Groups who push for progress against childhood cancer see President Donald Trump’s imminent signing of a bill to enhance research in the field as a significant win after they grew frustrated with Congress and the administration’s pursuit of higher-profile legislation to expand access to experimental treatments.
The cancer legislation would let the Health and Human Services Department set up demonstration projects for childhood cancer survivors. Even after successful treatment, these patients can experience effects from their cancer, such as cardiovascular issues, intellectual handicaps and emotional trauma.
“Two-thirds of childhood cancer survivors suffer late effects of either their disease or treatment,” said Michael P. Link, a professor of pediatric oncology at Stanford University and the co-chairman of the Alliance for Childhood Cancer, a patient advocacy group. “This law will spur needed progress.”
The bill would renew and tweak several programs at the National Institutes of Health and the Centers for Disease Control and Prevention. The NIH would be able to make grants for collecting medical specimens related to the childhood cancers with the least effective treatments available. The CDC’s registry for tracking childhood cancer would be reauthorized through 2022, and the CDC would be able to award grants to states to improve their registries. The bill would authorize $30 million a year to support all of these programs.
The Senate passed and the House cleared it, both by voice vote. Supporters say it will make a meaningful difference. It follows similar efforts in the 2017 Food and Drug Administration reauthorization act meant to encourage drug companies to conduct more early-stage research on cancer drugs for children.
“If Congress fully funds this bipartisan bill, it will be a game-changer in how scientists approach childhood cancer research and how the NIH works to make childhood cancer a top national priority,” said Danielle Leach, senior director for advocacy and government affairs at St. Baldrick’s Foundation, a nonprofit charity that provides grants for childhood cancer research.
‘Right to Try’ Law
This all comes on the heels of Trump signing an additional bill that virtually all cancer patient groups opposed: the so-called “Right to Try” law, intended to get patients access to experimental drugs when they don’t qualify for clinical trials and are out of treatment options. Under that law, drug companies can provide unapproved treatments, outside of a clinical trial, to patients with life-threatening conditions if the product has cleared the first phase of safety testing. Previously, the FDA had to sign off on such an arrangement; that is no longer required.
Ultimately, most patient groups don’t think that drug companies will be any more likely to provide their drugs under this framework, and argue that taking the FDA out of the process will put patients at risk of harm.
“Clinical research subject protections are in place when experimental products are being tested to ensure the safe and ethical treatment of research participants,” the American Cancer Society Action Network and other patient groups said in a letter to Congress as lawmakers considered the legislation earlier this year. “Patients seeking expanded access to unapproved therapies outside of clinical trials must be afforded the same ethical standards and protections as patients taking part in clinical trials.”
However, the main reason that patients weren’t able to access treatments outside of clinical trials hasn’t been the FDA but the drug companies themselves. To encourage companies to participate in this expanded access, the new law shields them from liability and prevents the FDA from considering the results of a drug’s use under the law as part of the formal approval data.
FDA Commissioner Scott Gottlieb has vowed to help carry out the law, but he’s also taking the agency in a direction that might reduce the need for it. In a speech Saturday to the American Society of Clinical Oncology’s annual meeting, Gottlieb suggested that cancer research and clinical practice needed to be more highly integrated, and patients should be able to access clinical trials more easily. He also said the FDA should approve more cancer drugs on the basis of more limited data than what’s traditionally been acceptable, as long as follow-up studies were conducted once the products are sold.
“There are critics who say we should hold drugs back from the market, and demand more pre-market studies proving overall survival endpoints, before we consider approving new drugs,” Gottlieb said. “I disagree. And I suspect some of the patients who face long odds, for whom available therapy gives them just a slim chance of long-term survival, might also disagree.”
He also highlighted a recent step to try to get more teen cancer patients into clinical trials. On Friday, the FDA issued a draft document providing guidance to the drug industry on when it’s appropriate to include adolescent patients in clinical trials normally reserved for adults.
The FDA’s rationale is that some cancers in adolescents are similar to cancers in adults, but pediatric trials typically take place years after drugs have already been approved for adults. By including teens in some trials, it could potentially give more younger people a chance to access cancer drugs sooner after their approval for adults.
The guidance says adolescent patients should be enrolled only after toxicity data is obtained from adult patients, and provides age-appropriate dosing recommendations.