Here are more reflections on the December 11th NYTimes article about the 11th-hour rescue of a dying 6-year-old girl with an investigational treatment. I hope you find them useful in the pursuit of Healthy Survivorship.
New Hope for Patients with Uncommon and Orphan Diseases. Running clinical trials is expensive. If only a small population of patients might benefit from a new treatment, pharmaceutical companies are less likely to invest in the research.
However, a new model is arising with some of the new, tailored therapies that "have a big, unmistakable impact on a small number of patients. Such home-run drugs can be approved more quickly and efficiently...with smaller studies than are needed for drugs with less obvious benefits."
Research often reveals the "what" before the "why." While this young girl's dramatic story inspires hope for the development of more effective treatments, "the research is still in its early stages, and many questions remain. The researchers are not entirely sure why the treatment works, or why it sometimes fails."
This article reminds us that science is a way of examining the world to get closer to the truth about what is happening and why it happens. Healthy Survivors put news like this in perspective by keeping in mind that developing safe and effective treatments depends on an understanding of "why."