U.S. company, Vertex Pharmaceuticals, is the creator of three treatments for cystic fibrosis: Orkambi, Symdeco, und Kalydeco. These drugs were unavailable in Switzerland, as Vertex and Swiss health officials were unable to come to an agreement on price. As Orkambi was approved four years ago, this battle has been very long, especially for those affected by cystic fibrosis in Switzerland. Romain Benicchio, a public health advocate whose eight year old son has the disease, talks about the difficulties of affording treatments for rare diseases.
About Cystic Fibrosis
Cystic fibrosis is a genetic disorder that is characterized by progressive damage to the respiratory and digestive systems. Those with cystic fibrosis do not have the slippery mucus that is normally found in the lungs. Instead they have thick and sticky mucus which builds up in their system. This buildup causes clogs in the airways, which then traps bacteria and causes breathing problems, infections, lung damage, and respiratory failure. It can also block digestive enzymes, which makes it difficult to absorb nutrients. Cystic fibrosis affects one in every 2,500 to 3,500 Caucasian babies, and it is even rarer in other ethnicities.
Cystic fibrosis is a recessive disorder, meaning that the mutated gene must be passed down by both parents. The gene responsible for this condition affects the protein that regulates salt movement. The mutation in the gene varies in severity as well.
Symptoms of cystic fibrosis affect the respiratory and digestive systems. They include persistent coughing and wheezing, shortness of breath, difficulty exercising, frequent lung infections, stuffy noses, trouble with gaining weight, constipation, male infertility, salty-tasting skin, and exercise intolerance.
Diagnosis often comes at birth, as screening for cystic fibrosis is standard across the United States. In order to complete a screening, doctors must perform blood sampling, genetic testing, and sweat tests. After a diagnosis is obtained, doctors select the correct treatment. The goal is to control infections, remove mucus from the lungs, prevent intestinal blockage, and provide proper nutrition. Methods include antibiotics, anti-inflammatory medications, medicine to induce the coughing up of mucus, bronchodilators, pancreatic enzymes, exercise therapy, chest physical therapy, vest therapy, lung transplants, and surgery to remove bowel blockages, remove nasal polyps, and place a feeding tube.
About the Deal
As of May 1st, the three treatments will be available on the Swiss drug specialties list, meaning that health insurance will reimburse patients. This is a big relief for those with cystic fibrosis, as they had been waiting for the clearance of this deal for four years.
Benicchio says that this is just an example of a broken system. Vertex insisted on an astronomical price of $164,000 per year, a price very few can afford. As Vertex is the major producer of treatments for CF, they have a big say in pricing. But because of the deal, 400 patients in Switzerland will now have access to these treatments, despite the fact that the Swiss government has still not disclosed the exact price that was agreed upon.
It was a grassroots movement of parents with children affected by the disease that led to this deal in the first place, proving that there are issues present in this healthcare system. While this situation was not ideal, they hope that they have set a precedent for future treatments.
In fact, Trikafta, also produced by Vertex, has recently submitted its application for marketing authorization in Switzerland. It has already received the fast track status, with urgency added by the COVID-19 pandemic. As those with cystic fibrosis are at a higher risk, treatments are absolutely necessary. People with cystic fibrosis and those close to them hope that it will not take another four years for this drug to become available to patients.
While this deal is a success, it also exposes a large flaw in the system.
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