Why Do Pediatric Drugs Take So Long to Develop? A Look into the Lag Time on Drug Approvals for Childhood Cancer & Other Illnesses

Unfortunately, Dr. Jackson explains, there is not a whole lot of incentive for pharmaceutical companies to study drugs for children. Children make up a very small number of people with chronic illnesses or conditions that require serious therapies. For example, when it comes to cancer, the American Cancer Society estimates that around 15,590 children and adolescents (ages zero to 19) were diagnosed with cancer in 2021; that’s compared to an estimated 1.9 million new cancer cases in total).

“Clinical development is done by for-profit companies,” Dr. Jackson explains. “Those for-profit companies sell drugs to help patients, but they also need to make a profit. The market in pediatrics is very, very small. Only 25% of the population is less than 18, and even fewer of those under 18 years old have chronic conditions that will require long-term therapy … there's no market advantage really.”

Because of this small number, the companies that make and study drugs may not see financial opportunity in putting resources into pediatric clinical trials and that’s a real issue. Legislation passed in the U.S. the Pediatric Research Equity Act (PREA) and Best Pharmaceuticals for Children Act (BPCA) aimed to incentivize drug makers to study treatments for children, and required these studies in certain situations, but there is still a lag.

“Were it not for those rules and regulations, these trials wouldn’t be done,” Dr. Jackson explains. “There’s very little incentive for pharmaceutical companies to do those trials, but the laws are very helpful.”

Why are clinical trials so important?

To understand why it’s important that companies conduct clinical trials in children and adolescents, it’s important to understand why these trials are so important in the first place.

A clinical trial is essentially a study that tests out new and innovative drugs on patients with certain illnesses. These patients are monitored closely by a team of doctors and the information that is obtained from these trials is critical to the advancement of new therapies.

“Clinical trials hopefully can benefit you, but is also providing very, very vital information to the whole scientific community about the effectiveness of these treatments,” Dr. Beth Karlan, a gynecologic oncologist at UCLA Medical Center, told SurvivorNet in a previous conversation.

Dr. Beth Karlan, of UCLA Medical Center, explains the importance of clinical trials.
“They can be lifesaving,” Dr. Karlan added. “We’ve seen many in the last few years of children and adults who have participated in trials and have had miraculous results.”

Dr. Jackson adds, “Clinical trials in children are necessary due to the physiological and developmental differences between the various age groups of in children vs. adults.”

SurvivorNet also has this tool to help patients looking for clinical trials find the right option.

What can be done about the lag in pediatric drug approvals?

One major obstacle to tackle is awareness. The majority of the world was aware of how quickly clinical research was done to advance the Covid-19 vaccine and approvals for children to get the vaccine came shortly after. But unfortunately, this is not the case for the vast majority of drugs.

“The general public has no idea that a lot of the drugs that are being used in children are off-label,” Dr. Jackson explains. “It’s not a terrible thing, it’s just not optimal.”

Off-label means that a doctor may prescribe a drug that is approved for one condition or in adults, but has not gone through the full approval process for other conditions its being prescribed for or been studied in children. It’s a very common practice.

“There are ways that we can prescribe these drugs safely especially older ones but it doesn’t allow children to take these new and innovative drugs, especially when it comes to chronic conditions” Dr. Jackson explains. “So, children are kind of these therapeutic orphans. Using off-label doesn’t gather any data … it’s this terrible catch-22 we get ourselves into. We don’t have drugs approved so we use them off-label and because we use them off-label, it’s harder to recruit for clinical trials.”

In addition to what I-ACT for Children is doing by creating connections between pediatric researchers and drug development stakeholders, pediatric clinical trials also need to be part of the culture when it comes to chronic conditions, Dr. Jackson explains.

Pediatric cancer happens to be an area where participation in clinical trials is encouraged, and often welcomed by families who have a sick child.

“There are some areas that are extraordinarily good at having children enroll in clinical trials something like 90% of children with cancer,” Dr. Jackson explains. “It's really high because it's become part of the culture, the culture of the training physicians. People just know they're going to be part of a trial. There's a very good trial network that's been set up for this.”

“Clinical research is a part of good clinical care,” she adds. “When you have good clinical care, you should be able to enroll in a clinical research trial.”

Learn more about SurvivorNet's rigorous medical review process.