The Hematologist

September-October 2015, Volume 12, Issue 5

Making Sense of New Drug Discovery

David A. Williams, MD Chief of Hematology/Oncology; Director of Clinical and Translational Research
Boston Children's Hospital, Boston, MA

Published on: August 10, 2015

The 2016 ASH annual meeting will mark 40 years that I have been attending ASH meetings. As a medical student at Indiana University, I presented at the 1976 ASH Annual Meeting, where I also first met one of my lifelong mentors, Dr. David Nathan. I was very impressed with the combination of science and clinical topics. Despite this long history of attendance, I was amazed at the myriad of exciting new drugs moving into the benign and malignant hematology space at the 2014 meeting in San Francisco. What explains this impressive new cadre of medicines we can offer our patients? Research! This is exemplified by this year’s Beutler Lecture and Prize which recognizes the basic research of proteasome biology and the derivative development of proteasome inhibitors in blood diseases. For decades, our field has led medicine in using scientific discoveries to better understand disease pathobiology and to develop new treatments based on this knowledge.

ASH has developed numerous resources to help clinicians and patients stay abreast of this fast-moving landscape. In May 2014, the Society, in collaboration with the U.S. Food and Drug Administration (FDA), launched the “ASH/FDA Updates” series with a webinar that focused on ibrutinib as a treatment for chronic lymphocytic lymphoma. ASH now regularly hosts educational webinars that feature presentations by experts in the field. These key opinion leaders cover current information on how best to diagnose and care for patients, and the webinar format provides an opportunity for speakers and participants to discuss relevant issues during a question-and-answer session. ASH CME webinars are offered live (learn more at, and recordings of the first seven webinars are archived and available free of charge at

At the 2014 ASH Annual Meeting, the Society introduced an innovative educational session focused on recently approved drugs used in hematology, moderated by Dr. Mikkael Sekeres from the Cleveland Clinic. The speakers discussed a selection of hematology drugs newly approved in 2014 and focused on issues clinicians face in treating patients with the new drugs, such as appropriate populations, dosing, side effects, drug/drug interactions, and off-label use. A recording of this presentation is also available at ASH will hold another Special Education Session on Newly Approved Drugs at the 2015 ASH Annual Meeting that will have a practical physician-to-physician focus.

ASH has additional resources on its website, including an overview of patient access programs for specific drugs, as well as templates for letters of appeal to insurance providers justifying patient coverage for the use of specialty tier drugs. And of course, Consult-a-Colleague is one of ASH’s longstanding member benefits that helps facilitate consultation on clinical cases between hematologists and their peers on a wide range of hematologic conditions.

Importantly, ASH continues to advocate for increased NIH funding of research into all diseases but also advocates for legislation that can enable patients to access high-cost hematology drugs. In May of this year, members of ASH’s Committee on Practice visited nearly 40 congressional offices to educate and urge elected officials to support the Cancer Drug Parity Act. This legislation requires private health plans that provide coverage for traditional chemotherapy treatment delivered parenterally to also provide coverage for orally administered and self-injectable anticancer medications at a cost to the patient that is comparable to that of institutional parenteral treatments (i.e., office visit co-pay). ASH also supports the Patients’ Access to Treatments Act. The bill proposes to prevent private health insurance plans from imposing higher co-insurance for medications in the specialty drug tier (Tier IV) and represents an important first step toward removing the burden of excessive cost-sharing for Americans who need high-cost, specialty-tier medications.

The pace of drug discovery in our field is extraordinary and offers new hopes for our patients. I hope that you will continue to think of ASH as a primary information resource as these new drugs are approved. In looking back on my early experience with the ASH annual meeting, I also hope you will provide an opportunity for your trainees to attend this year’s meeting in Orlando. It could change their lives – it did mine!

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