Evaluating the Usefulness of an "Innovative" Clinical Procedure - How Can We Really Know If It Works?, 8/1998

There is an old clinical axiom: If there are more than two treatments for a disorder, there is probably no good treatment. The treatment of several of the disabilities associated with cerebral palsy is a case in point.

We are now fortunate that there are several reasonably good treatments for minimal or moderate spasticity of limb muscles (arms; legs). None are perfect, but when used wisely and specifically, they often provide for significant improvement in function and in activities of daily living. Most established drugs or surgical interventions for limb spasticity are part of a treatment plan which also includes a program of physio-therapy, occupational therapy, speech therapy and/or exercise.

However, when there are problems of body trunk control (e.g.: athetosis) or control of impaired swallowing or of severe spasticity of limb muscles, there are very few interventions available -- if any -- that provide for a useful improvement in function. At best, established therapies generally are useful in these situations primarily for preventing secondary complications of spasticity; this is important but hardly ideal.

It is in these situations of more severe limitation of function that "innovative therapies" are often attempted; examples are conductive education, the Adeli suit, hyperbaria, functional electrical stimulation, biofeedback, herbs and nutrients. It is also in these situations where the useful evidence for evaluating the effectiveness of innovative therapies is poor or lacking.

Innovative therapies are generally characterized by periods (sometimes long periods) of special attention to the person with the disability; the unbridled enthusiasm and support of the person by a hopeful and frustrated parent or other care giver; a change in the environment of the person (going to a clinic -- sometimes in another city or country); participating in group sessions with other persons with a similar disability which often includes group self-encouragement; a short-term improvement in some function; and too often, a longer term return to the previous functional status. Unfortunately, they are also often characterized by a profound feeling of guilt on the part of parents or other care givers who feel that they failed to participate adequately in the home phase of the treatment program when the loss of function returns to its previous level.

Sound familiar?

However, in the growing list of innovative therapies, there may be interventions that might be useful generally, or for a specific type of functional limitation. We hope so. But the information usually available is the enthusiastic and theoretical explanations of the therapy's originators or therapists reinforced by the anecdotes of often desperate parents and care givers. This type of evidence is just not good enough to satisfy experienced clinicians, clinical scientists or third party payers. And it shouldn't be good enough for you!

However, the Foundation does understand the urgent need for better ways of improving functions. What is absolutely needed to answer the question of usefulness is a controlled clinical trial.

A controlled clinical trial usually requires that there are: 1) a group of persons with a defined disability who are available for study; 2) clinicians skilled in the innovative therapy dedicated to its evaluation; 3) two treatments, the outcomes of which are being compared; 4) a pre-established set of entry and exclusion criteria in order to insure that the comparisons among treatments are being done in similar groups of people; 5) an evaluator of the treatment results who does not know which treatment was used; 6) a specific time after completion of the treatment (or if a prolonged treatment, during the treatment) in which it is evaluated by a pre-established set of criteria; and 7) hopefully, a long term follow-up (3-5 years).

A "control group" is an essential ingredient of a clinical trial. The outcome (results) of the innovative therapy in one group (the study group) is systematically compared with the outcome in another group who did not receive the therapy being evaluated -- the control group. The control group may be defined as a historical experience (a relatively poor control group), a group receiving no therapy, or more commonly, a group receiving a generally accepted therapy. Sometimes the study group and the control group both receive the same therapy, but the study group in addition receives the therapy being evaluated. This procedure of a control group helps answer the question: the usefulness of the innovative therapy as compared to what?

In this way, the results of treatment are evaluated in two groups of similar persons by an expert who is "blind" to which treatment was administered. Is there a significant difference in results? Is the beneficial result maintained? This research technique is used in evaluating the usefulness of interventions in cancer, heart disease, stroke, prosthesis for amputees, etc. Why not for the innovative treatments being proposed for the disabilities associated with cerebral palsy?

The Foundation is prepared to be of help to persons (clinicians, advocates, organizations and care givers) seriously committed to organizing and conducting such studies. We have said so for several years.

Why there are no takers?