Horizon

Jeff Sherman

Jeffrey (Jeff) Sherman, MD, FACP, is a seasoned pharmaceutical executive with over 35 years of experience in regulatory and clinical strategy, as well as therapeutic development for rare diseases. He served as Executive Vice President and Chief Medical Officer (CMO) at Horizon Therapeutics from 2009 to 2023, overseeing innovative treatments for rare, autoimmune, and severe inflammatory diseases. His leadership contributed to Horizon’s growth into a global biotechnology company, now part of Amgen. Prior to Horizon, Dr. Sherman held senior roles at IDM Pharma, Takeda, NeoPharm, Searle/Pharmacia, and Squibb/Bristol-Myers Squibb, including securing European approval for MEPACT (mifamurtide) to treat osteosarcoma.

Dr. Sherman is actively involved in professional organizations, including the Center for Healthcare Innovation, CISCRP, and the Drug Information Association, where he is an inaugural fellow. He also serves on the boards of Xeris Biopharma, Larimar Therapeutics, and Sorriso Pharmaceuticals. An adjunct faculty member at Northwestern University’s Feinberg School of Medicine, Dr. Sherman advises programs at Stanford University and holds certifications from the National Board of Medical Examiners and the American Board of Internal Medicine. He earned his medical degree from the Chicago Medical School and completed advanced training at Northwestern University and the University of California, San Francisco.

Advancing Care and Research

There is need for a further Call to Action for Rare Diseases which includes genetic diseases and types of cancer. A rare disease in the U.S. affects fewer than 200,000 people. By all accounts, there are over 7,000 rare diseases and the number is growing with technological advances with over 25-30 million people in the U.S. living with a rare disease which is close to 1 in 10 individuals. Worldwide, it is estimated that 350 million people have a rare disease. Many of these rare diseases result in the premature death of infants or can be fatal in early childhood and carry significant morbidity for those who survive childhood. Most concerning is that more than 90% of rare diseases are still without an approved treatment and available treatments may have shortcomings.

More and better treatments are needed as well as further research to address these devastating diseases which have far-ranging impacts beyond the individuals affected. Rare disease may be difficult to diagnose and manage particularly if signs and symptoms of the disease are challenging or the healthcare provider may not have the knowledge or experience with rare diseases. This can be very stressful on the individual and their loved ones having unanswered questions and concerns about health and wellbeing.

In more personal terms, an individual with a devastating rare disease would trade for an unaffected person’s worse day. Activities of daily living, such as getting out of bed in the morning, going to the bathroom, and eating can be extremely taxing and not taken for granted. Each day that passes without a diagnosis and treatment, if available, can mean the progression of the disease which may be irreversible. Hence, there is a very real sense of urgency to address this growing unmet medical need. Furthermore, these diseases do not know or adhere to socioeconomic, gender, race and other boundaries.

Individuals may or may not be aware of resources available to them through the Undiagnosed Disease Network, funded by the National Institutes of Health (NIH) Common Fund, the National Organization for Rare Disorders (NORD), Global Genes, and many other organizations. An important part of the diagnostic work-up of many of these diseases is medical genetic testing and resources through various third parties may be available to cover the costs.

There is an opportunity to come together on Rare Disease Day, the last day of February, which is considered the rarest day on the calendar, to increase awareness and generate additional commitment to addressing Rare Diseases.