Some of the big pharmaceutical companies, which have made billions by selling widely bought products such as Lipitor and Viagra, have turned to "orphan drugs" which used to be unprofitable.
Orphan drugs are medications used to treat rare disorders. Some drugs — such as those used to treat rare cases in which medicines for cholesterol to help prevent strokes — are needed by seniors.
Some drug companies now have found they can charge enormous prices for orphan drugs, which makes some drugs for only rare diseases well worth the investment required to plunge into research and development.
Historically, rare diseases have been overlooked by most pharmaceutical companies. Companies naturally focused on drugs that would draw customers in the millions, rather than merely thousands of customers.
It's a strong incentive for the pharmaceutical companies when they can charge eye-opening prices for these drugs aimed at rare diseases. A drug to treat a rare disease can command a price tag of $200,000, for example.
A drug made by Sanofi SA became the second medication to get FDA approval in only two months to treat an often deadly, inherited, cholesterol disorder. The disorder is found among only a few thousand people. The drug, called Kynamro, will compete with juxtapid, made by Aegerion Pharmaceuticals.
Meanwhile, the drug giants, Pfizer and GlaxoSmithKline, are studying therapies for a muscular dystrophy that is inherited by only one in 3,600 boys.
Progress in understanding the molecular origins of diseases are helping to guide many drug companies in new research directions. The FDA's urge to develop orphan drugs means quicker approval. Also a protection from competition for seven years, instead of five years, is an extra inducement given to the drug companies.
According to the FDA, when Congress created the orphan-drug designation a quarter century ago, drug companies typically were working on only one such drug a year. Today, the agency says close to 200 rare-disease drugs are under development.
It also costs less to bring an orphan drug to market because orphan drugs require fewer patients for clinical trials.
Industry experts predict that competition will eventually bring down the prices of these very expensive drugs. Already, private health plans are agreeing to pay for these high-end drugs. Good news for seniors with rare ailments.
Tait Trussell is an old guy and fourth-generation professional journalist who writes extensively about aging issues among a myriad of diverse topics.