A Balanced Analysis
I propose a concept of a balanced analysis of risk and benefit. While we have explored standardized measures to express the subjective and dynamic term Quality of Life, few have served as well as the quality adjusted life year, or QALY, in referencing clinical benefit over time. Relating this measure to patient risk is the task at hand, in order to balance the precautionary perspective with a supplemental perspective regarding clinical utility or benefit of any medical product.
The medical industry has been shaped for decades by regulation, reimbursement constraints, and risk-aversion into competing regimes. These camps have been trained to respect, if not embrace, the costs and time applied to investigating product consequences in advance of product availability. Enormous amounts of time and mind power are spent on labeling and risk communication, with an ever-narrowing acceptability of the associated risks of use.
All this effort is to satisfy a market whose values often do not preclude the acceptance of risk, even unto death, in its struggle to obtain benefit, even potential benefit. The Abigail Alliance, an advocacy group comprised of late-stage cancer patients, is pointedly asking for access to cancer drugs under investigation regardless of the risks. However, the choice of taking unapproved drugs for even potential improvement in quality of life is not allowed.
With a medical industry that felt confident enough to offer a new paradigm prospectively to the FDA, there might be a pre-market submission composed of less risk-related data and more quantitative expression of the benefit of the proposed product within its intended uses.
The fact is that, although risk-mitigation is a zero-sum exercise, there is always some residual risk. Current efforts to amplify risk management throughout the life cycle of a medical product ought to be supplemented by achieving a working standard for measuring the intended and actual improvement in the quality of life, and for how long the improvement is likely to endure, so as to factor into premarket submissions and subsequently postmarket surveillance activities.
The FDA currently regulates for safety over effectiveness, and if the U.S. medical industry and its market desire to see improvements in medicine, for which our nation is certainly capable, then it will task itself with establishing a standard of risk acceptability as measured in the context of corresponding clinical benefit.
Upon completing this task, the process of drug development and regulatory market approval would be improved due to the increased clarity in decision-making. There could be far less waste, less cost, and less anguish in adopting such a standard and applying it to rule making. Such a system would increase our knowledge of risks as they relate to benefits over time, whether intended or unintended. This data, expressed comparatively or semi-quantitatively, would use scales that take into consideration that, in certain cases, the quality of life gained by the medical treatment may be considered worse than death.
Moreover, the utility of a medical treatment in prolonging such conditions and/or co-morbidities rather than improving the quality of healthful living is of a negative benefit. Such data, expressed in summary form for consideration by panels and the public, are valuable and urgently necessary. There are some models for using a new method of risk/benefit calculus for the life sciences. Before a new method is applied, however, the medical industry must be bold enough to demand it.