Selected quoted from the Director of FDA's CEDR division before congress July 29, 2021. Testimony of Patrizia Cavazzoni, M.D. Director - Center for Drug Evaluation and Research
We fully recognize the concerns that clinical trial participants share with us about the use of placebo-controlled studies, and understand their frustration with such designs. However, the track record for new drugs shows us that for every successful treatment, about 40 drugs failed — many of which were touted as highly promising.
A drug with a dramatic effect size might be adequately evaluated without a comparator, but to detect a more modest, yet very relevant benefit, only a controlled trial — comparing the candidate to placebo — can serve.
Although FDA has no involvement in the manufacturers’ or sponsors’ decisions to provide such access to eligible investigational drug products under expanded access, we do play a role in approving the requests. However, under the “Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017,” also known as the Right to Try Act, we generally do not have a role in access to investigational products, or approval of such requests, although we provide information for patients on our website.
A little over a year ago, CDER reorganized its Office of New Drugs (OND), and among other changes, we stood up an Office of Neuroscience. The reorganization of OND was the culmination of three years of effort. We undertook this reorganization to modernize OND in part to align interrelated disease areas and divisions with clearer and more focused areas of expertise. The Office of Neuroscience’s new structure allows the office to provide a more holistic approach to the regulation and review of neuroscience drugs, and to leverage expertise across the office so that all divisions within the office benefit from shared knowledge and ensure greater consistency in regulatory approaches.
We stand ready to use the expedited development and approval programs that are available to FDA in order to help bring new treatments to patients as quickly as possible. We recognize the impact these diseases have on patients and their loved ones, and share in the common goal of wanting to advance the development of treatments with a positive benefit-risk profile that can help patients.
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