Effective, safe, and as fast as reasonably possible


For readers who are interested in the processes and the speed with which potential new cancer drugs can be brought to market here in the USA (and other places around the world too), there in an important article in this week’s New England Journal of Medicine.

The article by Prowell et al. is entitled “Seamless oncology-drug development” and the three authors are all members of the staff of the Office of Hematology and Oncology Products at the U.S. Food and Drug Administration (including its director, Dr. Richard Pazdur). It addresses the complex questions that are faced by researchers, clinicians, patients, and regulators in finding an acceptable balance between getting effective and safe new drugs to market as fast as can possibly be done and ensuring that we do not approve such new drugs until we have accumulated enough information to have a strong belief that the overall benefits of this drug are greater than the overall risks associated with the use of this drug in one or more well defined groups of patients.

The so-called “breakthrough therapy” designation has been an absolute game-changer for the ways in which promising new cancer drugs can be “fast-tracked” through the regulatory approval process. Dr. Pazdur and his colleagues at the FDA have been among the most strenuous advocates for the application of this process when it is appropriate. But retaining the fine balance between efficacy, safety, and speed to market is hard.

Even the most skeptical readers need to appreciate that the Office of Hematology and Oncology Products at the FDA is highly committed to bringing valuable new cancer drugs to market as fast as they possibly can. But they practice this commitment with a very distinct point of view (one with which your sitemaster is in complete agreement):

Even as we strive to provide earlier access to highly effective anticancer agents, we cannot abandon our commitment to well-designed, well-conducted clinical trials. Such studies are the only way to obtain the high-quality efficacy and safety data that will enable clinicians to counsel patients about a drug’s risks and benefits, permit patients to make informed choices about their treatment, and ultimately facilitate widespread global access to highly effective new anticancer agents through regulatory approval and reimbursement.

Even though the FDA has no direct involvement in how drug therapy gets paid for here in America, approval of a drug for a specific indication is commonly a critical factor in ensuring that Medicare and other payors will actually cover some or all of the costs associated with the use of that drug for a specific indication. Dr. Pazdur and his colleagues are very well aware of this.

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