Cystic fibrosis and the new drug Kalydeco
Cystic fibrosis is genetic disease affecting the respiratory and digestive system, eventually leading to early death (Marieb, 2012). This disease is inherited from your parents who both must carry this gene (WebMD staff, 2012). According to Grossman and Grossman (2005) there are numerous variances that can occur to the gene CFTR resulting in the disease manifestation, but the majority has the defect F508. CFTR regulates the flow of chloride in and out of cells (Marieb, 2012). When this process is not working properly mucous is not thinned out appropriately becoming thick, sticky and clogging the lungs and pancreas (NCBI:Publicmed Health staff, ...view middle of the document...
Kalydeco’s estimated price tag per patient per year is approximately $294,000 (Pollack, 2012). This is not unusual for specialty drugs due to the targeting of a small population and the pharmaceutical company desiring recoupment for their research and design investment (Dickson, 2009). Those that have insurance will benefit once this drug is approved on their insurance formulary. However, having an expensive drug covered by insurance passes the cost of this drug on to everyone else having insurance by increasing co-payments and premiums (Schilling, 2012).
CFF staff (n.d.). About cystic fibrosis: What you need to know: What is cystic fibrosis? Retrieved March 9, 2013, from http://www.cff.org/AboutCF/
CFF staff (2012). Kalydeco. Retrieved March 9, 2013, from http://www.cff.org/treatments/Therapies/Kalydeco/#How_does_Kalydeco_work?
Dickson, M. (2009). The cost of new drug discovery and development. Retrieved March 9, 2013, from http://www.hks.harvard.edu/m-rcbg/fellows/T_Christian_Study_Group/Session%205/Cost_of_New_Drug_Discovery_and_Development.pdf
Grossman, S. & Grossman, L. (2005). Pathophysiology of cystic...