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RACE for Children Act Spurs Pharma to Study Molecularly Informed Drugs in Pediatric Cancer Patients


NEW YORK – An impact analysis of the RACE for Children Act has shown that the legislation is prompting drugmakers to study more molecularly informed drugs in pediatric cancer trials.

Based on the success of RACE, which took effect in August 2020, advocates are now pushing for a second bill that would require pharmaceutical companies to study combination therapies for pediatric cancers.

The impact analysis, presented at the American Association for Cancer Research's annual meeting on Monday, tracked the number of drugs for which sponsors had to complete pediatric cancer trials from 2019, the year prior to the RACE Act's implementation, to 2021.

Legislators passed the RACE (Research to Accelerate Cures and Equity) for Children Act in 2017, requiring that when drugmakers submitted new drug applications (NDAs) to the US Food and Drug Administration for adult cancers, they also study the agents in pediatric cancer patients if the underlying molecular mechanism was relevant for the population.

In the study presented at AACR, researchers led by Brittany McKelvey, a science policy analyst at Friends of Cancer Research, analyzed sponsors' willingness to conduct pediatric cancer trials for 19 drugs, a dozen approved pre-RACE implementation and seven approved after the bill became law. The proportion of NDAs submitted to the FDA with required pediatric study plans jumped from zero in 2019, to 43 percent after RACE went into effect.

"With the approach of the one-year anniversary [of implementation] last fall, we wanted to evaluate the impact of the RACE Act on the number and types of pediatric studies that were being required, specifically for orphan designated products," McKelvey said. "When we looked at the year after RACE implementation, almost half of the agents that were approved did have some type of required pediatric study. Of those agents that had required pediatric study, all had an orphan designation, highlighting the impact of the RACE Act."

The bill also closed a loophole that drugmakers had been using to avoid conducting pediatric studies if their therapeutics were for a rare cancer indication and had received orphan drug designation from the FDA.

Of all 19 therapies included in this analysis, 78.9 percent received an orphan drug designation and none of the drugs approved pre-RACE were studied in pediatric cancer trials. Post-RACE implementation, 71.4 percent of the therapies received an orphan drug designation and 60 percent of those were still required to do pediatric studies due to the drug's relevant mechanism of action.

McKelvey highlighted drugmakers' research efforts for two drugs to demonstrate the RACE Act's effect on closing the orphan designation loophole. In early 2020, the FDA cleared Incyte's FGFR2 inhibitor Pemazyre (pemigatinib) for FGFR2-altered cholangiocarcinoma patients. While FGFR2 alterations are also relevant to pediatric cancers, Incyte was not required to conduct pediatric studies because the drug was granted orphan designation.

After the RACE Act went into effect, the FDA approved QED Therapeutics and Helsinn Group's Truseltiq (infigratinib) for a similar indication in FGFR2-altered cholangiocarcinoma. However, because the law had changed, the companies made a pediatric study plan despite the orphan designation and will study Truseltiq in pediatric patients with FGFR2-altered advanced or metastatic solid tumors, an even broader population than the approved adult indication, McKelvey said.

However, a limitation of the RACE Act was that it only applied to single-agent cancer drugs. Advocates are now hoping to expand the FDA's authority to require that drug sponsors conduct pediatric studies of combination therapies with the Give Kids a Chance Act, which was introduced in the US House of Representatives in March.

In a presentation at the Children's Hospital of Philadelphia Pediatric Cancer Summit in March, Nancy Goodman, a patient advocate and founder and executive director of the nonprofit Kids v Cancer, explained why Give Kids a Chance is needed and is a logical next step after the RACE Act.

"If they have a novel therapy that they are submitting to the FDA for approval, and there's no theory of efficacy without a matched second drug as a combination or if there's other data indicating that a combination would dramatically increase the probability of a durable response, the FDA can ask a company to consider a combination [for pediatric cancer trials], but there's no requirement the FDA can impose here," Goodman said. "Some companies have been open to talking with the FDA. We want to make sure it's all companies."

When a drugmaker submits investigational new drug applications for a combination therapy with the FDA, the new bill would allow the agency to require the sponsor to study that regimen in relevant pediatric cancer patients based on available data. The regulator could also require companies to conduct combination pediatric cancer trials for single agents. Companies that submit single agent INDs could be asked to study the drug in combination with standard-of-care therapies, like chemotherapy, or other approved oncology drugs. Finally, the bill would give the FDA authority to ask companies to undertake preclinical studies of a drug combination that could lead to pediatric trials.

Goodman noted that she is "cautiously optimistic" that Give Kids a Chance will pass by September of this year. The bill will be attached to the omnibus spending bill that needs to be passed by the end of September. If passed, she and other advocates believe it would continue the RACE Act's impact of expanding the proportion of drugs studied in children with cancer.

In a House Committee on Energy and Commerce subcommittee on health hearing last month, Rep. G. K. Butterfield, D-N.C., who introduced the Give Kids a Chance Act, noted that the bill had "many" supporters among members of the health subcommittee.

In the same hearing, Cartier Esham, CSO of the Biotechnology Innovation Organization, acknowledged that pediatric studies present some challenges for the industry.

"Firstly, it is very rare, and the etiology and biology of cancers that occur in children can differ from those that occur in adults, so immediate extrapolation of efficacy and safety is not always possible," Esham said. "We don't want children being placed at a disadvantage of being enrolled in a clinical trial that has undue exposure to risks or does not provide the necessary healthcare."

However, Goodman said that some in the pharma and biotech industry may be against the bill requiring even more studies. She suggested some groups were pushing back on Give Kids a Chance, "it's not the direction I would choose, so we really need a counter it [to get this passed]," she said.

McKelvey said that it may be several years before the RACE Act leads to a meaningful increase in new cancer therapy options approved for pediatric cancers. Some of the studies required of sponsors in the first year of RACE will take at least five years to complete.

Her team at Friends of Cancer Research will continue to follow up this data to confirm whether more pediatric studies "translates to label expansions for pediatric patients," she said.

"Some possible solutions to potentially be able to address medical research in these populations are to encourage the use of master protocols and tissue-agnostic trials, to extrapolate safety and efficacy information from adult clinical trials, and include adolescents in pivotal registrational trials for relevant pediatric cancers," McKelvey said.