I recently came across a rather unusual, but quite interesting study that looked at the PatientsLikeMe Web site and analyzed how the information reported by patients on the site could be a useful tool for both doctors and their patients when it comes to prescribing medications off-label. 

Identifying the Problem

Although off-label prescribing may sound like a somewhat questionable activity, it is actually a legal and common practice in the U.S.  When a pharmaceutical manufacturer develops a new drug for a particular purpose, the FDA evaluates the medication's efficacy for that specified purpose.  However, once the drug has been approved and is on the market, physicians are free to prescribe the medication as they see fit, whether it is for the FDA-approved purpose or any other purpose.  In fact, it is estimated that 21% of all prescriptions are given for off-label purposes. 

The reason pharmaceutical companies often don't seek FDA approval for multiple purposes is that it takes several years and costs millions of dollars to conduct all of the clinical trials required for each new indication they want to add.  And once a drug goes generic, there is little financial motivation to put that much time an effort into adding another purpose for the medication. 

Antidepressants and anticonvulsants are two examples of drugs that are often prescribed off-label for certain chronic pain conditions.  The mechanism by which certain drugs in those categories work to relieve depression or prevent seizures can also be effective in relieving pain. 

The practice of off-label prescribing has its pros and cons:

• Pros:  Some conditions (like ME/CFS) have no FDA-approved drugs so the only medications that can be used for treating its symptoms must be prescribed off-label.  Even if a condition has one or more approved medications, they may only treat certain symptoms, while an off-label drug might help improve other symptoms.  Also, patients are different and for some the approved drugs may not be effective, making it necessary to try other off-label medications.

 

• Cons:   Often there is little scientific evidence underlying the use of a particular drug for a non-approved purpose.  Prescribing a medication based only on anecdotal information or small studies can be risky for patients.  An example cited by the study authors: “In the past, millions of women received prophylactic hormone treatment for the prevention of osteoporosis...However, systematic evaluation revealed no therapeutic benefit and elevated risks of cardiac damage.” 

Purpose of the Study

In an effort to identify an additional tool that could supplement traditional clinical trials and enhance the understanding of how medications and other medical products perform once they are actually on the market and used by a wide variety of patients, researchers evaluated the PatientsLikeMe Web site.

As described in the journal article, “PatientsLikeMe is a web-based community and research platform where patient members share details about their treatments, symptoms, and conditions, with the intention of improving their outcomes. Patients join communities designed specifically for their condition. At the time of writing, there were 11 distinct patient communities and over 70,000 patient members. The site synthesizes members’ data into interactive reports for review. Each member sees a graphical representation of their own and others’ function, treatments, and symptoms over time and can view reports of aggregated data. The site includes an interactive treatment report for each medication and intervention that patients add to the system. The report includes dosages taken, time on treatment, and evaluations of the treatment, including perceived efficacy, side effects, and burden. These treatment reports can suggest usage patterns and effectiveness rates for drugs across different purposes.”