BioWorld Today Columnist

Here we go again. Last month, this column discussed the poor decision making that went into the pricing of Makena, the injection to prevent preterm labor in certain at-risk patients marketed by K-V Pharmaceutical Co., of St. Louis. (See BioWorld Today, May 3, 2011.) I could have added something about the gout drug Colcrys, approved in October 2009 and manufactured by URL Pharma Inc., of Philadelphia, which has caught similar flak . . . but I didn't, because the cases are actually quite different.

It's true that, like Makena, the active ingredient in Colcrys is a long-used, generic drug that URL took through the regulatory hoops to win a formal approval. And like Makena, the approved drug was priced much higher than the generic alternative (in this case, about one order of magnitude higher, instead of two in the case of Makena). But unlike K-V Pharmaceutical, URL actually conducted a reasonably extensive and, by all accounts, fairly expensive research program. The program resulted in changes to how the drug is typically dosed, so the resulting product is not only manufactured to approved standards of purity and consistency, but is also likely to be less toxic when used according to the label. And perhaps more significantly, pricing was based on something other than simply targeting the highest level the market might bear. The price of Colcrys is at least somewhat comparable to that of other newer gout drugs, like Osaka, Japan-based Takeda Pharmaceutical Co. Ltd.'s Uloric. Moreover, URL appears to have done its homework with insurers and other payers before setting the price.

Unfortunately, none of this has stopped URL from suffering the wrath of Congress. The company has been called upon to provide information on the cost of its clinical studies and otherwise justify pricing in a May 23 letter from Sen. Herb Kohl (D-Wisc.), and Reps. Henry Waxman (D-Calif.), Frank Pallone (D-N.J.) and Diana DeGette (D-Co.). And URL can probably thank K-V for all the attention.

Nor does it stop there. The same members of Congress have sent a similar request to Avanir Pharmaceuticals Inc., of Aliso Viejo, Calif. The bone of contention here is Nuedexta, recently approved for the treatment of pseudobulbar affect, an ailment marked by uncontrollable outbursts of weeping or laughter. Unlike Makena and Colcrys, Nuedexta isn't an old pre-Kefauver drug that has been used for decades in regulatory limbo; it is a combination of generics, specifically dextromethorphan and quinidine. (Kefauver is the drug efficacy amendment to the Federal Food, Drug, and Cosmetic Act.) Certainly these are old drugs, but developing them into a novel treatment required a full clinical program. Multiple studies were required to find the right dosing mix and establish safety and efficacy. Even then, a 2006 submission received an approvable letter from the FDA, and the company had to do more clinical work.

So if we are now swinging into full witch hunt mode, who will be next?

After all, we've seen a number of drugs that are either pre-Kefauver medicines or combinations of older molecules move toward the marketplace recently. Executives at Orexigen Therapeutics Inc., of La Jolla, Calif., and Vivus Inc., of Mountain View, Calif., must already be considering the next potential obstacles for their respective weight loss drugs Contrave and Qnexa, even assuming they can eventually win regulatory approval. Both, after all, are combinations of older drugs.

The folks at Acorda Therapeutics Inc. of Hawthorne, N.Y., may be looking over their shoulders regarding the recently approved multiple sclerosis drug Ampyra, which is based on a long-available generic. Amarin Corp., of Dublin, Ireland, has raised a lot of excitement about its AMR-101, which is "just" an omega-3 fatty acid supplement.

But why stop there? Drug companies regularly create combination products to stave off the threat of generic competitions like Pfizer Inc.'s Caduet, which combines the now-generic high blood pressure drug Norvasc with the soon-to-be generic cholesterol drug Lipitor. Should the price of Caduet drop to the price of the generic constituents as soon as Lipitor is off patent? How about Gilead Sciences Inc., of Foster City, Calif., which has employed combination drugs as a core corporate strategy, both as a means of improving patient outcomes and as a means of patent extension?

Members of industry have for years been calling for ways to make it easier to develop around combination drugs, whether they are two or more novel compounds, two or more generics or some combination. The FDA has finally taken steps to streamline policy around combination cancer drugs, which most observers contend represent the best path forward in improving patient care. But developing combinations, in whatever form, is far from simple. The last thing industry needs is Congress second-guessing every pricing decision involving a generic chemical.

Karl Thiel, an analyst for the Motley Fool, can be reached at kthiel@qwest.net. His opinions do not necessarily reflect those of BioWorld Today.