The FDA Approval Process: A Primer

Peter Martin, M.D.

By Peter Martin, M.D.

In addition to providing the best possible care for people with lymphoma, one of our primary goals in the Weill Cornell Lymphoma Program is to help develop better treatments. To keep track of these approvals we have created the Lymphoma Therapy Approval Timeline page on our blog. The timelines lists all of the new Breakthrough Designations, and approvals generated by the FDA for therapies related to the treatment of lymphoma. More in-depth information about these approvals can be found  our blog.

So far, 2016 has been an eventful year, with six drugs receiving an updated approval status or Breakthrough Therapy Designation for the treatment of lymphoma. At the same time, recent circumstances have led to the closure of at least seven clinical trials evaluating the role of idelalisib. In the face of all this activity, it is clear that we are keeping the FDA busy. People in my clinic ask me almost every day, “Why does it take the FDA so long to approve promising new drugs?” To help answer this question I will write a series of blog posts summarizing the drug development process. An in-depth explanation of the process can be found on the FDA website.

Today, in the first post of the series, I thought it would be interesting to address whether the FDA is a bureaucratic wall standing between patients and potentially lifesaving cancer therapy, whether the FDA has become too lax in its approval process, resulting in approval of dangerous drugs or labels that are too broad, or whether it gets the balance just right.

In 2014 Yale University researchers published a study that sought to characterize how new treatments were approved by the FDA between 2005 and 2012. Of the 188 new treatments included in the study the largest group were cancer treatments at 41 (19.9%). Compared to other indications, cancer treatments were more frequently approved through less time consuming non-randomized, non-blinded studies, and accelerated approval programs. Instead of using standard clinical outcomes, nearly all of these trials measured surrogate end points like reduced tumor size or decreased biomarker levels. These endpoints can be reached in shorter periods of time than clinical outcomes like overall survival. The study authors noted that while treatments for other indications generally relied on 3 trials, “Most therapeutic agents approved for cancer indications were approved on the basis of a single trial.”

Some of these recent approvals likely reflect that fact that we are in the midst of a scientific revolution, with an explosion of new drugs that are clearly superior to older therapies becoming available at an unprecedented rate. But it is also clear that the FDA is aware that people with cancer face unique circumstances and are willing to move forward with new drugs at a faster rate. If you have any specific questions or experiences that you’d like us to cover in this series of posts, let me know and I’ll do my best to address them. Stay tuned!

Author: lymphomaprogram

Located on the Upper East Side of New York City, the Lymphoma Program at Weill Cornell Medical College/NewYork Presbyterian Hospital is internationally recognized for our efforts to enable patients with non-Hodgkin lymphoma, Hodgkin disease and related disorders to have the best possible clinical outcome, including cure when possible.

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