Policy Documents

Everyone Deserves the Right to Try: Empowering the Terminally Ill to Take Control of Their Treatment

Christina Corieri –
February 11, 2014

Anna was only 13 years old when she died of an embryonal sarcoma, a rare form of liver cancer.6 Six months before she died, she had exhausted all conventional therapies, and her doctors informed the family there was nothing more they could do. Her parents were not willing to accept the news without a fight. They began researching experimental medications and soon discovered a number of investigational drugs in clinical testing to treat sarcomas like Anna’s. Anna’s age and advanced diagnosis, however, disqualified her from participating in the clinical trials, leaving the Tomalis family with one only option – asking the FDA for permission for Anna to try investigational drugs through an expanded access program – the single patient IND.

For months, the family sought approval for expanded access for their daughter. However, the process was difficult, uncertain, and time consuming. Anna’s mother said, “I came into this process so naïve, thinking that those of us who seek compassionate use of drugs actually get them. It was a shock to find out I had been seriously misled.” By the time the FDA finally granted access, it was too late. Anna died three weeks later, leaving her grieving family wondering whether Anna could have won her battle if she had been granted access sooner.

The FDA strictly controls which medications are available in the United States. Before a drug can be made available to the general public, it must undergo a lengthy and expensive clinical trial process to determine its safety and efficacy, which takes on average 10 to 15 years and over $800 million dollars to complete. Terminally ill patients can request exemptions, but the exemption process can take several months and requires doctors to complete paperwork that the FDA itself notes will require more than 100 hours to complete. Ultimately, the decision still rests with the FDA.

These bureaucratic impediments violate an individual’s fundamental right to try to save his own life. Unfortunately, the federal government has shown little interest in reforming the FDA as bills to reform the process for terminal patients have been introduced, but have never received a vote in Congress. State legislators, however, have the opportunity to protect their citizens’ right to try investigational medications by enacting Right to Try measures. These measures would ensure the right to protect one’s life by returning medical decisions where they belong – to patients and doctors.