There’s so much in flux in healthcare now, it’s hard not to worry about people who need doctors and treatment for illness, trauma and other conditions, like pregnancy. Dozens of millions of Americans may lose their health insurance coverage, or be relegated to networks so narrow or Medicaid plans so sub-optimal they’ll skip preventive care and, if they become sick, might be denied expert care. Women will become infertile from complications of botched abortions. There will be more untreated disease if the ACA is repealed rather than advanced, as seems likely under Trump’s administration.
So you might ask how I support the 21st Century Cures Act, when I fear for loss of “the basics.” I’m reminded of a conversation from when I was in medical school: “Why do you want to do research when people in Bolivia can’t get penicillin?” a friend challenged me. His point was that as a civil and global society, we’d be better off raising the lower bar–for essential medicines and surgery–than working to improve outcomes in people with cancer and rare diseases.
The answer is we need and can afford both: to raise the bar so that everyone has access to modern healthcare, and to advance treatments through medical science. There’s no dichotomy between healthcare for all and research, including precision medicine and cancer research. We can afford to take good and better care of people who are ill, and also study ways of preventing and tempering diseases. The two goals are intertwined.
It’s true, the 21st Century Cures Act will be good for pharmaceutical companies. But it will also be good for patients. Among other things, this legislation supports that a larger fraction of research will come from public U.S. agencies such as the National Institutes of Health. I have some serious concerns about the bill, such as easing approval for stem cell therapies, and reducing requirements that pharmaceutical companies disclose payments to physicians. Those parts of the bill have reportedly been nixed, which is a good thing.
The main issue is exorbitant drug pricing, and the larger problem of healthcare’s unaffordability. Even with insurance, patients struggle to pay for their medications. So we’re back to an updated version of my friend’s question: Why develop better drugs and treatments if people can’t afford them? The 21st Century Cures Act does nothing to address this very real issue, of healthcare costs. I’m not sure it’s the best legislative bundle in which to remedy this huge problem, as suggested by Senators Bernie Sanders and Elizabeth Warren, but it does need address.
Given how much progress may come from the "Cures" bill, I think it would be a shame to deny this act and pull back on the U.S. government’s investment in medical research, what the 21st Century Cures Act is really about. In principle and over the long term, better medical treatments should lower treatment expenses for many patients, who will require less hospitalization for disease. With improved outcomes, many will live longer and more productive lives.
Some policy experts and doctors have expressed concern about greater speed at the FDA, and increased use of “real-world” data. But I think those are good, modern aims that will help patients. As I've said before, I believe we need a strong FDA, but that the agency’s main role should be in collecting and reporting on safety and toxicity, including post-marketing data. I don’t support free-standing stem cell clinics, places where vulnerable patients might receive who-knows-what organic and inorganic substances. But I do support patients’ having greater access to drugs manufactured carefully in monitored facilities by reputable companies.
One detail in my view on the FDA's role comes from my experiences as a patient, and as a peer patient. In general, I’m more concerned about easing medical device approvals than about expediting medication reviews. That’s because few patients, if any, can know what tools their surgeon will use in an operating room, or a gastroenterologist might use in an endoscopy suite. A cardiac pacemaker or defibrillator, once inserted, becomes hard to remove. Medications have risks, but usually those can be weighed before trying, and can be stopped if serious side effects occur. I don’t dismiss those risks. But I know that most patients are capable of weighing the risks of medications along with potential benefits.
All of this will require transparency, providers’ avoidance of conflicts of interest, and greater education of the public so they can make informed choices. Up-to-date information needs be available about all treatments, including mandatory post-marketing reports on toxicity. Modern patients want to consider their treatment options in discussion with peer patients and doctors, in context of their values and priorities. What citizens expect, and still need, is for the FDA to monitor the quality and manufacture of drugs and medical devices, so they can be assured of the veracity of products sold.
