“The Missing Voice of Patients” in Drug Safety Reporting

physiciansThat is the title of an important paper published in the New England Journal of Medicine just last week. You can read the full text article at your leisure, for free, by clicking on the link.

Each and every one of us who had to make therapy choices has agonized over adverse effect listed in clinical trial reports, worrying over the toxicity profiles and hoping that some how we will be among the lucky ones. That somehow we will be in the top of the class when it comes to response and remission, but the nasty stuff of adverse effects will pass us by with merely a nod and a wink.

Who collects the data on adverse effects? Who gets to say whether the fatigue was just garden variety tiredness that only a wimp would complain about or really mind-numbing nightmare that makes it hard just getting out of bed?

Pain and suffering are hard things to measure. We each of us experience the world through our own eyes.  Each of us registers pain through our own nerves, in our own bodies. Your bruised big toe is a lot more painful to you than someone else’s fractured leg.  All the same, I assumed some effortis made to collect adverse effect data based on what patients described, that there was some semblance of  authenticity to the reported data. I was naïve. I was wrong.

Watching Pain, Dispassionately

Here is how it is done. I must warn you, reading this review is going to be a little bit like watching sausages being made. Someone told me that once you watch the process you will never be able to eat sausages again – your innocence is gone forever. What do I know – I am a life long vegetarian. I never ate sausages anyway.

  • “Adverse effect information, largely collected during clinical trials, is based almost entirely on clinicians’ impressions of patients’ symptoms – not on patients’ own firsthand reports of their experiences with the drug”
  • The assumption is that it is safe to trust physicians report the subjective experience of their patients without bias.
  • Yet, as this paper points out, there is substantial evidence to disprove this assumption. Physicians systematically and consistently down-grade the severity of symptoms experienced by their patients.
  • It has been shown that patients’ self reports frequently list symptoms and adverse effects that physicians’ reports fail to capture at all, which means these adverse effects are not addressed at all in future generations of patients.

The Art of Listening

Call me a nostalgic romantic, but I am convinced there used to be a time not so long ago when physicians and patients had a much closer and more trusted relationship. We were not too intimidated to talk, and they took the time to listen, really hear what we were saying.

MSKK (Memorial Sloan-Kettering Cancer Center, New York) did an interesting study. 467 patients with breast, lung, urinary or gynecologic cancers were asked to report their symptoms at each clinic visit. The physicians and nurses treating these same patients were also asked to list adverse effects in the usual manner. Below is how some very common adverse effects were rated by the patients and their physicians. The gold curves are patient reports and the blue curves are physicians’ reports.

adverse reporting one

Adverse reporting two


I was blown away by how consistently the gold (patient) curves soared over the blue (physician) reports. Just for example, physicians reported a mere 5% of the patients experienced appetite loss 25 months from the start of the study. A whopping 35% of the patients reported the same adverse effect. The same trend is observed in each of the categories.

It is clear that physicians’ reports are not capturing all the adverse effects experienced by patients.  Patient generated adverse effects reports might have resulted in earlier detection of some highly publicized scandals, such as suicidal tendencies related to some antidepressants in younger patients.  So, why is this not standard practice, get cancer patients undergoing therapy to fill out adverse effect questionnaire each time they visit the clinic? You would think this is a pretty logical thing to do.

There have been numerous objections to adopting a patient-focused approach to safety monitoring. Opponents have trotted out “regulatory constraints”, “confidentiality issues”, “feasibility concerns” “increased administrative costs” and lack of properly constructed “questionnaires”. What a bogus list of complaints! Each and every one of these road blocks can be overcome, if only there was a real desire to hear what patients have to say.

Is there anything we can do to change this status-quo? I do not know. We need to brainstorm together as a community, get some consensus on how we feel about it, how strongly we feel about getting our first-person feedback heard when adverse profiles are being compiled. I want each of you to think about what you are willing to do, your individual contribution to the greater good of this patient community. Our ability to influence things is only as great as our determination to be a determined and cohesive group, each of us working to provide grassroots support for the advocacy efforts that our community takes on as a whole.

On A Personal Note…

I am struggling with the stressful job of packing. The date is set. I will be leaving my home in sunny Sedona (Arizona) and moving to Columbia (Maryland) in the first week of May. As I sort through papers and stuff, deciding what to keep and what to discard, I am going through a kind of emotional archaeological dig – debris from more than a couple of decades of my life with my husband PC. Some of the memories bring a smile to my face, some are downright painful. So it goes. But it is time for me to move on; downsize, minimize and simplify my life. And be closer to my daughter and son-in-law.

One of the advantages of living on the East Coast is that I will be in the thick of things, a mere stone’s throw (more or less) from the NIH and its impressive library etc. As soon as I am settled in my new home I will be volunteering to put up CLL patients visiting the NIH and needing a place to stay for a day or two.

I also plan to have monthly CLL meetings for patients who live in this general area. Over time, as we gather momentum, it will be fun to invite some of the East Coast CLL experts as guest speakers.

Living in Columbia, MD will also give me a chance to see more of you on a one-on-one basis. Sedona is a vacation destination and I have met some of you over the years while you were here on holidays. Now I can make myself available for personal meetings with more of you – those of you who want to.

The world being what it is today, my daughter worries about my security and insists that we retain full rights to control and limit participation in either the monthly meetings or individual consultations. At the very least, participants will be limited to our long term members / donors. That protects all the participants,  none of us want snake-oil scammers or drug company reps crashing our meetings. I will publish all the details a little later.

Wish me luck in my new home to be.