Paying for promising but unproven technologies

Abstract

Is the policy on “coverage with evidence development” a helpful way forward?

Promising new medical technologies, especially expensive ones, pose a difficult problem for healthcare systems. If there is not enough evidence for conventional systematic reviews and technology assessments to find benefit, the default decision is that a new technology, whether drug, device, or surgical procedure, is not “covered” and thus not paid for. This seems reasonable when we are talking about a screening test or other preventive manoeuvre, since most would agree that we should require a high level of proof before exposing a well population to expensive and potentially harmful interventions. But what about when the person is sick, perhaps with advanced cancer, and the promising technology is a potentially lifesaving chemotherapeutic agent, a new surgical procedure, or a drug already approved but for a different condition?

On the other hand, to allow payment for such technologies might break the bank if the new interventions are expensive, and of course they may not work at all. Everyone's favourite cautionary example is high-dose chemotherapy with autologous bone marrow transplantation for advanced breast cancer. Hugely …