Alberto Gutierrez, director of the Office of In Vitro Diagnostics and Radiological Health at FDA, recently described the regulatory environment for personalized medicine as “chaotic,” noting the need for FDA to provide better clarity on many issues.  Indeed, a strategic priority for the Center for Devices and Radiological Health (“CDRH”) in 2013 is to further develop its personalized medicine program.  According to these goals, stakeholders will see two key guidance documents in 2013:  a finalized version of a guidance on companion diagnostics by March 31, 2013, and a draft guidance addressing co-development of drugs and devices by September 30, 2013, although recent reports have suggested that the release of the companion diagnostics guidance will now be pushed to the second quarter of 2013. Stakeholders also may see continued developments regarding the regulation of laboratory developed tests (“LDTs”).

FDA’s existing Draft Guidance for Industry and Food and Drug Administration Staff—In Vitro Companion Diagnostic Devices was released in July of 2011 and met with some criticism.  One matter of debate was FDA’s statement that a therapeutic product and its companion in vitro diagnostic “ideally” be developed “contemporaneously, with the clinical performance and clinical significance of the IVD companion diagnostic device established using data from the clinical development program of the corresponding therapeutic product.”  Some industry stakeholders pointed out that this “ideal” scenario often was not realistic.  Stakeholders asked for further guidance on such issues as clinical validation of biomarkers after initiation of a phase III trial, designing retrospective studies, and use of banked samples.  Nevertheless, Liz Mansfield, Director of the Personalized Medicine Staff in CDRH’s Office of In Vitro Diagnostics and Radiological Health, has stated that the final guidance will not reflect many changes from the draft.

Moreover, despite regulatory uncertainties, FDA has continued to approve drugs with companion diagnostics.  In 2012, FDA approved a new indication for Erbitux with a companion KRAS test and approved Perjeta with two HER2 companion diagnostic tests.  In 2011, FDA approved two drugs with companion diagnostics:  Pfizer’s Xalkori and Roche’s Zelboraf.  Pharmaceutical companies and diagnostic manufacturers continue to partner on companion diagnostics and paired therapies, reflecting a continued interest in personalized medicine.  For example, Eli Lilly and Qiagen recently inked a deal to develop and commercialize companion diagnostic products.

Personalized medicine stakeholders also should continue to monitor developments regarding the regulation of LDTs.  FDA announced in June of 2010 that it intended to begin regulating LDTs as medical devices and held a public meeting on the issue.  It widely was reported that FDA would attempt to phase-in regulation of LDTs as medical devices through a series of guidance documents, but FDA has yet to issue any such document.  Under section 1143 of the Food and Drug Administration Safety and Innovation Act of 2012, FDA must now notify Congress 60 days before issuing a draft or final guidance document regarding the regulation of LDTs.  Members of Congress may continue to float legislative alternatives to FDA’s plans, such as Representative Michael Burgess’s bill, the Modernizing Laboratory Test Standards for Patients Act, and Senator Orrin Hatch’s draft bill, the Better Evaluation and Treatment Through Essential Regulatory Reform for Patient Care (BETTER) Act.