Horizon
Navigating Biotech And Patient Advocacy
Jeff Sherman
With over three decades of pharmaceutical experience, Jeff Sherman specializes in clinical and regulatory strategy and therapeutic development for rare diseases, including TEPEZZA (teprotumumabtrbw), for Thyroid Eye Disease (TED). Dr. Sherman served as Executive Vice President and Chief Medical Officer (CMO) at Horizon Therapeutics from 2009 to 2023. Previously, he was the Senior Vice President of Research and Development and CMO at IDM Pharma, leading the team that secured European Commission approval for MEPACT (mifamurtide) for osteosarcoma. He has also held leadership roles at Takeda Global Research & Development, NeoPharm, Searle/Pharmacia and Squibb/Bristol-Myers Squibb.
Dr. Sherman is actively involved in industry and academic initiatives. He serves on the boards of Xeris Biopharma, Larimar Therapeutics, and Sorriso Pharmaceuticals, as well as a board member of the Center for Healthcare Innovation (CHI) and the Center for Information and Study on Clinical Research Participation (CISCRP). A former FDA-sponsoredClinical Trial Transformation Initiative (CTTI) steering committee member, he is also an inaugural fellow and past board chair of the Drug Information Association (DIA). His collaborations extended to organizations such as Global Genes, the National Organization for Rare Diseases (NORD) and the European Organization for Rare Diseases (EURORDIS). In academia, he was an Adjunct Assistant Professor at Northwestern University Feinberg School of Medicine and advises Stanford University’s Master in Translational Research and Applied Medicine (MTRAM) program. He is a diplomat of the National Board of Medical Examiners and the American Board of Internal Medicine, demonstrating his commitment to advancing medical research and patient care.
Every Day Of Delay In Drug Approval Is A Day That Someone Unduly Suffers
Recognizing Dr. Sherman’s extensive experience in rare disease therapeutic development and patient advocacy, this exclusive interview offers invaluable insights into the challenges of advancing drug approval processes and strategies for improving patient care in the biopharmaceutical industry for rare diseases
Professional Journey And Current Role
I trained as a physician, completing an internal medicine internship, residency and chief medical residency at Northwestern University in Chicago, followed by an infectious diseases fellowship at the University of California, San Francisco (UCSF). I was a research associate at the Howard Hughes Medical Institute at UCSF in allergy and immunology, which fostered my long-standing interest in immunological diseases.
I began my career in clinical pharmacology at Squibb which later became Bristol Myers Squibb, transitioning into clinical research. From there, I moved to SearleMonsanto (which later became Pharmacia, now part of Pfizer), where I held various clinical research roles and medical affairs. My experience spans both large pharmaceutical companies like Takeda and smaller biotech companies such IDM Pharma and what was then Horizon Therapeutics.
In 2009, I joined Horizon Therapeutics when it was privately held and had no products on the market. Over the years, I played a role in its transformation into a successful biopharmaceutical company focused on rare diseases, culminating in its acquisition by Amgen in 2023. My role as Chief Medical Officer evolved over the years as Horizon grew. I also represented Horizon on the board of the Biotechnology Innovation Organization (BIO) trade organization and was deeply involved in patient advocacy and patient support initiatives. Much of Horizon’s focus—and now part of Amgen’s — is on rare diseases that affect no more than 200,000 individuals. These are severe conditions in which patients endure severe challenges, often struggling with routine activities such as getting up in the morning, brushing their teeth, and eating.
Every Day Of Delay In Drug Approval Is A Day That Someone Unduly Suffers
A significant focus is on thyroid eye disease (TED) and TEPEZZA (teprotumumab-tbrw), the first and only FDA-approved treatment for TED, a potentially sightthreatening condition associated with Graves’ disease of the thyroid. Thyroid diseases often go undiagnosed.
The integration of Horizon’s products into Amgen’s more extensive portfolio is a delicate process, mainly when dealing with rare diseases that impact vulnerable patient populations.
The advisory position has allowed me to continue having an impact on patient care.
Challenges Faced As A Chief Medical Officer
The challenges of being a chief medical officer can vary depending on a biopharmaceutical company’s size and financial state. Whether a company is focused on developing new products or managing those already in the marketplace and whether it is burning cash or generating cash impacts its strategic priorities.
For companies still burning cash, meaning they have products in development and no revenue stream, one of the biggest challenges is ensuring financial sustainability and securing funding to see projects through.
In addition to this, they must navigate complex clinical, regulatory and market access pathways for success. Statistics show that one out of five products, or sometimes as few as one in ten that enter the clinic, gain approval at the end of the day, meaning the majority don’t make it to the market. Despite significant advances, many products still fail to make it, and for companies without strong financial reserves, a single setback can be devastating, potentially forcing them out of business. The industry remains high-risk and uncertain, requiring careful risk management throughout development.
Success depends on navigating the regulatory approval process and then working through pricing and reimbursement. It’s a constant balancing act, where you must focus on advancing new drugs and innovations while ensuring the company’s financial health and sustainability and navigating the complexities of the regulatory environment.
Advice For Upcoming Professionals In The Industry
My key advice for anyone who is a chief medical officer or aspiring to be one is to be flexible. Never say never, and don’t lose sight of what truly matters— the patient. Any one of us could find ourselves in their position, and it’s essential to remember that patient care extends beyond the individual—it includes their families, friends, caregivers, and the medical and scientific community that supports them. A Chief Medical Officer also plays a crucial role in serving the broader communities they impact. The ultimate goal is to develop new treatments and improve healthcare for everyone. When we elevate healthcare standards, we all benefit.
One of the not-for-profits I’m involved with, the Center for Healthcare Innovation (CHI), is dedicated to addressing unmet needs. Indeed, the COVID pandemic highlighted some challenges, and we don’t want to repeat some shortcomings. As an investigator once said, “The enemy of good is perfect—plan for the worst, hope for the best.” Keeping an open mind, focusing on what’s essential and ensuring that new medicines reach patients as quickly as possible is critical. Every day of delay in drug approval is a day that someone unduly suffers. Health care, in general, and the role of a chief medical officer carries immense responsibility in making a meaningful impact.
