I read with interest, as I’m sure many of you did, the story of Josh Hardy, the critically ill seven-year-old from Virginia whose family petitioned Chimerix, a North Carolina biotech company to extend an unapproved drug to their son under the compassionate use regulations. You can read the story here.
What a heartbreaking story about a young boy who was diagnosed with cancer four times since the age of nine months. The good news is that Chimerix, a small biotech company with no approved drugs in its pipeline, was able to negotiate with the Food and Drug Administration (FDA) to launch a small pilot study of 20 patients in Josh’s indication. This allows the company to collect data for product launch and also provides access to the drug for Josh. It seems like a win-win…or is it?
The Washington Post published an interesting story discussing the ethical dilemma this case, and others, have brought to light.
What do you think? I’m interested in hearing the thoughts of our readers on this topic. What are your thoughts on this story?
Until next time,
“This may be a checklist item at your job, but it’s my LIFE.”
I read the most recent issue of the ACRP Wire and I found the following article very interesting:
TJ Sharpe shared his perspective on being a clinical trial participant at a recent Scope Summit conference in Miami, Florida. I wanted to share his story with you again here in case you missed it in the Wire.
I was so impressed with his perspective that I’ve started following TJ on Twitter and I’ve read his other blog posts, available here.
TJ’s perspective and writings share a glimpse of what it’s like to be living with a potentially fatal disease and some of the challenges associated with finding and participating in clinical trials. I think it’s an excellent reminder of what it’s like for those whose only hope is clinical trials. I believe there’s much to be learned from his writings, like this quote from the blog referenced above, dated February 6, 2014:
Raise your hand if, diagnosed tomorrow with a serious illness, you would be interested in the most advanced, cutting-edge treatments available? I’m betting there are lots of you… good. Keep those hands up if you know where to find those treatments. Ok, ok, probably still a decent number of you.
Now – suppose you are sitting in your doctor’s office looking at scans, or blood tests, or even worse, doing these things in the hospital preparing for/recovering from surgery. Say your doctor told you the “standard of care” was Drug A, and this option was preferable to Drug B, C, or D, and “we should get started soon.” Would you know enough to ask about Drugs X, Y, or Z – ones that haven’t made it onto the market yet because they are still in the clinical trial phase? If you are in the 98% of Americans who undergo standard-of-care treatments, chances are, you may not.
According to TJ, 98% of patients don’t know enough to ask about drugs in clinical trials. No wonder clinical research recruitment is an ongoing challenge! No wonder there are timeline delays associated with clinical trial recruitment, leading to cost overruns and potential product failures due to a lack of statistical significance! All of these things are important to me as a clinical researcher, but when I read TJ’s blog, what struck me most was his summary of the impact of that statistic. He very eloquently stated:
Still, the question from the conference remains: “Are we doing the right things for the consumer – for the patient?” They are one in the same, but regular consumers make selections and purchase products or services. Patients… we make selections, we consume goods and services, but there is so much more depth to our decision-making, and the stakes are infinitely higher. This isn’t like picking between a sedan or SUV, steak or seafood. These are “life-altering, life-extending, potentially life-saving” judgments, made under the influence of medical professionals who may not know that cutting-edge treatments are available in research. I am in the 2% that knew to find the treatment and lucky enough to get into it. How many of the 98% suffer or die because they don’t have access to the same trials?
I encourage you all to follow TJ’s journey and allow a participant’s perspective to supplement your own perspective as we continue to try and improve how we do clinical research in the future.
Until next time,