BioWorld Today Columnist

Editor's Note: This is part two of a two-part series on notable industry happenings. Part one ran in Thursday's issue.

In Thursday's issue, I left off my rant on biotech grievances with comments on the FDA tipping off the SEC to violations. I've got more to say about the agency.

• OK, maybe we don't really need efficacy either. In late 2004, Abbott asked FDA to approve Xinlay for advanced prostate cancer, even though the drug failed to provide statistically significant clinical benefit over placebo in two trials involving 1,097 patients. The company based the NDA on a "meta-analysis" that pooled data from the two trials, managing to show that "intent to treat" analysis showed a delay in time to disease progression.

Xinlay is a selective endothelin-A receptor antagonist, blocking one of the proteins thought to play a role in metastasis. Heck, just based on coolness of mechanism of action, it should work!

On Sept. 13, the FDA Oncology Drug Advisory Committee decided not to recommend approval of Xinlay. Nonetheless, Abbott continues to believe in their drug and is waiting for final word from FDA. Additional clinical trials in a different patient population also are ongoing.

Xinlay has run smack into the ongoing debate over just how much efficacy is enough for cancer drugs. AstraZeneca plc's Iressa threw a monkey wrench into the engine after an early approval in 2003 was later pulled when more extensive clinical trials failed to support that approval.

• And finally, the FDA has completely given in to political threats. In August, FDA once again refused to make a decision on whether to make emergency contraception available to women without a prescription - a blatant case of abortion politics interfering with the drug approval process. That refusal follows a 23-4 vote in favor of approval by FDA's advisory committee back in December 2003.

According to a scathing commentary in the Sept. 22 New England Journal of Medicine, FDA management was concerned not about safety, efficacy, nor SEC violations, but concerned that teenagers might have more sex if the medication was available at Long's Drugs. Meanwhile, aspirin and acetaminophen - favored OTC drugs for suicide attempts - are readily available.

Susan Wood, assistant commissioner for women's health at FDA, resigned over that dangerous behavior. Her resignation email stated, "I can no longer serve as staff when scientific and clinical evidence, fully evaluated and recommended for approval by the professional staff here, has been overruled."

Sadly, that makes total sense in an administration that doesn't believe in global warming, promoting condom use to prevent STDs, or evolution.

• California is still struggling to figure out how to handle its theoretical $3 billion stem cell grant program without running into conflict of interest. Lawsuits filed by opposing groups are claiming that 13 of the 16 research institutions that have received training grants from the program have a conflict because those institutions receive money from drug companies, have board members who work for biomedical companies, or have some other industry links.

OK, let's see how many research institutes we can name that have none of those characteristics and also are home to the top research teams that have the ability to generate something useful from this stem cell work. Anyone?

The stem cell initiative is bogged down under the completely predictable problems that follow big-bucks programs - worries over how $3 billion is distributed fairly, worries over how the state of California and its taxpayers reap any benefit, arguments over intellectual property ownership, etc. The state apparently can't raise the funds until the lawsuits are resolved.

The state has been run with a significant budget deficit (temporarily vanquished with hand-waving), and the underfunded public school system is under serious pressure to generate those highly educated employees needed to staff state biotech/high-tech industry. Maybe a $3 billion bond issue for a technology that is a decade or more away from clinical use is not the best focus for California. On the other hand, it's done a great job of stimulating lots of competitive funding discussion in other states! Stem cell research just may get the support it needs even if the California Institute for Regenerative Medicine never grants a single dollar.

• We have a great new idea for a low-risk, high-payoff biotech business model - counterfeit pharmaceuticals! Hey, if knockoff Prada purses and Gucchi gumballs are big biz, take a look at this: World Health Organization estimates that counterfeit drugs brought in $32 billion in 2003 global sales - that's 10 percent of worldwide drug sales!

An FDA official was quoted in The Wall Street Journal in early October as saying that "it's more lucrative to sell a counterfeit drug than it is a narcotic such as heroin." If the venture community got excited about selling pet food on the internet and back-burner drugs via specialty pharma plays, just imagine how excited they will get over the pro-formas of a global counterfeiting business model! None of that muss and fuss of regulatory processes and GMP - straight to the internet pharmacies.

• In another blow to big-bucks formularies, the Sept. 22, 2005, issue of New England Journal of Medicine published a report comparing five drugs to treat schizophrenia. The head-to-head study, conducted by the National Institute of Mental Health, part of the National Institutes of Health, found that the snazzy new drugs, based on molecular knowledge of specific neuronal receptors and costing a lot more than the old standby drug, didn't work much better. The oldest drug was as effective as three of the new drugs and had a side effect profile that was similar in severity. Zyprexa, a new drug from Eli Lilly and Co., had a slightly better efficacy coupled with serious side effects.

Two key issues were raised by that study. No. 1, the new drugs had been approved primarily on the basis of short-term studies that compared them to placebo, not to competing drugs, and No. 2, The New York Times was prompted to call for Congress to take note of the idea that there is value to forcing drug companies to test their new drugs against the existing treatments they seek to displace. Argh - clinical costs just went up exponentially.

In this age of cost-effectiveness, it may take more than simply being new.

• More fun with politics. In September, the Washington, D.C. Council made it illegal for pharma companies to sell prescription drugs in the district at "an excessive price" - defined as 30 percent over the comparative price in Germany, Canada, Australia or the UK. The bill passed unanimously.

In a surprise move, the Pharmaceutical Research and Manufacturers of America condemned the move, saying it would "stifle supply, smother innovation, and harm the District's business climate," according to The Washington Post. Following on, the Biotechnology Industry Organization sued the District of Columbia, claiming the act is unconstitutional.

BIO's measured response to the district's attempt to help its population - certainly not the most wealthy population in the country - and its decision to sue to prevent price controls is questionable. Surely BIO's members don't believe that is the only and best approach to helping the country and locals who are trying to cope with the reality of high drug prices.

• According to the PhRMA website home page, America's pharmaceutical companies have donated more than $120 million in drugs and money to relief efforts on the Gulf Coast (gosh, that's almost twice the amount donated to the lobby efforts to block Prop 79 in California!).

As you can see, there is an ever-growing list of bizarre, frustrating and challenging news events that will drive the biotech industry. Take these snippets as just a sampling of what is happening on a daily basis around the world.

The IBF Biotech Investing conference, concluding Thursday in Millbrae, Calif., included lively panel discussions that highlighted the fact that many of the issues are on the minds of people in and around the biotech sector. We will continue to highlight these topics and to ask the industry and its supporters to rise to the challenges presented.

Robbins-Roth, Ph.D., founding partner of BioVenture Consultants, can be reached at biogodess@earthlink.net. Her opinions are her own and do not necessarily reflect those of BioWorld Today.