Patient Advocacy – in Current Science – Guest Editorial by Mohua Chakraborty Choudhary
It took seven years for one patient to be correctly diagnosed with Pompe disease. This disease needs enzyme replacement therapy (ERT), which can cost up to Rs 1 crore per person per year. This was beyond the means of the patient’s father, and taking care of his daughter was a huge challenge. He first established the POMPE foundation and later the Organizations for Rare Diseases India (ORDI), an umbrella organization. ORDI along with another patient group, Lysosomal Storage Disorder Support Society (LSDSS), took every possible measure from filing Public Interest Litigations to appealing to the
pharma industry to provide accessible and subsidized treatment. As a result, in 2017, 240 patients with lysosomal storage disorders (LSD) received free ERT inIndia. ORDI was also instrumental in setting up the first Centre of Excellence for Rare Diseases at the Indira Gandhi Institute for Child Health, Bengaluru.
Download PDF of the article.