ASH Clinical News Focus on Rare Diseases | Page 3

OCTOBER 2016 AMERICAN SOCIETY OF HEMATOLOGY HEADQUARTERS 2021 L Street NW, Suite 900 Washington, DC 20036 www.hematology.org Tel: [phone] EDITOR-IN-CHIEF Mikkael Sekeres, MD, MS Vice-Chair for Clinical Research Director, Leukemia Program Cleveland Clinic, Taussig Cancer Institute Cleveland, OH ASSOCIATE EDITORS Beth Faiman, CNP, PhD Cleveland Clinic Cleveland, OH Alice Ma, MD University of North Carolina School of Medicine Chapel Hill, NC CONTENTS David Steensma, MD Dana-Farber Cancer Institute Boston, MA Keith Stewart, MBChB, MBA Mayo Clinic Scottsdale, AZ PUBLISHER American Medical Communications EDITORIAL MANAGING EDITORS Ariel Jones-DeMaio, AMC Karen Learner, ASH ASSOCIATE MANAGING EDITOR Kerri Fitzgerald, AMC ART DIRECTOR Ari Mihos ASSISTANT ART DIRECTOR Charlene DePrizio DIGITAL PROJECTS MANAGER Chris Gedikli IN THE PIPELINE POLICY UPDATES A review of FDA orphan drug approvals and breakthrough therapy designations Recent changes from the Centers for Medicare & Medicaid Services in physician payment and coverage of services for patients with rare diseases 3 CLINICAL TRIAL UPDATES Ongoing clinical trials for rare diseases in bone marrow failure disease, clotting and bleeding disorders, leukemia, and myeloma and lymphoma 13 15 MEETING COVERAGE A collection of news from recent hematology/oncology meetings, including the 2016 ASH Meeting on Hematologic Malignancies. 18 ADVERTISING ACCOUNT MANAGER Nick Luciano [email protected] Gene Conselyea [email protected] Recruitment advertising orders can be sent to: DIRECTOR, RECRUITMENT CLASSIFIEDS Lauren Morgan [email protected] ©2016 by the American Society of Hematology. All materials contained in this newsletter are protected by copyright laws and may not be used, reproduced, or otherwise exploited in any manner without the express prior written permission of ASH Clinical News. Any third-party materials communicated to ASH Clinical News become its copyrighted property and may be used, reproduced, or otherwise exploited by ASH Clinical News. All correspondence for the American Society of Hematology should be sent to: American Society of Hematology, 2021 L Street NW, Suite 900, Washington DC 20036. Neither the American Society of Hematology nor the publisher is responsible for statements made by any editor or contributor. Statements, editorials, or opinions expressed in ASH Clinical News magazine do not necessarily represent official policy of the American Society of Hematology unless so stated. No responsibility is assumed by the American Society of Hematology or the Publisher for any injury or damage to persons or property as a matter of product liability, negligence or otherwise or from any use or operation of any methods, products, instructions or ideas contained in the material herein.  Although all advertising material published in ASH Clinical News magazine is expected to conform to ethical (medical) standards, inclusion in this publication does not constitute a guarantee or endorsement by the American Society of Hematology or the Publisher of the quality or value of such product or of the claim made of it by its manufacturer. About the American Society of Hematology The American Society of Hematology (ASH) is the world’s largest professional society concerned with the causes and treatments of blood disorders. The mission of the Society is to further the understanding, diagnosis, treatment, and prevention of disorders affecting the blood, bone marrow, and the immunologic, hemostatic and vascular systems, by promoting research, clinical care, education, training, and advocacy in hematology.