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A unifying strategy for clinical trials in rare urological malignancy

Nature Clinical Practice Urology volume 5, pages 652–653 (2008)Cite this article

Why is the evidence base for uncommon urological malignancy so seldom derived from prospective trials? The answer might seem self-evident, but we know from our colleagues who deal with sarcoma, hematological malignancy and childhood cancer that it is possible to produce trial data for rare tumors. I will address some key issues in developing a trial strategy for rare urological malignancy.

No definition of 'rare' is entirely satisfactory. The European Union definition of rare disease is a condition that affects less than 5 people per 10,000, while in the US a rare disease is one that affects fewer than 200,000 individuals.1 'Rare cancer' is generally taken to mean cancer originating at an uncommon site, cancer at a common site with histology unusual for that site, or cancer in which the histology is itself a curiosity. It is important to consider that cancer that is rare in one part of the world can be rather less so in another—squamous bladder cancer in the West compared with the Middle East, for example, or cancer of the penis in the UK compared with India.

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References

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Author notes

  1. Department of Oncology, Leicester Royal Infirmary, Leicester LE1 5WW, UK wondersn@doctors.org.uk

Authors and Affiliations

  1. S Nicholson is a Senior Lecturer in Medical Oncology at the University Hospitals of Leicester, Leicester, UK.,

    Steve Nicholson

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  1. Steve Nicholson
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The author has declared that he is a Consultant for both Bayer and Roche.

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Nicholson, S. A unifying strategy for clinical trials in rare urological malignancy. Nat Rev Urol 5, 652–653 (2008). https://doi.org/10.1038/ncpuro1257

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