A Complex Personal and Societal Issue

Implications: The issues associated with expensive therapeutics are multifaceted and involve personal, professional and societal components. Value related to medical treatment is uniquely assessed by each individual with respect to his/her financial burden, overall lifestyle and quality of life, societal role, and belief structure. Society itself is beset with contrary stakeholders in the insurance, governmental, medical and belief-centered establishments. This article is important in that it lays out some fundamental questions that wealthy countries face with respect to cost vs. benefit for new therapies in an environment relatively little constrained by cost-containment in the medical arena.

Analysis: The risk vs. benefit to profit-making enterprises in the medical field is usually drawn sharply to the risk side of the equation when one looks at the number of preclnical and clinical programs initiated vs. the rate of succesful therapies introduced each year, and over five to ten year periods in the U.S. However, for those medical products which are approved, particulalry in the oncology arena, off-label uses greatly multiply the sales of drugs and biologics over what one would expect for the approved uses. Even for labeled conditions, sales can be massive, due to the ever-present hopefulness inherent in oncology patients and their physicians and the emotional toll that this disease complex takes on the paitnets and their famillies. The "real" benefit to the patient may be minimal or major, depending on the relative proportion of off-label vs. within-label uses of a therapeutic and the perception of benefit seen by the stakholders involved in the process.

In the real world of medicine benefit, real or imagined, may be of a greater magnitude than seen in pre-approval clnical trials or of a lesser magnitude. It is to pharmaceutical firms' benefit to prepare for these real-world issues when developing and marketing new and expensive therapies and to be careful of either deliberately or subliminally overseelling benefit. The newly institued and expanded clinical trials reporting laws and the enabling regulations to come will provide patients and helthcare providers with a new armamentarium to assess perceived benefit vs. cost for approved products. This should provide somewhat of a new tool for making decisions and use of new therapies.