Gaining an Understanding of Cystic Fibrosis Treatment Options

Cystic fibrosis is a debilitating disease without known cure. Daily research continues all over the world for the solution to cure those already diagnosed, and for ways to prevent this disease from ever occurring.

While there is not yet a cure for CF, there are meds and treatments which help people control their symptoms.

Knowing more about your treatments can make you an informed consumer. It’s been said that being an informed consumer helps put you in the driver’s seat; in this case, becoming informed about medication and treatment option helps increase your decision-making role in the treatment of CF.

According to Seeking Alpha (SA), three treatments for CF have been approved by the FDA, with two others to complete their phase two clinical trials this year (2017). It’s also encouraging to know that many new treatments for CF are also in the clinical and regulatory process, commonly referred to as the ‘pipeline’.

Drug studies and the importance of the FDA

After many false starts of drug studies worldwide, to include deaths and birth defects from the use of inadequately tested medications, the U.S. Federal Drug Administration has formed and evolved into a respected worldwide guide. Their approval is required for new medications to be placed on the U.S. market. The FDA is seen as a trusted authority to help physicians use the best possible medicines to treat their patients. The U.S. Food and Drug Administration has evolved as one of the world's foremost institutional authorities for conducting and evaluating controlled clinical drug trials. (USDA)

What to consider regarding popular treatment options

• Vertex’s drugs are mutation specific. So, they don’t have a positive effect on all CF patients; secondly, it’s a treatment, not a cure, and must be taken for your lifetime, along with other standard maintenance treatment.
• The annual cost of Kalydeco treatment is $300,000 and Orkambi is $259,000. By comparison, the annual cost of Pulmozyme is $24,000.
• There are probably more CF patients who could benefit from Vertex’s drugs but are not currently using them because of the exorbitant cost.
• For Galapagos and Protalix, their respective CF drug candidates have shown positive results in their phase 2 trials.

There’s no question that those with CF would benefit from price reductions on the meds they require for ongoing maintenance of their disease.

How could you help bring this about?

To learn about medications and treatments on the market for CF and also current studies underway, let’s take a fresh look at the drugs already being use by those with CF and two new ones in current clinical trials.
To do so, we’ll research 4 major drug companies.