NCHR’s Testimony to FDA on Pediatric Development Plans for Oncology Indications

Nina Zeldes, PhD, National Center for Health Research, June 17, 2020

Thank you for the opportunity to speak today on behalf of the National Center for Health Research. I am Dr. Nina Zeldes, a senior fellow at the center. Our center analyzes scientific and medical data to provide objective health information to patients, health professionals, and policy makers. We do not accept funding from drug or medical device companies, so I have no conflicts of interest.

We strongly support the FDA and Committee’s goals to suggest recommendations for clinical trials of treatments for pediatric cancers during these meetings taking place over the next two days. Rare and aggressive childhood cancers are often fatal. It is essential to carefully study benefits and risks to determine if the likely benefits of a specific indication outweigh the risks.

My statement today is relevant to all the drugs you are considering today and tomorrow.

When evaluating drugs for pediatric use, doses must be scrutinized cautiously for children of different ages and weights. Even when there are likely benefits for children on average, it is important to minimize risks whenever possible by determining which children are most and least likely to benefit.

It is also important to consider whether patient demographics affect the benefit or risk. With that in mind, we strongly urge you to recommend that all clinical trials should include subgroup analyses for sex, race, and age. We understand that this is difficult in rare diseases, but at least some demographic differences are likely to increase or reduce the risks or benefits. Risk-benefit profiles should, when possible, be assessed for each particular subgroup.

For example, given the known very serious adverse events of MRZ, to be discussed later today, it is important to determine which types of patients, in terms of demographics, are most likely to benefit. And, in addition to targeting patients most likely to benefit, are there other ways to mitigate risks? One of the discussion questions for MRZ addresses mitigating risks for pediatric patients. We suggest that risk mitigation be considered for all 4 drugs that will be discussed over the course of these next two days.

We understand the desire to get new treatments to patients who desperately need them as quickly as possible. But, it is important to make sure that clinical trials are appropriately designed to clearly answer questions of safety and efficacy. Poorly designed trials, trials with too few patients, or too few patients representing key demographic groups, or with poorly selected endpoints do not provide clinicians and patients the information that they need to make informed decisions. Parents want to have hope for their children, but no parent wants to subject their child to treatments with horrible side effects unless those treatments can eventually significantly improve how long they live or their quality of life. Efforts to design the best possible clinical trials and to protect patients who participate in those trials or who may eventually be prescribed cancer drugs are essential. Even if clinical trials take a little longer, but are more informative and conclusive, they will in the long run help more patients and harm fewer patients, which is everyone’s goal.