With the widely chronicled untimely death last year of Randy Pausch, author of the widely acclaimed “Last Lecture” and international figure thanks to the power of a YouTube video capturing his strength, wit, and laughter in the face of almost certain and imminent death from cancer, it seemed appropriate to reflect on what we as a society ought to be doing to save the next Randy Pausch.
Although I find it a somewhat overused cliché, if we really are ‘at war’ with cancer, we now wage it with guns and tanks from World War II. According to recent Congressional testimony from scientific experts, we are using forty-year old science and statistical methods to try to assess 21st century science and technology. It should be no surprise then, that we aren’t making more progress against cancer. We have made and continue to make tremendous progress in advancing our understanding of cancer and other life-threatening diseases. Where we fail to progress is in translating those advances into meaningful patient benefits, into increased therapeutic options to which doctors and patients have true access.
At this moment, Senator Ted Kennedy is fighting brain cancer in its deadliest form. There are few good options to fight his terrible diagnosis, just as Randy Pausch had little to turn to in his fight against pancreatic cancer. Senator Arlen Specter has good odds against his Hodgkin’s lymphoma, though returning cancer almost always seems to strike with an extra vengeance.
Anna Tomalis, a seventh grader from Maryland that you have likely never heard of, was a sweet, bubbly, 12-year-old girl who ought to have had a lifetime ahead of her, but instead, was told in March of last year that she should go home with her parents and siblings because there was nothing left for her to do but live out her last days. She chose to ignore that dead-end advice and fight on, with the indomitable spirit of a child and the love and fortitude of a mother with a master’s in public health who knows that highly promising options remain to give her daughter more life.
So they continued to fight for access to any one of half a dozen drugs in our research clinics today; drugs that have, on average, almost ten years of data in human clinical trials for Anna’s form of cancer. These drugs have long-since proven their safety, particularly when compared to the certain effects of Anna’s lethal disease. Some of these drugs have shown startling efficacy in treating Anna’s type of cancer, a rare Sarcoma, but she isn’t allowed to try them - instead our exclusionary regulatory system counsels her to go home and die. I was lucky enough to have met her earlier this year and consider myself better for having met her. On August 16th, 2008, Anna finally succumbed to her cancer.
Randy Pausch is now a statistic and was joined by more than 500,000 others who died from cancer in 2008. It may have been Randy’s last lecture, but it need not be for the current and future generations of cancer patients if we act quickly to accelerate our progress against cancer and other life-stealing diseases.
Unfortunately, in this time of ‘safety first, safety last, and safety everywhere in between’ for each new drug reviewed, today’s FDA is dedicating itself to compiling reams of data, piles of statistics, and endless clinical studies on each drug brought for review. This paralysis by analysis is partly a function of what FDA advisors note is a deficiency in scientific expertise regarding new technologies, leading risk averse reviewers to repeatedly request more and more data. It is also partly to protect FDA from the grandstanding of other famous members of Congress who have learned that railing against the FDA on “safety” gets their names and photographs in the news on almost a weekly basis. Hidden behind the intense and almost daily focus on the rare negative effects of a few approved medications, is the far greater loss of life caused by an inefficient, outdated, and out of touch regulatory system that puts the real-world risks faced by seriously-ill patients a distant second to the “data” they endlessly accumulate in hopes of defending themselves against those backseat drivers.
Today’s drug review process fails to adequately consider the very real risks of the disease when comparing the potential benefits of a new drug versus its risks. In a report issued last December, the FDA’s own top science advisors reported that FDA reviewers often don’t understand the science behind the treatments they review and are failing in their judgments of the merits and perils of new therapies. Those advisors noted in a hearing before the House Committee on Oversight and Investigations this January that “American lives are at risk” as the FDA continues to use these half-century old drug development and review concepts.
Industry must do more to work in overcoming intellectual property issues that hamper combination trials, work harder to include academia in reform and improvement discussions, and must continue to stand up for its view on how to accelerate progress, particularly by moving forward with advanced trial designs and statistical analysis methods.
The principle of “Do No Harm” cannot mean withholding a potentially lifesaving therapy, thus contributing to potential suffering and death for a patient. Promoting the public health, as is the FDA’s charge, demands more.
Science has made terrific progress in the lab and in the clinic, but it must be a moral imperative to translate that progress into meaningful patient benefits by providing more, better, and increasingly affordable new options by which patients can truly achieve the promise of personalized medicine. Let us hope that enough members of Congress and the public hear the pleas of these patients, educate and inform themselves, and respond with the speed and resolve necessary to truly accelerate progress.
Founder and Executive Director