When considered together, two apparently unrelated reports in the April 1996 issue of the Academy Bulletin raise an important issue about clinical research. The first report is the "Update" on the pedicle screw controversy. The second relates to the comments of several leading orthopaedic researchers regarding the importance of clinical trials to evaluate new technologies.
In their discussion of clinical trials, Drs. Victor Goldberg, Richard Coutts and Gary Friedlaender very correctly focus on the need for clinical trials to confirm and validate the results of basic research and technology development. There should be no argument about this. Recently, however, it has become apparent that it is often necessary to add a second level of clinical research - one which should become a step in the progression from technology development to wide-spread clinical dissemination.
The two levels of clinical research - efficacy and effectiveness - are distinct, and there is a logical progression. The clinical research advocated by Goldberg and colleagues represents efficacy research. After new technologies are developed at the basic science level, efficacy studies should be undertaken to ascertain if the proposed technology works in tightly-controlled clinical trials. Next, effectiveness research should be carried out to determine if the beneficial outcomes noted in efficacy studies are reconfirmed when the new technology is utilized in community practice. Prospective, community-based effectiveness studies evaluate patient-outcomes at the level of broad, general usage - a point where both physicians using the technology and their patients are much more variable than in randomized trials.
In orthopaedics, there are examples of apparently beneficial new technologies that never went through adequate efficacy research before wide dissemination - certain joint implants are an example of this. Chymopapain injection for lumbar disc herniation is an example of a technology which passed efficacy evaluation in a carefully controlled randomized trial. However, effectiveness studies were never conducted prior to general release of the drug. Once available, chymopapain therapy was rapidly implemented at the community level. In most physicians' hands it was ineffective and rapidly disappeared from general use, but not without significant medical, disability and other costs. There are many other examples.
This leads to consideration of the pedicle screw issue. There is little doubt that when properly applied for the proper indications, pedicle screws work well. However, efficacy and effectiveness studies were not conducted to evaluate this technology, and it became rapidly disseminated and implemented by surgeons across the country. The inability of experts in spine surgery to be able to document the efficacy and effectiveness of pedicle screws led the FDA to require that a retrospective study be undertaken. It is generally agreed that retrospective studies are much less definitive than well-developed prospective trials.
If the developers of this clearly useful technology had undertaken the clinical trials recommended by Goldberg and colleagues and followed those trials with community-based effectiveness research studies, much of the adverse publicity, public confusion and litigation which has occurred might have been minimized or even avoided.
The lessons learned from pedicle screws, chymopapain and other new technologies should make it clear that these methods should not be widely disseminated until they have been proven to be of value by a careful process of basic research and development, efficacy trials and finally, effectiveness studies. The potentially frustrating delays in permitting broad dissemination would be more than offset by the avoidance of the difficult and expensive process that has characterized pedicle screw controversy.
Robert B. Keller, MD
I have always been proud to be a member of such a forward-thinking Academy. Immediately grasping new trends, like arthroscopy, the establishment has never attempted to thwart progress even if it endangered their status. Through programs like OKU and the Orthopaedic Learning Center it pulled our specialty to the top of medicine.
I have just finished the course, "Winning at Risk," and my faith is reaffirmed. Rather than fight for the status quo or stick our heads in the sand, the Academy searches for the proper response to the ever changing environment. I applaud the effort. The course was exceptional. It gave the attendees a cynosure to follow, not just how to win but how to change the landscape. It called for us to be proactive.
Peter Senge talks about the tension between goals and the true reality as creative. This course was just that creative tension. Using that creative tension the orthopaedic community can use its knowledge-base to change the face of the present health care environment. Please thank all of those involved.
Warren F. MacDonald Jr., MD
Cape May Court House, N.J.