Reading some news reports, you might wonder about the benefits for patients of the 21st Century Cures Act. This massive piece of legislation focused on medical research and innovation, has been scripted and re-scripted. The latest version, a 996-page document, includes some odd provisions and compromises I’d prefer to scuttle.
That said, I’m in favor of the Cures Act. Strongly.
I’ve seen stories suggesting that “Cures” is all about perks for pharmaceutical companies, and that industry-funded advocacy groups are parading patients, like puppets, before legislators to promote the bill. Earlier this year, I attended a small meeting where healthcare journalists didn’t just question, but scoffed at the prospect of speeding the FDA’s approval process, which is part of the package. “Why would anyone support this bill?” a journalist asked.
The fact is, every patient with whom I have discussed the 21st Century Cures Act hopes it will move forward. Doctors too, tend to favor this bill, because it supports more government-funded research.
I support most of the “Cures” provisions, for three big-picture reasons. First, the legislation includes significant investment for President Obama’s Precision Medicine Initiative and the real Cancer Moonshot. I agree with oncologists who observe that we’re at a pivotal moment in cancer research, ready to turn in-hand progress in science and information technology into survival and quality-of-life advantages. Second, because this legislation will enable the FDA to modernize its ways of evaluating new treatments, including more input from patients. In this age of open information and Facebook groups with patients trading stories of medication toxicity, I’m less afraid of bad drugs getting approved than of having a bureaucracy block their availability to people who want to try those. Third, because this legislation has the potential to keep the U.S. at the forefront of medical progress, a draw for scientists and doctors around the world, a source of pride.
Janet Freeman-Daily is one of many patients with whom I’ve discussed the 21st Century Cures Act. At age 60, she’s an MIT and CIT-educated engineer who lives in Seattle with her husband. She’s been alive with advanced lung cancer for five years, and counting several beyond what she expected. The only thing she’s ever smoked is salmon, she said.
Her 2011 diagnosis came as a surprise, at age 55. After standard treatments with radiation and chemotherapy for stage 3 disease, metastatic disease became evident in bones and elsewhere. Fortunately, Freeman-Daily has family in Denver and sought consultation at the University of Colorado. An oncologist there advised sequencing of her tumor. Molecular studies revealed an altered ROS1 gene in her lung cancer cells. This mutation renders the tumor sensitive to crizotinib. This drug, sold by Pfizer as Xalkori, was FDA-approved for advanced lung cancer with ALK1 mutations in 2011. Its on-label use has since been extended to ROS1-mutated cases.
Freeman-Daily supports the 21st Century Cures Act because it will promote research and patients’ access to new drugs. “I like the focus on precision medicine, infectious diseases and pediatrics, with global studies,” she told me by phone. “The plan to include more patients’ voices is great,” she added. “Mainly it’s good for research. We’ve got to let our scientists do science and stop worrying about paperwork.”
“They’re talking about accelerating approval of drugs for patients with rare diseases and oncogene-driven cancers like mine.” Many of these are conditions are infrequent; it would be extremely difficult if not impossible to collect sufficient numbers of patients for separate clinical trials of each drug in each condition. “The nearest trial for me is one thousand miles away, in Denver.” Freeman-Daily travels to check in for her clinical trial. “Many patients can’t travel.” That’s why greater flexibility in trial design for evaluating precision medicine drugs is needed, she considered.
“I understand the concerns for rigor and safety,” she considered. “But if we wait until the results of phase 3 trials are available, how many of us are going to die in the meantime? If you’re out of other options, you’re willing to take a bit of a risk if you want to stay alive.”
Peter Pitts is a former associate FDA commissioner and co-founder of the Center for Medicine in the Public Interest. His experiences as a person with congestive heart failure influence but don’t define his views on medical care, he told me in a phone interview. “Like many people in the health policy field, I have a health condition,” he said. “I care deeply about these issues.”
“I’m a big supporter of the bill” Pitts said, referring to the 21st Century Cures legislation. “What it says for patients is that their voices will be heard more regularly and taken seriously,” Pitts said. “That is a good thing for public health.”
“There are no white hats and black hats in this,” he said. Health are is like an ecosystem, he suggested. It will only work if all the people involved, all the players–including physicians, payers, industry, regulators and patients themselves–take a role in helping patients to achieve better health.
Pitts takes a positive view of proposed changes for the FDA. “The agency is at center of innovation,” he said. “It does much more than expedite approvals and bring drugs to market. The agency is responsible for distributing truthful, accurate and non-misleading information, beyond what’s on the labels,” he said. “That is crucial for physicians and for patients.”
“The most important shift that is embodied in the 21st Century Cures act is that it goes beyond pure regulatory science, for the FDA, to understanding and incorporating what patients feel is appropriate,” Pitts said.
I asked Pitts about some concerns for the FDA lowering standards for drug approval. “The FDA is at a crossroads now,” he considered. “If some people think the FDA is moving too quickly, and some people think it’s too slowly, then it’s probably doing things just about right,” he said. “At the end of the day, I think patients want the FDA to be innovative, aggressive and prudent. That’s a nice public health triple play.”
