More than 400 people worldwide are still waiting for Kalydeco to become available.
This is close to 20% of the worldwide G551D Cystic Fibrosis population.
Several of these countries, including Australia and Canada, have been involved with reimbursement negotiations for over a year. These negotiations have stalled due to the $300,000+ price tag for Kalydeco.
According to Vertex, Kalydeco is currently available to approximately 1000 patients in the US and 1000 patients in Europe. Kalydeco is available in the US, England, Scotland, Northern Ireland, Wales, the Republic of Ireland, France, Germany, the Netherlands, Austria, Denmark, Sweden, Norway, Greece and Italy.
This means Kalydeco is available in 15 countries, however according to the Cystic Fibrosis Worldwide member list, there are 45 countries that potentially have G551D patients who do not have access. Some of these countries cannot afford basic CF medications like Pulmozyme, so how are they going to afford $300,000+ per year per patient?
As a CF patient living in Australia, it concerns me that there is a growing worldwide divide between countries which can afford Kalydeco and those that cannot. I would like to know how Vertex plan to approach access in these forgotten countries, particularly South Africa and South America?
Those who do not have insurance in the US can access Kalydeco for free through a Vertex Patient Access Program. Could a similar program be an option for some of the remaining countries, where Kalydeco is very unlikely to be approved at even half the current price?
The reality is that whilst these negotiations occur and access is delayed, patients are left with few options, 1) attempt to raise an extraordinary amount of money, 2) move to a different country and hope they qualify for Kalydeco access or 3) continue getting sicker. People with CF all over the world need help and implore Vertex to find a way to give them access to Kalydeco before it’s too late.
The Associations / Countries in this list were contacted: Cystic Fibrosis Worldwide Member Countries.
I was unable to source information from these countries, which may also have G551D patients: Armenia, Azerbaijan Republic, Bahrain, Colombia, Costa Rica, Cuba, Dominican Republic, Ecuador, Egypt, El Salvador, Estonia, Finland, Georgia, Guatemala, Iceland, India, Israel, Jordan, Latvia, Lebanon, Lithuania, Luxembourg, Mexico, Panama, Paraguay, Portugal, Qatar, Romania, Russia, Saudi Arabia, Switzerland & Uruguay. Bulgaria, Hungary, Macedonia & Turkey have no recorded G551D patients.
Note: I have branched out from my standard blog content as I strongly believe attention needs to be drawn to the 20% of the world who do not have access to Kalydeco.
It was announced recently that Canada has a tentative price agreement and that Spain has agreed to a price. This means that close to 90% of the worldwide G551D population will have access to Kalydeco (17 countries).