According to a story from Cystic Fibrosis News Today, Elizabeth Rogers, a cystic fibrosis patient, is just one of the many who have been disappointed by the lack of impact of many of the latest medications that have been introduced to the market in the last few years. Such drugs include Orkambi and Symdeko, both developed by Boston-based Vertex Pharmaceuticals.
About Cystic Fibrosis
Cystic fibrosis is a type of genetic disorder which can have impacts throughout the body, but it is most characterized by the build up of abnormally thick, sticky mucus in the lungs. This mucus becomes a fertile breeding ground and habitat for potentially infectious bacteria. Many patients must take antibiotics for much of their lives. This disorder is caused by mutations of the CFTR gene. Symptoms of cystic fibrosis include progressive decline in lung function, lung and sinus infections, coughing up mucus, fatty stool, poor growth, infertility in males, clubbed digits, and digestive problems. Treatment includes antibiotics and medications or procedures intended to maintain lung function. Lung transplant is an option when lung function declines severely. Life expectancy ranges into the 40s and 50s with good care. To learn more about cystic fibrosis, click here.
Elizabeth tried Orkambi first, but the serious side effects wound up being the primary impact that the drug had on her. The most unpleasant was a feeling of tightness in her chest that made it feel as if she couldn’t breathe. Meanwhile, the only real benefit she saw from the drug was a small reduction in the frequency of her lung infections. She did not see any meaningful improvement to her lung function. She then tried Symdeko, which had less severe side effects. Still, the benefit for her was minimal.
Over-hyped and Overpriced?
Orkambi and Symdeko have both gained widespread attention as game changers in cystic fibrosis treatment, and some patients do see substantial benefit from taking them. However, Elizabeth is not alone in feeling that the impact of these therapies was over-hyped in the media. It is also worth noting that we are talking about therapies that run into the hundreds of thousands of dollars for a year’s treatment.
The NHS in England has received sharp criticism for rejecting coverage for Orkambi. One of the reasons for this hesitancy was skepticism about the cost-effectiveness of the drug. Perhaps these concerns were not entirely unfounded.