By: Robert Justice, M.D., M.S.
The growth in drug development has begun to bear fruit, particularly for the treatment of breast cancer. As Commissioner Hamburg pointed out in her blog earlier this month, 40 percent of all drug development is in cancer research, and a significant part of that work is focused on breast cancer. And we’ve learned a lot about breast cancer in the last decade, including that one therapy does not treat all. Despite many advances in treatment, the need for new and effective therapies remains.
Accelerating development of effective new drugs for breast cancer patients has been a top FDA priority for some time. In recent months, FDA has been examining the use of novel anti-cancer therapies prior to surgery. This is known as neoadjuvant therapy, which we believe has the potential to advance drug therapy for breast cancer even further, including the possibility of converting an inoperable breast cancer tumor into an operable one. Preoperative therapy could also make it possible to identify—early in treatment—tumors that are resistant to chemotherapy, enabling doctors to discontinue ineffective therapy and better assess the likely course and outcome of the disease.
We’ve come a long way… but there is still a ways to go
To continue to develop these new preoperative therapies, we need new and innovative ways to test their effectiveness and safety. To evaluate this innovative approach, a new endpoint is needed — an outcome from a clinical trial that FDA will accept as evidence that the drug will be effective. This means some new ways of thinking about what kind of information from clinical studies constitutes enough evidence to consider a drug both safe and effective.
FDA has shared its thoughts and views with the pharmaceutical industry on this subject via a document called a draft guidance, which discusses endpoints for clinical trials that may be considered when studying new drugs intended for neoadjuvant therapy.
In the years ahead, patients may have access to entirely new classes of drugs specifically designed to treat their breast cancer while sparing them some of the often harsh side effects of traditional anti-cancer therapies. For our part, we are continuing to learn all we can about the newest therapies being developed. We are also working with both academic researchers and drug makers on innovative clinical trial designs so that safe and effective drugs can reach the public as soon as possible.
Robert Justice, M.D., M.S., is the director of the Division of Oncology Products 1 in the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research