Medical news

 

ORKAMBI approved!

The drug combo targeting the double f508del mutation has been approved for use in the US and EU!

Unfortunately, it will not be reimbursed in Poland, as its per-patient cost is estimated at USD 250,000 per year.

http://investors.vrtx.com/releasedetail.cfm?ReleaseID=856185

 

http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Summary_for_the_public/human/003954/WC500197614.pdf

 

KALYDECO approved

Kalydeco™ is a new oral medication for the treatment of cystic fibrosis, approved by the U.S. Food and Drug Administration (FDA) in January 2012 for CF patients with the G551D mutation of CF (4% of all CF sufferers).

http://www.cff.org/treatments/therapies/kalydeco/

http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Summary_for_the_public/human/002494/WC500130744.pdf

 

 

IV treatments 

The most important legislative development in Poland is the legalization of home intravenous antibiotic therapy in September 2012. However, as no secondary legislation has been enacted, the new law is non-operational, and patients in Poland are still waiting to gain access to this procedure. Inadequate standards of hygiene in hospitals (not a single one of the hospitals in Poland providing treatment for CF patients is able to meet the international requirements regarding patient isolation) means that each hospital stay exposes patients to the risk of cross-infections. This accounts for the ten-year difference in average life expectancy between CF sufferers in Poland and in Western Europe / the United States.