RACE Act Implementation: Our Comments on Draft Guidance
The Food and Drug Administration (FDA) recently published draft guidance on implementation of the FDA Reauthorization Act of 2017 (FDARA) as it relates to pediatric studies of molecularly targeted oncology drugs. Through February 12th, the FDA is accepting comments on the draft guidance. Children’s Cancer Cause submitted our comments this week, which you can read in full here.
The 2017 passage of FDARA included provisions from the Research to Accelerate Cures and Equity for Children (RACE) Act, which improved opportunities for more studies in childhood cancer by amending the Pediatric Research Equity Act (PREA). This is a drug development issue that Children’s Cancer Cause has been engaged in since PREA was enacted back in 2003.
PREA - and its partner legislation known as BPCA - provided critically important information on the safe and effective use of medications in the pediatric population. But these laws had a very modest impact on childhood cancer. In fact, exemptions carved out resulted in PREA having virtually no effect in stimulating new treatments for childhood cancer. The RACE Act eliminated those exemptions - marking a huge victory for our kids on an issue we have been working on for years.
The new draft FDA guidance lays the groundwork for new rules that will go into effect this August because of this legislation - rules that will accelerate early pediatric evaluation of molecularly targeted cancer drugs.
Read More: Fostering Oncology Product Development for Kids with Cancer (FDA Law Blog)
Below are highlights from our own comments about this guidance. We encourage others to submit comments (you may borrow from our own), which can be done by visiting the Regulations.gov comment page.
The FDA is to be commended for stressing international collaboration agreements about pediatric plans with the European Medicines Agency (EMA) and regulatory agencies from other countries. Hopefully, such coordination will encourage companies to advance international alignment and prevent duplication in planning pediatric trials. We are especially pleased that the European-based ACCELERATE project is cited as a trusted forum for further defining the parameters of molecularly-driven pediatric cancer evaluations.
FDA states that it will consider pediatric waivers for agents within the class when competing studies with the same mechanisms of action are undertaken and there is no evidence that they have proven relevant in pediatric settings. This approach clearly will protect children from unnecessary exposure to agents for which there may already be effective therapy. It may also prevent companies from conducting pediatric trials of agents that do not propose clinical advances for children but rather fulfill a pediatric requirement for the sole purpose of seeking extended exclusivity for an adult indication.
We are pleased to see guidance describe a clear pathway for FDA to issue a Written Request once an Initial Pediatric Study Plan and Proposed Pediatric Study Request are evaluated. We would hope that the ‘carrot’ of a possible six months of patent exclusivity for a new agent will work in the new regulatory context to incentivize companies to move early and efficiently to gather the evidence necessary for submitting pediatric plans.
We urge the FDA to be strict with timelines and deadlines and to strongly encourage companies to thoroughly investigate academic, NCI, NIH, and other resources necessary to provide evidence for their pediatric plans in a timely manner. Similarly, if an applicant is awarded a deferral or waiver, a determination of when that designation might be revisited - or not - would provide industry with certainty about expectations and resources required, and ensure that current science informs decisions about pediatric therapeutic relevance about previously eliminated agents.