Back to—and from—the books

Some of you might have been wondering about my uncharacteristic silence since the 2^nd edition of my book was born. No—it’s not from post-partum depression. From June until Thanksgiving I have either been working caring for patients or been immersed in the ritual hazing known as preparing for the “Boards.” I am now coming back to my usual self and a more normal life. I have just taken the ABIM’s Infectious Diseases computer-graded subspecialty examination, yet won’t know the results for months. While recovering from this miserable rite of passage, I thought I would get back to commenting on recent notable events.

Two of my favorite pharma scandals to follow have been intermittently in the news. First is the ongoing saga of Pfizer’s Trovan trials, now of Wikileaks fame. Guess apologies are due for my previous guarded defense of Pfizer. Sorry…

Genzyme is often in my thoughts. Perhaps their story reminds me of karma. They were one of the darlings of biotechs. First, they had great respect, having developed life-saving drugs for orphan diseases, Gaucher’s, Fabry’s, and Pompe’s disease, each caused by in-born errors in metabolism.

Then the company’s avarice caught up with them, and they exploited their position as the sole provider of treatment, as I’ve written about here, both in the US and in Latin America, where courts have upheld the constitutional right of access to medication, regardless of the cost.

Last spring, Genzyme’s products were found to have viral contaminants and even bits of trash, prompting a shutdown of its plant and rationing of the life-saving drugs. This has led to the FDA’s issuing a costly consent decree and fast-tracking a rival’s drug. The rationing has gone on for 18 months (for Fabrazyme) and no end is in sight.

Genzyme has also been the target of a takeover bid from Sanofi Aventis in recent months.

So, patients petitioned the NIH and DHHS to override Genzyme’s patent exclusivity, given that the NIH funded the research at Mt. Sinai, which then licensed Fabrazyme only to Genzyme. Last week, NIH turned down this appeal for licensing rights.

Given that patients have no other source of vitally needed medicines, that the drug development was funded by the public, and that Genzyme has not been a good steward of its patent monopoly, do you think pro-business laws like the Bayh-Dole act should be reconsidered? Should NIH now reject patent exclusivity in favor of patient access? What do you think?

Birthing a New...Book!

Longer than the usual gestation, the 2^nd edition of Conducting Clinical Research just came into being. A bit overdue, the past 10 months have been an immersion of reading, research, and writing to bring you an expanded, revised, and fully updated edition.

CCR 2^nd shares the basic genetic makeup of its older sibling—a pragmatic, step-by-step, how-to manual for both experienced medical professionals and newbies, and shares the dominant wry humor gene.

There are notable differences, with many updates including recent regulations affecting research, subject injury clauses, tips on surviving audits and legal landmines. New topics include the unique needs of device and vaccine trials, perspective on the globalization and outsourcing of clinical trials, cultural competency and health literacy—keys to understanding and succeeding with the changing dynamics of the industry.

My first-born was greeted with much critical acclaim:

"Successfully covers . . . clinical trials. Practical considerations, substantial background and resource information. The text is well written . . . fills an important need."

—Annals of Internal Medicine, April 2007

“5 Stars! (100 out of 100)… This is a must have for novice or experienced clinical researchers.”

Doody's Review: Expert Review 2007

Sibling rivalry portends even greater success for the new addition.

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To find out more, click here for an excerpt, table of contents, and complete description.

Please share our joy with your friends.

The Rise of the Superbugs

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This week has brought news of new superbugs--daptomycin-resistant VRE (Vancomycin-resistant Enterococcus) isolates and linezolid-resistant MRSA.

I've cared for patients this week with MRSA, VRE, and ESBL gram-negative rods resistant to almost every antibiotic.

Many factors fuel the rise in resistant organisms. Some that are not often addressed include inadequate education (e.g. that asymptomatic Foley catheter related cultures should not be treated);

fear of peer review or legal liability for not treating a culture;

the demands of family or religious groups that “everything be done” even when the patient is clearly terminal and the treatment is futile—and where the treatment of one such patient puts many others at unnecessary risk.

A excellent overview of some of the other issues can be found at the Center for Global Development’s drug resistance site.

As an Infectious Disease physician who has been involved in clinical research for new antibiotics as well as patient care, I am very familiar with the difficulties of bringing a new drug to market. In fact, I participated in early clinical trials for both Daptomycin and Linezolid. It angers and saddens me to see these valuable and critical resources squandered by irresponsible detailing and prescribing patterns and because of liability concerns. We need serious guidelines and restrictions on antibiotic use if we are not to soon enter the post-antibiotic era. There is no time to wait for new drugs to be developed.

Rare Diseases Day

Just a quick note to remind you today is Rare Diseases Day. A rare disease is one that affects less than 1 in 2000 people. Because of the infrequent occurrence, these diseases tend to be overlooked by many, including Pharma, as there is generally no financial incentive, as the potential market is so limited (Genzyme is an exception--see the previous posts about Gaucher's disease and orphan drugs).

You can learn more about this from the European Organisation for Rare Diseases, EURORDIS, an umbrella group of organizations addressing problems of specific rare diseases and encouraging research in these areas. Check it out. They have great information./a>