A Sick Bureaucracy
September 4, 2013
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As executive director of the Texas Advocacy Project, Andrea Sloan spends her days defending victims of domestic violence. It’s a job she loves. Recently, however, Andrea has been forced to spend more time advocating for herself, fighting for the breakthrough drug that could potentially save her life, but which none of her doctors are permitted to give her.
For the past seven years, Andrea has been battling stage three ovarian cancer. She has already endured two rounds of chemotherapy, five surgeries, and a stem cell transplant. Unfortunately, Andrea’s cancer has returned and she and her doctors at MD Anderson, one of the best cancer centers in the country, have few options left.
There is one drug they believe could help, however. It’s called BMN 673, and it’s right there in the hospital where Andrea is undergoing treatment and being administered to patients in the next room. Andrea’s doctor even told her that “this drug is amazing and is the drug I wish I could give you.” It has shown very promising results in patients who have the same type of ovarian cancer as Andrea, known as BRCA1.
Unfortunately for her, this drug is not yet approved by the Food and Drug Administration. Consequently, the only patients receiving BMN 673 are those enrolled in clinical trials conducted by the drug’s maker, BioMarin, in order to gain FDA approval.
BioMarin, however, is not currently accepting patients like Andrea into its clinical trials. This leaves her with only one hope of obtaining BMN 673: being granted “compassionate use” by the company and receiving a waiver from the FDA. FDA officials have already told Andrea that she would likely receive such approval from the agency, provided BioMarin agrees to let her have it as well.
So far, its executives are refusing. Apparently granting access to a new drug in the middle of a billion-dollar effort to win FDA approval is a risk they are unprepared to take, at the expense of Andrea’s future. The company released a statement that said in part, “It is too early to know if the experimental therapy is safe or effective, or will even prolong life, until we conduct the appropriate Phase 3 trials.” Interestingly, BioMarin is simultaneously preparing to present data at the European Cancer Congress, including early dose expansion data from the ongoing study, with more mature data in breast and ovarian cancers.
Andrea has remained optimistic despite BioMarin’s refusal thus far, continuing her efforts to persuade the company. She says she is thankful to BioMarin for creating the drug and hopes to be a “poster child” for its potential. A petition at change.org has already gathered more than 18,000 signatures requesting the company to give Andrea access to the breakthrough treatment. You can sign the petition here.
Andrea is not alone in her struggle to obtain potentially-life saving breakthrough drugs which are unnecessarily delayed. Every year, thousands of Americans die waiting for treatments to clear the FDA approval process, which now takes 15 years on average from the beginning of clinical trials.
This immense regulatory hurdle is partly responsible for the skyrocketing cost of drug development, which further retards innovation. Avik Roy of the Manhattan Institute reports that these costs rose from $100 million per drug in 1975 to more than $1.3 billion in 2005, adjusted for inflation. Very few companies can afford to bear such costs.
Another victim of this bureaucratic delay was Abigail Burroughs, a bright young 21-year-old student at the University of Virginia who died in 2001 after being denied drugs which might have helped her cancer but had not yet received FDA approval. Her case came to national attention through a heartbreaking profile in the Washington Post and the efforts of her father, who started an organization called Abigail Alliance to advocate improved access to experimental drugs for patients who had no other options.
Abigail’s case was the perfect example of the bureaucratic delays we must fix. One of the drugs she sought access to, Erbitux, was discovered in 1983, when she was three years old. It took a decade to find a company to carry it through the approval process. Not until 2003 did it receive FDA approval for treatment of colorectal cancer. Even more alarming, it was not approved for the late stage head and neck cancer Abigail suffered from until 2011 (ten years after she passed away). That’s a total span of 28 years from discovery to the final approval Abigail needed.
Everyone wants to ensure our drugs are safe, but we must do something to speed up the bureaucratic process which keeps breakthroughs mired in studies and red tape for decades, or nips them in the bud entirely. The regulatory system originally intended to keep Americans safe has become so slow and ineffective that it is killing them.
We must find a way for Americans like Andrea and Abigail who are seeking relief from debilitating diseases to access breakthrough drugs once they have exhausted other options. The outmoded system we have today is keeping treatments out of the hands of those who need them most, often for decades.
One important step would be the Patient Choice Act of 2013, introduced in May by Representatives Scott Peters, Morgan Griffith, and Michael McCaul. This bill would give terminally ill patients access to breakthrough drugs that have received preliminary approval from the FDA or have been approved in Europe (where many treatments are available years before they come to market in the U.S.).
Americans should speak up and support these and other reforms to speed access to medical breakthroughs. In the meantime, please please sign Andrea’s petition and keep her in your thoughts and prayers.