AACR urges guidance on co-development process
This summer, the U.S. Food and Drug Administration (FDA) approved two new medicines for cancer: crizotinib (Xalkori) and vemurafenib (Zelboraf). These two medicines are examples of the shifting paradigm of drug development in which therapies are rationally designed to target the molecular defects found in particular cancer cells.
The foundation of personalized medicine lies in being able to identify these defects and detect them in individual patients. To that end, both Zelboraf, approved for the treatment of metastatic melanoma expressing a particular mutation, BRAFV600, and Xalkori, approved for the treatment of late-stage non-small cell lung cancers expressing an abnormal ALK protein, were approved with a companion diagnostic. The diagnostic tools are tests that detect whether a patient’s cancer has the defects that make it likely to respond to these treatments. The tests were approved by the FDA on the same day as the drug.
This is a glimpse of the future, but there are many considerations for a companion diagnostic to be developed alongside a therapeutic. In a long-awaited draft guidance released in July, FDA clarified its policy with respect to companion diagnostics and their corresponding therapies. The guidance defines a companion diagnostic and states that under most circumstances FDA will require that the diagnostic be approved at the same time as the therapeutic, if a diagnostic is required for the safe and effective use of a therapeutic.
In formal comments to FDA, the AACR expressed its general support for the draft guidance as a welcome step forward in addressing the need for transparency and consistency in how FDA approaches this burgeoning scientific area. The AACR comments sought clarity about FDA’s requirements with respect to those tests currently used in standard-of-care cancer treatments that may now fit the definition of companion diagnostics according to the draft guidance but that are not FDA approved or cleared. This point is relevant when testing experimental therapies in clinical trials where such a standard-of-care test is used with a therapy either in the control arm or in combination with the agent being tested.
The draft guidance implies that the therapeutic and diagnostic will have to be co-developed, but does not discuss the process by which this will occur. The regulatory uncertainty surrounding co-development is widely cited as a primary disincentive for industry to pursue such therapies and companion diagnostics. In its comments, the AACR strongly urged FDA to expedite the development of guidance on the process of co-development.
In recent reports on regulatory science and innovation, FDA has described its intention to enhance its capacity to deal with new medical products that result from advances in science and technology. The AACR has become actively engaged in supporting FDA as it seeks to incorporate advances in science into the regulatory framework. In fact, the AACR Task Force on Regulatory Science and Policy was convened in early 2011 to develop initiatives to help FDA address issues such as companion diagnostics, therapeutic/diagnostic co-development, and other regulatory science issues. This task force is spearheading a new track at the AACR Annual Meeting 2012 that will highlight cutting-edge regulatory science and the related policy issues that cancer researchers face today.
Read more from the October Edition of the AACR Cancer Policy Monitor: