Tuesday, March 7, 2017

The Value of Oversight in a Century of Promise & 'Cures'

This post, by CEC member Paul McLean, originally appeared on WBUR's CommonHealth blog.
The blood-thinner heparin is not a 21st-century cure. It was discovered 100 years ago by a scientist looking for something else entirely, and is one of the oldest drugs still in regular use.
After my daughter was diagnosed with a potentially fatal blood disorder, heparin played a key daily role in her treatment. We’d wash our hands meticulously, lay out gloves and antiseptic wipes, saline flushes for the access lines to her fragile immune system, and finally the sealed heparin syringe.
For many months, we went through boxes of heparin and never questioned its safety. Never had reason to.
But in 2008, after my daughter was officially declared cured and we’d used heparin for the last time, contamination in the supply from China killed 19 Americans and harmed many others. Writing that sentence still gives me the chills. My daughter survived thanks to medicine, but it also could have killed her.
So you can understand why, as the 21st Century Cures Act sailed to passage, I experienced both excitement and dread.
That heparin contamination was in part due to lax oversight of the drug supply chain. It reminds me why I do not want to see the work of the Food and Drug Administration compromised by overeager drug companies taking advantage of the hopes of desperate patients, and taking shortcuts on safety.
The “giant piƱata” of a bill, as science blogger Derek Lowe aptly described the 21st Century Cures Act, is destined to explode in unexpected ways.
I can’t predict whether it will prove to be mainly a bounty of new treatments, a bouquet of hope for patients, or a lollipop for Martin Shkreli, the hated face of pharma profiteering.
As a medical ethicist, I’m most concerned about this potential fallout: that the FDA will be seen as a barrier rather than a protector, and become too quick with the rubber stamp; and that the DNA of the National Institutes of Health will be edited into a form that diminishes basic science.
And as a father, a husband and a patient myself, I’m most concerned that one day, I’ll ask my doctor, “Is this new drug safe? Is it going to work?” And he’ll shrug.
The act undoubtedly will help with the addiction crisis, improve treatment of the mentally ill, and perhaps move research toward a cure for Alzheimer’s and other brain diseases. That is why it has been championed by the American Psychological Association, the American Society for Human Genetics and the American Society for Clinical Oncology among many others, not to mention President Obama.
But in highlighting the act’s benefits, many of the bill’s backers downplayed its potential harms. Particularly when it comes to the FDA, and the bill’s acceleration of the approval process for new drugs and medical devices.
Is the FDA, in fact, a barrier or a protector? I hadn’t thought to frame the question quite that way until reading about another recent news event. At Standing Rock, in the days before the Dakota Access oil pipeline’s setback, some of people gathered there expressed consternation over the media’s frequent description of Native Americans and their supporters as “protesters.” You see, they perceive themselves as "water protectors."
The 21st Century Cures Act sees the FDA as a barrier to drug makers, blocking the companies' pipeline to patients. What I see is that the agency has long served as a protector — and one thing it has protected is trust in the doctor-patient relationship.
Drugs don’t get approved until proven safe and effective — so doctors know they're unlikely to harm a patient and likely to do some good. Doctors need that confidence in the FDA, but it could be put at serious risk by the act's acceleration of the drug-approval process.
Fueling the bill’s attack on the FDA was an unfounded suspicion that the agency keeps readily available treatments and cures from desperate patients. On the contrary, the FDA has responded meaningfully to the urgency of patients and advocacy groups by creating more avenues for expanded access to experimental treatments.
Still, the 21st Century Cures Act portrays the FDA as too tight, too restrictive. Some backers of the bill portrayed safe and effective treatments as the enemies of compassion. But I worry that choosing that type of compassion over caution can cause harm, both immediate and long-term — to patients and to science alike.
My own bias in assessing the bill can be traced to a cure whose credit belongs largely to the previous century. My daughter had severe aplastic anemia, a failure of the bone marrow that leaves a person defenseless against disease.
There was really nothing doctors could do about it until the second half of the 20th century, when scientists — painfully, methodically — gained understanding of the need for compatible tissues, how to keep certain T cells at bay with aggressive treatment, how to get chemo and radiation doses right, and how to make the donor blood supply safe.
Cyclosporine, which suppresses the immune system, changed everything. In 1983, more than a decade after its discovery in Norwegian soil fungus, it was determined to be safe and effective — by the FDA.
Now, aplastic anemia is often curable, as it was for my daughter. Cyclosporine helped her body make peace with her donor’s stem cells. She benefited not only from emergent knowledge of her rare disease but from myriad clinical trials into other diseases that increased knowledge relevant to her illness.
So when I hear about the billions that the 21st Century Cures Act invests in the “Cancer Moonshot," I know cancer patients won’t be the only beneficiaries. A broader common good will be served, even as individual patients are given new hope and sometimes cured.
But my daughter’s cure was grounded in real scientific knowledge, which randomized clinical trials are uniquely able to provide. And though the 21st Century Cares Act includes $4.8 billion for new research at the NIH, that’s press-release money. The real funding will be determined by Congress over the next decade.
Sen. Elizabeth Warren of Massachusetts, who opposed the bill, warned of its potential systemic harms: “Medical breakthroughs come from increasing investments in basic research,” she said. “Right now, Congress is choking off investments in the NIH.”
My annual physical is coming up. In the past, discussing prescription drugs with my doctor, I’ve never felt the need to ask, “Is this drug safe?” I may need to start. I hope he never answers with a shrug.