Orphan drug market set to climb above $200B: report

Orphan drug sales have nearly doubled since 2008, a trend set to continue through 2022 as biopharma companies increasingly focus on meds to treat a variety of rare diseases. Sales for the superpricey drug class will nearly double—again—by 2022 to $209 billion, according to a new industry analysis.

In its new World Preview report, life science commercial intelligence firm Evaluate projects orphan drug sales will represent 32% of pharma’s total growth through 2022. After reaching $115 billion in sales last year, the class will add $95 billion more by 2022, according to the analysts.

As Evaluate’s report notes, the trend comes as drugmakers increasingly look to rare disease meds because they can address a greater unmet need and offer a “more compelling market access value proposition.” Further, drugmakers can win development incentives for advancing meds to treat rare diseases.

RELATED: FDA's orphan drug program under GAO scrutiny after senators allege abuse

The last point has been under scrutiny lately, as some members in Congress believe some pharma companies are gaming current laws to win development incentives for meds that treat large patient populations.

Earlier this year, Senators Orrin Hatch (R-Utah), Chuck Grassley (R-Iowa) and Tom Cotton (R-Arkansas) wrote to the U.S. Government Accountability Office head asking for an investigation of the Orphan Drug Act. First enacted in 1983, the law offers incentives to spur development in disease areas that might otherwise be ignored.

But reports have highlighted how drug companies can legally use the law to reel in sizable development incentives for mass-market meds. Some best-selling drugs notched initial approvals for diseases with broad patient populations and then, when the drugmakers applied for approval in rare diseases, brought in benefits such as tax breaks and monopoly pricing power. That list includes AstraZeneca's cholesterol med Crestor, Otsuka's antipsychotic Abilify, Roche's cancer med Herceptin and AbbVie’s rheumatoid arthritis med Humira.

RELATED: With FDA nod, BioMarin prices orphan drug Brineura at $702K, but plans hefty discounts

The GAO accepted the senators’ request for an investigation, a spokesperson said in March; he added that the inquiry “won’t get underway for several months” due to other responsibilities at the agency.

As orphan drugs address diseases with small patient pools where there are few other options, they command high prices. For instance, BioMarin recently set Brineura, which treats the ultrarare CLN2 disease, at a price before discounts of $702,000.

The meds also present an opportunity for drugmakers to hit the market running with newly approved products, as payers have increasingly gotten tough on new mass market meds that offer incremental gains.