According to a story from BBC, Health ministers are starting to put pressure on Vertex Pharmaceuticals to lower the cost of its treatment Orkambi. The drug is indicated for the treatment of cystic fibrosis, a fatal, genetic lung disease. Currently, year of treatment with Orkambi costs a staggering £100,000. The immense price tag has kept the drug from being covered on the UK’s National Health Service.
Cystic fibrosis is a lung disorder that can also have long ranging impacts in other areas of the body as well. This is a long term, progressive disease that results in a gradual decrease in lung function over time and ultimately leads to a shortened lifespan for those that have it. The disease is characterized by heavy mucus production that creates a potential habitat for infectious bacteria. As a result, patients are much more vulnerable to lung infections in comparison to an unaffected individual. Most patients experience difficulty breathing as well as clubbed digits, sinus infections, fatty stool, poor growth, and infertility in males. People with cystic fibrosis often have to take antibiotics for much of their lives. While there are several other procedures and therapies that can offer temporary improvements in lung function, none of these options can prevent disease progression. A lung transplant may be an option if lung function has worsened severely. To learn more about cystic fibrosis, click here.
Vertex has claimed that it wants to reach an agreement to get the drug on the NHS, but the company has rejected the first offer from the service. The company claims that the offer would not allow for funding of future research efforts. While the company and the NHS continue to attempt to workout these negotiations, desperate patients are stuck waiting on the sidelines; most patients currently can’t afford the medication anyway and are exhausting the limited treatment options that are available.
The issue of getting Orkambi onto the NHS was raised in the House of Commons just last month. Health ministers Steve Brine and Lord O’Shaughnessy have sent a letter the Vertex’s Simon Bedson, a senior vice president at the company. In response, the company has issued a statement saying that it plans to meeting with the ministers soon. Hopefully, they will be quick enough to save the patients whose lives hang in the balance.
What are your thoughts on the issue of high prices for rare disease drugs? Share your stories, thoughts, and hopes, with the Patient Worthy community!
To find out more about CF, check out our partners Cystic Life and Strawfie Challenge.
