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Do-It-Yourself Clinical Trials

Single-patient clinical trials may get the right drugs sooner to patients who are willing to do some extra work.
August 1, 2001

For all the science behind today’s drugs, it’s often a trial-and-error process to find the ones that work best for you. A new clinical-trial system promises to speed this process by testing how individual patients respond to prescription drugs.

Beginning this fall, Opt-e-scrip, a mail-order pharmacy in Morristown, NJ, will release patented test kits for up to 16 common chronic conditions, such as allergic rhinitis (runny nose), osteoarthritis (painful knees) and gastroesophageal reflux (heartburn). The test kits, containing blister packs of pills and a daily diary for patients to record their reactions, will be available by prescription.

No Magic Bullets

The idea is to help doctors make better choices when prescribing medications. Even though prescription drugs go through a rigorous set of clinical trials before they are approved for sale, it is hard to predict which patients they will help. Reactions to drugs vary by age, sex, activity level, biology and other factors. Some drugs work for only 50 percent or fewer patients, and placebos or “sugar pills” can sometimes work for almost as many.

For example, clinical-trial data from Schering-Plough indicated that patients taking its allergy drug Claritin showed a 46-percent improvement in symptoms. But patients unknowingly taking a placebo reported a 35-percent improvement.

Such results often leave doctors relying on their wits when prescribing drugs. The Opt-e-scrip test kit system could eliminate some of the guesswork.

Clinical Trial in a Box

The Opt-e-scrip system works much like a regular prescription, but with a few more steps. The doctor writes a prescription for a specific test kit, specifying the drug and protocol to be used. Three kinds of kits will be available: the test drug compared to a therapeutic substitute such as a generic drug, the test drug versus a placebo and the test drug at different dosages.

The patient takes a series of unlabeled pills, which look alike, for a month and records each day’s reactions in a diary. A kit comparing two allergy medicines, for example, might involve taking Claritin for one week and an over-the-counter drug like Chlor-Trimeton the following week; the two-week cycle is then repeated. When the course is complete, the patient sends the diary to Opt-e-scrip, which analyzes the responses and sends a report to the doctor.

The diary includes a section for rating symptoms that are present before each drug dose, such as sneezing or runny nose for allergic rhinitis. Another section asks the patient to rate the severity of adverse effects such as headache, sleepiness or dry mouth. A third asks for a ranking of symptom relief on a scale from poor to excellent.

Opt-e-scrip claims accuracy is improved by comparing new test results against its database of results from previous single-patient clinical trials. (Patients sign a consent form before their information is put into the database.) This method gives better statistics for an individual patient, says Dr. Donald Reitberg, company co-founder and scientific affairs president.

Data analysis might show, for example, that patients who have a demographic profile and test results similar to those of the new patient have a 90-percent positive response rate when they are prescribed the recommended regimen of drugs, versus only 40 percent if they take an alternate drug.

“This system identifies the best care for the patient,” says Dr. Nardo Zaias, a dermatologist who tested experimental Opt-e-scrip kits on patients needing antihistamines. Zaias also runs a clinical testing service in Miami Beach, FL.

A Simple Idea Overdue

Opt-e-scrip received a patent in June for its method of administering single-patient tests and aggregating test results to indicate how patients of a certain age, gender, race and health profile react to a given medication.

While it might seem like an obvious invention, Opt-e-scrip claims to be the first company to pool individual test results in a systematic way. To prescribe medications, doctors commonly rely on observations from their own experience, as well as anecdotal information from colleagues. So far they have had little else to confirm the claims made by pharmaceutical companies.

“Physicians usually prescribe medicines based on a lot of trial and error. So the notion of comparing two medicines, or comparing a medicine to a placebo, is very compelling,” says Dr. Steven Peskin, president of Nelson Managed Solutions, a Lawrenceville, NJ, health-care consulting company.

The Opt-e-scrip system adds “scientific rigor” to the process of finding human preferences for medicines, he says.

One limitation to the system, however, is suggested by Dr. David Berenbeim, vice president of clinical solutions at PacifiCare Health System’s Prescription Solutions subsidiary in Santa Ana, CA. Single-patient trials are more suited to stable, chronic diseases such as allergic rhinitis or migraine headaches, he says. He would not recommend them for unstable or progressive diseases such as diabetes or Alzheimer’s, which require more frequent monitoring.

Cost Savings for HMOs

Spending on prescription drugs increased by a record 17.4 percent in 2000, according to a recent study by Express Scripts, a St. Louis drug-benefits company.

Opt-e-scrip says HMOs could reduce costs by objectively evaluating chronic-care drugs and switching patients to less expensive drugs when warranted. One large HMO has already signed a letter of intent to try the Opt-e-scrip system, says Reitberg.

Test kit prices will vary based on the therapy, but in each case the price will be based on the total number of doses in the kit, multiplied by the price of the most expensive drug. For HMOs, this should produce the same cost and co-payment as using the expensive drug itself for the time of the trial.

Overcoming Inertia

The big question for the approach is whether doctors and patients will be willing to take on any added effort.

“In theory it sounds like a nifty idea. But if it’s more complicated than tying your shoes, you’re not going to get widespread adoption,” says Robert Seidman, chief pharmacy officer at WellPoint Health Networks in Woodland Hills, CA.

Zaias, the dermatologist who tested the Opt-e-scrip kit, thinks the idea will take off in the next couple of years, after doctors see it in use. “This took a little more of my time, but the patients were extremely grateful,” he says.

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