The Benefits of Clinical Trials

April 14, 2000 (Washington) -- A remarkablestatistic arrived the other day from a company that recruits people toparticipate in clinical studies: 80% of clinical trials fail to enroll therequired number of patients on time.

The statisticdefies logic. People who enroll in these studies often getbetter medical care than most other patients, can get anadvanced therapythat generally is notavailable to other patients, and usually receivefollow-up monitoring to assure theirsafety.

Why, then, isit so tough to find people willing to enroll in a clinicaltrial?

There seems tobe a number of barriers:

  • The medicalcriteria usually are strict. A clinical study can reachvalid scientific conclusions only if all the patientshave similar medicalconditions and meetvery exacting requirements. Sometimes it's hard to findpatients who meet all the criteria.
  • Many patientsbelieve that they may get a placebo, or dummy pill, if theyenroll in a clinical study. It is true that many studiesareplacebo-controlled -- that is, somepatients receive a placebo and othersreceive the drug being studied. It is also true thatneither the patientnor the physician mayknow which patients are receiving the active drug. But not all studiesareplacebo-controlled. In studies formedical conditions for which effective and safe treatments already exist, noone receives a placebo; patients get either the new drug being tested, or theaccepted, approvedtreatment. Beforedeciding whether to participate in a clinicaltrial, a patient should know whether the alternativetreatment is a placebo.
  • Recent newscoverage of gene therapy trials, in which one teenager died duringa study, may be scaring off many prospective enrollees.This isunfortunate, but the reality isthat recruitment was difficult before this death occurred.
  • Clinicaltrials can't even get respect in the theater. A play called"Wit," which recently appeared in Washington, isa powerful drama about aterminal ovariancancer patient ("I'm at stage IV; there is no stageV," she says) who is enrolled in a study of anexperimental drug. Thecharacter'sphysicians treat her more like a test subjectthan a human being. No one would want to be in aclinical trial afterseeing thisplay.

Experts whoknow how clinical trials actually function are in disbelief thatthe American public views them negatively. Carolyn R.Aldige, president andfounder of the CancerResearch Foundation of America, tells WebMD: "Anyonewith a cancer that cannot be effectively treated shouldaggressively seekout participation in aclinical trial. The chances of receiving the latestand best treatment is so muchhigher."

Aldige'sorganization is sponsoring a national survey in hopes of gaining abetterunderstanding of why people arereluctant to enroll in cancer trials. In cancer trials, she notes,patients always receive either the best standardtreatment or an additionaltreatment thatmay be even more effective.

Aldige pointsout that clinical trials have a high degree of protection forpatients. Every trial is approved by the FDA to assurethat the productbeing tested will be safe,and by an institutional review board, consistingof experts at the institution where the trial is takingplace. The reviewboard, among otherthings, assures that patients receive theinformation they need to decide whether toparticipate.

A patient mustsign an informed consent form to participate in a study. A good informedconsent form will tell them what the therapy is for, whetherall patients will receive an active drug, what thepossible or expectedbenefits are, and whatthe risks may be.

Duringclinical studies, extensive information is gatheredfrom the patients -- their disease or condition, howthey react to thetreatment, and potentialside effects, which will be monitored.

The healthsections of newspapers are filled with advertisements forclinical trials, for everything from sleep disorders todepression to highcholesterol tomenopausal symptoms. Drug investigators must advertise torecruit patients with the exact criteria they areseeking. Increasingly, the Internet is beingused for recruitment. The National Institutes of Healthrecently launched its own web page to listclinical trials and the medical criteria forentry:

Aldige andother experts advise people with diseases that do not have fullyeffective treatments to explore -- through theirphysicians, newspapers, or the Internet -- the possibility of enrolling inclinicaltrials. Not only will thesepatients benefit, but the more people who enroll, themore quickly new therapies will become available forpeople who may need them in the future.