For people who are seriously ill, every day counts. Yet until recently, the traditional process of developing and getting new medicines approved by the U.S. Food and Drug Administration (FDA) could take too long for people with serious or life-threatening diseases. I was fortunate to have a front-row seat watching researchers and regulators collaborate to develop a speedier regulatory review and approval pathway in 2012: the “Breakthrough Therapy Designation” or BTD, which has brought new, effective medicines to patients who need them months or years faster than before.

This new pathway was the latest in several reforms to the FDA’s regulatory process. Since the 1980s, patient advocates and health care providers had pushed the agency to become more efficient. At that time, it often took the FDA three years or more to review a drug license application. By 1992, the intense need to treat patients during the AIDS epidemic galvanized the FDA and Congress to pass the first Prescription Drug User Fee Act (PDUFA) - legislation that would provide more resources to help the FDA expedite their review and approval of new medicines. PDUFA, combined with other legislation, created a few fast-tracked paths to prioritize medicines that treat serious conditions or unmet medical needs.

But by 2011, even those pathways seemed too slow. Progress in research was accelerating and the landscape of drug development was fundamentally changing with a greater understanding of the underlying biology of disease. More new medicines were showing potential in earlier studies and demonstrating significant effects in targeting disease. That year, Dr. Ellen Sigal, who founded a group called Friends of Cancer Research after her sister died of breast cancer, held an important panel with scientists and regulators to consider how approval for those medicines could happen faster.

At the Friends of Cancer Research panel, one question before the scientists was basically whether it was possible, using data, to identify molecules that showed a great benefit early on and tag them for quicker review and potential approval. Sitting in the front of the room, I watched as Dr. Gracie Lieberman, then a statistician at Genentech, participated as a key member of the panel and presented information about the development path of recently approved medicines, questioning whether the traditional development pathways were still applicable for medicines with significant treatment effects. She concluded that if a medicine, say, shrank a tumor by 80 percent in early clinical studies, it could not be a random event, but rather the medicine could be transforming, a “breakthrough.”

It was clear to her, and to the rest of us, that if a new medicine for a serious illness showed a dramatic effect, it should be an all-hands-on-deck situation, with the FDA and the company collaborating to speed up the development and evaluation of the safety and efficacy of the medicine to get it to patients as soon as possible. Fortunately, PDUFA was designed to evolve with progress in research, allowing regulations to keep pace with science. Dr. Richard Pazdur, acting director of the FDA’s Oncology Center of Excellence, along with other FDA leaders, have been major advocates for patients and were key champions in working with Congress to move the legislation forward. With the support of their leadership, the FDA agreed that medicines that show strong early results should have faster trials and approval processes. Ellen and her collaborators were models of how fast approval can work: her group, along with the FDA, lawmakers, and companies, moved the idea of a “breakthrough” designation from innovative concept to proposal and into law in just over a year. On July 9, 2012, President Barack Obama signed into law the Food and Drug Administration Safety and Innovation Act (FDASIA), which included the new BTD for medicines for serious diseases that show early, important effects compared to other existing therapies.

With this new review pathway, the FDA provides more resources and access to senior officials for collaboration with developers throughout the clinical trial process, with the goal of expediting the review and potential approval of a medicine that shows dramatic, early promise for treating people with serious or life-threatening diseases. That means more frequent meetings with a dedicated FDA senior management team and the ability to submit data to the FDA as they become available, which can speed up the process by making it more efficient. Since the BTD legislation was created, the FDA has received more than 360 requests for BTD, granted more than 120, and approved nearly 50 medicines with the designation. Today, BTD medicines are approved in an average of three and a half years from early stage studies, which is less than half of the seven to eight years it takes medicines that don’t have BTD.

Since we at Genentech had supported the BTD effort early on, it was very exciting when Genentech’s Gazyva^® (obinutuzumab) was the first medicine granted BTD to be approved. When we got the news, it was a momentous occasion, one that made our efforts – all the research, statistical analyses, and meetings – more than worthwhile. But beyond being one of those times when you want to hug all of your teammates who worked on it, it was a proud, yet humbling, moment to think about how we can now help get medicines to the people who need them sooner.

^{Title image: Genentech employees celebrate new medicine approvals by gathering and ringing bells. Here, team members mark the approval of Gazyva with the Breakthrough Therapy Designation.}