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European Biopharmaceutical Review

Pruning the Branches of Rare Cancer

Orphan drugs and orphan diseases have often been a hidden corner of research and development. In cancer, I think that the distinction is becoming less meaningful. We need to shift from the idea that there is a one-size-fits-all approach for most cancers, and treat a few rare cancers differently. All cancers need therapies that target the underlying biology of each person’s disease, and diagnostics that help doctors determine the right treatments for each patient.

Rare Cancers and Orphan Drugs

We have always known there are rare cancers, and treatments have progressed that would have been unimaginable at the turn of the century. 20 years ago, it was very difficult to run trials: the saga of one of a group of bone marrow disorders shows just how difficult identifying a condition can be. Authorities agreed that the disorder merited orphan drug indication, but went back and forth over the benefits and risks of one treatment option. In 2008, Professor Mario Cazzola, now of the University of Pavia, Italy, wrote, “as a doctor, I have no certainties, some hopes, and many doubts on this subject now, and find it extremely difficult to provide patients with accurate information about this treatment option” (1).

It wasn’t any lack of expertise or attention on Professor Cazzola’s part. Indeed, he is a world renowned expert, and studied all of the available data. It was the complexity of diagnosis, recruitment, and monitoring in an age before Big Data that meant trials had small numbers of patients, and took a long time to recruit and complete. Now, as the EMA said last year, “Big Data are extremely large, rapidly accumulating datasets captured across multiple settings and devices.” They

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Dr Johanna Bendell is the Head of Oncology at Roche Pharmaceutical Research and Early Development in Basel, Switzerland. She joined recently from the Sarah Cannon Research Institute in Nashville, Tennessee, US, where she served as the Chief Development Officer, Director of the Drug Development Unit, and Director of the Gastrointestinal Oncology Research Program.

Johanna earned her undergraduate and medical degrees from the University of Chicago, US, and completed her internship and residency at Brigham and Women’s Hospital, and Fellowship in Adult Oncology at the Dana-Farber Cancer Institute, both in Boston, MA, US. She is an author of nearly 200 journal publications.
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Dr Johanna Bendell
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