Orphan drugs: Stronger orientation towards medical need required

The number of drugs approved for rare diseases (orphan drugs) has increased significantly in Europe over the last 20 years. This is partly the result of support at the EU level, which aims to encourage orphan drug development for therapeutic indications where there are no or very limited treatment options. But has this been successful?

In early 2002, the German Institute for Quality and Efficiency in Health Care (IQWiG) published a working paper analysing decisions on HTAs of orphan drugs and concluded that in about half of the cases, there was no added benefit over standard care. In a new publication in the International Journal of Technology Assessment in Health Care, an IQWiG team led by Philip Kranz now presents further analyses.

Orphan drugs are intended in particular to address areas of unmet medical need, i.e. diseases for which there are no established treatments. However, this is often not the case: 58% of the research questions studied already had established treatments at the time of assessment. For oncological indications, this was the case for 88% of the research questions, compared to 24% for non-oncological indications.

In addition, oncological indications - where prices tend to be particularly high - are strongly overrepresented in drug approvals, while patients with many other rare diseases still have no new drugs. The probability of an added benefit in regular HTAs of orphan drugs for non-oncological indications is higher than for oncological indications, which shows that it increases in indications without established treatments.

Finally, the IQWiG team discusses how the research infrastructure and funding landscape for rare diseases could be improved so that incentives for orphan drug development reach indications where they are most urgently needed.