Remembering the Thalidomide Tragedy at ICPE 2011

Things mostly had a familiar feel at this year’s International Conference on Pharmacoepidemiology and Therapeutic Risk Management (ICPE) in Chicago. The agenda was crammed with quick-paced research paper presentations, followed by cross-ex that seemed a bit gentler than usual, and the aisles were packed with posters frequently depicting the most esoteric and nuanced research topics, which sometimes taxed the capabilities of the non-epidemiologic mind to even discern the shades of differences between them.

Except for the mid-meeting plenary session entitled “Thalidomide:  50 Years Later” which brought a collective, highly-emotional hush and pause to all the bustle for a couple of hours.

Any mention of the Thalidomide tragedy is always accompanied by some of those heart-wrenching images of babies with phocomelia, and the awful realization of how this must have affected mothers, families, and caregivers, as well as the victims themselves. This occasion was punctuated by the personal history of Tsugumichi Sato, who, after having been born with this condition, dedicated his life to the science of pharmacoepidemiology and drug safety to help avoid the recurrence of such catastrophes.

Yet it’s hard for us today to imagine how this product could have been approved as safe in 46 countries, and how it was promoted so widely as a wonder drug at the time, such as in West Germany when all general practitioners received a letter promoting its use during pregnancy. And in today’s Internet age, we can’t possibly understand how it could have taken three years after approval before a case-control study finally proved a causal relationship between the drug and its devastating effects. Or how the Swedish government could possibly have delayed communicating that causality because, as Barbro Westerholm, MD,  said with chagrin, “they didn’t want to worry expectant mothers.” Of course, we can too easily imagine the public and political pressures withstood by Francis Kelsey, MD, of FDA, whose steadfast approval to deny approval resulted in only 18 cases in the United States, versus more than 3,000 in Germany alone (where up to one million people were taking the drug at one point).

And, amidst all of this horror, it’s also ironic to think of how the drug has experienced a resurgence for its efficacy for conditions such as leprosy and myeloma—when used under the proper controlled conditions to balance its benefits against its egregious risks.

So we should all stop and think now and then of why we do all that what we do in new drug development. We sometimes rail against all of the barriers, bureaucracy, busy-work, and controls that are placed before us in the development and marketing of new drugs, so it’s good to have a reminder of why these exist, how important it is for all of us in academia, industry, government, and even the public to collect such careful data, to follow the rules and thoroughly weigh the benefits and risks, and what the world was like before them.  Or, to paraphrase the famous Orwell quote, which applies to heroes like Francis Kelsey and Barbro Westerholm as much as it does to the military: “We sleep soundly in our beds because they stand ready in the night.”