Regulatory Submission (Chapter 4)
The FDA and other regulatory agencies are conservative by design. Drugs are approved based on their safety and efficacy (historically their ability to address a particular disease or patient condition) regardless of a patient’s genomic profile. Pharmaceutical companies have attempted to follow proven regulatory pathways as opposed to proposing or negotiating new ones which could add both time and risk.
Personalized Medicine Implications
For two recent personalized medicines approved by the FDA, Xalkori (Pfizer) and Zelboraf (Genentech & Daiichi Sankyo), the Agency approved the following labels:
- XALKORI is a kinase inhibitor indicated for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) that is anaplastic lymphoma kinase (ALK)-positive as detected by an FDA-approved test. This indication is based on response rate. There are no data available demonstrating improvement in patient reported outcomes or survival with XALKORI.
- ZELBORAF is a kinase inhibitor indicated for the treatment of patients with unresectable or metastatic melanoma with BRAFV600E mutation as detected by an FDA-approved test.
Based on these precedents, if a pharmaceutical manufacturer wants to get a personalized medicine approved, it had better have “an FDA-approved test” to detect the genetic profile or risk the consequences.
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