Education And Debate

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

BMJ 2002; 324 doi: https://doi.org/10.1136/bmj.324.7350.1389 (Published 08 June 2002) Cite this as: BMJ 2002;324:1389
  1. P P Koopmans, Specialist in internal diseases, clinical pharmacology, and infectious diseases (p.koopmans@aig.azn.nl)
  1. Department General Internal Medicine, University Medical Center St Radboud, Nijmegen, Netherlands

    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

    Endpoints

    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 …

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