Patient Advocacy

Generic medicines are the backbone of the US prescription drug market, supplying more than 9 out of every 10 prescriptions. And the production of generic medicines overall has been remarkably stable—even during challenges such as the COVID-19 pandemic.

Total drug spending in the United States, based on invoice prices, reached $425 billion in 2015, according to the IMS Institute for Healthcare Informatics report, “Medicines Use and Spending in the U.S.: A Review of 2015 and Outlook to 2020.” When adjusted for net price spending, $310 billion went to drug and biotech companies, but where the other $115 billion went is often omitted from media coverage.

On April 25, 2016, an FDA advisory committee voted not to recommend the approval of eteplirsen, an experimental drug that targets one of many genetic mutations causing Duchenne muscular dystrophy (DMD), a deadly degenerative disease that has no cure. After agreeing to study the real-world effects of eteplir­sen, the FDA advisory committee rejected findings that patients who have been taking eteplirsen since 2011 were still able to walk because the clinical data did not meet the FDA requirements for a well-controlled study.

I never thought it would happen in the United States, but it has. We are critically close to running out of some chemotherapy drugs. It came as a shock to all of us, when we realized how severely the problem of drug shortages has started to affect our patients. The following case is but one example.