May 15th, 2013 - This year at ASCO hundreds of presentations will feature promising investigational medicines (those being studied by researchers but not yet approved by the FDA). Understandably, after presenting new data at a meeting like ASCO, we see an increase in requests from people asking about how they can be treated with unapproved medicines.
It’s a great responsibility, and one we as a company – and I personally – need to get right.
Whether or not we provide access to an investigational medicine is a complex decision, made in collaboration with doctors and health authorities (like the FDA), and with many factors to be weighed.
In my role as Chief Medical Officer, I’m often the one who makes the final decision on such requests.
Sometimes the Answer Isn’t Obvious
As a doctor, my first sentiment is to do everything in my power to help the sick person in front of me. But I have to resist acting on instinct alone. Instead, our ethics compel us to make consistent decisions in the best interest of the greatest number of patients.
"We believe clinical trials are the best way for patients to access unapproved medicines. But it’s imperative to strike a balance. We need to avoid any interference with FDA-mandated clinical trial and approval processes, while maintaining responsible and ethical access to unapproved treatments." - Brandon Hayes-Lattin, MD, Senior Medical Advisor, LIVESTRONG Foundation.
Sometimes, the answer isn’t obvious. To help guide our actions in these situations, we have developed a clear policy about access to investigational medicines. You can read more about it here. The policy was written with the help of patient advocacy groups and is designed to adhere to FDA guidelines.
Our priority is to help the greatest number of patients possible by ensuring our medicines receive approval around the world, as only approval by health authorities can ensure the broadest, most appropriate access. This means that we need to ensure that our decisions do not interfere with the path a medicine must take to approval in the United States, Germany, Japan or any other country, to avoid the risk of a medicine not being approved at all.
What We are Able to Do, and When We’re Able to Do It
Providing access to an unapproved medicine outside of a clinical trial can actually prevent us from truly answering the question of whether a medicine helps patients.
We focus on enrolling appropriate patients into clinical trials, so we can collect the evidence needed to answer questions about the potential risks and benefits of a new medicine. There are far too many examples of medicines that have had promising results in early, smaller studies, but those results failed to replicate in larger studies.
In certain circumstances, we’ll open what is called an Expanded Access Program (EAP). An EAP is a study designed to provide a group of people with a medicine prior to its approval. To be able to open an EAP, certain conditions must be met both by participating patients and the study medicine. Importantly, we must be reasonably confident that the medicine will be approved based on the data we’ve already collected and that the potential benefits outweigh the potential risks.
The most difficult situations, which always affect me deeply, are requests from people who have exhausted all of the available options, including clinical trials or EAPs. In such circumstances, we may be able allow “compassionate use.” Compassionate use allows us to provide an investigational medicine to an individual on humanitarian grounds. We can do this if, and only if, doctors at Genentech, the patient’s doctor and an ethics committee at the patient’s treating hospital (called an Institutional Review Board) all approve the use of the investigational medicine.
As we learn more about treating cancer, and increase our use of personalized medicines (those specifically designed to work only in patients with specific forms of a disease) or combinations of medicines, we expect these decisions to become even more complex.
When faced with these situations, a clear policy helps us pressure test every decision to make sure we are doing the right thing in each individual case. I feel humbled when I hear from patients who have been helped as a result of compassionate use, but I am also mindful that there is always a risk that our medicine could harm someone.
The best way for us to make new cancer medicines widely available is to get our new drugs approved quickly with more efficient clinical trials. But when there is doubt, I need to be confident in both the “yes” and the “no” decisions.
As an industry, we will see requests for access to investigational medicines increase as patients rightfully take a more active role in their treatments. Also, as we learn more about cancer, and present exciting data on tomorrow’s breakthrough treatments, patients around the world will listen with hope that these treatments may help. I want those affected to know that there is reason to be hopeful, and we care deeply about making the right decisions. Those of us at Genentech, along with our colleagues from ASCO and other companies, have devoted our lives to turning scientific advancements into new treatments.
Dr. Barron is the Executive Vice President, Head of Global Product Development and Chief Medical Officer, responsible for the products of the combined portfolio of Roche and Genentech. Hal is a member of the Genentech Executive Committee (GEC), the Pharma Medicines Leadership Team (MLT), and co-chair of the late stage portfolio committee (LSPC).
Barron joined Genentech in 1996 as a clinical scientist. During the next several years, he held positions of increasing responsibility and leadership within Cardiovascular Research and Specialty Therapeutics. In 2002 Barron was promoted to vice president, Medical Affairs. In 2003 he became the senior vice president of Development and in 2004 he was appointed chief medical officer. In 2009 he was appointed executive vice president, Head of Product Development: Oncology, Inflammation/Tissue Growth Repair and Virology.
Prior to joining Genentech, Barron received his Bachelor of Science in physics from Washington University in St. Louis, his Medical Degree from Yale University and completed his training in medicine and cardiology at the University of California San Francisco.
Barron’s academic positions include Associate Adjunct Professor of Epidemiology and Biostatistics and Associate Clinical Professor of Medicine/Cardiology at the University of California, San Francisco. He has been issued several patents for his work in thrombosis and angiogenesis and has published more than 90 papers in peer-reviewed scientific journals. He is also a member of the board of directors of Alexza Pharmaceuticals, Inc.