Clinical Trials

Look what you can do without any medical training whatsoever. A couple of weeks ago I opened the new Clinical Trials Unit at Wellington Hospital and for my efforts got a plaque on the wall. For that privilege, I had to entertain the guests for about 20 mins. Extracts of my speech follow.

"One of the decisions a cancer patient has to make is whether or not to take the advice of all the well-meaning friends and family members who prescribe cures so numerous that they would fill several lifetimes attempting to try them out.

Amongst the more benign are various fruit or vegetable concoctions, reiki, acupuncture, meditation, prayer and just thinking nice thoughts. And at the other extreme are vegan diets, the swallowing of hydrogen peroxide, and the excessive consumption of apricot kernels, with accompanying risk of cyanide poisoning. And these remedies are just what my friends have suggested!

As a scientist, I guess I instinctively gravitate to what some people call conventional medicine. There have been many wonderful discoveries in medicine. There have been antibiotics, vaccination, chemotherapy and the aspirin. But the possibly the greatest medical discovery, greater than all those things, is the randomized, double blind, placebo-controlled test. You get see if the treatment actually made a difference.

That’s it. That’s how scientific medicine has to work. It sounds simple once you grasp it, but it actually is rocket science. And it is rocket science because of human complexity, and especially because of that measurable phenomenon, the placebo effect. And when you design the test, carry out the study without bias, and then analyse the statistics properly, you get real evidence.

By the way, in my own scientific profession of physics, we seldom do statistics. Unlike the infinite variability of biology, and human patients in particular, atoms are atoms are atoms. Ernest Rutherford, who was never one to mince his words, said, “If your experiment needs statistics, you ought to have done a better experiment.”

He was referring to physics. But in biology, n=1 simply won’t do. And in drug trials, hundreds or even thousands of patients are the norm..........

.....Finally I want to make some very personal remarks, based on my own experience. You have a beautiful hospital, and yes, I know, you need more capital equipment. You have dedicated professional staff, staff who I suspect are overworked and probably underpaid, but the standard of care they give, at least in my experience, is mostly excellent. You have superb IT systems-just visit an Australian hospital at random and see the file boxes and cartons of documents all over the place. Take a blood test, a radiological image, and it is there at the physicians demand. People might call me Paul Callaghan, but I know my true identity. It is EYD7212. When I flash my neutropenic alert card at Accident and Emergency, and utter the magic words EYD7212, they know to whip me past the Courtenay place casualties and straight to the intravenous drip line through the double doors.

But there is also a culture here that we should not take for granted. The personal kindness and care that I have had here has been remarkable. In the Blood and Cancer centre, and what a marvelous place that is, I get to sit among the comrades, greeted by staff who know you personally, who, when asked the cost of the drugs they are pumping into you, feign complete ignorance and unconcern. The district nurses call you at home to check that you are coping well, and they smooth the information flows with the oncologists. And then there are the nurses on the wards. How can anyone be bored for a moment in this hospital, especially with the theatre of the consultants rounds? And what consultants! They have taken the time to talk through difficult issues, treating their patient, at least this one who wanted that, as worthy of an informed conversation, and when asked, they were prepared to partner with other hospitals or the private radiologists and consultants, and all with the help of the cancer society and its programmes that are able to address broader patient needs over and above what can be provided in the busy operation across the road. I have been incredibly lucky.

There is much to be proud of here, and in the profession you practice. And speaking personally, I have come to like the eccentricities of medical practice. The idiosyncracies of surgeons particularly-what a craft that is, beyond contemplation, no double blind placebo-controlled stuff there. Just daring and skill and fearless opinion. I should know, my son is one.

And then there are the oncologists with their difficult role in patient dialogue, telling it tough, -no sugar coated pills there. Thank goodness I know about right-skewed survival statistics (let’s face it, the lower bound is zero, they can’t tell you that you will die yesterday, but of course the upper bound is decades), so bugger the median I say, I’m aiming for the right skew, but bless ‘em, no oncologist will give you false hope by pointing to its existence.

For my first course of chemotherapy, and just to get me started, my oncologist referred me to adjuvant online so that I could read the data for myself. I’ve been a fan for clinical trials and their data ever since. Oxaliplatin, Capecitabine, Irinotecan. Cetuximab, Avastin, I’ve read them all. And for each there is an evidence base for efficacy based on clinical trials.

It doesn’t mean they will work for every individual and that fact is honestly declared, but the fact it that each one has passed a Kaplan-Meier analysis gives me that confidence that it is worth trying it, that it won’t unduly harm me, and it might actually do me some good.

That’s more than can be said for apricot kernels and hydrogen peroxide."

http://tvnz.co.nz/health-news/cutting-ribbon-research-4147548/video