Posted by on November 25, 2014 - 4:16pm

In response to the call for more sex inclusion data in drug studies, the FDA has developed  Drug Trials Snapshot a pilot project to provide information about the sex, age, race and ethnicity of clinical trial participants for a small group of recently approved drugs. In addition to information about who participates in the trial, each Snapshot also includes information on how the study was designed, results of the efficacy and safety studies and, if known, differences in efficacy and side effects among sex, race and age (referred to as subgroups).

While this is certainly an important step toward inclusion, the recently posted six examples reinforce the lack of racial minorities in all studies and the lack of women in many studies.   These drugs were approved over a two month period in 2014.  In summary, of the six examples provided:

  • All studies, except one, had more males than females in the clinical pool
  • All studies except one, reported that the drug achieved the desired response (efficacy) in both in men and women (one study showed the the drug trended in favor of females though only 23% of the study subjects were female)
  • Four studies indicated the side effect (safety) profile was similar in both sexes; one study did not evaluate safety; and one study found increased risk for women even though only 24% of the study subjects were female).
  • All studies showed a considerable lack of minority race/ethnic participants .

"‘This new report provides a clarion call to action for the scientific, medical and regulatory communities to ensure representational science, medicine and the approval process. By taking strong, decisive action today, we can be assured a healthier tomorrow for all people." says Teresa K. Woodruff, PhD, director, Women's Health Research Institute and a national leader in the movement toward sex equity in science.


Posted by on March 5, 2010 - 12:07pm

Throughout my career, I have been a strong proponent of medical research through consumer advocacy and educational activities. But recently, I had the opportunity to actually participate as a research subject!  As an enrollee in the Illinois Women's Health Registry, my health profile matched the criteria that a researcher was seeking for an osteoarthritis (OA) study.  The project is called the MAK-3 Study and its purpose is to explore whether people with stronger hip muscles have a slower rate of OA progression and whether different factors of knee instability are related to knee OA progression.

My camera flash picked up the reflector balls/markers taped to my joints ---  could this be the new look in jewelry!!

My Registry profile included "knee pain" which flagged me as a potential match for this study.  I gave permission for the Registry staff to give my name to the research coordinator for the study and I was selected!  The study is being done at Northwestern University and it involves 2 or 3 visits for a variety of non-invasive evaluation procedures.   They would be done at baseline (this past month) and repeated two years later.   This is what they call an "observational" study.  It will not directly benefit me, but it would help scientists better understand this condition and the factors that influence it.  Since my family was riddled with osteoarthritis, I wanted to participate.

I chose to do the evaluations in three rather than two sessions because it fit my work schedule better.   At the first session, I filled out numerous surveys about pain, my medications and my exercise habits.    A physical therapist watched me walk down the hall several times to observe my "gait".    I was then hooked up to equipment that measured muscle strength ( it looked like much of the equipment you see at your local gym, just with gauges to measure strength). They asked me to perform some typical knee and hip movements like leg lifts and recorded my muscle strength.  After that a physician examined my legs and hips and reviewed my medication history.  I was asked if I would provide an optional urine sample that would be stored to eventually be used to test any new biomarker tests for OA that may be developed.  I was provided a diary where I recorded by physical activity for a week at home.

At the second session I met the team in a research lab that was equipped with sensors along the ceiling and a hollow floor that ran wires to a lot of computerized equipment. They taped small reflective balls to my joints and had me walk up and down the room many times while the sensors picked up the signals from these balls  (see picture!)    When I finished, the researcher showed me what the sensors were detecting.    The computer screen showed a stick figure (me) with multicolored dots on my joints (representing the reflecting balls) moving across the screen.   They were recording my alignment and gait.   It was very interesting to see my stick figure move across the screen.    Once they pulled off the sensor balls, they measured the speed of my normal walk by making me walk down a hallway and timing me.

A few weeks later I went to a different location where I had a special x-ray taken that included, on one film, my ankle, knee and hip joint to see how they lined up.  This was followed by rather long MRIs on each of my knees.   This was the hardest (but tolerable) part of the study, I needed to stay still for 40 minutes per knee.   At least they gave me headphones and my head was outside the MRI machine where I watched a bird trying to get through the window.  Throughout this experience the research coordinator explained what would happen and why it was done.   Since I was going to physical therapy for my knee at the time, it was helpful to have her explain where they saw strengths and weakness in my hips and knees.  It made me appreciate why the PT was having me do certain exercises on these related muscle groups.

