Selecting Treatment Options

Reported by Joyce Graff

On April 23, 2010, I attended a conference on Benefit-Risk Considerations in making drug Regulatory Decision-Making, co-sponsored by the U.S. Food and Drug Administration (FDA) and the New York Academy of Sciences. 

There were twelve very fine presentations from many different perspectives – FDA regulators, doctors, scientists, pharmaceutical companies, consultants, and even patients, from the U.S., Canada, and Europe.  It all came down to a few essential criteria – define “benefit” and define “risk”.

Dr. Nananda Col from the Maine Medical Center gave us a glimpse into the “informed decision making” that she goes through as a physician.  She used the example of recommending a course of action for a patient with osteoporosis.  What should she prescribe?  What are the pros and cons of each of the medications on the market?  What is the easiest way for a physician or a patient to obtain this information?  On Lowe’s or Home Depot’s websites you can select two or three candidate refrigerators or dishwashers and ask them to compare them for you – build a grid that shows you at a glance the characteristics of each, laid out in a similar way for easy comparison.  No such tool for drugs.  She went to WebMD, HealthCentral, and a variety of other websites and found articles about each of the many possible therapies, but nothing that would easily compare them.  There is a convenience factor -- What is the most convenient way to administer the drug for this person (as a pill, an injection, an infusion, once a day/week/month/year)? 

Since most of the drugs have very similar benefits (reduce the possibility of bone breakage), the real job is to choose among the various risks – for this patient, are we willing to accept an increased risk of heart attack, blood clot, stroke, cancer, stomach ulcers?  It would be helpful to have statistics to indicate the level of risk (2% or 20% or 60%?) of each of these things, or whether people with a specific set of characteristics were at greater or lesser risk for these negative consequences. 

Nananda Col., MD, MPH, FACP, Director of the Center for Outcomes Research and Evaluation at the Maine Medical Center Research Institute, Porland, Maine

Dr. Col asked in particular that the FDA provide a mechanism for reporting outcomes so that better information can be collected on the adverse events experienced by people taking any drug.  This information is currently held by the pharmaceutical companies, and is often downplayed in the drug information fact sheets.  “Blood clots have been reported” is not as informative as information about what percentage of people with a history of what kinds of health issues experienced problems.  She feels that the government is in the best position to do this, since no one else can be relied upon to be truly neutral – the medical resource sites gain their revenue from the drug manufacturers, which gives them a certain bias toward a drug-based solution (rather than simply exercise and Vitamin D), and potentially toward a particular client.

Until that happens, she suggests that patients should read the fact sheet that goes with the drug, and ask the doctor or the pharmacist to explain anything that is unclear.  If the patient sees something worrisome in the fact sheet, it is best to discuss it with the doctor.  

There are no perfect drugs with all benefit and no risk.  Every medication comes with a certain level of potential harm.  We choose to accept the risk in order to gain the benefit.  But each doctor and patient must weigh the possible benefit against the risk and decide … in this situation, with this patient, and this person’s total state of health, is the possible benefit to be gained worth the potential harm?  These are never easy questions, there is always a great deal of uncertainty – both around the benefit and around the risk.  Will the full benefit be achieved?  Will any of the possible risks ever occur?

We are all working to reduce the uncertainty and improve our ability to predict both the benefits and the risks.  This is true of all drugs, and will be critically important as VHL enters the Brave New World of drug therapies for VHL tumors.

Dr. Gladys Glenn of the U.S. National Institutes of Health reminds us of two things we need when using any drug that is new to your body:

(1)  Close monitoring early (blood & urine tests; rule out new symptoms) during the initial phase of the treatment, so as to identify early any developing adverse effects.
(2)  Early assessment to determine anti-tumor activity (using imaging and biomarker(s)).

Be well . . . and be a Powerful Patient!

As printed in the VHL Family Forum 18:2, May 2010. For permission to reprint, please contact VHL Family Alliance, editor@vhl.org. Further information is available from the VHL Family Alliance, info@vhl.org.