Bridging the Gap

Recognising rare diseases' impracticability of having extensive clinical trials necessary for regulatory approval, the US Orphan Drug Act appropriately facilitates the commercialization of "orphan drugs". However, even common cancers also have rather specific but uncommon clinical situations for which extensive clinical trials are hardly practicable. Moreover, although such very costly and extensive trials often depend on industry support, uncommon situations are unattractive. Eventually, many specific treatments for designated clinical oncology situations often lack regulatory approvals merely because of suboptimal trial data. Now, this EBM (evidence-based medicine) book by expert authors with first-hand clinical experience is about off-label but acceptable treatment options. The attempt is to patch up the low response rates and considerable adverse events of novel immunotherapy. Currently, without any such off-label treatments, some cancer patients may well be left out in the cold. Although off-label treatments lack regulatory approval, they are backed by EBM and have good safety profiles. It's inappropriate to announce "nothing else can be done" merely because all approved options have already been exhausted. In expert hands, despite the "on compassionate grounds" proviso, the safety profiles of such off-label prescriptions may even compare favourably with orthodox chemotherapy given at top doses.