In 2008, New Zealand lecturer Sarah Broom developed a cough while she was pregnant with her third child. She had to deliver her baby three months early in order to get a biopsy, but by then the lung cancer had become advanced-stage and not much could be done. Chemotherapy didn’t help– doctors soon found a new plum-sized tumor in her ovary. Broom contacted friends all over the world to ask for help, including someone in Boston who managed to enroll her in a clinical trial in Australia for a brand-new compound called crizotinib that had only been tested in two patients before. This drug worked briefly. Two years later, the tumors came back and Broom developed new ones in her brain after receiving treatment in Boston. Her last hope was a drug called LDK378– however, it had not been released yet because it was still going through clinical trials.

The idea behind clinical trials is that a new drug is tested on patients who have not been responding to other treatments; the company that developed the drug would track the patients’ condition to see if the drug is effective, and if it causes side effects. Each drug has to go through three stages of trials before it can be released to the public. In the United States, this process could take about 10 years, and the process of applying for a clinical trial is a huge pain for patients. Just look at this flow chart about the application process (don’t read it– I have no idea what these words mean):

In the end, Broom and her family wrote letters to Novartis, the drug company that developed LDK378, to request compassionate access (when everything is skipped and patients get immediate access to the medicine). The drug worked, and everything turned out fine.

But clinical trials aren’t just another example of paperwork and bureaucracy– there have been examples of drugs that were thought to be effective, only for people to learn that the side effects are worse than the diseases they were supposed to treat. Interferon gamma, which was developed for a lung disease, actually caused respiratory infections that killed patients faster. The sedative thalidomide, which was used for women in labor, was responsible for thousands of birth defects. Clinical trials could have discovered this long before these drugs were released.

Source:

http://www.nytimes.com/2013/11/03/magazine/how-dying-patients-get-access-to-experimental-drugs.html?pagewanted=4&_r=0&ref=science