Thomson Reuters | The New Blockbusters: Orphan Drugs

4 Sep 2012

The New Blockbusters: Orphan Drugs

Our analysis proves the economic viability of orphan drugs positioned for unmet medical needs

The New Blockbusters: Orphan Drugs It’s long been surmised that orphan drugs, developed to treat rare diseases affecting up to 200,000 people, are replacing the blockbusters of prior decades in their revenue-generating power. Yet, to date, there hasn’t been proof of their economic viability. That is, until tour new research and analysis, which verifies they have the potential to generate as much lifetime revenue as drugs used for more common health conditions.

Kiran Meekings, Ph.D., Life Sciences consultant, and John Arrowsmith, Ph.D., Life Sciences executive advisor, both with Thomson Reuters, conducted research, in conjunction with Cory Williams, MD, Ph.D., Pfizer, on how orphan drugs compared to past blockbusters. What they found is that they are comparable and, in fact, they have a higher compounded annual growth rate (CAGR) than non-orphan therapies.

The findings are featured in "The Economic Power of Orphan Drugs," and in Drug Discovery Today.

Developmental drivers such as government incentives, shorter clinical trials and high rates of regulatory success make top orphan drugs as economically viable as non-orphan ones, even though the number of patients benefiting from them is significantly smaller than those benefiting from non-orphan medicine. Rare diseases include illnesses such as Cystic fibrosis, Wilson’s disease, and Homozygous familial hypercholesterolemia.

Current estimates indicate that 250 new rare diseases are identified annually. This report provides extensive and tangible evidence that orphan drug development is an important component of biopharmaceutical R&D strategy and that a number of orphan drug therapies are already achieving blockbuster status.