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.
Information Sources
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.
9/6/14 Update
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).
sixtyfiveroses 
Where is the individual in this wonderful new era of “individualized medicine”? This amazing scientific progress can’t be made in a vacuum! ACCESS must be as important as the science itself! There must be a way to bring the value of these “forgotten lives” into the price negotiations. Access discrepancies have turned Kalydeco into an absolute miracle for some, and a cruel and unusual form of torture for those who are left without.
Hola ¡¡ aqui en Latinoamerica la inequidad y el olvido es
desgarrador¡¡ en todos los sentidos, en los seguros de salud y
obras sociales, en la falta de diagnostico pernatal, en el faltante
de tratamientos y medicacion adecuada, la falta de informacion.la
falta de centros multidisciplinarios, la falta de concientizacion y
apoyo a la FQ, la falta de gestion a la integracion con la FQ del
primer mundo ,la falta de union (no hay congresos latinoamericanos
de FQ) LA FLATA DE UNION EN LAS IINSTITUCIONES DE AYUDA, sumado
todo esto a la ineficacia de los antibioticos que no combaten las
pseudomonas sino todo lo contrario la hacen mas resistentes y
mucosas bajando las defensas y el sistema inmune logrando acelerar
el deterioro pulmonar y logarndo la conjuncion de todos estos
factores que la expectativa de vida aqui sea exactamente la mitad
de la del primer mundo ¡¡¡ Acaso una vida de un joven ,un niño
latinoamericano vale menos que la de un niño o joven del primer
mundo por su lugar de nacimeinto ?? No¡¡ los derechos humanos en
algun lado deben decir que son iguales en todos lados del planeta
pero no es asi ¡¡ Nosotros tenemos hace un año exactamente un grupo
en face (casi 4000 miembros) casi 100 Instituciones de ayuda a la
FQ en el cual comentamos el olvido de Vertex y la falta de gestion
de nuestros gobiernosy de nuestras instituciones para lograr los
ensayos clinicos minimamente para G551 por ahora ¡¡ nadie nos
escucha ¡¡¡ los nombres de Instituciones que la worldwide coloca
(Sr Terry) en el listado no es cierto ,desconocen la worldwide , no
tienen contacto con la worldwide y si lo tienen es meramente
protocolar o de saludo . No existe un plan que avance en ele sntido
de gestionar kalydeco a latinoamrica ¡¡ no existen Instituciones
UNIDAS o una Asociacion Latinoamericana que nos represente como en
el primer mundo ¡¡ La individualidad es moneda corriente y la falta
de UNION reitero es la constante . Los invito a incorporrase al
grupo lalmado KALYDECO PARA LATINOAMERICA.TESTIMONIOS . el cual
comenza con una carpeta de 200 hojas donde se trataba de entusismar
a laALAPE (asoc latinoamerica de pediatria) que incluya y nos ayude
en la getsion ante los gobiernos pero fue infructuoso todo no nos
atendieron ,los medicos no se involucran ¡¡ los gobiernos no se
involucran , las instituciones no se involucran y nuestro niños
falelcen de a 1 por mes en cada pais de latinoameirca (promedio)
por ello me sorprende este articulo es la primera vez en tanto
tiempo que se nombra LATINOAMERICA y se asocia con Kalydeco . no
deja de ser un gran paso ¡¡ M Gracias ¡¡ A disposicion ¡ Jorge
Precerutti administrador del grupo de face Abuelo de una nietita FQ
: ARGENTINA .
Your post made me happy. It’s always good to know that
someone remembers us from Latin Ameria. The party has started and
we can not accept that it is only for VIP guests.
Reblogged this on mukomama… and commented:
Kalydeco, wspanialy lek, dla tych ktorzy moga. My czekamy niecierpliwie na takie ktory pomoze Kubuniowi…