I was very encouraged by news this week that the FDA has issued final rules which will help make investigational drugs more widely available to patients and also helps clarify the procedure for access. While it is heartening to see the FDA continually update and expand its position on providing important medicines for people with life threatening diseases, it also speaks to me on a very personal level.
My dad has a terminal lung disease called Interstitial Pulmonary Fibrosis (IPF). It hardens or scars the tissue in his lungs and makes it very difficult to breath. He uses oxygen and takes an oral steroid every day for the inflammation in his lungs. The biggest heartbreak has been watching a once very active man reduced to brief forays into his beloved garden which last only moments before he is exhausted, running out of breath and reduced to racking coughs. He's always tethered to his oxygen now and he spends most of his time sitting in his recliner watching news or sports or maybe reading a book. Playing weekly tennis and golf are only distant memories, but he still devours them on TV. And while I know that my weekly visits are good medicine for his heart, there is no approved medicine for his disease.
However, there are ongoing clinical trials investigating treatments for IPF, and as a family, we discussed and considered the possibility of my dad entering a trial, but we learned he would have to stop taking his steroid...and for us the risk of him getting a placebo was just too high. Now that the FDA has expanded the opportunity, we may be able to get my dad experimental medicine outside of a trial...and I will make sure to explore all of his options to the fullest. The new FDA website for consumers is very helpful for patients and patient advocates, guiding them through the process and offering considerations for pursuing an investigational treatment.
While there are still many hurdles for people like my dad--people with chronic, life-threatening diseases--to actually get investigational medicines outside of a clinical trial, these steps by the FDA make it a little bit easier and I'm grateful for the progress.