In recent years, treatment options for cystic fibrosis, a rare disease, have increased significantly in number and in effectiveness. However, many of the most effective treatments, developed by Vertex Pharmaceuticals, are extremely expensive. The organizations Just Treatment and Vertex Save Us are spearheading a new campaign intended to reduce costs and improve treatment access. With no plan to offer access to the drug in developing countries, Vertex has been criticized for price-gouging and a lack of empathy for patients.

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 buildup 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. More useful treatments for the disorder have been introduced in recent years. Lung transplant is an option when lung function declines severely. Life expectancy ranges into the 40s and 50s and beyond with good care and the most advanced therapies. To learn more about cystic fibrosis, click here.

Cystic Fibrosis Patients are Dying

A recent study found that only around 12 percent of 162,000 patients currently can access the latest therapies from Vertex. Just Treatment and Vertex Save Us have submitted an open letter to the CEO of the company, Dr. Reshma Kewalremani, with three demands:

1. The release of a plan for universal access to treatments by the end of the year;
2. The dropping of patent claims in low to middle income countries to enable generic manufacturers to meet the needs of patients in those regions;
3. Find agreements within the health systems of all developed countries that enable access within three months; or facilitate access to generics in those countries

At The North American Cystic Fibrosis Conference, a study estimated the production cost of Trikafta, the company’s most effective therapy, at $5,700. This is a marked contrast to its listed US price of $311,000. The same study concluded that there was no sign that patients outside of the developed world would see improvements to access. The study also concluded that generic production of these therapies was completely feasible and could help reduce the price and enhance treatment access.

Furthermore, other drug companies have made greater efforts to enhance access to medicines in lower income countries through the issuance of voluntary licenses; Vertex hasn’t made similar plans. This lack of action can be quantified in the needless deaths of hundreds of patients around the world. 

Patients in the UK, such as Tim Wotton, have been sharing about how Trikafta has transformed their lives, but they want the rest of the world to get access.

Noted cystic fibrosis patient and advocate Marc Cotterill has also been participating in the campaign for greater access.

The campaign also includes a petition calling for Vertex to take action and improve treatment access. Click here to add your signature.