Just after her second birthday, additional testing, including an MRI of her brain and spine followed by a genetic analysis, revealed that our daughter hadย metachromatic leukodystrophy. This lysosomal storage disorder is anย autosomal recessive genetic diseaseย that interferes with the bodyโs production of a single enzyme, arylsulfatase A.
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Shire Pharmaceuticals has developed a therapy for the disease and has even found a way to deliver it across the blood-brain barrier, which is no mean feat. The company has even completed a multicenter Phase 1/2 trial of the drug, called SHP-611.
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It meant that Radha should qualify for what the Food and Drug Administration calls itsย expanded access program, also known as compassionate use. It governs the use of an investigational medicine that has not been approved by the FDA outside of a clinical trial.
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Radhaโs physicians followed Shireโs protocol for applying for compassionate use exactly as directed onย the companyโs website. Within a day or two, their request was denied, without any legitimate medical reason given.
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Many [companies]ย refuse to grant accessย to investigational drugs outside of clinical trials, and efforts to lobby them to release the medication as part of compassionate use are often rebuffed.
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From Radhaโs perspective, they are nothing more than a cruel joke, dangling a potential lifesaving therapy just out of her reach.
Read full, original post:ย The โcruel jokeโ of compassionate use and right to try: Pharma companies donโt have to comply
