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Where’s the evidence?

Modern medicine is evidence based, so new therapies or procedures need clear evidence of benefit to be accepted into medical practice. Any new drug seeking a listing on the Pharmaceutical Benefits Schedule (PBS) needs a robust body of evidence for efficacy and safety supported by pharmaco-economic evidence of cost effectiveness. A similar framework underlies applications for new listings on the Medicare Benefits Schedule (MBS).

02 Sep 2012

Modern medicine is evidence based, so new therapies or procedures need clear evidence of benefit to be accepted into medical practice. Any new drug seeking a listing on the Pharmaceutical Benefits Schedule (PBS) needs a robust body of evidence for efficacy and safety supported by pharmaco-economic evidence of cost effectiveness. A similar framework underlies applications for new listings on the Medicare Benefits Schedule (MBS).

Such an approach is consistent with prudent use of the taxpayers’ healthcare dollar, but where is the evidence to come from? In its absence, Australians stand to be deprived of life enhancing or life prolonging drugs and procedures, but calls for evidence can easily become a dead weight on progress and innovation.

The advent of the Independent Hospital Pricing Authority (IHPA) has further potential to stifle innovation. If hospital admission for a disease process or procedure is founded on its historic cost there is less incentive to innovate, especially if innovation carries higher initial costs even though there is increased patient benefit, and perhaps future savings to the cost of health care.

The history of innovation in health care is positive. Costs associated with research, development, passing regulatory hurdles and initial marketing are amortised over time. Benefits associated with reduced hospital length of stay or reduced hospitalisation persist. But innovation needs investment today for the dividends to accrue in the future. With funding for research and development in short supply, and likely to come under even more pressure, a new approach and new funding sources are needed.

This may be a good time to re-think our approach to these difficult issues and provide a robust evidence base for new treatments. When there is some sound evidence of benefit, but perhaps not sufficiently robust to justify PBS or MBS listing or funding through the IHPA, the treatment could be provided to half of those with agreed indications and the funds saved by not treating the other half used to support the clinical trial infrastructure needed to assess the efficacy, safety and cost effectiveness of the treatment in an Australian setting. These trials should have patient-centred outcomes, such as survival or quality of life. The same approach could also be used if drugs or MBS items are being considered for de-listing.

A seasonally suitable example might be the use of vaccines for preventing influenza in the elderly. This intervention is recommended worldwide for individuals aged 65 years or older as a means of reducing the risk of complications in a vulnerable population. However, in 2010 a Cochrane review of 75 studies into influenza vaccination in the elderly found only one randomised clinical trial, and this did not detect any significant effect on influenza complications. The other studies were all of poor quality, so the authors were unable to reach any conclusion about the efficacy of influenza vaccines in the elderly. This doesn’t mean that influenza vaccines don’t work, just that there isn’t good evidence that they do. It’s not surprising, therefore, that the authors recommend that, to resolve the uncertainty, an adequately powered randomised clinical trial is needed. But who is to fund it?

A lot of work would need to be done, in consultation with those running clinical trials and with health consumers, to develop the necessary ethical framework and research infrastructure. Public education would be another challenge. This is not an easy option. Nevertheless, public debate about the funding of health innovation is a debate that perhaps we have to have.

Australia has many groups with expertise in running pragmatic clinical trials with patient-centred outcomes, so we should be confident there is the capacity to do it well. Providing new treatments only within the context of a clinical trial is not a new concept, but doing it on this scale may be seen as controversial. However, if the alternatives are to either prevent or restrict the access to new treatments in Australia, or fund them on the basis of imperfect evidence, the clinical trial should be preferred.

Worldwide, countries are struggling to meet the escalating costs of health care. As a profession, we advocate for as much funding as possible to be devoted to health care, but we also have a responsibility to ensure the dollars are well spent. The aging of the baby boomer generation is about to put greater pressure on all health budgets and, in this context, striving for effectiveness - and not just the ‘efficiency’ that is the focus of bureaucratic attention - will do much to ensure our health system is sustainable.


Published: 02 Sep 2012