Editor's Notes:

This article chronicles the growing number of FDA approvals for so–called "orphan diseases", or medicines used to treat diseases that afflict 200,000 or fewer Americans annually. The Pink Sheet notes that the FDA is on pace for a record number of orphan drug approvals in 2006. It credits the growth in orphan drugs over the last 20 years to advances in biotechnology combined with marketing and tax incentives for orphan drug research.

"A better understanding of molecular and genetic causes of disease has given us new tools to explore rare diseases, which are often more complex than more common diseases," [a recent PhRMA report states], but [PhRMA] gives the lion's share of the credit for upward trends in rare disease R&D and approvals to the Orphan Drug Act.

"This legislation is credited with the explosion in drug approvals for rare diseases after the Act was passed in 1983," the report states. The law's provisions include seven years of marketing exclusivity for orphan-designated indications, as well as other incentives for drug development.

"Over 160 drugs were approved during the past decade (1995-2005) to treat rare or 'orphan' diseases that affect 200,000 or fewer people in the United States," PhRMA notes. "This compares with 108 approvals in the decade before (1984-1994) and fewer than 10 in the 1970s." "Since the Orphan Drug Act, over 1,450 drugs in development have been designated as orphan products," the PhRMA report notes.

The Orphan Drug Act has, with relatively little fanfare, revolutionized the treatment of rare diseases in the U.S. and has become a model for similar legislation around the world.

Editor's Notes:

As the saying goes, no good deed goes unpunished. In Washington D.C., no good legislation ever gets passed without first being hammered by the opposition. This is certainly the case with Senator Enzi's association health plan legislation, which would allow small employers to band together on the national level to buy health insurance for their employees outside of heavily regulated state markets. Democrats paint the legislation as enabling a race to the bottom where small businesses would offer worse–than–nothing coverage to their employees.

This argument is pernicious for many reasons. First, many small employers can't afford any insurance today, pricing their employees out of the market entirely. Regulations that keep insurance too expensive for these small companies don't help anyone. Second, federal employees (including Washington legislators) are already exempt from state mandates, so it is disingenous for legislators to say that there aren't affordable, effective plans without state mandates. Finally, in a competitive national market, health associations for small businesses would use their new market leverage to compete for labor with larger employers (who are also exempt from state mandates). Health associations would have every incentive to offer plans that would attract the most number of employees, not engage in a self-defeating "race to the bottom."

The bottom line is that the Democrats' opposition is more about election year politics than real insurance concerns. Politics as usual in Washington, perhaps, but dispiriting nonetheless.

Under the proposal endorsed by President Bush, businesses could buy insurance through regional or national trade associations. The insurance would be free of many state mandates. That could make it a cheaper alternative for businesses and workers wanting to buy more scaled–back coverage.

By a vote of 55-43, Democrats blocked a motion that would have limited debate. They provided several arguments for their action; for example, Republicans' refusal to allow votes on amendments, such as extending Monday's Medicare enrollment deadline, that they considered a priority.

Democrats also said the bill stripped away a state's ability to determine which health care screenings and treatments insurers must cover. They said states have worked for such protections for years, and it would not be right for lawmakers in Washington to take them away. Democrats said commonly required screenings for cancer, such as mammograms and colorectal exams, save lives and money. State should have the freedom to require them, they said.

Sen. Patrick Leahy, D-Vt., said the bill would pre-empt requirements in his state that insurers cover treatment for alcoholism, contraceptives, diabetes supplies and home health care. "It kind of makes you wonder what's going to be left to be covered," Leahy said.

Sen. Mike Enzi, R-Wyo, led the effort to pass the health plans. He said employers would not cut benefits for workers who already have insurance, but they might be able to afford to provide coverage for more of those without.

"What they're trying to nail you on is that this bill eliminates mandates. Well it doesn't really, because every association is going to make sure your employees have the things that they need for their health care," Enzi said when talking to small business owners on Thursday.