Orphan Drug Forecast Notes Uncertain Policy Environment

Rare Daily Staff

By 2030, orphan drugs will make up a fifth of the forecasted $1.6 trillion in worldwide prescription drug sales, a doubling of their share over the last decade, a new report from Evaluate finds.

But while orphan drugs are expected to continue outpacing mainstream pharmaceuticals (10 percent vs. 7.5 percent compounded annual growth rate), Evaluate said the growth advantage is expected to shrink to just 1 percent by the end of the decade, which could mark the end of what it called “an extraordinary run.”

“Buoyed by considerable regulatory and exclusivity advantages, some orphan drugs now resemble mainstream pharmaceutical blockbusters, and dynamics within some niche markets mirror those across more prevalent disease areas,” writes report author Melanie Senior. “With growth rates for orphan and non-orphan categories also converging, this begs the question: should orphans still be considered a class apart?”

Over the rest of the decade, orphan drug candidates are expected to account for 20 percent of the global pipeline forecast sales.

The report did note the uncertain policy environment in the United States, which it said complicates forecasting.

“The Trump administration has begun to fire FDA staff as part of its wider efficiency drive, depressing staff morale and perhaps prompting further, voluntary departures among hard-to-replace experts,” wrote Senior. “Until now, FDA has supported cell and gene therapies, and pushed for accelerated access to novel rare disease therapies, for instance with a new rare diseases hub coordinating action across its CDER and CBER approval divisions. These initiatives are now under threat.”