The growing number and costs of drugs for rare diseases (orphan drugs) are straining healthcare budgets. Should the NHS continue to pay for them and how will this affect other health services, ask two articles in this week's British Medical Journal (BMJ)?
Orphan drugs are often expensive to produce and benefit only small numbers of patients. Historically, the NHS has paid for them because treatments for these diseases were so rare that the effect on health services was negligible.
But, as more and more orphan drugs come on to the market, the impact on other health services is becoming substantial.
In the first article, experts argue that the cost effectiveness of orphan drugs should be treated in the same way as for other healthcare technologies.
They believe that the costs of production and the value of innovation cannot justify special treatment, and that arguments about the measurement and valuation of health outcomes apply equally to orphan drugs and drugs for more common conditions. "Why should a person's health be valued less simply because the condition is not rare?"
Special status for orphan drugs in resource allocation will avoid difficult and unpopular decisions, but it may impose substantial and increasing costs on the healthcare system, they write.. The costs will be borne by other patients with more common diseases who will be unable to access effective and cost effective treatment as a result.
The second article describes how a national decision to provide therapies for a group of rare disorders prevented local funding for other equally vital services in the West Midlands.
The new commissioning arrangement came with no extra funding. The cost to primary care trusts doubled from £3.2m to £6.7m, limiting budgets available to commission and develop other services.
It is time to educate ourselves, policy makers, and the public, say the authors. "We need to learn how to make trade-offs between equity and efficiency that are explicit, principled, and generalisable and how to admit openly when there are treatments and services that are not being funded."