Orphan Drugs’ Transformation From Neglected Niche To Tantalizing Moneymaker
Critics are worried that a law that was originally created as an incentive for the industry to develop drugs to treat rare diseases has backfired, because it offers companies a period of market exclusivity that prevents competition.
The Washington Post:
High Prices Make Once-Neglected ‘Orphan’ Drugs A Booming Business
Three decades ago, Congress listened to the plight of Americans sick with diseases so rare many people had never heard of them. They were victims of a pharmaceutical market failure — “orphans” ignored by drug companies because, the thinking went, tiny groups of patients would lead to trifling sales. To make the business viable, Congress — pushed by patients and a popular television show that highlighted rare diseases — passed the Orphan Drug Act. The 1983 law offered drug companies attractive tax credits and monopolies to develop treatments for rare diseases, radically transforming the pipeline of orphan drugs. Now, rare diseases are no longer a neglected niche of the pharmaceutical business; they are a tantalizing moneymaking opportunity. (Johnson, 8/4)
In other pharmaceutical news —
The Boston Globe:
Momenta Halts Pancreatic Cancer Drug Trial
Momenta Pharmaceuticals Inc. said Thursday it halted a clinical trial of a pancreatic cancer drug after an independent panel concluded the experimental therapy wasn’t effective enough to justify continuing the study. The move was a setback for Momenta, a 16-year-old Cambridge company that markets two generic medicines and is developing generic biotech drugs and its own therapies. The pancreatic cancer drug, called necuparanib, was its lead novel drug candidate, targeting a disease that has been stubbornly resistant to treatment. (Weisman, 8/4)
Stat:
Groups Opposing Medicare Part B Overhaul Received Pharma Dollars
After the Obama administration proposed overhauling Medicare Part B earlier this year, a plethora of patient advocacy groups quickly expressed opposition. A new analysis, however, finds that the vast majority of those groups received funding from the pharmaceutical industry, which may come out losers if the proposal goes into effect. (Silverman, 8/4)
Stat:
FDA Warns Chinese Drug Maker For Lying
Pharmaceutical and ingredients manufacturers in China and India have come under intense scrutiny in recent years due to a series of disturbing events. In 2008, for instance, 246 deaths in the United States were linked to a fake ingredient from China that was found in the blood thinner heparin. China is now the world’s largest supplier of active pharmaceutical ingredients. (Silverman, 8/4)