Commentary: No more death by federal red tape
March 25, 2009
Here’s hoping that as Dr. Margaret Hamburg takes over the Food and Drug Administration FDA, she will remember Anna Tomalis, a seventh-grader from Maryland who died last August from cancer.
Archaic FDA rules prevented Tomalis from getting a new drug in the late stages of development that doctors believed would have meaningfully extended her life.
And despite considerable scientific advances in understanding cancers, the FDA is presiding over a period of plummeting approvals. The result is that other drugs that could help patients like Anna never make it from the lab to the medicine cabinet.
To prevent the FDA from blocking medical progress and innovation, the new commissioner must reform and modernize the review process so 21st-century science can be turned into valuable new medicines.
Despite progress in research, 2008 was a dark year for actually fighting cancer. Only 10 cancer drugs got market approval from the FDA but only three of those were actually new, the rest being approved drugs receiving further approval to treat additional cancers.
This mirrors a wider decline: Only 19 new medicines of any kind were approved in 2007, the lowest figure for 24 years. That was a bad blip, but averages are steadily falling.
This decline is inversely proportional to the high levels of scientific discovery and investment. Between 1990 and 2007, the private sector tripled research and development budgets to $45 billion.
According to Tufts University research, the number of drugs entering clinical development more than doubled between 2000 and 2005, but only 8 percent of these candidates finally won market approval from the FDA, down from 20 percent for the period 1993-97.
Something is clearly wrong with the way we regulate drug development. Huge financial and intellectual resources are being poured into one end of the pipeline but very little comes out the other. The FDA’s methods are now almost five decades old and far behind current science.
There are also problems with the way the FDA reviews and approves clinical trials. A recent study by Vanderbilt University showed that some cancer trials take more than 800 days before they are even allowed to begin, with more than half of all process steps adding nothing to the assessment.
Between 1999 and 2006, the average length of a clinical trial increased by 70 percent but delivered little for patients. As Dr. Vincent DeVita, one of the country’s leading oncologists, wrote in Nature journal in December: “At my own institution I reside on many committees involved in protocol review, and I can say with authority that we are hopelessly over-regulated.”
As a result, bringing a new drug to market now takes between 12 and 15 years and costs around $1.2 billion, effectively excluding all but huge companies.
This makes it uneconomic to develop new treatments for many less common afflictions such as rare cancers. The increasing regulatory burden now stifles research, forcing it overseas to countries such as China and India.
One easy reform for the new leaders at the Department of Health and Human Services and the FDA would be to speed up access to the most promising new therapies when their benefit to a patient outweighs the risk from the disease.
Currently, the system considers a drug unsafe and ineffective until the day it is approved, when in reality its effects are known long beforehand. The FDA thus arbitrarily denies terminal patients access to experimental drugs that could extend their lives.
As a large, centralized bureaucracy, the FDA should recognize it cannot keep pace with science to the degree necessary to assess new drugs and diagnostics promptly and efficiently.
The FDA should therefore restrict itself to what it does best, such as advising on best practice and monitoring new drugs as they become widely used.
Actual drug approval must be delegated to those closer to the science and the patients, such as the National Cancer Institute, at the forefront of research and treatment and with experience of partnership with the FDA on training.
Dr. Hamburg must sweep away bureaucratic inertia and commit herself to bringing the FDA into the 21st century. Reform of drug approval and regulation is not just a dry subject for academics and bureaucrats, nor is it pro- or anti-industry: It is pro-patients — and life or death for many.
Scott Riccio is founder and director of Accelerate Progress, the nonprofit Center to Accelerate Progress Against Cancer and Other Life-Threatening Diseases.
