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.
I got an opportunity to do my second panel discussion in the last 10 days, at the SCOPE Summit in Miami. The conference brings together stakeholders from the clinical trial landscape – pharmaceutical heavyweights, research institutions, clinical research operations organizations, biotechnology companies, and others in the healthcare field. There were about 600 people registered, and the conference included the typical “exhibit hall” set up with booths to chat up potential clients, distribute information, and hand out little tchotchke giveaways (Josie got a wind-up dancing robot, Tommy a branded stress-relief globe ball; and yes, I had to look up how to spell tchotchke). It was surprising how many for-profit businesses were associated with the machine that is clinical research.
I was part of a four-person panel discussing our role as the “customer” in clinical trials and the struggles we faced finding and enrolling in those trials. We were slotted as the second keynote speaker, after an excellent presentation by Jamie Heywood from PatientsLikeMe.com on real-time patient data measurement – it’s a fascinating site that aggregates patients’ shared medical and treatment information, so others with similar conditions can become more informed. I would absolutely recommend it for anyone facing a serious illness or chronic disease (there are nearly 100 conditions listed; besides the biggies like AIDS and cancers, it includes Fibromyalgia, Migraines, Hepatitis, Asthma, Glaucoma, Autism, PTSD, and everyone’s favorite, ED). There is literally something for everyone here, and it breaks down patient data into a comprehensive, yet extremely easy to understand, set of graphics, plus links to treatment information. My part of the discussion followed Jamie’s; for the second week in a row, it was the speaker’s version of a golf pro-am.
Presenting a small part of my story in a room full of people (a LOT of people… I wouldn’t say I was nervous, but there was a butterfly or two heading up there) was a great experience. The discussion was led by Christine Pierre from the Society for Clinical Research Sites; her organization represents the clinical trial sites (i.e. Moffitt and Holy Cross, but really anywhere a clinical trial is being held). She lobbed us softballs we had already discussed, which made opening up easier. The most difficult part was being short and sweet, as my readers and Philly.com editors know isn’t my strong suit. Each patient got a chance to share our challenges with the clinical trial process in a room full of people who sponsor and execute them – a great opportunity to give some real consumer feedback. Wouldn’t you like to get in front of Bill Gates when your PC acts up, or the late Steve Jobs when your iPhone can’t hold a charge through the commute home, or even Ed Snider when the Flyers pick up another overpriced sluggish defenseman? I got that chance on Tuesday.
The stories seemed very well-received; the other patients told about being discouraged by their doctors to explore clinical trial options, of a father who learned about clinical trial treatments too late to help him, of having to navigate the clinical trial landscape on their own with limited knowledge and support. I offered my story of the first trial being delayed waiting for legal approval, and threw some poor soul at Bristol-Myers Squibb under the bus in describing the eternal wait we endured for his or her rubber-stamp CYA approval – going so far as to use LinkedIn to bombard the BMS Legal Department with inquiries. (If you’re that BMS attorney or their admin assistant, I owe you a beer.) The part that resonated with the group was my saying, “This may be a checklist item at your job, but it’s my LIFE.” The phrase “Time to market” might be used inside the industry, but “time to treatment” is how patients – the customers – view the sometimes-agonizing delays from conception to availability. These delays may cost Big Pharma real dollars, but they also cost real lives – just ask Nick Auden’s family.
Look, clinical trials aren’t for everyone. There is a level of uncertainty with it that presents both physical and mental challenges. A quick primer: pretty much any prescription drug (and, I am guessing, almost all OTC ones, too) has undergone the clinical trial process. After lab testing is complete (which takes many years and significant capital), clinical trials are designed and VERY thoroughly reviewed for safety and compliance with standards. They are then opened up to sites that enroll patients. Data is collected and submitted to the FDA, whose job it is to ultimately decide whether a compound provides effective treatment without adverse effects.
Not surprisingly, this is a slow, expensive proposition with plenty of pitfalls if not done according to specific guidelines. It also operates very inefficiently; many trials cannot enroll enough patients to fulfill data, and those that do have a fairly dismal success rate getting through all three phases and FDA approval. It’s a catch-22; trials must prove via scientific method the drug is safe AND effective, but proving that requires “test subjects” (that’s me!) who are willing to forgo treatments with known outcomes for ones that may be no help at all, and could potentially have toxic side effects. No wonder there is a cottage industry connecting patients and trials (http://curelauncher.com/ is one free resource that is like the Match.com of clinical trials). In a capitalist nation, supply and demand continually push progress.
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?
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