Clinical Research as Care: Cut the Patient off Post Clinical Trial—A Lose-Lose in the End

Deborah Cohen of the BBC reports that patients in the UK who contributed to orphan studies only to be cut from ongoing access once the study is completed.  The patient receives care from the clinical trial only to be cut once the trial is over and the drug approved.

The Patient

Louise Moorhouse is a 35 year-old teacher from Birmingham, UK. Afflicted with a rare genetic condition known as phenylketonuria (PKU) she must survive off diet pills and bad-tasting shakes. It turns out PKU makes it difficult for patients to digest an amino acid known as phenylalanine. These building blocks of protein are broken down by the body to make its own proteins.  In the case of PKU levels accumulate and brain damage can ensue reports the BBC. Her life is far harder in the years she has lost access to a drug known as Kuvan.

Patients Contribute to the Approval of The Investigational Drug

An orphan drug made by San Francisco Bay Area-based biotech BioMarin would let Ms. Moorehouse eat normal again. In fact, she was a subject in a BioMarin clinical trial for three years. During that time her life became more normal and the overall quality of life high.  The drug, Kuvan was approved by the F...

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