham and Women's Hospital, Boston, MA
²Division of Hematologic Malignancies and Hematopoietic Stem Cell Transplantation, Sidney Kimmel Cancer Center, Thomas Jefferson University, Philadelphia, PA
³Department of Environmental Medicine and Public Health, Icahn School of Medicine at Mount Sinai, New York, NY

Amanda Wilson^1*, Ahmed Daak² and Jun Su³

¹Sanofi, Cambridge, MA
²Clinical Development, Sanofi, Waltham, MA
³Medical Affairs, Sanofi, Cambridge, MA

Ian J. Hooley, BS^1*, Kathleen Maignan, NP^1,2*, Alexandra Jacob^1*, Angelica Medina, MSRA^1*, Lauren Benderoff^1*, Robert Chen, PhD¹ and Scott F. Huntington, MD, MPH³

¹Flatiron Health Inc, New York, NY
²Weill Cornell Medicine, New York, NY
³Yale Cancer Center, Yale University School of Medicine, New Haven, CT