A Standard of Perfection; a Plan for Action

Merriam-Webster defines idea as "a standard of perfection; a plan for action."

Mizner had it right, to a degree. Stealing the best ideas from many authors is research. But only if you take the best of those ideas and push them further, at times escaping their boundaries, and flirt with the best that humanity must give.

One of the best new ideas that I have recently encountered is regarding our health care system and drug trials, particularly for children. It is no secret to my friends that I listen to National Public Radio (thanks Mom). What caught my attention so quickly was the brief reference to Duchenne muscular dystrophy; my French teacher’s son, Matthew, has Duchenne, and we grew close on a trip to France two years ago.

I digress. The idea in NPR’s broadcast was that patients and their parents, not drug companies, are developing and setting the standards for new drug trials based on their own individual needs. The idea is just so simple; I am not sure why we have not thought of this before. Patients and their families, as most of the patients in question are children, are guiding the research performed by pharmaceutical companies and researchers regarding drug treatments and other protocols for medical research and diagnostics. Prior to this, “big pharma”, as the pharmaceutical companies are known, pitched a drug to the Food and Drug Administration (FDA), who then spent months and years determining if the drug is safe and effective. Before patient influence of drug trials and research, the hope was that big pharma’s goals would line up with patients’ needs. That was before patients’ families started renting out lab spaces at Harvard and tracking down mutated genes in their free time. Now, patient needs can be at the forefront of research and, in time, research and treatments can become more individualized.

What made this new idea possible? Well, it has taken the efforts of many parents advocating for their children. And children advocating for themselves when possible. One of the individuals in the story aired on public radio was of a 14-year-old girl, Lindsey, who had already faced a kidney transplant. Lindsey did not want to take medications multiple times per day. In addition, she wanted the transplanted kidney to function well for the remainder of her life, and she wanted the transplant drugs to be less toxic to her body. At fourteen, Lindsey has already faced and conquered so much more than many adults. And she was speaking clearly and confidently to a panel at the FDA. Amazing! These are just two small changes that have opened the door for what will need to be many more incremental steps forward; parents and kids must keep pushing boundaries to get what they need, what we need, from giant pharmaceutical companies. But beyond that, pharmaceutical companies have been willing to take a chance and listen to patients – to take a chance on a few of the one-in-a-million treatments. It’s so exciting to see these options become more possible as the pharmaceutical and medical technology fields grow ever more advanced and specialized.