IGA Blog

Charitable access success

Atif Taha, Qatar

Six years ago, my family received devastating news. Two of my three children were diagnosed with Gaucher disease, a very rare condition that affects 1 in 50,000 people around the world.

The fact that the disease has viable, albeit lifelong, treatment is a great blessing. The big problem, however, was the exorbitantly expensive cost. Figures that are out of reach of any family in the world except, perhaps, for the richest 0.1%. And we have not one, but two children requiring this medication. As far as we know, no Gaucher patient in the world is paying for treatment out of pocket. During these very testing 6 years we managed to secure intermittent doses of the medication for our eldest child who was showing more advanced symptoms. The other child was left untreated. A decision no family should have to make.

We got in touch with Mrs. Tanya Collin-Histed from the IGA who lobbied on our behalf and sped up our application to Sanofi, the drug manufacturer, to receive the medication. Unfortunately, our application was rejected multiple times on the grounds that it did not meet the criteria per Sanofi’s policy at that time. But Mrs. Collin-Histed, Tanya, did not give up. She set up meetings for us with the lead Gaucher specialist in the UK who wrote medical reports to support our case. Throughout these 6 years, Tanya never stopped advocating for our kids. They were much more than just case files or data points to her. They were two promising young children that deserved a chance for a healthy life.

One exceptionally hot and humid late June afternoon, we received the long awaited news. We were informed that, following a change of policy, Sanofi is now offering the drug, free of charge, to our children as long as their conditions require. Apparently, Tanya’s relentless advocacy, through the IGA’s channels, finally managed to move the mountain of impossibility. She was instrumental in Sanofi’s policy update.

No words can express our gratitude for what Tanya and the IGA team has done for our family. The six years of tireless campaigning on our behalf. They believed that our story could have a happy ending, and in doing so gave us hope.

The IGA always say "Rare but not alone", they mean it genuinely and we lived it. The IGA inspired us to start a new chapter in our life. Supporting ones in need and lending a helping hand is our ultimate goal. We started with our home country Sudan with the IGA full arsenal assisting us.

Tanya adds:

I have been privileged to be a member of the Gaucher community for 27 years as mum to Maddie and 25 years as a patient advocate. Every day there are highs and lows, and you meet amazing and strong people. Some days are more special and 30th June was one of those days. Working in collaboration with Sanofi and in particular with Bonnie and Jasmina these two siblings from Qatar were approved for treatment through Sanofi’s charitable access programme. These cases have been a huge challenge but the first rule of advocacy is don’t give up and never take the first answer given if it’s NO.

The IGA and Sanofi have worked together on many cases where access to treatment has been a challenge. Sanofi’s International Cerezyme programme has treated and continues to treat hundreds and hundreds of Gaucher patient around the world. I would like to congratulate Sanofi for recognising that due to demand and a constantly changing world, they have been open and responsive to reviewing their charitable programmes and we are delighted that these two siblings were able to meet their criteria and be approved for treatment.

Sadly there are still hundreds of Gaucher patients around the world without treatment and as a community we still have so much to do.

 Visit our website for more information on charitable access