This issue of Joints includes three articles that focus on the treatment of degenerative knee disease.
Priano[1] describes a study of HYADD®4 (a hyaluronic acid derivative-based hydrogel) and its
effects on symptoms and joint function in patients with symptomatic knee osteoarthritis.
Adriani et al[2] evaluates the safety and efficacy of autologous aspirated and purified fat tissue
injected percutaneously into the knee joint for the treatment of symptomatic osteoarthritis,
while Rosa et al[3] reports the findings of a systematic literature review on the treatment of failed
cartilage repair procedures.
Joint preservation–I refer not only to minimally invasive surgery but also to conservative
treatments and damage prevention measures—is currently a very widespread and topical
concept relevant not only to the knee joint. However, joint preservation strategies
are often expensive and not validated for efficacy and safety, with the result that
the value of these treatments is still debated.
Michael Porter, of Harvard Business School, defined patient value as outcome divided
by cost.[4] On the basis of his analysis, Porter formulated a concept that he termed “value-based
medicine,” wherein value is the best possible relationship between the optimal outcome
of a treatment (i.e., restoration of the best possible health status attainable) and
the expenditure incurred to achieve that result. However, if an individual patient
perceives his or her health to be priceless, then cost becomes irrelevant.[5] But who should foot the bill? The national healthcare system of a country plays
a decisive role in the selection of treatments, given that the costs of unnecessary
treatments may ultimately be borne by the community. Unfortunately, Porter's theory
has a weakness that he himself recognized. In short, to calculate the value of a treatment,
it is necessary to measure and evaluate its outcome, and he found that doctors, in
most cases, did not know how to do this.
How should the result of a treatment be measured? There already exists a surfeit of
outcome measures, some subjective and others objective, and both types carry risks
of bias. With regard to patient-reported outcomes (PROs), patient expectation is known
to be significantly correlated in a positive sense with subjective outcome. In other
words, patients who expect more from the proposed treatment will feel that they have
done better. Information, too, is significantly correlated with outcome, and this
often depends not just on the communication skills of the physician and the tools
he/she uses but also on the patient's ability to interpret the information given,
a factor that seems to be strictly correlated with the patient's sociocultural status
and education. Furthermore, patient expectations and patient information are significantly
correlated with each other. It should also be added that the validity of a treatment,
as can be inferred from the evidence of its efficacy reported in the literature, depends,
in theory, on its integrity or fidelity, i.e., the extent to which it is implemented
as intended.[6] Treatment integrity is an essential condition for empirical evaluation of an intervention's
efficacy, as it allows unambiguous interpretation of the results obtained. Without
it, the results obtained could actually be showing a correlation with the treatment
as it was administered in reality, rather than with the treatment as it was theoretically
meant to have been administered. This is a hugely important concept in outcome research,
particularly in areas, such as surgery and rehabilitation, where the administration
of treatments may be subjected to uncontrolled variability both between and within
the subjects. In practice, if a validated treatment is administered with a high level
of integrity, it is probably more likely to give outcomes similar to those reported
in the original efficacy study.
Even the costs are not as easy to calculate as one might imagine. For example, apart
from the direct and indirect costs of a treatment, which are often underestimated,
consideration should also be given to costs that are incurred as a result of doctors'
decisions,[7] and in this regard, how do we determine whether the treatment chosen by a doctor
is the right one for a particular patient? Recently, Kibler, in an interesting reflection
on the value of treatment in orthopaedic surgery, stressed the importance of a correct
diagnosis to obtain a good result, remarking that “there is a linear, almost cause-and-effect
relationship between the diagnosis and the outcome.”[8] As explained by Rossi et al[9] in a recent editorial published in Arthroscopy that to achieve the best outcome, we need to choose the best treatment, and to choose
the best treatment, we need to make an accurate diagnosis.
In outcome research, there is a lot of talk of efficacy and safety, but not enough
is said about adequacy. Adequacy, basically, refers to the ability to identify the
type of patient who may really benefit from a given treatment.
In the light of all these considerations, the choice of a treatment should, nowadays,
be based on an individualized and shared weighing up process, involving both the physician
and the patient, rather than purely on evidence-based data. Shared decision making
gives both the patient and the physician the chance to decide, together, what should
be done after first discussing the various options and their risks and benefits, and
considering all the aspects linked to the value of the treatment, to preferences,
and to the individual patient's circumstances. Naturally, all this rests on the assumption
that the information needed to reach a decision is valid, i.e., that the diagnosis
is correct; the integrity of the proposed treatment can be guaranteed; the expected
benefits are genuinely valid in safety, effectiveness, and adequacy terms; the estimate
of the direct and indirect costs is reliable; and the doctor–patient relationship
is good and underpinned by proper bidirectional communication. The question to be
asked, though, is: Are we truly equipped to guarantee all this?
