Bright line or lottery? On significance and value in medical decision making

Abstract

It is widely acknowledged that using p-value thresholds as the basis for making decision on health care spending is not appropriate. In the context of medical decision making, we argue that patient preferences need to be a stronger factor. Depending on attitudes to risk, patients might prefer a medical treatment that performs on average worse than a comparator but offers a small probability of a large gain such as a cure. However, what has been labeled 'value of hope' is not yet fully reflected in the decision-making process of drug approval and health technology assessment (HTA). Therefore, patient risk preferences should be formally incorporated within the decision-making framework for regulatory and reimbursement decisions.

The philosophy of pharmaceutical regulation—Paternalism or freedom of choice?

“…(3) Valuation of Risk: Regarding risky situation i.e., when outcomes are associated with a quantifiable probability, the question is how to value risk. The term "value of hope" for instance refers to risk seeking individuals who prefer outcomes that are associated with a high variance because they value the potential gains more than potential losses (3). A drug that offers on average a lower life expectancy than the established standard of care and is even riskier in that the variance of life expectancy is higher might still be preferred by patients because the high variance offers sufficient opportunities that are of value for patients.…”