When and how should new therapies become routine clinical practice?
Following on from my last post about when to adopt new therapies – a wonderful colleague of mine (who shall remain nameless, but You Know I Know Who You Are) sent me a copy of this paper from a physiotherapy journal. Bo and Herbert argue that the current way that new therapies become integrated into our daily clinical work is ‘far from optimal because innovative therapies still become accepted practice on the basis of laboratory research alone.’ I agree. Worse still, old therapies that have little evidence to support them continue to be used – even in the face of clinical studies demonstrating that they have no greater effect than placebo.
Bo and Herbert suggest there are several ways that new therapies are adopted within physiotherapy practice. I suggest that there is little difference between the situation in physiotherapy and other health professions!
Clinical experience - this is the traditional way practice in health care evolved. Experienced therapists hand down ‘what works’ on the basis of their observations within their own practice. Sometimes this is more formalised within workshops or conferences, and sometimes case reports are published. The basis for adoption is mainly on the reputation (or charisma) of the founder of the method. This situation continues today within most health professions, but fails to account for biases that are present in clinical practice. Things like patient expectation, natural course of the disorder, placebo, reduction of distress and failing to control for other external sources of influence mean the ‘expert’ can be mistaken. There are some ways to ameliorate these biases such as using good outcome measures and making time for long-term follow ups, but clinical experience alone is insufficient to make good generalisations about ‘what works’.
Theories based on basic science or ‘preclinical’ research (laboratory findings)
In this paper, Bo and Herbert suggest that as physiotherapists began to develop an academic arm, researchers based their experiments on their knowledge of the basic sciences underlying the profession. Often the laboratory-based experiments lead almost immediately to alteration of clinical practice – alternatively, laboratory findings were used to justify existing practice if the results obtained were consistent.
The problem is that while laboratory findings can inspire further study, perhaps leading to the testing of new hypotheses, findings don’t directly translate to what occurs in a real clinical patient. Patients don’t conform to the very tight parameters required for experiments. There are multiple variables involved in their clinical presentation that may be quite important but too complex to be incorporated into basic science experiments. Bo and Herbert suggest that laboratory studies measure impairment-level outcomes, while treatments need to measure disease-specific or disability/quality of life changes. Clinical practice is very different from a laboratory!
The ‘gold standard’ for identifying whether an intervention provides an effect in real people is the randomised controlled trial. When they are well-designed, they can be used to clearly demonstrate that treatment X and only treatment X influences the outcome. I won’t detail why the RCT is such an important method today, only to say that it remains the best way to ensure extraneous variables are controlled for – but they’re expensive, time-consuming and difficult to conduct in a clinical setting. It’s not surprising that I don’t think I’ve ever read about RCT’s for raised toilet seats!
Bo and Herbert go on to describe a six stage protocol to be used when a new therapy is being considered.
Stage 1 – clinical observation, laboratory studies – development phase
A clinician or researcher observes something interesting. Preliminary studies demonstrate that this ‘interesting finding’ is able to be consistently obtained. Some hypotheses are developed and tested in a controlled environment.
Stage 2 – clinical exploration – development phase
The hypotheses are explored in a clinical setting, maybe a prototypical treatment is carried out amongst volunteers, trial and error (or ongoing hypothesis testing based on a theory or model) shapes the treatment. This is a strongly exploratory phase – and quite exciting!
Stage 3 – pilot studies – development phase
Once one or two specific interventions become confirmed, pilot studies in a controlled but clinical environment are conducted. These might be small-scale group studies, case series studies, or small RCT studies. Further refinement of the practicalities of this treatment is made.
Stage 4 – randomised clinical trials – testing phase
If pilot studies are ‘promising’, RCT’s are carried out. A single swallow does not a summer make, however, so one study is rarely sufficient data on which to base wholesale adoption of a new treatment. Replications with different settings, different clinicians, different patients need to be made.
Stage 5 – refinement – refinement and dissemination phase
This stage may involve ‘head to head’ comparison of the new approach with other, more established treatments. Larger RCT’s are needed to identify how subgroups respond to the intervention.
Stage 6 – active dissemination
Now the word can be spread using guidelines, teaching curricula, continuing education – other professionals will learn about it, patients can be informed of the option and even the general population can be advised.
Now I have absolutely no argument with this staged approach to developing therapy. I will, however, suggest that there are very few physiotherapy, occupational therapy, or even psychology interventions that have reached Stage 5 before the treatment is already adopted and described as ‘evidence-based’!
Lack of research expertise notwithstanding, obtaining funding for these studies is difficult. The hoops that need to be gone through, at least in New Zealand, to prepare a research proposal for even a Stage 1 or 2 study makes it challenging for therapists in full-time clinical work to contemplate conducting even basic observational studies. There is an inherent lack of interest from managers of health services to allow clinicians to spend time on non-treatment-related activities, it simply doesn’t pay. Allied health clinicians rarely have the expertise to carry out methodologically strong studies without requiring support from within the profession, within the clinical adminstration and management, and without support from an academic institution.
I’ll jump off that soapbox quickly now!
Where does that leave clinicians when thinking about adopting a new therapy? More tomorrow on what to say to patients, but in the meantime I want to leave you with this thought: until an RCT is able to demonstrate that treatment X has an effect attributable only to itself, and is applicable to the kind of patients within the kind of setting the therapist is working in, all treatments are really ‘experimental’.
BO, K., & HERBERT, R. (2009). When and how should new therapies become routine clinical practice? Physiotherapy, 95 (1), 51-57 DOI: 10.1016/j.physio.2008.12.001