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Patient Advocacy Case Studies2019-07-30T10:18:29+01:00

Helping advocacy group support the treatment needs of patients in outlier countries

Challenge

A rare disease patient advocacy group based in the US was receiving requests from patients in outlier countries who wanted to gain access to an EMA- and FDA-approved product not commercially available in their home countries. These patients had no other treatment options available to them and were at risk of experiencing worsening symptoms which could lead to emergency surgery. The advocacy group wanted to help these patients but did […]

Approaching pharma on behalf of a patient advocacy group

Challenge

A patient advocacy group supporting patients with a rare metabolic disorder in the western Pacific wanted to help provide access to effective treatments. There are currently no approved medicines available for this condition and, although there are promising treatments in development, there were no clinical trial sites set up in this region of the world, at the time. The advocacy group had heard of patients in the US experiencing marked […]

Working with drug manufacturer to fulfil patient request for much-needed treatment

Challenge

A patient living in the Caribbean had been suffering from a genetic lung disorder since a young age and had found there were no effective treatments available in his home country. His local physician decided to refer the patient to a specialist in the US who prescribed him an FDA-approved prescription inhaled antibiotic used to improve breathing symptoms. However, the drug was not approved in the patient’s country and there […]

Importing drug into a patient’s home country when it is not yet licensed there

Challenge

A patient in the US suffering from a seriously debilitating disease was struggling to manage his condition. The patient had tried all available treatment options in the US, but none had been effective. The patient was desperate to find some alternative, so his physician looked into treatments being used in Europe that were not yet available in the US. The physician found a promising drug that had EMA-approval and suggested […]

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