FDA Is Slowing Down Necessary Drugs and TreatmentsFor those looking for fresh evidence that companies and physicians are pushing cancer treatment into new frontiers, look no further than last week's annual American Society of Clinical Oncology meeting in Chicago. A new treatment designed to attack genetic abnormalities in a subset of lung cancer patients shrank or stabilized tumors in about 90 percent of patients in one study. Another clinical trial for a drug designed to boost the immune system extended survival for patients with melanoma, a deadly form of skin cancer that—unless it's caught early—is quickly fatal. Without a doubt, enormous public and private investments into the genetic causes of chronic disease are producing effective new treatments.
June 17, 2010
But by the time promising treatments like those presented at ASCO actually gain FDA approval and reach patients, far too many patients will have died waiting. New discoveries move from the lab to patients only as fast as the FDA can certify that they're safe and effective, and it's taking the FDA too long to do so.
One reason why is money: the amount spent on improving regulatory science at the FDA is a pittance-just $18 million for the FDA's Critical Path Initiative (the agency's national strategy for modernizing how new medical products are developed and evaluated) in 2010, including for the veterinary and food centers at the agency—compared to the over $30 billion we spend on basic research at the National Institutes of Health, and the $65 billion drug companies annually spend on R&D.
This is the equivalent of building a state-of-the-art racecar, fitting it with a Model T engine, and expecting it to break the sound barrier.
If the FDA doesn't have the scientific base to create flexible regulations that keep up with the pace with of new research, some vital discoveries will languish or be lost entirely. Prioritizing new cures—and accelerating patient access to innovative therapies—means recognizing that the FDA is not just a regulatory agency, but a scientific agency that plays a critical role in medical innovation. On average, it already takes drug companies over a decade, conducting tests and clinical trials, to bring a single new treatment to market. The FDA's drug-development regulations must be streamlined and modernized to help bring more innovative products to market faster.
Fortunately, the FDA is acutely aware of the problem. In 2007, the agency's science board said that "FDA's inability to keep up with scientific advances means that American lives are at risk. While the world of drug discovery and development has undergone revolutionary change... FDA's evaluation methods have remained largely unchanged over the last half century."
A June report from Battelle, a technology consulting group, sponsored by the Council for American Medical Innovation, reached a similar conclusion: "In the midst of the explosion of scientific knowledge and improvements, we have allowed our regulatory system to fall behind in its scientific skills and tools and instead become mired in processes that are unable to predictably balance the need for safety as well as patient benefits."
Streamlining how drug development is regulated could have enormous benefits for patients. In a recent report for the Manhattan Institute, health economists Tomas Philipson, from the University of Chicago, and Eric Sun, from Stanford University, calculated the value of three FDA-approved drugs to patients: highly active antiretroviral treatment (HAART), for the treatment of HIV/AIDS; trastuzumab (Herceptin), for the treatment of breast cancer; and rituximab (Rituxan), for the treatment of non-Hodgkin's lymphoma.
They estimate that HAART was worth $330 billion to AIDS patients; rituximab, $8 billion to lymphoma patients; and trastuzumab, $137 billion to patients with breast cancer.
But those benefits would've been even greater if patients had gotten access to these therapies just one year earlier. For HIV/AIDS patients, the value of one year's earlier access would've been $19 billion. For breast cancer patients being treated with trastuzumab, the value of one year's earlier access would be $8 billion. For patients diagnosed with non-Hodgkin's lymphoma, a year's earlier access to rituximab would be worth $310 million. For all three medicines, this totals over $27 billion. That dwarfs the benefits to drug companies from earlier market access—including reduced clinical trials costs and increasing profits—which stands at a comparatively modest $5 billion.
Quantifying the costs of drug development delays to patients-and the benefits of quicker access- should give policymakers and regulators an added incentive to look for ways to streamline drug development.
Still, there are legitimate concerns that speeding up the drug development process might present safety risks. But the FDA has at its disposal several tools that should mitigate those concerns, like post-market surveillance to rapidly remove unsafe drugs from the market; using biomarkers to detect patients who are likely to experience serious side effects; and authority to require post-market drug studies to better identify potential risks.
But the best argument for overhauling drug-development regulations is that safety and innovation are two sides of the same coin. As regulatory science advances, companies and regulators will be able to identify the most promising treatment candidates—and winnow out weak ones—much faster than they can now. Increased clarity about relative risks and benefits from new medicines will reassure patients and policymakers that accelerated access isn't coming at the price of public safety.
We know what we need to do. What is lacking now is simply a sense of urgency in Congress. Advocacy groups representing patients suffering from serious and life-threatening chronic diseases must demand that the FDA receive the funding it needs to advance regulatory science and accelerate access to innovative therapies. For patients suffering and dying from diseases like Alzheimer's and pancreatic cancer, there is not a second to waste.
Paul Howard is the director of the Center for Medical Progress at the Manhattan Institute and managing editor of MedicalProgressToday.com.