Editorial commentAre randomised trials needed in the era of rapidly evolving technologies?

Avicenna or more authentically Abu Ali-Al Hussain Ibn Abdallah Ibn Sina, the 11th century Persian physician and philosopher, is credited to be the father of the concept of testing the effectiveness of new drugs by trial. From this concept of the testing of treatment efficacy based on rational thinking, observation and outcome assessment, there evolved, 10 centuries later, the current evidence based medicine using scientific, mathematical and statistical methods and relying heavily on randomised controlled trial. Throughout the ages, the evaluation of a new drug or treatment method by trials or other means have existed. There also existed guidelines, ‘rules’ and ‘regulations’. Sina’s AL-Qanun Fi Tibb (The Canon of Medicine) [1] sets up rules and principles of testing the efficacy of new drugs, which basically remain valid for present day clinical trials. Similarly, the 16th century French surgeon Ambroise Parè’s writings lay down rules for the treatment of wounds. These were based on observations and experimentation in order to give credibility to the barber surgeons methods, vis a vis ‘academically qualified’ doctors [2]. The history of medicine and surgery is crowded with such examples. So, is randomised controlled trial anything other than the adaptation and updating of the ‘rules’ of validation of ‘efficacy of treatment’ to fit our 21st century setting? The answer is that it is not. Randomised controlled trial with its other attribute, blinding, seems to be the rational and natural progression of the concept of validation of therapeutic methods of bygone centuries to the present time evidence based medicine. Why then are surgeons reluctant to embrace randomised controlled trials and seem unable to conform to the Guideline (Statement) of the CONSORT (Consolidated Standard of Reporting Trials) group [3,4]? The main reason I suggest is that in cases such as those of rapidly evolving treatment methods (surgery or otherwise) execution of randomised controlled trials and the practical application of its criteria become difficult and/or impossible. With good reason Tom Treasure questions in this issue of the journal (P 473) [5] if ‘in the era of rapidly evolving technology randomised controlled trial is needed’. As a cardio-thoracic surgeon, and given that the speciality is one with rapidly growing technology, he draws his examples from that discipline; but this question can be applied to a number of other interventions and technologies (such as photodynamic medicine and its applications to cancer). He is not denouncing evidence based medicine, for which he is one of its standard bearers, but he rightly opens the debate focusing attention to the two ends of the spectrum and a wide gap between the fanatics of randomised controlled trials and the non-believers. The fanatics uphold the view that randomised controlled trials (with its accessory; blinding) is the only way of establishing the validity of a treatment method. The non-believers would deny the necessity of an objective outcome measurement for as long as they, judging by their experience, and the patients are satisfied with the result. There are however those like myself who believe that evidence based medicine is a mandatory requirement to validate a new therapeutic procedure, but feel that not all treatment methods can be measured for validation, by the parameters, which are used for ‘drug testing’. Tom Treasure outlines some of the issues for debate. From my own experience in many clinical trials I would also throw into the debate the following: In the era of informed consent to treatment, is it practical to offer the patient an interventional therapy, irrespective of its degree of invasiveness, using criteria of randomised controlled trial with its ‘blinding’ and placebo prerequisites? In the current climate of patient choice can we apply the rules of randomisation? What if the patient does not accept the treatment to which he/she is randomly allocated? I have experience of this [6]. Evidence based medicine based on randomised controlled trials does not consider the needs of an individual patient. Randomised controlled trials do not consider the relationship between expertise/skill of the operator and outcome, particularly concerning trials in which sample size demands multicentre participation. There has always been a fundamental difference between methods using drugs to achieve treatment objectives and those that employed procedures to mend injuries, and deformities by incision, excision, ablation and repair. Initially physicians were dealing with both kinds of therapy which were referred to by a generic term: medicine. Surgery has evolved from procedures that were designed by masters knowledgeable in anatomy and anatomical pathology and by those skilled in the use of instrumentation. The very word ‘surgery’ (originating from Latin chirurgia and Greek cheros (hand) and ergon (work)) echoes this development. Logically, therefore, the benchmark which applies to the evaluation of drugs needs to be modified for the interventional procedures. Finally in reference to surgical research and ‘Comic Opera’ [7], it is relevant to note that as a genre comic opera (opera buffa) represents a valid expression of an art form which historically nearly overwhelmed the opera seria, and was taken on by some geniuses amongst composers, including Mozart. Therefore the simile might not be quite as apt as the author [8] intended.