Orphan drugs and the NHS

Editor—Orphan drugs pose several dilemmas for policymakers, not all of which are addressed by discussing cost effectiveness.¹

To be reimbursed by a publicly funded healthcare system a drug needs to be effective, safe in animal testing, effective in humans, and a cost effective use of public resources.

Orphan drugs will not generate sufficient sales to recoup the cost of jumping these hurdles. One solution is to offer special incentives to companies to develop orphan drugs—for example, a higher cost per quality of life years (QALY) threshold. This is inflationary, since it effectively increases the price that the NHS is willing to pay for certain types of drugs.

Another solution might be to reduce the costs of development. The hurdles that drugs must cross are designed to protect the public from potentially harmful drugs. Not every person with an illness can give his or her consent to a new or experimental treatment, so clinical trials are conducted in a minority who are prepared to do so.

Figure 1.

Credit: PENNY TWEDDIE/SPL

With a very rare illness it may be possible to obtain consent from every person (or most people) with the illness. If most people with a rare illness are prepared to consent to accepting a drug that has crossed lower regulatory thresholds, the ethical position seems stronger. Indeed, would we have the right to deny such patients a drug?

The onus is placed on the pharmaceutical manufacturer to identify the community to which it is responsible (those with the disease) and secure informed consent. The appropriate threshold for consent should be that the majority of those believed to suffer from the disease (or their guardians) have given consent. Without the consent of the majority the pharmaceutical manufacturer must comply with the usual regulations. With consent the industry can proceed—in effect being regulated by the patients themselves.