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They are relatively cheap to develop, since clinical trials are small and “the lack of alternative treatments give orphan agents an advantage when up for regulatory review,” according to a report by Evaluate, a life science market intelligence company that provides forecasts on that sector. The company’s second annual EvaluatePharma Orphan Drug Report says that orphan drug sales will make up 19% of prescription drug sales by 2020, totaling about $176 billion.

Insurers will have their hands full because even though the populations that need orphan drugs are small “they represent big per-patient outlays, and insurers will be looking carefully at new tools to arrest cost growth as more and more orphan drugs launch,” says the study. The cost per orphan drug per patient is six times that of non-orphan drugs.

Worldwide Orphan Drug Sales & Share of Prescription Drug Market (2000-20)

Average Cost per Patient per Year 2010-14

Source: “EvaluatePharma Orphan Drug Report 2014,” Evaluate 

Contributing Voices

Princeton’s Uwe Reinhardt, PhD, renowned health care economist, sits down with Managing Editor Frank Diamond to discuss the economic effects of the Affordable Care Act, wellness programs, and the state of health care in the United States in general.

Contributing Voices

The vast majority of Part D plans follow a tiered cost-sharing structure with incentives for members to use less expensive generic and preferred brand-name drugs. Cost-sharing has increased since 2006, but the Kaiser Family Foundation reports in “Analysis of Medicare Prescription Drug Plans in 2011 and Key Trends Since 2006” that there was barely a change between 2010 and 2011.” The foundation reports that since 2006, median cost sharing for a 30-day supply of nonpreferred brand name drugs in stand-alone prescription drug plans (PDPs) increased by 42 percent, from $55 to $78. Preferred brand costs increased 50 percent, from $28 to $42. But since 2010, cost sharing has been stable.

About half of PDP enrollees and over 75 percent of MA-PD plan enrollees are in plans that charge 33 percent coinsurance for specialty drugs. Compared to 2009, this share is down modestly for PDPs but up substantially for MA-PD plans. In contrast, only 4 of the 35 national or near-national PDPs charged a 33 percent coinsurance rate for specialty tier drugs in 2006.