Fast-track to where? PharmaTimes May 2006
There has been a lot of publicity lately about a new drug for the treatment of breast cancer, Roche`s Herceptin, and whether or not National Health Service organisations have a right to decide if they will fund the use of this drug in women with early-stage disease ahead of a license in this indication.
Herceptin is currently licensed solely for advanced breast cancer, although an application for early-stage use was given the nod by Europe`s scientific advisory board just as PharmaTimes Magazine went to press. Meanwhile, there are major potential side effects and the consequences both of long-term treatment and outcome are not yet established.
Last November, the King`s Fund published a short paper highlighting two important issues raised by this situation.
1. Fast-tracking [pushing drugs with an unmet need through the regulatory process in a shorter time frame] might equate to more risk [to the patient]; and
Without denying the importance of these two issues, I believe that there are other implications that are significant for all pharmaceutical companies and not just to patients and NHS management.
There are processes across Europe and North America that have a high degree of commonality and are based on long-established principles that hold across the spectrum of scientific research and teaching. It is these processes that are the foundation of the pharmaceutical industry and which form the principal platform in differentiating prescription medications from `alternative therapies`, which - it is fair to say - do not have the equivalent established and scientifically accepted body of evidence for their use and effectiveness.
The major costs involved in bringing a new therapy to market are predominantly generated by fulfilling these processes to a standard that satisfies the regulatory bodies. And it is those costs - both of the successful compounds and of the very many that fall by the wayside - that are used to justify the high premium costs to healthcare providers. All that research and validation has to be paid for, otherwise there would be no new drugs and the quality of healthcare technology would remain static.
Undermining conventional practice
That being the case, it is clear that, not only are patients put at a greater (or unknown) risk in being prescribed a drug that has not fulfilled the established criteria, but the whole principal that has underpinned `conventional` medical practice for the last hundred years or so is undermined. Either there is evidence that meets the necessary established criteria, or there isn`t. If there is evidence, then logically there is no need to go to court to compel healthcare organisations to provide the drug. If there isn`t the evidence, then the implications of such an action are that, if accepted, they undermine the whole scientific foundation of healthcare.
So where does this leave the world`s pharmaceutical companies? The suggestion is that the accepted and established evaluative processes are no longer the `highest authority` for deciding what is safe and effective. If that is the case, is it therefore a waste of time and resources to gather the evidence that is currently considered essential for a new drug`s approval and launch?
The implications for pharmaceutical companies from the second factor identified in the King`s Fund paper - that local decisions are being undermined - are equally disturbing for very similar reasons. `Policy affects prescribing` is now generally accepted, and the days of clinical autonomy are no longer. Increasing focus has been given to putting the right messages about products to those managers who define and implement management policy, on the understanding that there will be major gains from `getting it right` with local policy. The obvious implications for `getting it right` at a national level are enormous: a potential massive win - or a potential massive loss. However, there could be much that is counter-productive, even for a company that is selling the `winner`. How does a company set about marketing a potentially profitable but `non-sexy` product that has a clear evidence-base and clinical advantage, but to managers who can have their policy and autonomy over-ridden either by a scientifically arbitrary diktat or by an equally nonevidenced legal decision?
Differentiation between products has, for many years, been based either on the positive experiences of the practitioner being sold to, or those of respected colleagues treating similar patients. Differentiation for a manager has been based on a combination of health economic arguments around relative benefits to the local population and the opinion of local practitioners with specialist knowledge of the disease. How will a company be able to sell its product if there is no scientific evidence acceptable to the established processes? The end result would seem to me to be marketing anarchy for all companies and healthcare organisations.
The potential implications of the action that has been pursued, I feel, are of the utmost significance to the pharmaceutical industry. That being the case, I would like to refer to one more point that was identified in the King`s Fund paper: that patients bringing such action may have the direct or indirect support of the company producing the drug in question. It is my belief that such support will, in fact, be to the detriment of that company`s competitors within the clinical area under question, but also, ultimately to itself and possibly the whole pharmaceutical industry as we know it. Serious thought should be given to where this fast-track could lead to.
Dr Matthew McGlennon