It is generally accepted that people affected by ultra-rare diseases across the globe face challenges getting reimbursed high cost market approved treatments.
Like many LSD patient organisations across the World the MPS Society receives 100–200 emails each year from patients and their families pleading for help to access enzyme replacement therapy (ERT) for MPSI, MPSII, MPSIVA, MPSVI and Fabry disease. For most we can only respond with a level of empathy, kindly advice about joining forces with others in their country similarly affected and engaging with clinicians and the Departments of Health. Many of these enquiries come from the Middle East, India, Pakistan and Bangladesh where mistakenly people are under the impression that everyone else in the World is receiving reimbursed ERT unhindered. As many families even in Australia, New Zealand, Canada, South Africa and parts of Europe know this could not be further from the truth.
But there is a chink of light for some MPSII and Fabry patients as Shire and Direct Relief, a medical relief non-profit, are working together to improve access to therapy for patients living with Lysosomal Storage Disorders in countries where Shire’s humanitarian program is available. Clinicians in Ecuador, Dominican Republic, Egypt, Albania, Belarus, Tunisia, Malaysia and Pakistan are invited to apply for their patients online here.
Read more on p28 of Summer 2016 edition of the MPS Magazine.