Clinical endpoints in trials of drugs for cancer: time for a rethink?

There is a continual debate between health authorities, doctors, researchers, the pharmaceutical industry, and regulatory authorities over the question of when a new drug should be admitted to the market and when it should be prescribed or reimbursed.1 Divergent interests often obscure the scientific debate, but it is in the interest of all parties that valuable and effective drugs should be licensed without undue delay. 2 3 Yet it may take many years (mostly 10-15) from the time when a clinical development plan for a drug (mapping out how to investigate it) is established to its being prescribed for patients. This is frustrating for all drugs but is particularly so for drugs for fatal diseases such as cancer. I argue here that the process can be improved and focus particularly on three issues: the endpoints, side effects, and more collaboration between the parties, and especially the participation of patients. #### Summary points The introduction to market of drugs for lethal diseases such as cancer can be improved More attention should be given to clinical symptoms and quality of life and less to survival Surrogate endpoints and biomarkers should be used as support for proof of effectiveness Clinical endpoints such as survival can be investigated after the drug's introduction Licensing authorities, pharmaceutical companies, and patients should agree in advance the relevant endpoints and desired effect sizes The major points at issue in drugs for cancer are the desired endpoint and the degree of change required over existing treatment.4 Licensing authorities require that drugs have a clinically meaningful effect. In the case of cancer drugs this is often translated as meaning an increase in survival compared with standard treatment. In potentially lethal diseases, however, waiting to assess these clinical endpoints (for example, the effect on five year survival) poses a dilemma as the …