Finally, the best part of this experience was knowing that I may actually be contributing to science, with little interruption (total 7- 8 hours) in my life.   They will have me back in two years to repeat these tests and determine the status of my OA symptoms.  By the way, I was given a stipend to cover my expenses like parking---and  just enough  to take a few girlfriends out for a nice lunch and get them to join the Illinois Women's Health Registry!

Posted by on February 5, 2010 - 5:07pm

Every day we hear about a new drug, screening tool or technology that promises to improve our knowledge of health and disease.    Generally, the news is good, or at least hopeful, reassuring the public that important research is progressing.  Often, the new breakthroughs  have been demonstrated in the lab or in animal models and has not yet been studied in humans,  a process that could take years.  Sometimes the lab findings do not translate well to the human and the researcher may have to start all over with a new strategy.   This is particularly frustrating for individuals who have a disease or condition that is debilitating and could be deadly.  The new movie, Extraordinary Measures,  is the story about a father who self-funds a researcher to accelerate the process for a promising drug for Pompe disease because his children have the condition.  It is a great example of how driven and focused we can become when our own loved ones are ill.

The research community is challenged when what we  know now (based on the science technology and data available at the time), changes when new technology allows us to further explore a Slide23particular issue.  Or when there is more data in a statistically significant pool that truly reflects our population diversity and allows us to determine differences that may exist among different races and ethnicities.  This could lead to different approaches for certain populations.    We'd like to think of research as finite but it is a constantly revolving cycle.  One important example is the recent mammography guideline debate.   Several years ago, mammography screening became mainstream:  most women knew the guidelines, advocates worked to pass laws to assure that all women had access,  and early studies showed that finding breast cancer early, in many cases, improved survival rates.   Last November the U.S Preventive Task Force new mammography guidelines that were posted by Candace  questioned the benefit of the current guidelines as applied to the general population.  It should be noted that this is the first update by the Task Force since 2002.   This led to conflicting reports of support verses outrage.  There are some valid arguments on both sides and the new report was based on additional data.  The outrage of strong proponents of mammography often overlooked the details of the report that acknowledged that high-risk women may want to discuss their screening intervals with their health care provider.   On the other hand, the team behind the report were criticized for not fully explaining that there were high-risk populations that required further study.   These exceptions would not change their overall recommendations, but they could have made it clearer that there could be exceptions that do not apply to the population at large and that they were sensitive to the emotional backlash this report would produce.

Advocacy is a powerful activity that has led to increases in research dollars and more sensitive care.   But sometimes advocates are so focused that they do not see the big picture.  An example of advocacy compromising good science is the debate in the 1990's about High-Dose Chemotherapy with Bone Marrow Transplant or Stem Cell Support (HDCBMTSCS) as a treatment for breast cancer.  HDCBMTSCS was seen as a promising aggressive treatment for the growing number of women who were being diagnosed each year with this disease.   As appropriate, randomized controlled clinical studies (the gold standard that compared a new treatment with the latest standard of care) opened and tried to recruit women.   However, many interested women did not want to be in the control group.   After all, more is better!  To respond to this demand from advocates, many hospitals opened bone marrow transplant units and insurance companies were pressured to cover this "experimental"  treatment despite the lack of clinical trial evidence.  And what happened?    Women by-passed the trials and went to the institutions that offered the treatment outside of clinical trials.  This delayed the evidence-based findings that eventually concluded that HDCBMTSCS was not better than standard treatment for breast cancer  (it does offer a good option for certain other cancers) and that many women suffered far more severe side effects (that sometimes resulted in death) with little improvement in outcome.

So what do we do?   It is important for men and women to understand enough science to enable us to interpret studies without the bias of headlines.   Programs like Project Lead sponsored by the National Breast Cancer Coalition trains consumers to understand the basic scientific principles behind research studies.   We need greater participation in well-designed clinical trials so we can get answers sooner so better therapies become available sooner.     Finally,  look for portals to research studies that not only study disease, they also look for better ways to prevent disease.   One such portal is the Illinois Women's Health Registry.  Hopefully, more of these gateways will become available.